Clinical trial NCT03717415
Conditions: Locally Advanced or Metastatic Solid Tumor
Keywords: rebastinib breast cancer ovarian cancer mesothelioma
Sponsor: Deciphera Pharmaceuticals, LLC
Start date: 2019-01-02; Completion date: 2022-11-01
Enrollment: 70 participants; Allocation: RANDOMIZED; Intervention model: SEQUENTIAL

Brief summary:
This is an open-label Phase 1b/2 multicenter study of rebastinib (DCC-2036) in combination with carboplatin designed to evaluate the safety, tolerability, and pharmacokinetics (PK) in participants with advanced or metastatic solid tumors.

Detailed description:

Arms: arm 1: Dose escalation with rebastinib 50 milligram (mg) twice daily (BID) orally (PO) in 21-day cycles in combination with carboplatin administered by intravenous (IV) infusion at area under the curve (AUC)5 at Day 1 of each 21-day cycle. arm 2: Dose escalation with rebastinib 100 mg BID PO in 21-day cycles in combination with carboplatin administered by IV infusion at AUC5 at Day 1 of each 21-day cycle. arm 3: Dose escalation with rebastinib 100 mg BID PO in 21-day cycles in combination with carboplatin administered by IV infusion at AUC6 at Day 1 of each 21-day cycle. arm 4: Dose expansion in triple-negative breast cancer (TNBC) participants. Rebastinib 50 mg BID PO in 21-day cycles in combination with carboplatin administered by IV infusion at AUC5 at Day 1 of each 21-day cycle. arm 5: Dose expansion in TNBC participants. Rebastinib 100 mg BID PO in 21-day cycles in combination with carboplatin administered by IV infusion at AUC5 at Day 1 of each 21-day cycle. arm 6: Dose expansion in platinum-sensitive ovarian cancer participants. Rebastinib 50 mg BID PO in 21-day cycles in combination with carboplatin administered by IV infusion at AUC5 at Day 1 of each 21-day cycle. arm 7: Dose expansion in platinum-sensitive ovarian cancer participants. Rebastinib 100 mg BID PO in 21-day cycles in combination with carboplatin administered by IV infusion at AUC5 at Day 1 of each 21-day cycle. arm 8: Dose expansion in mesothelioma participants. Rebastinib 50 mg BID PO in 21-day cycles in combination with carboplatin administered by IV infusion at AUC5 at Day 1 of each 21-day cycle. arm 9: Dose expansion in mesothelioma participants. Rebastinib 100 mg BID PO in 21-day cycles in combination with carboplatin administered by IV infusion at AUC5 at Day 1 of each 21-day cycle.
Interventions: intervention 1: Rebastinib intervention 2: Carboplatin
Intervention descriptions: intervention 1: Administered orally intervention 2: Administered by IV infusion

Protocol documents:
Prot_000.pdf: 
 
  
 
Protocol: DCC-2036-01-004  
 
Official Title: An Open-Label, Multicenter, Phase 1b/2 Study of Rebastinib (DCC-2036) in Combination 
with Carboplatin to Assess Safety, Tolerability, and Pharmacokinetics in Patients with Advanced or 
Metastatic Solid Tumors 
 
NCT Number:  NCT03717415 
 
Approval Date: 24 FEB 2020 


24 FEBRUARY 2020 
CONFIDENTIAL 
Deciphera Pharmaceuticals, LLC 
CLINICAL STUDY PROTOCOL 
Protocol DCC-2036-01-004 
An Open-Label, Multicenter, Phase 1b/2 Study of Rebastinib (DCC-2036) 
in Combination with Carboplatin to Assess Safety, Tolerability, and 
Pharmacokinetics in Patients with Advanced or Metastatic Solid Tumors 
This study will be conducted according to the protocol and in compliance with  
Good Clinical Practice, the ethical principles stated in the Declaration of Helsinki, 
and other applicable regulatory requirements. 
Study Sponsor: 
Deciphera Pharmaceuticals, LLC 
Headquarters: 
200 Smith Street 
Waltham, MA 02451 
Phone: 
 
Fax: 
 
Research Facility: 
643 Massachusetts St., Suite 200 
Lawrence, KS 66044 
Phone: 
 
Fax: 
 
IND Number: 
128702 
Document Version (Date): 
Original (12 JUL 2018) 
Amendment 1 (02 OCT 2018) 
Amendment 2 (15 NOV 2018) 
Amendment 3 (07 MAY 2019) 
Amendment 4 (24 FEB 2020) 
NCT #: NCT03717415
PPD
PPD
PPD
PPD


Clinical Study Protocol DCC-2036-01-004, Amendment 4 
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SPONSOR SIGNATURE 
Date 
24 FEBRUARY 2020 
CONFIDENTIAL 
Deciphera Pharmaceuticals, LLC 
PPD
PPD


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Deciphera Pharmaceuticals, LLC 
INVESTIGATOR STATEMENT 
I understand that all documentation provided to me by Deciphera Pharmaceuticals, LLC or 
its designated representative(s) concerning this study that has not been published 
previously will be kept in the strictest confidence. This documentation includes the study 
protocol, Investigator Brochure, case report forms, and other scientific data. 
This study will not commence without the prior written approval of a properly constituted 
Institutional Review Board/Independent Ethics Committee/Research Ethics Board. No 
changes will be made to the study protocol without the prior written approval of Deciphera 
Pharmaceuticals, LLC and the Institutional Review Board, except where necessary to 
eliminate an immediate hazard to the patient. 
I have read, understood, and agree to abide by all the conditions and instructions contained 
in this protocol. 
 
Investigator Name 
 
Investigator Signature 
 
Date 
 
 
 
 
 
Name of Investigational Site 
 


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Deciphera Pharmaceuticals, LLC 
CLINICAL STUDY SYNOPSIS 
Protocol Title: 
An Open-Label, Multicenter, Phase 1b/2 Study of Rebastinib (DCC-2036) in 
Combination with Carboplatin to Assess Safety, Tolerability, and 
Pharmacokinetics in Patients with Advanced or Metastatic Solid Tumors 
Protocol Number: 
DCC-2036-01-004 
Study Phase: 
1b/2 
Study Centers: 
Dose Escalation: Approximately 4 centers in the United States (US) 
Dose Expansion: Up to 18 centers in the US 
Number of Patients 
Planned: 
Dose Escalation: Approximately 18 patients to determine the recommended 
Phase 2 dose (RP2D) 
Dose Expansion: Approximately 99 patients in indication-specific cohorts  
Objectives: 
Dose Escalation: 
Primary Objectives: 
• 
To establish the maximum-tolerated dose (MTD) or RP2D of 
rebastinib and carboplatin in combination 
• 
To evaluate the safety and tolerability of rebastinib when 
administered in combination with carboplatin 
Secondary Objectives: 
• 
To assess the pharmacokinetics (PK) of rebastinib and carboplatin 
when administered in combination 
• 
To assess the preliminary efficacy of rebastinib administered in 
combination with carboplatin 
Dose Expansion: 
Primary Objectives: 
• 
To evaluate the objective response rate (ORR) as the primary 
efficacy measure of rebastinib in combination with carboplatin 
• 
To evaluate the safety and tolerability of rebastinib when 
administered in combination with carboplatin 
Secondary Objectives: 
• 
To assess the PK of rebastinib and carboplatin when administered in 
combination 
• 
To evaluate efficacy measures, such as progression-free survival 
(PFS), clinical benefit rate (CBR), response duration, time to 
response, time to progression (TTP), and overall survival (OS) of 
rebastinib in combination with carboplatin 


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Deciphera Pharmaceuticals, LLC 
Dose Escalation and Dose Expansion:  
Exploratory Objectives: 
• 
Assess the quality of life (QOL) impact of rebastinib administered in 
combination with carboplatin using patient reported outcome (PRO) 
measures  
• 
To evaluate changes in select blood and plasma biomarkers when 
rebastinib is administered in combination with carboplatin 
• 
To evaluate changes in the tumor tissue microenvironment 
(e.g., changes in the composition of infiltrating mononuclear cells) 
when rebastinib is administered in combination with carboplatin 
• 
To assess polymorphisms in genes encoding drug metabolic 
enzymes and/or transporters involved in metabolism and disposition 
of rebastinib in combination with carboplatin 
Study Design: 
This is an open-label Phase 1b/2 multicenter study in patients with advanced 
or metastatic solid tumors who have exhausted available, approved therapies 
and for which carboplatin is considered appropriate treatment. 
Adverse events (AEs) will be assessed, and laboratory values, vital sign 
measurements, and electrocardiograms (ECGs) will be obtained to evaluate 
the safety and tolerability of rebastinib when administered in combination 
with carboplatin. AEs will be graded according to the National Cancer 
Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) 
Version 5.0. Tumor response will be assessed according to Response 
Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 or modified 
Response Evaluation Criteria in Solid Tumors (mRECIST; pleural 
mesothelioma only). Biomarker and PK samples will be collected at 
prespecified time points. PROs will be collected electronically in accordance 
with the guidelines from the organization that developed the instrument. 
Rebastinib will be administered orally (PO) twice daily (BID) and 
carboplatin will be dosed on Day 1 of each cycle. Each cycle is 21 days, 
however, initiation of subsequent cycles after Cycle 1 is dependent upon 
carboplatin dosing. Carboplatin dosing may be delayed due to 
treatment-related AEs up to 14 days without dose reduction of carboplatin. If 
carboplatin dosing is delayed more than 14 days but less than 21 days, the 
dose will be reduced by one dose level. If the dose delay is 21 days or more, 
then the patient will be discontinued from the study unless the Investigator 
and the Sponsor agree that the patient is getting a clinical benefit from study 
treatment. However, if a patient completes 4 cycles of study drug 
combination treatment (80% of planned rebastinib doses per cycle) and can 
no longer tolerate carboplatin, the patient may continue rebastinib as single 
agent treatment. 
The study consists of two parts: Dose Escalation and Dose Expansion.  
Dose Escalation 
In the Dose Escalation phase, doses of rebastinib and carboplatin will be 
escalated using modified 3 + 3 dose escalation rules starting with rebastinib 
at 50 mg BID in combination with AUC5 of carboplatin. The next cohort is 
rebastinib at 100 mg BID with AUC5 of carboplatin. The third planned 
cohort will be rebastinib at 100 mg BID with AUC6 of carboplatin. Based on 
the safety and tolerability, other combinations of doses such as rebastinib at 
50 mg BID with AUC6 of carboplatin may be evaluated if, at least, 


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rebastinib at 50 mg BID with AUC5 of carboplatin is deemed safe. In 
addition, the rebastinib dose may be increased to 150 mg BID with AUC5 
and/or AUC6 of carboplatin if 100 mg BID of rebastinib with AUC5 and 
AUC6 of carboplatin is tolerated. Alternatively, 75 mg BID of rebastinib 
may be evaluated if 100 mg BID is not tolerated and 50 mg BID is deemed 
safe. Dose escalation will proceed to the next dose level if no dose-limiting 
toxicity (DLT) is observed in a minimum of 3 patients completing 1 cycle. If 
a DLT is observed in only 1 of 3 patients, an additional 3 patients will be 
enrolled up to a total of at least 6 patients at this dose level. Dose escalation 
will only proceed if no more than 1 patient in the cohort of at least 6 patients 
has experienced a DLT during the first cycle of treatment in the dose 
escalation phase. If a DLT is observed in 2 or more patients in a cohort of 
3 to 6 patients, dose escalation will stop. The determination of the MTD or 
an RP2D will be based on the safety and tolerability of at least 6 patients at a 
dose level of the combination. As necessary for evaluation of safety and 
tolerability, up to 12 patients may be enrolled per cohort. Enrollment of 
additional patients to further explore safety and tolerability may take place 
simultaneously while the cohort at the next dose level is enrolling patients 
for dose escalation. A comparison of the PK and pharmacodynamic (PD) 
data, as well as preliminary efficacy data obtained in the Dose Escalation 
phase, may be used to refine the RP2D for each Dose Expansion cohort. The 
MTD is defined as the highest dose level of rebastinib and carboplatin at 
which no more than 1 of 6 patients experiences a DLT during the first cycle. 
The RP2D will be a dose level of rebastinib and carboplatin deemed safe and 
tolerable on the basis of the totality of safety, tolerability, PK, and 
preliminary efficacy data. The RP2D will not exceed the MTD. 
Dose Expansion 
Upon determination of the MTD or an RP2D, the Dose Expansion phase will 
be initiated to enroll approximately 99 patients in three indication-specific 
cohorts. A Simon’s two-stage design will be applied to the Dose Expansion 
phase to further evaluate the safety, tolerability, and preliminary efficacy of 
rebastinib in combination with carboplatin in triple negative breast cancer 
(Cohort 1), ovarian cancer (Cohort 2), and mesothelioma (Cohort 3). An 
RP2D used for each cohort will be chosen based on agreement between the 
Investigators and the Sponsor.  
Each cohort in the Dose Expansion phase will initially enroll up to 
18 patients in the first stage. The decision to enroll patients beyond the first 
stage will be based on response assessments obtained after the first post-dose 
response assessment of the last patient enrolled in the first stage of a cohort. 
If >4 responses (defined as partial response [PR] or complete response [CR] 
as best response) are seen in a cohort, additional patients will be enrolled for 
a total of up to 33 patients. If ≤4 responses are seen in a cohort, the cohort 
will be terminated. If >4 responses are seen prior to the last evaluable patient 
in the first stage, expanding the cohort may be triggered earlier. Patients who 
meet criteria defined in Section 8.3 will be replaced and not be included in 
the responder analysis. There will be an enrollment pause between the first 
and second stage for evaluation of response. 


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Dose-Limiting 
Toxicities 
A DLT will be defined as any one of the following AEs during the first cycle 
of treatment occurring during Dose Escalation of the study up until the time 
of establishing the MTD or an RP2D, unless it is clearly and incontrovertibly 
due to disease progression or other identifiable extraneous causes. 
• Any AE preventing administration of ≥80% of planned doses of 
rebastinib during the first cycle 
• A delay in the initiation of Cycle 2 more than 2 weeks due to a lack of 
adequate recovery from toxicity (see Section 5.3)  
• Hematologic AEs: 
o Grade 4 neutropenia (<500/mm3; <0.5 x 10e9/L) ≥7 days  
o ≥Grade 3 febrile neutropenia  
o Grade 3 thrombocytopenia, associated with bleeding that requires 
transfusion therapy 
o Grade 4 thrombocytopenia 
• Non-hematologic AEs: 
o Any Grade ≥3 non-hematologic toxicity will be considered a 
DLT except: 
o Grade 3 nausea or vomiting lasting <7 days 
o Grade 3 diarrhea lasting <7 days 
o Grade 3 fatigue 
o Isolated, asymptomatic Grade 3 abnormalities in chemistry 
laboratory values that last for ≤7 days. This includes 
electrolyte abnormalities that respond to medical 
intervention 
o ALT or AST elevation of >3X ULN with total bilirubin elevation 
of >2X ULN will be considered as a DLT if absence of initial 
findings of cholestasis such as alkaline phosphatase elevation of 
<2X ULN and no other reason can be found to explain 
simultaneous elevation of ALT or AST and total bilirubin 
• Any AE at least possibly related to the study drugs rebastinib or 
carboplatin, regardless of NCI CTCAE grade, leading to dose 
modification of rebastinib or carboplatin in the first cycle 
Study Population: 
Inclusion Criteria 
Patients must meet all of the following criteria to be eligible to enroll in the 
study: 
1. Male or female patients ≥18 years of age at the time of informed 
consent. 
2. Dose Escalation 
i. 
Histologically confirmed diagnosis of a locally advanced or 
metastatic solid tumor for which carboplatin is considered 
appropriate treatment. 
ii. 
Patients who have progressed despite standard therapies, or 
for whom conventional therapy is not considered effective 
or tolerable, as judged appropriate by the Investigator. 


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3. Dose Expansion, All Cohorts 
A. Cohort 1: Triple-negative Breast Cancer 
i. 
Histologically confirmed metastatic triple-negative breast 
cancer based on the American Society of Clinical Oncology 
(ASCO)/CAP guidelines (1). 
ii. 
Received at least one prior line but no more than three prior 
lines of systemic chemotherapy in the metastatic setting. 
B. Cohort 2: Ovarian Cancer 
i. 
Histologically confirmed, recurrent epithelial ovarian, 
peritoneal or fallopian tube carcinoma. Note: patients with 
low grade serous, mucinous or clear cell histology will be 
excluded. 
ii. 
Achieved a PR or CR following treatment with at least one 
of the prior platinum-based regimens. 
iii. 
Recurrent platinum-sensitive disease, defined as disease 
progression ≥6 months after completing a minimum of 
4 cycles in the most recent previous platinum-containing 
regimen. 
iv. 
Received no more than five prior lines of systemic 
chemotherapy. 
a. Neoadjuvant and/or adjuvant is considered one 
regimen. 
b. Maintenance therapy including poly (ADP-ribose) 
polymerase (PARP) inhibitors is considered part of 
the preceding regimen. 
c. Hormonal therapy is not considered a prior systemic 
chemotherapy.  
v. 
Must have received prior treatment with a PARP inhibitor if 
patients have a BRCA1 or 2 germline or somatic 
mutation(s). Patients who have refused therapy with a PARP 
inhibitor may be considered for enrollment, following 
consultation with the Sponsor. 
C. Cohort 3: Mesothelioma 
i. 
Histologically confirmed pleural or peritoneal malignant 
mesothelioma not eligible for curative surgery. Note: 
patients with pericardial, and tunica vaginalis testis 
mesotheliomas will be excluded. 
ii. 
Received at least one prior systemic chemotherapy.   
4. At least one measurable lesion according to RECIST Version 1.1, or 
mRECIST for pleural mesothelioma.  
5. Eastern Cooperative Oncology Group (ECOG) performance status 
(PS) of ≤2 at screening. 
6. Able to provide an archival tumor tissue sample; if an archival tumor 
tissue sample is unavailable, a fresh tumor biopsy is required prior to 


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the first dose of study drug, only if tumor biopsy is safe and 
accessible for biopsy as judged by the Investigator.  
7. Adequate organ function and bone marrow reserve as indicated by 
the following laboratory assessments performed within 14 days prior 
to the first dose of study drug:  
i. 
Bone marrow function: absolute neutrophil count (ANC) 
≥1500/µL; hemoglobin ≥9 g/dL; platelet count 
≥100,000/µL. 
ii. 
Hepatic function: total bilirubin ≤1.5 × the 
upper limit of normal (ULN) or <3× ULN for Gilbert’s 
syndrome; aspartate transaminase 
(AST)/alanine transaminase (ALT) ≤2.5× ULN (≤5× ULN 
in the presence of hepatic metastases). 
iii. 
Renal function: creatinine clearance (CL) ≥50 mL/min based 
either on urine collection or Cockcroft Gault estimation. 
iv. 
Coagulation profile: prothrombin time adjusted for the 
international normalized ratio (PT-INR) and 
partial thromboplastin time (PTT) ≤1.5× ULN. Patients on a 
stable, maintenance regimen of anticoagulant therapy for at 
least 30 days prior to study drug administration may have 
PT-INR measurements >1.5× ULN if, in the opinion of the 
Investigator, the patient is suitable for the study. An 
adequate rationale must be provided to the Sponsor prior to 
enrollment. 
8. If a female of childbearing potential, must have a negative serum 
beta-human chorionic gonadotropin (β-hCG) pregnancy test at 
screening, and agree to use two methods of contraception with, one 
of them being highly effective, prior to the first dose of study drug 
and for at least 120 days following the last dose of study drug as 
outlined in Section 6.8.10. 
9. If male, must agree to practice effective barrier contraception, and 
refrain from sperm donation prior to the first dose of study drug 
through 120 days following the last dose of study drug as outlined in 
Section 6.8.10. 
10. Patient must provide signed consent to participate in the study and 
be willing to comply with study-specific procedures. 
Exclusion Criteria 
Patients meeting any of the following criteria will be excluded from the 
study: 
1. Received prior anticancer or other investigational therapy within 
28 days or 5× the half-life (whichever is shorter) prior to the first 
dose of study drug. See Section 5.11.6 for further details. 
2. Not recovered from all toxicities from prior therapy to Grade ≤1 
(or baseline) within 1 week prior to first dose of study drug 
(excluding alopecia and ≤Grade 3 clinically asymptomatic alkaline 
phosphatase).  
 


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3. Patients with a prior or concurrent malignancy whose natural history 
or treatment have the potential to interfere with the safety or efficacy 
assessment of this clinical trial.  
4. Known active central nervous system (CNS) metastases defined as:  
i. 
Unstable (i.e., evidence of progression by 
magnetic resonance imaging [MRI]) within 4 weeks 
prior to the first dose of study drug.  
ii. 
Neurologic symptoms within 2 weeks prior to the first 
dose of study drug and required use of enzyme-inducing 
antiepileptic drugs. 
iii. 
Patients who require steroids must be on a stable dose 
for 2 weeks prior to the first dose of study drug. 
5. Use of systemic corticosteroids within 7 days prior to the first dose 
of study treatment or an existing condition that requires the 
concomitant use during the course of the study, unless the dose is no 
more than the equivalent of prednisone 15 mg/day. Inhaled, 
intranasal, intraocular, topical, and intraarticular injections are 
allowed. NOTE: premedication with steroids, in accordance with 
institutional practice (including dexamethasone), is permitted prior 
to carboplatin dosing. 
6. Known retinal neovascularization, macular edema or macular 
degeneration. 
7. History or presence of clinically relevant cardiovascular 
abnormalities such as uncontrolled hypertension, history of class III 
or IV congestive heart failure according to New York Heart 
Association classification, unstable angina or poorly controlled 
arrhythmia as determined by the Investigator, or myocardial 
infarction within 6 months prior to the first dose of study drug. 
8. QT interval corrected for heart rate at screening using 
Fridericia’s formula (QTcF) >450 ms in males or >470 ms in 
females or history of QT interval corrected for heart rate (QTc) 
prolongation. 
9. Left ventricular ejection fraction (LVEF) <50% at screening. 
10. Arterial thrombotic or embolic events such as cerebrovascular 
accident (including ischemic attacks) or hemoptysis (more than 
mild) within 6 months prior to the first dose of study drug. 
11. Symptomatic venous thrombotic event (e.g., deep vein thrombosis) 
within the 3 months prior to the first dose of study drug. Following a 
symptomatic venous thrombotic event of ≥3 months prior to the first 
dose of study drug, must be on a stable dose of anticoagulation 
therapy if clinically indicated.  
12. Active infection ≥Grade 3 requiring IV anti-infective treatment 
within 7 days prior to the first dose of study drug.  
13. Known human immunodeficiency virus or hepatitis C infection only 
if the patient is required to take medications that are excluded per 
protocol. 


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14. Active hepatitis B or active hepatitis C infection.  
15. Use of proton pump inhibitors (PPI) within 4 days prior to the first 
dose of study drug or an existing condition that requires the 
concomitant use of a proton pump inhibitor during the course of the 
study. 
16. If female, the patient is pregnant or lactating. 
17. Major surgery ≤4 weeks prior to dosing; all surgical wounds must be 
healed and free of infection or dehiscence. 
18. A manifestation of malabsorption due to prior gastrointestinal 
surgery, disease or other illness which could affect oral absorption as 
judged by Investigator and Sponsor. 
19. Known allergy or hypersensitivity to any component of rebastinib or 
any of its excipients. 
20. Any other clinically significant comorbidities, such as uncontrolled 
pulmonary disease, active infection, or any other condition which, in 
the judgment of the Investigator, could compromise compliance with 
the protocol, interfere with the interpretation of study results, or 
predispose the patient to safety risks. 
21. Peripheral neuropathy of any etiology >Grade 1. 
Study Drug, 
Formulation, Dose 
and Route of 
Administration: 
Rebastinib will be administered in combination with carboplatin. 
Rebastinib will be provided by the Sponsor as 25 mg and 75 mg formulated 
tablets for oral administration. 
Rebastinib will be dosed BID continuously throughout each cycle. In the 
Dose Escalation phase, rebastinib will be escalated from 50 mg to 100 mg in 
combination with carboplatin. In addition, doses of 150 mg and 75 mg may 
also be explored.   
Carboplatin will be provided by the Sponsor and will be administered by IV 
infusion over approximately 60 minutes in accordance with institutional 
practices including premedication, if required, on Day 1 of each cycle and at 
least 21 days apart. A carboplatin dose will be calculated using the Calvert 
formula. The maximum carboplatin dose should not exceed AUC 
(mg x min/mL) x 150 mL/min. On Day 1 of Cycles 1 and 2 when serial PK 
samples will be collected, patients must take the dose of carboplatin 2 hours 
(+/-30 minutes) after rebastinib dosing.  
In the Dose Escalation phase, carboplatin will be dose-escalated from AUC5 
to AUC6 as described. In the Dose Expansion phase, the MTD or an RP2D 
of rebastinib and carboplatin will be administered. A different dose level not 
exceeding the MTD may be chosen for each cohort.  
Study Endpoints: 
Dose Escalation and Dose Expansion Endpoints: 
Safety: 
• 
DLTs in the Dose Escalation Phase 
• 
AEs 
• 
Serious adverse events (SAEs) 


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• 
Adverse events of special interest (AESIs) 
• 
Dose reduction or discontinuation of study drug due to toxicity 
• 
Physical examinations 
• 
Eastern Cooperative Oncology Group Performance Status 
(ECOG PS) 
• 
Ophthalmic examinations 
• 
Changes from baseline in laboratory parameters 
• 
ECGs 
• 
Echocardiograms/multigated acquisition scans (MUGAs) 
• 
Vital signs 
Pharmacokinetics: 
PK endpoints when rebastinib is administered in combination with 
carboplatin and as a single agent include, but are not limited to: 
• 
Time to maximum observed concentration (Tmax: rebastinib only)  
• 
Time to maximum observed concentration at steady state (Tmax,ss: 
rebastinib only) 
• 
Maximum observed concentration (Cmax) 
• 
Maximum observed concentration at steady state (Cmax,ss) 
• 
Concentration observed at the end of the dosing interval (Cmin, 
trough concentration) 
• 
Concentration observed at the end of the dosing interval at 
steady state (Cmin,ss) 
• 
AUC 
• 
T1/2 
• 
Volume of distribution (Vd) 
• 
CL 
Efficacy Endpoints: 
Radiographic tumor assessments (computed tomography [CT] or MRI) will 
be performed by RECIST Version 1.1, or mRECIST for patients with pleural 
mesothelioma. Additionally in the Dose Expansion phase, patients with 
ovarian cancer will be assessed using both RECIST Version 1.1 and 
Gynecologic Cancer Intergroup [GCIG] Cancer Antigen 125 [CA-125] 
criteria. The endpoints for preliminary assessment of antitumor activity 
include:   
• 
Objective response rate (complete response [CR] + partial response 
[PR]) 
• 
CBR (CBR = CR + PR + stable disease [SD]) at 6, 12, and 18 weeks 
of the combination therapy 
• 
Best overall response 


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• 
Time to response (defined as time from Cycle 1 Day 1 to PR or CR) 
• 
PFS (defined as time from Cycle 1 Day 1 to disease progression or 
death due to any cause) 
• 
TTP (defined as time from Cycle 1 Day 1 to the first documentation 
of progressive disease) 
• 
Duration of response (DOR; time from first PR, CR to disease 
progression or death due to any cause). This endpoint will be 
determined for responses only, defined as time of first 
documentation of response to first documentation of disease 
progression or death of any cause 
• 
OS 
Pharmacogenomics: 
The pharmacogenomics endpoints of the study include, but are not limited to: 
• 
Assessment of polymorphisms in genes that may be associated with 
clinical response and/or study drug-related toxicity 
Pharmacodynamics: 
The pharmacodynamic endpoints of the study include, but are not limited to: 
• 
Assess changes of plasma chemokines/cytokines upon treatment  
• 
Assess changes in monocyte population in peripheral blood 
• 
Evaluate changes in tumor microenvironment, using tumor tissue, if 
obtained, including but not limited to tumor associated macrophage, 
tumor infiltrating lymphocytes using immunohistochemical (IHC), 
in situ hybridization (ISH) or other fit-for-purpose assays 
Patient Reported Outcomes (PROs): 
• 
Assess the safety profile of rebastinib in combination with 
carboplatin using certain questions from the NCI 
Patient-Reported Outcomes Version of the 
Common Terminology Criteria for Adverse Events (PRO-CTCAE), 
a “treatment-bother” question (GP5) from 
Functional Assessment of Chronic Illness Therapy’s (FACIT) 
Functional Assessment of Cancer Therapy – General (FACT-G), as 
well as the European Organisation for Research and Treatment of 
Cancer Quality of Life Questionnaire for Cancer 30-item 
(EORTC QLQ-C30) 
Statistical 
Considerations: 
General 
Descriptive statistics (number of patients, mean, standard deviation, median, 
minimum, and maximum) will be used to describe continuous variables. 
Time-to-Event variables such as PFS will be described using medians with 
90% confidence intervals. Categorical variables will be summarized using 
frequency distributions and percentages. All available data will be included 
in data listings. Data may be displayed by rebastinib, carboplatin, and overall 
combination of the treatment. In the Dose Expansion phase, each 
indication-specific cohort will be analyzed independently.  


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Given that this is a Phase 1b/2 study, no formal hypothesis testing will be 
performed. 
Analysis Populations 
Enrolled Population: The Enrolled Population contains all patients who 
signed the ICF.  
Safety Population: All patients who are exposed to any amount of either 
study drug. 
Modified Intent-to-Treat Population (mITT): All patients who had at least 
one full dose of the combined study drugs, had measurable disease at 
baseline, and had at least 1 postbaseline disease assessment unless the patient 
discontinued prior to the post-baseline disease assessment due to an AE at 
least possibly related to rebastinib or due to clinical progression. 
PK Population: The PK population will include all patients who received at 
least one dose of either study drug and had at least one measurable 
concentration in plasma for either study drug. Additionally, this population 
will be used for analysis of PD data, if post-dose PD data is available. 
Analysis of Efficacy Endpoints 
Efficacy Endpoint: ORR is the primary and secondary efficacy endpoint for 
the Dose Expansion phase and the Dose Escalation phase, respectively. The 
secondary endpoints include CBR, time to response, PFS, TTP, DOR, and 
OS. All response endpoints will be defined based on RECIST Version 1.1 or 
mRECIST (pleural mesothelioma only).  
Primary Endpoint: The primary endpoint, ORR, defined as the proportion of 
patients with a CR or PR will be analyzed in the mITT population as the 
primary analysis. Results will be summarized for each cohort with the 
proportion and exact 2-sided 90% CIs. 
Secondary Endpoint: CBR will be calculated and summarized with n and 
percentage at 6, 12, and 18 weeks. Proportions with exact 2-sided 90% CIs 
will be reported for each cohort. For time-to-event endpoints, summaries for 
each cohort will be provided using the methods of Kaplan-Meier (KM), and 
will include medians with 2-sided 90% CIs. 
Sample Size Justification 
Dose Escalation: The Dose Escalation phase will primarily be to determine 
the MTD or an RP2D and evaluate the safety and tolerability of the 
combination using modified 3+3 dose escalation rules. Three dose escalation 
cohorts are planned, and additional cohorts may be added. Approximately 
18 patients will be enrolled in the Dose Escalation phase. 
Dose Expansion: A Simon’s two-stage design will apply to this phase of the 
study. The number of patients required for each cohort was calculated to 
demonstrate 20% improvement in ORR (from 20% historical ORR in the 
setting to 40% for the combination) under 80% power and one-sided alpha of 
0.05. In the initial stage, up to 18 patients will be evaluated. Greater than 
4 responses will be required to enroll additional patients to demonstrate the 
target efficacy of >10 responses in a total of 33 patients. Thus, this part of 
the study may enroll approximately 99 patients (33 patients per indication-
specific cohort). Patients who meet criteria defined in Section 8.3 will be 
replaced and not be included in the responder analysis. For example, if 10% 


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of patients will be replaced, approximately 110 patients may be enrolled in 
the expansion phase. 
Duration of Study: 
Patients will receive study treatment until they develop progressive disease, 
experience unacceptable toxicity, or withdraw consent. 
Patients will be eligible to receive study treatment as long as the Investigator 
and the Sponsor agree that the patient is showing clinical benefit and for as 
long as rebastinib is being developed to support the indication, and 
continuation of treatment does not conflict with the Sponsor’s right to 
terminate the study. The study will end following the last patient's last visit. 


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Figure 1: 
Study Schema 
 
BID = twice daily; MTD = maximum tolerated dose; AUC = area under the concentration-time curve; RP2D = recommended phase 2 dose. 
In dose escalation, rebastinib at 50 mg BID with AUC6 of carboplatin may be evaluated if, at least, rebastinib at 50 mg BID with AUC5 of carboplatin is deemed 
safe. In addition, the rebastinib dose may be increased to 150 mg BID with AUC5 or AUC6 of carboplatin. Alternatively, 75 mg BID of rebastinib may be 
evaluated if 100 mg BID is not tolerated and 50 mg BID is deemed safe. Enrollment of additional patients for determination of an RP2D or evaluation of safety 
and tolerability may take place simultaneously while a cohort at the next dose level is enrolling patients for dose escalation. 


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Table 1: 
Schedule of Assessments 
Assessments / Procedures1 
Screening 
Cycle 1 
Cycles ≥2 
EOT Visit 
FSV23 
Cycle Day 
-28 to -1 
1 
(Baseline) 
8 
(±1 day) 
13 
(within 14 days of 
the decision to 
stop study drug) 
(30 days [±7 days] 
after the last dose 
of study drug) 
Informed Consent 
X 
 
 
 
 
 
Inclusion/Exclusion Criteria 
X 
 
 
 
 
 
Medical and Cancer History 
X 
 
 
 
 
 
Prior Medications/Procedures5 
X 
 
 
 
 
 
Pregnancy Test6 
X 
X 
 
X 
X 
 
Hematology 
X2 
X4 
X 
X4 
X 
 
Serum Chemistry 
X2 
X4 
X 
X4 
X 
 
Coagulation7 
X2 
X4 
 
X4 
X 
 
Urinalysis8 
X2 
X4 
X 
X4 
X 
 
Physical Examination 
X 
Examinations will be driven by clinical findings 
and/or patient complaints 
X 
 
ECOG PS 
X 
X4 
 
X4 
X 
 
Vital Signs and Weight9 
X 
X 
X 
X 
X 
 
Height 
X 
 
 
 
 
 
12-lead ECG10 
X 
X 
 
X 
X 
 
ECHO/MUGA11 
X 
 
 
X 
X 
 
Ophthalmologic Examination12 
X 
 
 
X 
X 
 
Adverse Event Reporting 
Continuous from signing informed consent through FSV or initiation of new anticancer therapy 
Concomitant Medications/Procedures 
Continuous from first dose of study drug through FSV or initiation of new anticancer therapy 
Dosing of Rebastinib13 
 
Continuous from Cycle 1 Day 1 (Baseline) through EOT 
 
Dosing of Carboplatin14 
 
X 
 
X 
 
 
Radiologic Imaging15 
X 
 
 
X 
X16 
 
CA-125 Tumor Marker (Ovarian only)17 
X 
 
 
X 
X 
 
Tumor Marker Data18 
Tumor marker data is collected if being done as part of standard care18 
Rebastinib PK Sampling19 
 
X 
 
X25 
 X 
 
Carboplatin PK Sampling19 
 
X 
 
X25 
X 
 
Archival Tissue Sample20 
X 
 
 
 
 
 
Fresh Tumor Biopsy21 
 
 
 
X 
X (optional) 
 


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Assessments / Procedures1 
Screening 
Cycle 1 
Cycles ≥2 
EOT Visit 
FSV23 
Cycle Day 
-28 to -1 
1 
(Baseline) 
8 
(±1 day) 
13 
(within 14 days of 
the decision to 
stop study drug) 
(30 days [±7 days] 
after the last dose 
of study drug) 
Pharmacogenomics 
 
X 
 
 
 
 
Whole Blood Immunophenotyping Biomarker 
Samples22 
 
X 
X 
X25 
X 
 
PD Plasma Biomarker Samples22 
 
X 
X 
X25 
X 
 
PRO-CTCAE24 
X 
X 
X 
X25 
X 
 
GP5 from FACT-G24 
X 
X 
X 
X25 
X 
 
EORTC QLQ-C3024 
X 
X 
X 
X25 
X 
 
AE = adverse event; BID = twice daily; CA-125 = cancer antigen 125, CNS = central nervous system; CT = computed tomography; ECG = electrocardiogram; 
ECHO = echocardiogram; ECOG PS = Eastern Cooperative Oncology Group Performance Status; EOT = End of Treatment; EORTC QLQ-C30 = European Organisation for 
Research and Treatment of Cancer Quality of Life Questionnaire for Cancer 30-item; ePRO = electronic Patient Reported Outcomes; FACT-G = Functional Assessment of 
Cancer Therapy – General; FSV = Follow-up Safety Visit; MRI = magnetic resonance imaging; MUGA = multigated acquisition; PRO-CTCAE = Patient-Reported Outcomes 
Version of the Common Terminology Criteria for Adverse Events; PD = pharmacodynamic(s); PK = pharmacokinetic(s); PS = performance status; RECIST = Response 
Evaluation Criteria in Solid Tumors. 
1. 
All assessments must be performed pre-dose, unless otherwise specified. Additional unscheduled safety or efficacy assessments may be performed at any time 
as clinically indicated to determine the relevance of specific findings and/or the duration of events. 
2. 
Screening samples for hematology, serum chemistry, coagulation, and urinalysis must be drawn within 14 days prior to the first dose of study drug.  
3. 
As of Cycle 2 and subsequent cycles, Day 1 of the cycle is dependent upon carboplatin dosing. If a patient continues rebastinib as a single agent treatment after 
completing 4 cycles, a cycle will become 21 days and visits should be scheduled and performed in a 21-day interval (+/-2 days). 
4. 
If the screening clinical laboratory testing and ECOG was performed within 3 days before the Cycle 1 Day 1 dose, the testing need not be repeated on Cycle 1 
Day 1. Similarly, if clinical laboratory testing and ECOG was performed within 3 days prior to the start of a new cycle, then the testing need not be repeated on 
Day 1.  
5. 
Any medication or non-drug therapy or procedure taken or performed within 30 days prior to screening and before the first dose of study drug.  
6. 
A serum β-hCG test to rule out pregnancy in women of childbearing potential will be obtained at screening (see Section 6.8.9). In addition, a urine or serum 
pregnancy test will be performed pre-dose on Day 1 of every cycle and at the EOT Visit. Patients must be counseled to inform the Investigator of any 
pregnancy that occurs during study treatment and for 120 days after the last dose of the study drug.  
7. 
Coagulation will be performed at screening, Day 1 of each cycle, EOT visit, and at the time of a tumor biopsy. Patients taking anticoagulants will have 
additional testing performed on Cycle 1 Day 8. 
8. 
If urinalysis testing is abnormal, a microscopic analysis will be performed. 


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9. 
Vital sign measurements will be collected after the patient has been at rest (seated or supine position) for at least 5 minutes. Measurements include sitting 
blood pressure, heart rate, respiratory rate, and temperature. On days when serial PK sampling is performed, vital sign measurements should be obtained prior 
to each PK sampling time point. In addition, weight will be obtained at each study visit.  
10. 
Single 12-lead ECGs will be performed during screening, Day 1 of each cycle, and at the EOT Visit, unless deemed clinically appropriate at other times. All 
12-lead ECGs will be performed with central over-reading after the patient has been at rest (supine or semi-recumbent position) for at least 5 minutes (see 
Section 6.8.5). The rest period begins after the placement of the ECG leads.  
11. 
An ECHO or MUGA scan will be performed during screening, on Day 1 of Cycle 4 and every third cycle thereafter (i.e., Cycles 7, 10, 13, etc.) (within 
14 days), and at the EOT Visit. The same modality (ECHO or MUGA) used during screening should be used for all subsequent assessments (see 
Section 6.8.6). ECHO/MUGAs while on active treatment may be performed up to 3 days prior to the corresponding visit. The ECHO/MUGA is not required at 
the EOT visit if already performed within 14 days of the last dose of all study drug. A MUGA scan should not be performed on the same day as the draw of the 
PK sample. If a MUGA scan is chosen, it must be performed at least 24 hours before the corresponding PK sample. 
12. 
Ophthalmologic examinations will be performed during screening, on Day 1 of Cycle 4 and every third cycle thereafter (i.e., Cycles 7, 10, 13 etc.) (within 
14 days), and at the EOT Visit (+/-7 days), unless deemed clinically appropriate at other times. This examination does not have to be repeated during screening 
if there is documentation of an examination that met protocol criteria within 60 days prior to the first dose of study drug and after the last dose of previous 
anticancer treatment. An examination is not required at the EOT visit if one has been done within 30 days prior to the last dose of all study drug. 
13. 
Starting with Cycle 1 Day 1 (Baseline), rebastinib will be administered orally per assigned dose BID continuously throughout each cycle. Study drug must be 
taken at approximately the same time each day. On days of planned study visits, patients will be informed to take the study drug at the study site after pre-dose 
assessments are completed and prior to the carboplatin infusion.  
14. 
Carboplatin will be administered by intravenous (IV) infusion over approximately 60 minutes on Day 1 of each cycle. It is acceptable to administer the 
carboplatin premedications prior to or after rebastinib dosing, if required. If a scheduled carboplatin dose needs to be delayed, see Section 5.3. If carboplatin is 
not tolerated after completing 4 cycles of study treatment, the patient may continue rebastinib treatment as single agent (see Section 5.3). On Day 1 of Cycles 1 
and 2 when serial PK samples will be collected, patients must take the dose of carboplatin 2 hours (+/-30 minutes) after rebastinib dosing. 
15. 
CT or MRI scans of the chest, abdomen, and pelvis will be performed at screening. Subsequent scans will be performed on Day 1 of Cycles 3, 5, every 3 cycles 
thereafter (i.e., Day 1 of Cycles 8, 11, etc.), and at the EOT Visit. Determination of objective tumor response will be performed by the investigator according to 
the RECIST Version 1.1 or mRECIST (pleural mesothelioma only). Assessments should be completed regardless of dosing delays or additional unscheduled 
imaging assessments. An MRI of the brain will be performed at screening and subsequent imaging time points if CNS metastases are present at screening or 
signs and symptom suggests CNS metastases. Imaging may be performed up to 14 days prior to the corresponding study visit. MRI or CT scans without 
contrast may be used for patients who are allergic to radiographic contrast media. Throughout the study, the same assessment technique should be used (see 
Section 6.10.1).  
16. 
EOT imaging will only be performed on patients if imaging was not performed within the previous 4 weeks. 
17. 
CA-125 will be measured for ovarian cancer patients at screening, and at the same time they undergo radiologic assessment, and at the EOT visit if patients 
have elevated CA-125 at screening (≥2 times of the ULN). Ovarian cancer patients experiencing CA-125 response must have a confirmatory test performed at 
least 28 days after the initial response is documented.  
18. 
Results of tumor marker(s) performed as standard of care for other disease indications will be collected.  


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19. 
Rebastinib and carboplatin PK sampling will be performed according to the schedule in Table 2. An unscheduled PK sample may be taken at the time of the 
onset of a new suspected, treatment-related adverse event if requested by the Sponsor. 
20. 
Archival tumor tissue samples should be collected for all patients enrolled in the study if possible. If an archival tumor tissue sample is unavailable, a fresh 
tumor biopsy is required prior to the first dose of study drug. Fresh biopsies will be performed for patients whose tumor is anatomically accessible and should 
only be collected if a patient qualifies for the study based on all other entry criteria.    
21. 
A fresh tumor biopsy will be collected at Cycle 3 Day 1 (within 14 days). An optional tumor biopsy may be performed at EOT if a patient had responded to 
treatment and then progressed, and has provided consent to undergo EOT tumor biopsy. Fresh biopsies will be performed for patients whose tumor is 
anatomically accessible. Cytology samples, such as fine-needle aspirates, are not acceptable. 
22. 
Whole blood immunophenotyping and PD plasma biomarker samples will be collected pre-dose at Cycle 1 Day 1 (Baseline), Cycle 1 Day 8, Cycle 2 Day 1, 
Cycle 3 Day 1, every 4 cycles thereafter, and at the EOT visit.   
23. 
Patients will be contacted for the FSV 30 (+/-7) days after receiving their last dose of study drug or before the start of new anticancer therapy, whichever 
occurs first. This visit can be performed over the phone.  
24. 
All patients will be asked to complete the EORTC QLQ-C30, the GP5 from FACT-G, as well as questions from the PRO-CTCAE item library using an 
electronic data capture system (ePRO device) (see Sections 6.13.1, 6.13.2, and 6.13.3). Only English-speaking patients will complete the questionnaires.  
Patient entered data will not be modified by the Investigator or site staff, ePRO vendor, CRO, or Sponsor. 
25. 
Once the patient is deemed eligible to proceed with carboplatin dosing, these assessments will be performed. 
 


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Table 2: 
Schedule of Electrocardiograms, Vitals and Pharmacokinetic Sampling  
Study Visit 
Cycle 1, Day 1 (Baseline) 
Cycle 2, Day 1a 
Cycle ≥3, Day 1a & EOT 
Time Point (hours) 
ECGb 
Vitalsb 
Rebastinib 
PKe 
Carboplatin 
PKe 
ECGb 
Vitalsb 
Rebastinib 
PKe 
Carboplatin 
PKe 
ECGb 
Vitalsb 
Rebastinib 
PKe 
Carboplatin 
PKe 
Pre-rebastinib dosec 
X 
X 
X 
  
X 
X 
X 
 
X 
X 
X 
 
Pre-carboplatin infusiond 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
 
 
End of carboplatin infusiond 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
 
 
1 hour after end of carboplatin infusion 
(± 10 min) 
X 
X 
X 
X 
 
 
 
 
 
 
 
 
2 hours after end of carboplatin 
infusion dose (± 10 min) 
X 
X 
X 
X 
X 
X 
X 
X 
 
 
 
 
AE = adverse event; ECG = electrocardiogram; min = minute; PK = pharmacokinetic. 
a. 
These assessments will be performed only if carboplatin is infused. 
b. 
ECG and vital sign assessments will be completed before the collection of the corresponding PK sample. 
c. 
Pre-rebastinib dose assessments should be obtained within 2 hours prior to rebastinib administration.  
d. 
Rebastinib will be dosed 2 hours (+/-30 minutes) prior to carboplatin infusion. A PK sample should be obtained within 10 minutes prior to the start and after 
the end-of-infusion. 
e. 
An unscheduled PK sample may be taken at the time of the onset of a new suspected, treatment-related AE. 
 


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LIST OF ABBREVIATIONS  
Abbreviation 
Definition 
ADL 
Activities of daily living 
AE 
Adverse event 
AESI 
Adverse event of special interest 
ALT 
Alanine transaminase  
AML 
Acute myeloid leukemia 
ANC 
Absolute neutrophil count 
ANG 
Angiopoietin  
ASCO 
American Society of Clinical Oncology 
AST 
Aspartate transaminase  
AUC 
Area under the plasma concentration-time curve  
AUC0-4h 
AUC from 0 to 4 hours 
β-hCG 
Beta-human chorionic gonadotropin 
BID 
Twice daily  
CA 
Cancer antigen 
CA-125 
Cancer antigen-125 
CAP 
College of American Pathologists 
CBR 
Clinical benefit rate 
CI 
Confidence interval 
CL 
Clearance 
Cmax 
Maximum plasma concentration  
Cmax,ss 
Maximum plasma concentration at steady state 
CMC 
Carboxymethylcellulose 
Cmin 
Minimum plasma concentration 
Cmin,ss 
Minimum plasma concentration at steady state 
CML 
Chronic myeloid leukemia  
CNS 
Central nervous system  
CR 
Complete response 
CT 
Computed tomography 
CTC 
Circulating tumor cells  
CTCAE 
Common Terminology Criteria for Adverse Events 
CYP 
Cytochrome P450  
DFS 
Disease-free survival  
DLT 
Dose-limiting toxicity 
DOR 
Duration of response 


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Abbreviation 
Definition 
ECG 
Electrocardiogram 
ECOG PS 
Eastern Cooperative Oncology Group Performance Status 
eCRF 
Electronic case report form  
EORTC QLQ-C30 
European Organisation for Research and Treatment of Cancer Quality 
of Life Questionnaire for Cancer 30-item 
EOT 
End of Treatment 
ePRO 
Electronic patient reported outcomes 
FACIT 
Functional Assessment of Chronic Illness Therapy 
FACT-G 
Functional Assessment of Cancer Therapy – General  
FDA 
Food and Drug Administration 
FIH 
First-in-human 
FLT3 
FMS-related tyrosine kinase 3 ligand 
FSH 
Follicle-stimulating hormone 
FSV 
Follow Up Safety Visit 
GCIG 
Gynecological Cancer Intergroup  
GCP 
Good Clinical Practice 
G-CSF 
Granulocyte-colony stimulating factor 
GI 
Gastrointestinal  
GLP 
Good Laboratory Practice 
GM-CSF 
Granulocyte macrophage-colony stimulating factor 
HER2 
Human epidermal growth factor receptor 2  
hERG 
Human ether-à-go-go related gene  
Hgb 
Hemoglobin 
HPLC/MS 
High performance liquid chromatography/mass spectrometry  
HR 
Hazard ratio  
IB 
Investigator’s Brochure  
ICF 
Informed consent form  
ICH 
International Conference on Harmonization 
IHC 
Immunohistochemical  
IOP 
Intraocular pressure 
IRB 
Institutional Review Board  
IRT 
Interactive response technology 
ISH 
In situ hybridization 
ITD 
Internal tandem duplication 
IV 
Intravenous 
KM 
Kaplan-Meier 


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Abbreviation 
Definition 
LVEF  
Left ventricular ejection fraction  
MBC 
Metastatic breast cancer 
MDR1 
Multidrug resistance protein 1 
MedDRA 
Medical Dictionary for Regulatory Activities 
mITT 
Modified Intent to Treat 
MPM 
Malignant pleural mesothelioma 
mRECIST 
Modified Response Evaluation Criteria in Solid Tumors 
MTD 
Maximum-tolerated dose  
MRI 
Magnetic resonance imaging 
MUGA 
Multigated acquisition  
NCI 
National Cancer Institute 
NOAEL 
No-observed-adverse-effect level  
NSCLC 
Non-small cell lung cancer 
ORR 
Objective response rate  
OS 
Overall survival  
PARP 
Poly (ADP-ribose) polymerase 
PD 
Pharmacodynamics 
PFI 
Platinum-free interval 
PFS 
Progression-free survival  
P-gp 
P-glycoprotein  
Ph+ 
Philadelphia chromosome positive 
PIC 
Powder-in-capsule  
PK 
Pharmacokinetic 
PO 
Orally 
PPI 
Proton pump inhibitors  
PR 
Partial response 
PRO 
Patient reported outcome 
PRO-CTCAE 
Patient Reported Outcomes Version of the Common Terminology 
Criteria for Adverse Events  
PT-INR 
Prothrombin time adjusted for the international normalized ratio 
PTT 
Partial thromboplastin time  
PyMT 
Polyoma middle T antigen 
QD 
Once daily  
QOL 
Quality of life 
QTc 
QT interval corrected 
QTcF 
QT interval corrected by Fridericia’s formula 


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Abbreviation 
Definition 
RECIST 
Response Evaluation Criteria in Solid Tumors 
RP2D 
Recommended Phase 2 dose  
SAE 
Serious adverse event 
SD 
Stable disease 
SOC 
System organ class 
SUSAR 
Serious Unexpected Suspected Adverse Reaction 
T1/2 
Half-life  
T315I 
Threonine 315 to isoleucine  
TdP 
Torsades de Pointes 
TEAE 
Treatment-emergent adverse event 
TEMs 
TIE2-expressing macrophages  
TIE2  
Tunica internal endothelial cell kinase 2 
TKI 
Tyrosine kinase inhibitor 
Tmax 
Time to maximum plasma concentration 
Tmax,ss 
Time to maximum plasma concentration at steady state 
TMEM 
Tumor microenvironment of metastasis 
TNBC 
Triple-negative breast cancer 
TTP 
Time to progression  
ULN 
Upper limit of normal  
US 
United States 
USP 
United States Pharmacopeia 
Vd 
Volume of distribution 
VEGF 
Vascular endothelial growth factor  
WBC 
White blood cell 


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TABLE OF CONTENTS 
1 
INTRODUCTION AND RATIONALE ......................................................................31 
1.1 
Introduction ............................................................................................................31 
1.2 
Clinical Indications ................................................................................................31 
1.2.1 
Triple-Negative Breast Cancer ........................................................................32 
1.2.2 
Ovarian Cancer ................................................................................................32 
1.2.3 
Mesothelioma ...................................................................................................32 
1.3 
Overview of Rebastinib .........................................................................................33 
1.3.1 
Nonclinical Experience ....................................................................................33 
1.3.2 
Clinical Experience ..........................................................................................35 
1.4 
Overview of Carboplatin........................................................................................36 
1.5 
Rationale ................................................................................................................36 
1.5.1 
Study Rationale ................................................................................................36 
1.5.2 
Dose, Regimen, and Treatment Duration Rationale ........................................37 
2 
STUDY OBJECTIVES ................................................................................................41 
2.1 
Dose Escalation ......................................................................................................41 
2.1.1 
Primary Objectives...........................................................................................41 
2.1.2 
Secondary Objectives.......................................................................................41 
2.2 
Dose Expansion .....................................................................................................41 
2.2.1 
Primary Objectives...........................................................................................41 
2.2.2 
Secondary Objectives.......................................................................................41 
2.3 
Dose Escalation and Dose Expansion ....................................................................41 
2.3.1 
Exploratory Objectives ....................................................................................41 
3 
STUDY DESIGN.........................................................................................................42 
3.1 
Overview of Study Design .....................................................................................42 
3.1.1 
Dose Escalation ................................................................................................42 
3.1.2 
Dose Expansion ...............................................................................................43 
3.2 
Number of Patients ................................................................................................43 
3.3 
Duration of Study ...................................................................................................43 
4 
STUDY POPULATION ..............................................................................................44 
4.1 
Inclusion Criteria ...................................................................................................44 
4.2 
Exclusion Criteria ..................................................................................................45 
5 
STUDY DRUG ADMINISTRATION AND MANAGEMENT .................................48 
5.1 
Study Drug Description .........................................................................................48 
5.1.1 
Rebastinib ........................................................................................................48 
5.1.2 
Carboplatin .......................................................................................................48 
5.2 
Study Drug Dose and Administration ....................................................................48 
5.2.1 
Rebastinib ........................................................................................................48 
5.2.2 
Carboplatin .......................................................................................................49 
5.3 
Dose Interruption/Modification and Management of Toxicities ...........................49 
5.4 
Packaging and Labeling .........................................................................................61 
5.5 
Storage Conditions .................................................................................................61 


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5.6 
Study Drug Compliance .........................................................................................61 
5.7 
Study Drug Accountability ....................................................................................61 
5.8 
Disposal, Return, or Retention of Unused Study Drug ..........................................62 
5.9 
Method of Assigning Patients to Treatment ..........................................................62 
5.10 
Blinding..................................................................................................................62 
5.11 
Prior and Concomitant Treatment and Procedures ................................................62 
5.11.1 
Prior Medications and Procedures ...................................................................62 
5.11.2 
Prior Anticancer Medications and Procedures .................................................62 
5.11.3 
Concomitant Medications ................................................................................62 
5.11.4 
Permitted Medication .......................................................................................62 
5.11.5 
Medications to Avoid or Take with Caution ....................................................63 
5.11.6 
Prohibited Medications and Substances ...........................................................63 
5.11.7 
Concomitant Procedures ..................................................................................64 
5.12 
Other Precautions ...................................................................................................64 
6 
STUDY ASSESSMENTS ...........................................................................................65 
6.1 
Screening................................................................................................................65 
6.2 
Treatment Period ....................................................................................................65 
6.3 
Follow Up Safety Visit ..........................................................................................65 
6.4 
Lost to Follow Up ..................................................................................................65 
6.5 
Informed Consent Procedure .................................................................................65 
6.6 
Patient Identification and Registration...................................................................66 
6.7 
Demographics and Medical History ......................................................................66 
6.8 
Safety .....................................................................................................................66 
6.8.1 
Dose-Limiting Toxicities .................................................................................66 
6.8.2 
Physical Examinations and Height and Weight ...............................................67 
6.8.3 
Eastern Cooperative Oncology Group (ECOG) Performance Status ..............67 
6.8.4 
Vital Signs, Weight, and Height ......................................................................68 
6.8.5 
Electrocardiograms ..........................................................................................68 
6.8.6 
Echocardiograms/Multigated Acquisition Scans .............................................68 
6.8.7 
Ophthalmologic Examination ..........................................................................69 
6.8.8 
Clinical Laboratory Tests .................................................................................69 
6.8.9 
Pregnancy Test .................................................................................................70 
6.8.10 
Contraception and Pregnancy Avoidance Measures ........................................70 
6.8.11 
Adverse Events ................................................................................................72 
6.8.12 
Safety Follow Up .............................................................................................72 
6.9 
Pharmacokinetics ...................................................................................................72 
6.9.1 
Sample Collection ............................................................................................72 
6.9.2 
Sample Assessment ..........................................................................................73 
6.10 
Efficacy ..................................................................................................................73 
6.10.1 
Radiologic Imaging ..........................................................................................73 
6.10.2 
Response Assessment Using CA-125 (Ovarian Cancer Patients Only) ..........73 
6.10.3 
Relevant Tumor Markers .................................................................................73 
6.11 
Biomarker Research Studies ..................................................................................73 


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6.11.1 
Sample Collection ............................................................................................73 
6.11.2 
Sample Assessments ........................................................................................74 
6.12 
Pharmacogenomic Measurements .........................................................................74 
6.12.1 
Sample Collection ............................................................................................74 
6.12.2 
Sample Assessment ..........................................................................................74 
6.13 
Patient Reported Outcome (PRO) Measurements .................................................75 
6.13.1 
National Cancer Institute (NCI) Patient-Reported Outcomes Version of the 
Common Terminology Criteria for Adverse Events (PRO-CTCAE) ..............75 
6.13.2 
European Organisation for Research and Treatment of Cancer Quality of 
Life Questionnaire for Cancer 30-item (EORTC QLQ-C30) ..........................75 
6.13.3 
GP5 from Functional Assessment of Cancer Therapy – General (FACT-G) ..75 
7 
ADVERSE EVENT AND SERIOUS ADVERSE EVENT DOCUMENTATION, 
SEVERITY GRADING, AND REPORTING .............................................................77 
7.1 
Adverse Events ......................................................................................................77 
7.2 
Severity Assessment ..............................................................................................77 
7.3 
Causality Assessment.............................................................................................78 
7.4 
Study Drug Action Taken ......................................................................................78 
7.5 
Adverse Event Outcome ........................................................................................79 
7.6 
Treatment Given ....................................................................................................79 
7.7 
Additional Points to Consider for Adverse Events ................................................79 
7.7.1 
Clinically Significant Assessments ..................................................................79 
7.8 
Serious Adverse Events .........................................................................................80 
7.9 
Adverse Events of Special Interest for Study Drug ...............................................80 
7.10 
Adverse Event Reporting Periods ..........................................................................81 
7.11 
Adverse Event and Serious Adverse Event Reporting Requirements ...................81 
7.12 
Abuse, Misuse, Overdose, and Medication Error ..................................................81 
8 
DISCONTINUATION AND REPLACEMENT OF PATIENTS ...............................83 
8.1 
Discontinuation of Treatment ................................................................................83 
8.2 
Follow Up Safety Visit / End of Study ..................................................................83 
8.3 
Replacement of Patients .........................................................................................84 
9 
STATISTICAL CONSIDERATIONS.........................................................................85 
9.1 
Determination of Sample Size ...............................................................................85 
9.2 
Analysis Endpoints ................................................................................................85 
9.2.1 
Safety ...............................................................................................................85 
9.2.2 
Pharmacokinetics .............................................................................................86 
9.2.3 
Efficacy ............................................................................................................86 
9.2.4 
Pharmacogenomics ..........................................................................................86 
9.2.5 
Pharmacodynamics ..........................................................................................87 
9.2.6 
Patient Reported Outcomes..............................................................................87 
9.3 
Populations for Analysis ........................................................................................87 
9.4 
Procedures for Handling Missing, Unused, and Spurious Data.............................87 
9.5 
Interim Analyses ....................................................................................................88 
9.6 
Adjustment for Multiple Comparisons ..................................................................88 


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9.7 
Blinding..................................................................................................................88 
9.8 
Statistical Methods .................................................................................................88 
9.8.1 
General Methods ..............................................................................................88 
9.8.2 
Disposition of Patients .....................................................................................89 
9.8.3 
Demographic and Baseline Characteristics .....................................................89 
9.8.4 
Extent of Exposure ...........................................................................................89 
9.9 
Efficacy Analysis ...................................................................................................89 
9.9.1 
Primary Endpoint .............................................................................................89 
9.10 
Secondary Endpoints .............................................................................................89 
9.10.1 
Progression-free Survival.................................................................................89 
9.10.2 
Time to Tumor Progression .............................................................................90 
9.10.3 
Overall Survival ...............................................................................................90 
9.10.4 
Time to Response .............................................................................................90 
9.10.5 
Clinical Benefit Rate ........................................................................................90 
9.10.6 
Duration of Response .......................................................................................90 
9.11 
Safety Analysis ......................................................................................................90 
9.11.1 
Adverse Events ................................................................................................90 
9.11.2 
Eastern Cooperative Oncology Group Performance Status .............................91 
9.11.3 
Vital Signs ........................................................................................................91 
9.11.4 
Echocardiogram/Multigated Acquisition Scans ..............................................91 
9.11.5 
Ophthalmologic Assessments ..........................................................................91 
9.11.6 
Clinical Laboratory Parameters .......................................................................91 
9.12 
Pharmacokinetic Analysis ......................................................................................91 
9.12.1 
Tumor Markers Analysis .................................................................................91 
9.12.2 
Biomarker and Pharmacodynamic Analysis ....................................................91 
9.13 
Pharmacogenomic Analysis ...................................................................................91 
9.14 
Procedures for Reporting Deviations to Original Statistical Analysis Plan ..........91 
10 
QUALITY CONTROL AND QUALITY ASSURANCE ...........................................92 
10.1 
Study Site Monitoring Visits .................................................................................92 
10.2 
Protocol Compliance ..............................................................................................92 
11 
DATA HANDLING AND RECORD KEEPING .......................................................93 
11.1 
Electronic Case Report Form .................................................................................93 
11.2 
Record Retention ...................................................................................................93 
12 
ETHICS........................................................................................................................94 
12.1 
Ethical Conduct of the Study .................................................................................94 
12.2 
Patient Information and Consent ...........................................................................94 
12.3 
IRB .........................................................................................................................94 
12.4 
Patient Confidentiality ...........................................................................................94 
12.5 
Reporting of Safety Issues or Serious Breaches of the Protocol or International 
Conference on Harmonization Good Clinical Practice ..........................................95 
12.6 
Liability and Insurance ..........................................................................................95 
13 
STUDY TERMINATION ...........................................................................................96 
13.1 
Criteria for Suspension or Premature Termination of the Study ...........................96 


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13.2 
Criteria for Premature Termination or Suspension of Investigational Sites ..........96 
13.3 
Procedures for Premature Termination or Suspension of the Study or the 
Participation of Investigational Site(s)...................................................................96 
14 
PUBLICATION OF STUDY RESULTS ....................................................................97 
15 
REFERENCES ............................................................................................................98 
 
LIST OF TABLES IN THE TEXT 
Table 1: 
Schedule of Assessments .................................................................................17 
Table 2: 
Schedule of Electrocardiograms, Vitals and Pharmacokinetic Sampling ........21 
Table 3: 
Treatment-emergent Adverse Events of Special Interest by Worst CTCAE 
Grade and Rebastinib Cmax and AUC0-4h Subgroup during Cycles 1 and 2 .....39 
Table 4: 
Dose Reduction Steps for Rebastinib ..............................................................51 
Table 5: 
Dose Reduction Steps for Carboplatin .............................................................51 
Table 6: 
Recommended Dose Modification for Hematological Adverse Events ..........52 
Table 7: 
Recommended Dose Modification for Peripheral Sensory Neuropathy ..........53 
Table 8: 
Recommended Dose Modification for Muscular Weakness ...........................54 
Table 9: 
Recommended Dose Modification for Ocular Events (Except 
Asymptomatic Intraocular Pressure Increases) ................................................55 
Table 10: 
Recommended Dose Modification and Management of Asymptomatic 
Intraocular Pressure Increases..........................................................................56 
Table 11: 
Recommended Dose Modification for Cardiovascular Events ........................58 
Table 12: 
Recommended Dose Modification for Gastrointestinal Adverse Events ........59 
Table 13: 
Recommended Dose Modification for Other Grade 3 Non-Hematological 
Adverse Events ................................................................................................60 
Table 14: 
Safety Laboratory Tests ...................................................................................70 
Table 15: 
National Cancer Institute (NCI) Common Terminology Criteria for 
Adverse Events (CTCAE) Version 5.0 Severity Grading Scale ......................77 
Table 16: 
Relationship to Study Drug Criteria ................................................................78 
Table 17: 
Classification for Study Drug Action Taken with Regard to an Adverse 
Event ................................................................................................................78 
Table 18: 
Classifications for Outcome of an Adverse Event ...........................................79 
Table 19: 
Partial or Missing Date Algorithms .................................................................88 
 
LIST OF FIGURES IN THE TEXT 
Figure 1: 
Study Schema...................................................................................................16 
 


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1 
INTRODUCTION AND RATIONALE 
1.1 
Introduction 
The angiopoietin (ANG)/tunica internal endothelial cell kinase 2 (TIE2) kinase signaling 
pathway is a pivotal angiogenic signaling axis in endothelial cells. TIE2 is also expressed on 
a subset of macrophages (i.e., TIE2-expressing macrophages [TEMs]) which not only 
promote cancer growth, cancer cell survival, and motility, but also can limit the efficacy of 
the tumor response to chemotherapy or radiotherapy. In particular, TEMs are known to be 
proangiogenic, prometastatic, and immunosuppressive in the tumor microenvironment 
(2,3,4,5,6,7,8,9,10). Since TIE2 expression is restricted to endothelial cells and highly 
protumoral TEMs, TIE2 is an attractive target for interrupting tumor cell/microenvironment 
interactions (11,12). Regarding its role in tumor angiogenesis, TIE2 signaling is necessary 
for upregulation and function of protumoral TEMs that mediate angiogenesis in breast cancer 
and pancreatic islet tumors syngeneic mouse models, and adaptive or evasive 
revascularization in pancreatic neuroendocrine cancer treated with anti-vascular endothelial 
growth factor (VEGF) therapy (13,14). Proangiogenic TEMs have also been shown to limit 
therapeutic effectiveness of vascular disrupting agents (14,15).  
It has been demonstrated that TEMs mediate invasion and metastasis in the polyoma 
middle T antigen (PyMT) syngeneic breast cancer model (2), and that TIE2 expression 
correlates with poor overall survival (OS) and a high risk of metastasis in breast cancer 
patients (4,16). Perivascular TEMs assemble into special structures with endothelial and 
tumor cells termed tumor microenvironment of metastasis (TMEM) to promote tumor cell 
intravasation into the blood stream, leading to circulating tumor cells (CTCs) and systemic 
dissemination and metastasis (3,5). Moreover, chemotherapy increases the density and 
activity of TMEM sites and promotes distant metastasis in the PyMT and patient-derived 
xenografts (17). Chemotherapy-induced TMEM activity and cancer cell dissemination were 
reversed by administration of the TIE2 inhibitor rebastinib. Analysis of clinical breast cancer 
specimens obtained from neoadjuvant treatment with paclitaxel, doxorubicin, or 
cyclophosphamide shows that TMEM density increased by treatment (15). Taken together, 
inhibition of TMEM function may improve clinical benefits of chemotherapy in the 
neoadjuvant setting or in metastatic disease.  
1.2 
Clinical Indications 
In this study, rebastinib will be evaluated for the treatment of patients with advanced or 
metastatic solid tumors in combination with carboplatin. In the Dose Escalation phase, 
patients with advanced or metastatic solid tumors who have exhausted available, approved 
therapies and for which carboplatin is considered appropriate treatment will be enrolled. In 
the Dose Expansion phase, three indication-specific cohorts in triple-negative breast cancer, 
ovarian cancer, and mesothelioma will be open once the recommended Phase 2 dose (RP2D) 
of rebastinib in combination with carboplatin is established. Although the aforementioned 
role of TEMs in tumor growth and metastasis has been demonstrated in models of breast 
cancer and pancreatic neuroendocrine tumor, the role of TEMs and the effect of rebastinib 
are considered as indication-independent. Diseases to be evaluated in the Dose Expansion 
phase have been selected mainly based on use of carboplatin and treatment options available 
currently. 


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1.2.1 
Triple-Negative Breast Cancer 
Triple-negative breast cancer (TNBC) accounts for approximately 12% to 17% of women 
with breast cancer (18). It lacks expression of estrogen receptor, progesterone receptor, and 
human epidermal growth factor receptor 2 (HER2). It is more common in young black and 
Hispanic women than in other racial groups. It exhibits aggressive clinical behavior, and 
patients with the disease have a relatively poor outcome and cannot be treated with endocrine 
therapy or trastuzumab. Currently, chemotherapy is the primary systemic medical treatment 
for TNBC, although there is no preferred standard form of chemotherapy. 
Carboplatin is rarely used as a single agent in metastatic breast cancer (MBC), but it is used 
in combination with other drugs. Although carboplatin is not approved for breast cancer, it is 
used as an off-label drug in combination with paclitaxel or docetaxel, and trastuzumab in 
HER2-positive MBC. The off-label use dosage is targeted at AUC 6 every 3 weeks in 
combination with paclitaxel and trastuzumab, or AUC 6 every 3 weeks in combination with 
docetaxel and trastuzumab. AUC 6 every 3 – 4 weeks is recommended for recurrent HER2 
negative disease (19). In a randomized phase III trial, carboplatin was equal in efficacy to 
docetaxel in the first-line metastatic setting among unselected patients with metastatic TNBC 
(objective response rate [ORR] as 30-35%) (20). However, carboplatin yielded a superior 
response rate (68%) and progression-free survival (PFS) (6.8 months from 3.1 months 
without BRCA mutations) compared with docetaxel in BRCA mutation-associated TNBC. 
1.2.2 
Ovarian Cancer 
Advanced ovarian cancer is treated with carboplatin-based combination therapies including 
carboplatin/paclitaxel combination. Despite initial therapy, the majority of women with 
advanced-stage ovarian cancer will relapse and require additional treatment. Subsequent 
management of patients is based on the amount of time that has elapsed between the 
completion of platinum-based treatment and the detection of relapse, known as the 
platinum-free interval (PFI). If PFI is 6 months or more, the disease is considered as platinum 
sensitive. Those platinum sensitive patients will receive platinum-based combination therapy 
with or without bevacizumab, an anti-VEGF antibody. Platinum-based combination therapy 
has demonstrated consistent improvements in PFS with ORR from 50 to 60 percent, with 
additional benefit from the addition of bevacizumab. If PFI is less than 6 months 
(platinum-resistant), patients are recommended to be treated with paclitaxel with or without 
bevacizumab. Other agents used in this setting include doxorubicin, gemcitabine, topotecan 
and etoposide. 
Once patients progress on treatment for the recurrent disease, no clear guidance for treatment 
exists. A recent survey has found that platinum-based therapies are still used in the third or 
fourth line settings. Bevacizumab is also given as single agent or in combination with 
chemotherapy in these settings (21). Response to platinum-containing regimens declines 
dramatically after 2 prior lines, even in patients who were initially platinum-sensitive (22). 
In one study of 63 patients who received at least 3 lines of chemotherapy, only 11.9% had a 
clinical response to third line chemotherapy, although 52% had responded to second line 
(23).  
1.2.3 
Mesothelioma 
Malignant mesothelioma is rare with a poor prognosis. It arises from mesothelial surfaces of 
the pleural cavity, peritoneal cavity, tunica vaginalis, or pericardium. Malignant pleural 
mesothelioma (MPM) is the most common type and can be difficult to treat because most 


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patients have advanced disease at presentation. Carboplatin is not approved to treat 
mesothelioma. However, carboplatin is used in combination with pemetrexed as a systemic 
chemotherapy to treat unresectable MPM. Pemetrexed plus cisplatin is the standard treatment 
for patients with unresectable malignant mesothelioma; however, carboplatin is often 
substituted for cisplatin, particularly in the palliative setting, due to its less toxicity. In a 
phase II trial in patients with MPM, treatment with pemetrexed and carboplatin was found to 
be as effective as treatment with pemetrexed and cisplatin (24). An ORR was achieved at 
18.6% (95% CI, 11.6% to 27.5%). Median time to progression was 6.5 months and median 
OS was 12.7 months. In another non-randomized, open-label trial of patients with peritoneal 
mesothelioma, pemetrexed in combination with carboplatin achieved a response rate of 
24.1% (95% CI, 10.3% to 43.5%) compared with 12.5% (95% CI, 3.5% to 29.0%) in the 
pemetrexed only group and 20.0% (95% CI, 7.7% to 38.6%) in the pemetrexed and cisplatin 
group (25). 
1.3 
Overview of Rebastinib 
1.3.1 
Nonclinical Experience 
Please refer to the Investigator’s Brochure (IB) for a more detailed summary of the 
nonclinical experience with rebastinib. 
1.3.1.1 
Pharmacology 
1.3.1.1.1 Primary Pharmacodynamic Studies   
The pharmacological properties of rebastinib were investigated in a series of experiments 
using cell-free enzyme assays, whole cell assays, and animal cancer models. Rebastinib 
potently inhibited recombinant TIE2 protein kinase in vitro. TIE2 activity was also inhibited 
in a series of cellular assays designed to determine the phosphorylation state of the protein. 
Rebastinib blocked capillary tube formation driven by TIE2 in endothelial cell lines, and 
inhibited TEM-dependent tumor cell intravasation through an endothelial monolayer. 
In vivo, rebastinib was evaluated in the PyMT syngeneic breast cancer model in mice. Tumor 
vascularization and metastasis in this model are known to be modulated by TEMs (26). 
Rebastinib treatment led to a significant decrease in the growth rate of the primary breast 
cancers, and to a decrease in the levels of tumoral TIE2 staining by immunohistochemical 
(IHC) analysis. Single-agent rebastinib also led to a significant decrease in the rate of 
occurrence of lung metastases in this model. Furthermore, rebastinib led to a dramatic 
reduction in the function of perivascular TEMs and in levels of CTCs. 
Recently it has been demonstrated that chemotherapy such as paclitaxel induces the structure 
terms TMEM consisting of invasive tumor cells, TEMs, and endothelial cells facilitating 
systemic dissemination of tumor cells. In addition, the combination of rebastinib and 
paclitaxel led to a significant, dose-dependent decrease in the number of lung metastases 
compared with treatment with paclitaxel alone. Rebastinib led to reduction or ablation of 
primary tumor vascular permeability and CTCs in the PyMT spontaneous breast tumor model 
(26). Rebastinib was also evaluated in combination with eribulin, another marketed inhibitor 
of microtubules. Rebastinib in combination with eribulin led to a significant increase in 
survival compared with single agent eribulin therapy in the PyMT implant breast tumor 
model (26). 


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Preliminary work in a syngeneic mesenteric mesothelioma mouse model has shown that the 
combination of rebastinib with carboplatin and pemetrexed leads to a significant increase in 
survival of mice versus treatment with carboplatin and pemetrexed alone. Rebastinib as a 
single agent, decreased primary tumor growth and blocked lung metastasis in this model, as 
well as led to an increase in the adaptive immune response to the tumor. Further study of 
combination of rebastinib with carboplatin in this model is ongoing. 
1.3.1.1.2 Safety Pharmacology 
Rebastinib was evaluated in a series of core battery studies according to International 
Conference on Harmonization (ICH) S7A guidelines that included evaluation of 
central nervous system (CNS) effects and respiratory function in rats, cardiovascular effects 
in dogs, and an in vitro human ether-à-go-go related gene (hERG) assay.  
There were no significant changes in respiratory function in rats up to a maximum dose of 
100 mg/kg (600 mg/m2), in the CNS in rats up to a maximum dose of 100 mg/kg 
(600 mg/m2), and in cardiovascular function in dogs up to a maximum dose of 200 mg/kg 
(4000 mg/m2).  
Cardiac electrophysiological parameters also were not affected in vitro in HEK-293 cells 
expressing the hERG-encoded potassium channel when exposed to concentrations of up to 
10,000 nM. 
1.3.1.2 
Pharmacokinetics and Absorption, Distribution, Metabolism, and Excretion 
Profile 
Preliminary screening studies indicated high plasma protein binding of rebastinib. In 
addition, membrane permeability studies indicated that the parent free base of rebastinib 
readily crosses biological membranes by passive diffusion.  
Rebastinib is a substrate for P-glycoprotein (P-gp) and has the potential to interact with other 
drugs known to be substrates or inhibitors of P-gp (e.g., ketoconazole, verapamil, and 
erythromycin).  
Rebastinib was found to be a potent inhibitor of cytochrome P450 (CYP)2C9 and to a lesser 
degree CYP2C19, indicating a potential for interference with clearance (CL) of known 
substrates of these isoenzymes. Primary routes of metabolism were hydroxylation of the 
t-butyl group and on the quinoline moiety. The carboxylate metabolite derived from further 
oxidation of the t-butyl group was also observed. Secondary metabolites were found in bile 
after oral dosing in bile duct cannulated rats. These metabolites were tentatively identified by 
high performance liquid chromatography/mass spectrometry (HPLC/MS) as the glucuronide 
conjugates of the t-butyl hydroxyl metabolite and the glucuronide ester of the carboxylate 
metabolite. Cytochrome P450 (CYP) reaction profiling indicated that hepatic metabolism of 
rebastinib occurs primarily by CYP3A4 oxidation with minor oxidation by CYP2D6. These 
results indicate that rebastinib exposure could potentially be altered by co-administration of a 
CYP3A4 inhibitor or inducer.  
In vivo studies in rats indicated no significant induction of hepatic metabolism from repeated 
dosing with rebastinib at 10 and 75 mg/kg by oral gavage daily for 5 days.  


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1.3.1.3 
Toxicology 
Exploratory multi-dose studies were conducted with rats and dogs, and definitive 4-week and 
13 week multi-dose studies were conducted in rats and dogs in compliance with 
Good Laboratory Practice (GLP) guidelines. Exploratory and definitive 4- and 13-week 
toxicology studies were conducted using orally administered rebastinib in 0.5% 
carboxymethylcellulose (CMC [vehicle]). 
The repeated dose no-observed-adverse-effect level (NOAEL) was 10 mg/kg/day 
(60 mg/m2/day) in rats and 25 mg/kg/day (500 mg/m2/day) in dogs.  
Deaths were seen with repeated dosing at ≥100 mg/kg (600 mg/m2) in rats. Clinical signs in 
animals at 75 and 125 mg/kg in the 13-week studies primarily included thin appearance, 
decreased activity and changes in fecal color or consistency. Dogs did not show the 
abdominal hardness or distension seen in some rats. These data indicated that dosing of 
rebastinib should be carefully monitored or discontinued if significant weight loss, serious 
gastrointestinal (GI) stasis or distress, or unexpected overall weaknesses are observed. 
Although generally mild in severity and reversible, liver findings in the nonclinical testing 
suggest that liver function should be monitored in rebastinib clinical trials. 
There were no ocular findings in the animal safety studies. Degenerative cardiomyopathy 
was seen at doses of ≥25 mg/kg (150 mg/m2) in rats but not in dogs. Exposures at those doses 
were substantially above the exposures obtained in the first-in-human (FIH) trial of patients 
with chronic myeloid leukemia (CML) and acute myeloid leukemia (AML). Rebastinib also 
did not result in adverse alterations in the ICH-recommended battery of genetic toxicology 
tests. Based on extensive nonclinical safety experience, clinical dosage/exposure 
extrapolations and on previous clinical experience in CML and AML, these data support the 
evaluation of rebastinib in further clinical trials. 
1.3.2 
Clinical Experience 
Please refer to the IB for a more detailed summary of the clinical experience with rebastinib. 
Clinical Study DCC-2036-01-001 (NCT00827138) was a FIH, multicenter Phase 1 study to 
determine the safety, tolerability, and pharmacokinetic (PK) profile of rebastinib in patients 
with Philadelphia chromosome positive (Ph+) leukemia or (FMS-related tyrosine kinase 3 
ligand [FLT3]/internal tandem duplication [ITD]) + AML. Patients with Ph+ CML who 
1) had the threonine 315 to isoleucine (T315I) mutation (BCR-ABL mutant, imatinib, 
dasatinib, nilotinib resistant), or 2) were refractory to or intolerant of more than two tyrosine 
kinase inhibitor [TKI] treatments, or 3) were refractory to or intolerant of 1 TKI and were 
unwilling or unable to receive treatment with other TKIs were enrolled.  
Study DCC-2036-01-003 is an open-label Phase 1b/2 multicenter study in patients with 
advanced or metastatic solid tumors where paclitaxel is considered appropriate treatment. 
Rebastinib is administered in combination with weekly paclitaxel in repeated 28-day cycles. 
The study consists of two parts (Part 1 and Part 2). The primary objectives of Part 1 are to 
evaluate the safety and tolerability of 50 mg and 100 mg of rebastinib twice daily (BID) 
when administered in combination with paclitaxel, and to determine the recommended phase 
2 dose (RP2D) of rebastinib in combination with paclitaxel. The primary objectives of Part 2 
are to evaluate the safety and tolerability of rebastinib at the RP2D in combination with 
paclitaxel, and to evaluate efficacy of rebastinib in combination with paclitaxel in 5 different 
diseases. 


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Study DCC-2036-01-004 is an open label Phase 1b/2 multicenter study in patients with 
advanced or metastatic solid tumors for which carboplatin is considered appropriate 
treatment. Rebastinib will be administered in combination with carboplatin in repeated at 
least 21-day cycles. The study consists of two parts: dose escalation and dose expansion. The 
primary objectives of the dose escalation phase are to establish the maximum tolerated dose 
(MTD) or RP2D of rebastinib and carboplatin in combination, and to evaluate the safety and 
tolerability of rebastinib when administered in combination with carboplatin. The primary 
objectives of the dose expansion phase are to further evaluate the safety and tolerability of 
rebastinib when administered in combination with carboplatin and evaluate the efficacy of 
rebastinib in combination with carboplatin in 3 different diseases. 
Additionally, an Investigator sponsored Phase 1b study (NCT02824575) entitled “Phase Ib 
study of rebastinib plus antitubulin therapy with paclitaxel or eribulin in patients with 
metastatic breast cancer” has tested rebastinib at 50 and 100 mg BID in combination with 
paclitaxel or eribulin in metastatic breast cancer. The study has two phases: dose escalation 
and dose expansion phases (27).  
1.4 
Overview of Carboplatin 
Carboplatin is a cisplatin analogue that also induces DNA adduct formation and interstrand 
cross-linking. Despite a similar mechanism of action to cisplatin, the toxicity profile is 
significantly different, with myelosuppression being the DLT. Thrombocytopenia may be 
more prominent that neutropenia, although all lineages may be suppressed in combination 
therapy. Emesis and chemical hepatitis may occur in greater than 25% of patients, and renal 
insufficiency and neuropathy in less than 10%. Carboplatin is dosed in most cases according 
to glomerular function, targeting a specific area under the curve. Acute hypersensitivity 
reactions occur in <5% of patients but can be dramatic, including flushing, rashes, itchy 
palms, nausea, dyspnea back pain, hypotension, and tachycardia (28). In US, carboplatin is 
approved to treat ovarian cancer. However, carboplatin is used to treat other cancer types 
such as non-small cell lung cancer (NSCLC), breast cancer, gastric/esophageal cancer, 
mesothelioma and sarcoma, mostly in combination with other anti-cancer agents.  
1.5 
Rationale 
1.5.1 
Study Rationale 
The ANG/TIE2 kinase signaling pathway is a pivotal angiogenic signaling axis in endothelial 
cells. TIE2 is also expressed on a subset of macrophages (i.e., TEMs). TEMs not only 
promote cancer growth, cancer cell survival, and motility, but can limit the efficacy of the 
tumor response to chemotherapy or radiotherapy. In particular, TEMs are known to be 
proangiogenic, prometastatic, and immunosuppressive in the tumor microenvironment. 
Rebastinib is a potent, small molecule inhibitor of TIE2. Although rebastinib has much 
higher activity against TIE2 compared to BCR-ABL and FLT3 (approximately 100 fold more 
selective), the FIH Phase 1 Study DCC-2036-01-001 (NCT00827138) of rebastinib was 
conducted by enrolling patients with Ph+ CML (52 patients) and FLT3-ITD positive AML 
(5 patients). Exposure of rebastinib was dose-proportional. A retrospective analysis of levels 
of ANG2, a ligand for TIE2, in serum samples obtained from patients in study 
DCC-2036-01-001 pre- and post-treatment with rebastinib (100 - 200 mg BID) revealed 
more than 2-fold increases in circulating ANG2 levels, indicating TIE2 receptor engagement 
in these patients. An MTD was established as 150 mg BID.  


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A rationale of testing rebastinib in combination with chemotherapy was first established with 
paclitaxel. Paclitaxel has demonstrated clinical activity both as single-agent therapy and in 
combination with other chemotherapeutic agents in a variety of cancer types, including breast 
cancer, NSCLC, ovarian cancer, and pancreatic cancer. In addition to arresting tumor growth, 
paclitaxel also disrupts the vascular endothelium and produces a hypoxic tumor environment. 
Hypoxia induces recruitment of bone marrow derived TEMs to reestablish the tumor 
vasculature, leading to tumor regrowth. Rebastinib in combination with paclitaxel 
significantly reduced tumor growth in a murine model of breast cancer. This anti-tumor 
activity correlated with a reduction in TEMs in the tumor and that in the formation of lung 
metastases. As described above, perivascular TEMs assemble into special structures with 
endothelial and tumor cells termed TMEM to promote tumor cell intravasation into the blood 
stream, leading to CTCs and systemic dissemination and metastasis. Moreover, 
chemotherapy including paclitaxel increases the density and activity of TMEM sites and 
promotes distant metastasis in the PyMT and patient-derived xenografts. 
Chemotherapy-induced TMEM activity and cancer cell dissemination were reversed by 
administration of the TIE2 inhibitor rebastinib. Analysis of clinical breast cancer specimens 
obtained from neo-adjuvant treatment with paclitaxel, doxorubicin, or cyclophosphamide 
shows that TMEM density increased by treatment suggesting that the mechanism of TMEM 
induction is treatment-agnostic and carboplatin should exert the same effect as what has been 
demonstrated by paclitaxel and the other agents (14). 
Based on these findings, the Investigator sponsored Phase 1b study entitled “Phase Ib study 
of rebastinib plus antitubulin therapy with paclitaxel or eribulin in patients with metastatic 
breast cancer” was initiated. As described above, rebastinib at 50 and 100 mg BID in 
combination with weekly dosed paclitaxel was tolerated and showed a hint of clinical activity 
in breast cancer. Thus, although this Investigator sponsored study will continue evaluating 
the combination with eribulin, the Sponsor is conducting the Phase 1b/2 study 
(DCC-2036-01-003) to evaluate the safety, efficacy, PK and pharmacodynamics (PD) of 
rebastinib in combination with weekly-dosed paclitaxel. In the DCC-2036-01-003 study, 
breast and gynecological cancers will be explored in the Phase 2 portion of the study. 
This study will evaluate the effect of rebastinib in combination of carboplatin, a 
chemotherapy agent commonly used in a variety of cancer including breast cancer, ovarian 
cancer, NSCLC, and mesothelioma. The purpose of this study is to determine an MTD or 
RP2D of rebastinib and carboplatin in combination and demonstrate preliminary efficacy in 
3 cancer types, TNBC, ovarian cancer, and mesothelioma where carboplatin is used as 
standard of care. 
1.5.2 
Dose, Regimen, and Treatment Duration Rationale 
In the FIH study of rebastinib, an MTD of 150 mg BID was established as described above. 
This study was performed in CML/AML patients, investigating rebastinib as a BCR-ABL 
and FLT3 inhibitor. In the current study, rebastinib will be investigated as a TIE2 receptor 
tyrosine kinase inhibitor with a picomolar potency for blocking not only TIE2 enzymatic 
activity, but also functional cellular activity in TEMs and endothelial cells. Due to the 
~100-fold higher potency against TIE2 compared to BCR-ABL or FLT3, it is expected that 
the optimum biologic dose is below the MTD established from that CML/AML clinical 
study. In addition, ANG2 levels were examined in CML and AML patients before and after 
treatment with 100-200 mg BID rebastinib. On Day 22 after starting rebastinib treatment, 
increases in ANG2 were observed in 19/20 patients and a >2-fold increase in ANG2 plasma 


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levels was observed in 14/20 (70%) patients suggesting pharmacological inhibition of TIE2 
by rebastinib at 100 mg BID. 
Based on safety analysis in the same study, muscular weakness/musculoskeletal disorders, 
peripheral neuropathy, cardiomyopathy/cardiac disorders, and visual abnormalities/eye 
disorders were considered to be of particular clinical interest and were further analyzed as 
Adverse Events of Special Interest (AESIs). Exposure-response relationship analysis for 
rebastinib and selected AESIs listed below were conducted (Table 3). The values for either 
Cmax or AUC from 0 to 4 hours (AUC0-4h) of evaluable patients were divided into low, 
middle, and high thirds. Patients were analyzed based on the highest toxicity grade for an AE 
category within each Cmax or AUC0-4h value. Incidences of AESIs with higher grades were 
increased in patients exposed to the high range of Cmax or AUC0-4h. PK analysis shows that 
geometric mean Cmax and AUC0-4h values of 100 and 150 mg BID are in the low and medium 
range, respectively. 
In addition, in the Investigator sponsored Phase 1b study, the combination with paclitaxel 
was found tolerable in 3 patients at the 50 mg BID dose level, and in 3 patients at the 100 mg 
BID dose level (as described in Section 1.3.2). 
Taken together, although 150 mg BID was determined as the MTD in the study with CML 
and AML patients, 100 mg BID should be deemed as an appropriate dose for the 
combination with carboplatin given the positive PD effect on ANG2 levels and the exposure-
dependent safety profile with a significantly reduced frequency of Grade 3 or 4 AEs at the 
dose level. However, 50 mg BID is chosen as the starting dose of rebastinib in Dose 
Escalation for determining the RP2D to provide additional safety as this study is the first 
study of the combination between rebastinib and carboplatin. The dose of rebastinib will be 
escalated to 100 mg BID, if tolerated, up to 150 mg BID. In addition, 75 mg BID may be 
tested, if 100 mg BID is not tolerated and 50 mg BID is deemed safe.  
Carboplatin is infused at AUC5 or 6 in most indications. Thus, the starting dose of 
carboplatin will be at AUC5. 
 


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Table 3: 
Treatment-emergent Adverse Events of Special Interest by Worst CTCAE Grade and Rebastinib Cmax and 
AUC0-4h Subgroup during Cycles 1 and 2 
  
  
Low 
Medium 
High 
SOC 
Worst CTC 
Grade of 
Adverse 
Event 
Cmax 
Subgroup 
(N=17) 
n (%) 
AUC0-4h 
Subgroup 
(N=15) 
n (%) 
Cmax 
Subgroup 
(N=15) 
n (%) 
AUC0-4h 
Subgroup 
(N=14) 
n (%) 
Cmax 
Subgroup 
(N=18) 
n (%) 
AUC0-4h 
Subgroup 
(N=18) 
n (%) 
Cardiac disorders 
Grade 1 
1 (6%) 
1 (7%) 
1 (7%) 
1 (7%) 
2 (11%) 
2 (11%) 
Grade 2 
0 
0 
0 
0 
0 
0 
Grade 3 
1 (6%) 
1 (7%) 
0 
0 
1 (6%) 
1 (6%) 
Grade 4 
0 
0 
0 
0 
0 
0 
Grade 5 
0 
0 
0 
0 
0 
0 
Total 
2 (12%) 
2 (13%) 
1 (7%) 
1 (7%) 
3 (17%) 
3 (17%) 
Eye Disorders 
Grade 1 
3 (18%) 
3 (20%) 
5 (33%) 
4 (29%) 
2 (11%) 
2 (11%) 
Grade 2 
0 
0 
0 
0 
2 (11%) 
1 (6%) 
Grade 3 
0 
0 
0 
0 
1 (6%) 
1 (6%) 
Grade 4 
0 
0 
0 
0 
0 
0 
Grade 5 
0 
0 
0 
0 
0 
0 
Total 
3 (18%) 
3 (20%) 
5 (33%) 
4 (29%) 
5 (28%) 
4 (22%) 
Musculo-skeletal 
Disorders 
Grade 1 
4 (24%) 
3 (20%) 
1 (7%) 
1 (7%) 
3 (17%) 
4 (22%) 
Grade 2 
0 
0 
2 (13%) 
2 (14%) 
2 (11%) 
2 (11%) 
Grade 3 
1 (6%) 
0 
4 (27%) 
4 (29%) 
5 (28%) 
3 (17%) 
Grade 4 
0 
0 
0 
0 
0 
0 
Grade 5 
0 
0 
0 
0 
0 
0 
Total 
5 (29%) 
3 (20%) 
7 (47%) 
7 (50%) 
10 (56%) 
9 (50%) 


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Low 
Medium 
High 
SOC 
Worst CTC 
Grade of 
Adverse 
Event 
Cmax 
Subgroup 
(N=17) 
n (%) 
AUC0-4h 
Subgroup 
(N=15) 
n (%) 
Cmax 
Subgroup 
(N=15) 
n (%) 
AUC0-4h 
Subgroup 
(N=14) 
n (%) 
Cmax 
Subgroup 
(N=18) 
n (%) 
AUC0-4h 
Subgroup 
(N=18) 
n (%) 
Nervous 
system 
Disorders 
Grade 1 
2 (12%) 
2 (13%) 
5 (33%) 
5 (36%) 
6 (33%) 
6 (33%) 
Grade 2 
1 (6%) 
1 (7%) 
1 (7%) 
0 
3 (17%) 
3 (17%) 
Grade 3 
0 
0 
1 (7%) 
1 (7%) 
1 (6%) 
1 (6%) 
Grade 4 
0 
0 
0 
0 
0 
0 
Grade 5 
0 
0 
0 
0 
0 
0 
Total 
3 (18%) 
3 (20%) 
7 (47%) 
6 (43%) 
10 (56%) 
10 (56%) 
Any of the 
above 
Grade 1 
6 (35%) 
5 (33%) 
6 (40%) 
6 (43%) 
6 (33%) 
7 (39%) 
Grade 2 
0 
0 
3 (20%) 
2 (14%) 
2 (11%) 
3 (17%) 
Grade 3 
2 (12%) 
1 (7%) 
4 (27%) 
4 (29%) 
7 (39%) 
5 (28%) 
Grade 4 
0 
0 
0 
0 
0 
0 
Grade 5 
0 
0 
0 
0 
0 
0 
Total 
8 (47%) 
6 (40%) 
13 (87%) 
12 (86%) 
15 (83%) 
15 (83%) 
AUC0-4h = area under the plasma concentration-time curve from zero to 4 hours; Cmax = mean peak plasma concentration; CTCAE = Common Terminology 
Criteria for Adverse Events; SOC = system organ class; CTCAE Version 3.0 
 


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2 
STUDY OBJECTIVES 
2.1 
Dose Escalation 
2.1.1 
Primary Objectives  
• To establish the MTD or RP2D of rebastinib and carboplatin in combination 
• To evaluate the safety and tolerability of rebastinib when administered in combination 
with carboplatin 
2.1.2 
Secondary Objectives  
• To assess the PK of rebastinib and carboplatin when administered in combination 
• To assess the preliminary efficacy of rebastinib administered in combination with 
carboplatin 
2.2 
Dose Expansion 
2.2.1 
Primary Objectives  
• To evaluate the ORR as the primary efficacy measure of rebastinib in combination with 
carboplatin  
• To evaluate the safety and tolerability of rebastinib when administered in combination 
with carboplatin 
2.2.2 
Secondary Objectives  
• To assess the PK of rebastinib and carboplatin when administered in combination 
• To evaluate efficacy measures, such as PFS, clinical benefit rate (CBR), response 
duration, time to response, time to progression (TTP), and OS of rebastinib in 
combination with carboplatin 
2.3 
Dose Escalation and Dose Expansion 
2.3.1 
Exploratory Objectives  
• Assess the quality of life (QOL) impact of rebastinib administered in combination with 
carboplatin using patient reported outcome (PRO) measures  
• To evaluate changes in select blood and plasma biomarkers when rebastinib is 
administered in combination with carboplatin 
• To evaluate changes in tumor tissue microenvironment (e.g., changes in the composition 
of infiltrating mononuclear cells) when rebastinib is administered in combination with 
carboplatin 
• To assess polymorphisms in genes encoding drug metabolic enzymes and/or transporters 
involved in metabolism and disposition of rebastinib in combination with carboplatin 


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3 
STUDY DESIGN 
3.1 
Overview of Study Design 
This is an open label Phase 1b/2 multicenter study in patients with advanced or metastatic 
solid tumors who have exhausted available, approved therapies and for which carboplatin is 
considered appropriate treatment. AEs will be assessed, and laboratory values, vital sign 
measurements, and electrocardiograms (ECGs) will be obtained to evaluate the safety and 
tolerability of rebastinib when administered in combination with carboplatin. AEs will be 
graded according to the National Cancer Institute (NCI) Common Terminology Criteria for 
Adverse Events (CTCAE) Version 5.0. Tumor response will be assessed according to 
Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 or modified Response 
Evaluation Criteria in Solid Tumors (mRECIST; pleural mesothelioma only) (29). Biomarker 
and PK samples will be collected at prespecified time points. The patient’s experience is 
captured using a selection of PROs (mainly the European Organisation for Research and 
Treatment of Cancer Quality of Life Questionnaire for Cancer 30-item [EORTC QLQ-C30] 
and the NCI Patient-Reported Outcomes Version of the Common Terminology Criteria for 
Adverse Events [PRO-CTCAE] items) and analyzed in accordance with the 
recommendations of the developers of the tools. Rebastinib will be administered PO BID and 
carboplatin will be dosed on Day 1 of each cycle. Each cycle is 21 days, however, initiation 
of subsequent cycles after Cycle 1 is dependent upon carboplatin dosing. Carboplatin dosing 
may be delayed due to treatment-related AEs up to 14 days without dose reduction of 
carboplatin. If carboplatin dosing is delayed more than 14 days but less than 21 days, the 
dose will be reduced by one dose level. If the dose delay is 21 days or more, then the patient 
will be discontinued from the study unless the Investigator and the Sponsor agree that the 
patient is getting a clinical benefit from study treatment. However, if a patient completes 
4 cycles of study drug combination treatment (80% of planned rebastinib doses per cycle) 
and can no longer tolerate carboplatin, the patient may continue rebastinib as single agent 
treatment.  
The study consists of two parts: Dose Escalation and Dose Expansion.  
3.1.1 
Dose Escalation 
In the Dose Escalation phase, doses of rebastinib and carboplatin will be escalated using 
modified 3 + 3 dose escalation rules starting with rebastinib at 50 mg BID in combination 
with AUC5 of carboplatin. The next cohort is rebastinib at 100 mg BID with AUC5 of 
carboplatin. The third planned cohort will be rebastinib at 100 mg BID with AUC6 of 
carboplatin. Based on the safety and tolerability, other combinations of doses such as 
rebastinib at 50 mg BID with AUC6 of carboplatin may be evaluated if, at least, rebastinib at 
50 mg BID with AUC5 of carboplatin is deemed safe. In addition, the rebastinib dose may be 
increased to 150 mg BID with AUC5 and/or AUC6 of carboplatin if 100 mg BID of 
rebastinib with AUC5 and AUC6 of carboplatin is tolerated. Alternatively, 75 mg BID of 
rebastinib may be evaluated if 100 mg BID is not tolerated and 50 mg BID is deemed safe. 
Dose escalation will proceed to the next dose level if no DLT is observed in a minimum of 
3 patients completing 1 cycle. If a DLT is observed in only 1 of 3 patients, an additional 
3 patients will be enrolled up to a total of at least 6 patients at this dose level. Dose escalation 
will only proceed if no more than 1 patient in the cohort of at least 6 patients has experienced 
a DLT during the first cycle of treatment in the dose escalation phase. If a DLT is observed 
in 2 or more patients in a cohort of 3 to 6 patients, dose escalation will stop. The 
determination of the MTD or an RP2D will be based on the safety and tolerability of at least 


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6 patients at a dose level of the combination. As necessary for evaluation of safety and 
tolerability, up to 12 patients may be enrolled per cohort. Enrollment of additional patients to 
further explore safety and tolerability may take place simultaneously while the cohort at the 
next dose level is enrolling patients for dose escalation. A comparison of the PK and 
pharmacodynamic (PD) data, as well as preliminary efficacy data obtained in the Dose 
Escalation phase, may be used to refine the RP2D for each Dose Expansion Cohort. The 
MTD is defined as the highest dose level of rebastinib and carboplatin at which no more than 
1 of 6 patients experiences a DLT during the first cycle. The RP2D will be a dose level of 
rebastinib and carboplatin deemed safe and tolerable on the basis of the totality of safety, 
tolerability, PK, and preliminary efficacy data. The RP2D will not exceed the MTD. 
3.1.2 
Dose Expansion 
Upon determination of the MTD or an RP2D, the Dose Expansion phase will be initiated to 
enroll approximately 99 patients in three indication-specific cohorts. A Simon’s two-stage 
design will be applied to the Dose Expansion phase to further evaluate the safety, tolerability, 
and preliminary efficacy of rebastinib in combination with carboplatin in triple negative 
breast cancer (Cohort 1), ovarian cancer (Cohort 2), and mesothelioma (Cohort 3). An RP2D 
used for each cohort will be chosen based on agreement between the Investigators and 
Sponsor. Each cohort in the Dose Expansion phase will initially enroll up to 18 patients in 
the first stage. The decision to enroll patients beyond the first stage will be based on response 
assessments obtained after the first post-dose response assessment of the last patient enrolled 
in the first stage of a cohort. If >4 responses (defined as partial response [PR] or complete 
response [CR] as best response) are seen in a cohort, additional patients will be enrolled for a 
total of up to 33 patients. If ≤4 responses are seen in a cohort, the cohort will be terminated. 
If >4 responses are seen prior to the last evaluable patient in the first stage, expanding the 
cohort may be triggered earlier. Patients who meet criteria defined in Section 8.3 will be 
replaced and not be included in the responder analysis. There will be an enrollment pause 
between the first and second stage for evaluation of response. 
3.2 
Number of Patients 
A total of approximately 117 patients (approximately 18 patients in the Dose Escalation 
phase and approximately 99 patients in the Dose Expansion phase) may be enrolled in this 
study. The study will be conducted at approximately 4 centers in the US during the Dose 
Escalation phase and up to 18 centers in the US during the Dose Expansion phase. 
3.3 
Duration of Study 
Patients will receive study treatment until they develop progressive disease, experience 
unacceptable toxicity, or withdraw consent. 
Patients will be eligible to receive study treatment as long as the Investigator and the Sponsor 
agree that the patient is showing clinical benefit and for as long as rebastinib is being 
developed to support the indication, and continuation of treatment does not conflict with the 
Sponsor’s right to terminate the study. The study will end following the last patient's last 
visit. 


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4 
STUDY POPULATION 
4.1 
Inclusion Criteria 
Patients must meet all of the following criteria to be eligible to enroll in the study: 
1. Male or female patients ≥18 years of age at the time of informed consent. 
2. Dose Escalation 
i. 
Histologically confirmed diagnosis of a locally advanced or metastatic 
solid tumor for which carboplatin is considered appropriate treatment. 
ii. 
Patients who have progressed despite standard therapies, or for whom 
conventional therapy is not considered effective or tolerable, as judged 
appropriate by the Investigator. 
3. Dose Expansion, All Cohorts 
A. Cohort 1: Triple-negative Breast Cancer 
i. 
Histologically confirmed metastatic triple-negative breast cancer based on 
the American Society of Clinical Oncology (ASCO)/College of American 
Pathologists (CAP) guidelines (1). 
ii. 
Received at least one prior line but no more than three prior lines of 
systemic chemotherapy in the metastatic setting. 
B.  Cohort 2: Ovarian Cancer 
i. 
Histologically confirmed, recurrent epithelial ovarian, peritoneal or 
fallopian tube carcinoma. Note: patients with low grade serous, mucinous 
or clear cell histology will be excluded. 
ii. 
Achieved a PR or CR following treatment with at least one of the prior 
platinum-based regimens. 
iii. 
Recurrent platinum-sensitive disease, defined as disease progression ≥6 
months after completing a minimum of 4 cycles in the most recent 
previous platinum-containing regimen. 
iv. 
Received no more than five prior lines of systemic chemotherapy. 
a. Neoadjuvant and/or adjuvant is considered one regimen. 
b. Maintenance therapy, including poly (ADP-ribose) polymerase 
(PARP) inhibitors is considered part of the preceding regimen. 
c. Hormonal therapy is not considered a prior systemic 
chemotherapy.  
v. 
Must have received prior treatment with a PARP inhibitor if patients have 
a BRCA1 or 2 germline or somatic mutation(s). Patients who have refused 
therapy with a PARP inhibitor may be considered for enrollment, 
following consultation with the Sponsor. 
C.  Cohort 3: Mesothelioma 
i. 
Histologically confirmed pleural or peritoneal malignant mesothelioma 
not eligible for curative surgery. Note: patients with pericardial, and tunica 
vaginalis testis mesotheliomas will be excluded.  


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ii. 
Received at least one prior systemic chemotherapy.  
4. At least one measurable lesion according to RECIST Version 1.1 or mRECIST 
for pleural mesothelioma. 
5. ECOG PS of ≤2 at screening. 
6. Able to provide an archival tumor tissue sample; if an archival tumor tissue 
sample is unavailable, a fresh tumor biopsy is required prior to the first dose of 
study drug, only if tumor biopsy is safe and accessible for biopsy as judged by the 
Investigator. 
7. Adequate organ function and bone marrow reserve as indicated by the following 
laboratory assessments performed within 14 days prior to the first dose of study 
drug:  
i. 
Bone marrow function: absolute neutrophil count (ANC) ≥1500/µL; 
hemoglobin ≥9 g/dL; platelet count ≥100,000/µL. 
ii. 
Hepatic function: total bilirubin ≤1.5× the upper limit of normal (ULN) or 
<3× ULN for Gilbert’s syndrome; aspartate transaminase 
(AST)/alanine transaminase (ALT) ≤2.5× ULN (≤5× ULN in the presence 
of hepatic metastases). 
iii. 
Renal function: creatinine CL ≥50 mL/min based either on urine collection 
or Cockcroft Gault estimation. 
iv. 
Coagulation profile: prothrombin time adjusted for the 
international normalized ratio (PT-INR) and partial thromboplastin time 
(PTT) ≤1.5× ULN. Patients on a stable, maintenance regimen of 
anticoagulant therapy for at least 30 days prior to study drug 
administration may have PT-INR measurements >1.5× ULN if, in the 
opinion of the Investigator, the patient is suitable for the study. An 
adequate rationale must be provided to the Sponsor prior to enrollment. 
8. If a female of childbearing potential, must have a negative serum beta-human 
chorionic gonadotropin (β-hCG) pregnancy test at screening, and agree to use two 
methods of contraception, with one of them being highly effective prior to the 
first dose of study drug and for at least 120 days following the last dose of study 
drug as outlined in Section 6.8.10. 
9. If male, must agree to practice effective barrier contraception, and refrain from 
sperm donation prior to the first dose of study drug through 120 days following 
the last dose of study drug as outlined in Section 6.8.10. 
10. Patient must provide signed consent to participate in the study and be willing to 
comply with study-specific procedures. 
4.2 
Exclusion Criteria 
Patients meeting any of the following criteria will be excluded from the study: 
1. Received prior anticancer or other investigational therapy within 28 days or 5× the 
half-life (whichever is shorter) prior to the first dose of study drug. See Section 
5.11.6 for further details.   


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2. Not recovered from all toxicities from prior therapy to Grade ≤1 (or baseline) 
within 1 week prior to first dose of study drug (excluding alopecia and ≤Grade 3 
clinically asymptomatic alkaline phosphatase). 
3. Patients with a prior or concurrent malignancy whose natural history or treatment 
have the potential to interfere with the safety or efficacy assessment of this clinical 
trial. 
4. Known active CNS metastases defined as:  
i. 
Unstable (i.e., evidence of progression by magnetic resonance imaging 
[MRI]) within 4 weeks prior to the first dose of study drug. 
ii. 
Neurologic symptoms within 2 weeks prior to the first dose of study drug 
and required use of enzyme-inducing antiepileptic drugs. 
iii. 
Patients who require steroids must be on a stable dose for 2 weeks prior to 
the first dose of study drug. 
5. Use of systemic corticosteroids within 7 days prior to the first dose of study 
treatment or an existing condition that requires the concomitant use during the 
course of the study, unless the dose is no more than the equivalent of prednisone 
15 mg/day. Inhaled, intranasal, intraocular, topical, and intraarticular injections are 
allowed. NOTE: premedication with steroids, in accordance with institutional 
practice (including dexamethasone), is permitted prior to carboplatin dosing. 
6. Known retinal neovascularization, macular edema or macular degeneration. 
7. History or presence of clinically relevant cardiovascular abnormalities such as 
uncontrolled hypertension, history of class III or IV congestive heart failure 
according to New York Heart Association classification, unstable angina or poorly 
controlled arrhythmia as determined by the Investigator, or myocardial infarction 
within 6 months prior to the first dose of study drug. 
8. QT interval corrected for heart rate at screening using Fridericia’s formula (QTcF) 
>450 ms in males or >470 ms in females or history of QT interval corrected for 
heart rate (QTc) prolongation. 
9. Left ventricular ejection fraction (LVEF) <50% at screening. 
10. Arterial thrombotic or embolic events such as cerebrovascular accident (including 
ischemic attacks) or hemoptysis (more than mild) within 6 months prior to the first 
dose of study drug. 
11. Symptomatic venous thrombotic event (e.g., deep vein thrombosis) within the 
3 months prior to the first dose of study drug. Following a symptomatic venous 
thrombotic event of ≥3 months prior to the first dose of study drug, must be on a 
stable dose of anticoagulation therapy if clinically indicated.  
12. Active infection ≥Grade 3 requiring IV anti-infective treatment within 7 days prior 
to the first dose of study drug.  
13. Known human immunodeficiency virus or hepatitis C infection only if the patient 
is required to take medications that are excluded per protocol. 
14. Active hepatitis B or active hepatitis C infection.  


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15. Use of proton pump inhibitors (PPI) within 4 days prior to the first dose of study 
drug or an existing condition that requires the concomitant use of a proton pump 
inhibitor during the course of the study.  
16. If female, the patient is pregnant or lactating. 
17. Major surgery ≤4 weeks prior to dosing; all surgical wounds must be healed and 
free of infection or dehiscence. 
18. A manifestation of malabsorption due to prior gastrointestinal surgery, disease or 
other illness which could affect oral absorption as judged by Investigator and 
Sponsor. 
19. Known allergy or hypersensitivity to any component of rebastinib or any of its 
excipients. 
20. Any other clinically significant comorbidities, such as uncontrolled pulmonary 
disease, active infection, or any other condition which, in the judgment of the 
Investigator, could compromise compliance with the protocol, interfere with the 
interpretation of study results, or predispose the patient to safety risks. 
21. Peripheral neuropathy of any etiology >Grade 1. 


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5 
STUDY DRUG ADMINISTRATION AND MANAGEMENT 
Study drug will refer to rebastinib and carboplatin. In cases where there is a distinction in 
how they are to be administered, details for each will be specified. 
5.1 
Study Drug Description 
Study drug will be labeled in accordance to applicable local and national regulations. It will 
be dispensed via the Interactive Response Technology (IRT).   
5.1.1 
Rebastinib 
Rebastinib will be provided by the Sponsor as tablets for oral administration containing 
25 mg and 75 mg of active rebastinib. The tablets also contain microcrystalline cellulose, 
lactose, polyethylene glycol 3350, poloxamer 407, crospovidone, colloidal silicon dioxide, 
PRUV®, and butylated hydroxytoluene. 
5.1.2 
Carboplatin 
Carboplatin will be provided by the Sponsor as a sterile, pyrogen-free, aqueous solution in its 
original form and packaging. Each mL contains 10 mg carboplatin and Water for Injection, 
USP. 
5.2 
Study Drug Dose and Administration 
5.2.1 
Rebastinib 
Rebastinib may be dispensed only under the supervision of the Investigator or an authorized 
designee and only for administration to study patients. Rebastinib will be dosed BID 
continuously throughout each cycle. In the Dose Escalation phase, rebastinib will be 
escalated from 50 mg to 100 mg in combination with carboplatin. In addition, doses of 
150 mg and 75 mg may also be explored. For patients that are unable to tolerate carboplatin 
after completion of at least 4 cycles of study drug combination treatment (80% of planned 
rebastinib doses per cycle), rebastinib may be administered as a single agent. 
The Investigator or designee must instruct the patient to take rebastinib as per protocol. 
• Patients should be instructed to take their assigned dose at the same time each day, 
approximately 12 hours apart. 
o If a patient forgets to take a dose at the scheduled time, the patient may take 
the scheduled dose if taken within 6 hours of the scheduled time that was 
missed. If more than 6 hours have passed after the scheduled time, then that 
missed dose must be recorded and the patient should continue treatment with 
the next scheduled dose. 
• Patients should take their study drug dose with a 6-ounce glass of water. 
• All doses of rebastinib should be taken at least 1 hour before or at least 2 hours after a 
meal.  
• Patients must be instructed to swallow the tablets whole. Tablets must not be crushed, 
chewed, or dissolved in liquid or food.  


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• On days of scheduled visits, the morning dose of study drug must be administered at the 
site after pre-dose assessments have been completed. The date, amount taken and time of 
study drug administration must be recorded in the patient’s dosing diary. 
• A dosing diary will be provided by Sponsor for the patient to record the date, amount 
taken and time of each rebastinib administration.  
• On days of carboplatin administration, patients must take the dose of rebastinib prior to 
infusion of carboplatin. On Day 1 of Cycles 1 and 2 when serial PK samples will be 
collected, patients must take the dose of rebastinib 2 hours (+/-30 minutes) prior to 
infusion of carboplatin. Premedication for carboplatin may be administered prior to or 
after the rebastinib dose, if required. The same administration order should be maintained 
throughout the study. 
• If vomiting occurs immediately after taking a dose, that dose must not be “made up,” and 
the patient may be offered prophylactic anti-emetics prior to their next dose. For 
information on overdose, refer to Section 7.12. 
• Patients must be instructed to avoid medications that increase gastric pH (e.g., H2 
receptor antagonists and antacids) within 2 hours before or after administration of 
rebastinib. H2 receptor antagonists administered during the required premedication for 
each carboplatin infusion are allowed. 
5.2.2 
Carboplatin 
Carboplatin will be administered by IV infusion over approximately 60 minutes in 
accordance with institutional practices including premedication, if required, on Day 1 of each 
cycle and at least 21 days apart. A carboplatin dose will be calculated using the Calvert 
formula. The maximum carboplatin dose should not exceed AUC (mg x min/mL) x 
150 mL/min (GFR + 25) as the maximum dose is based on a GFR estimate that is capped at 
125 mL/min. In the Dose Escalation phase, carboplatin will be dose-escalated from AUC5 to 
AUC6 as described. In the Dose Expansion phase, the MTD or an RP2D of carboplatin in 
combination with rebastinib will be administered. A different dose level not exceeding the 
MTD may be chosen for each cohort. Rebastinib will be administered prior to IV infusion of 
carboplatin.  
To initiate carboplatin infusion, the following is needed:  
• ANC ≥1,000/µL 
• Platelets ≥75,000/µL 
5.3 
Dose Interruption/Modification and Management of Toxicities 
Study drugs may be interrupted or modified (i.e., dose reduced) at the discretion of the 
Investigator at any time due to AE, to accommodate palliative treatment, or for other reasons 
after consultation with the Sponsor. Dose reduction steps of rebastinib and carboplatin will 
be completed according to Table 4 and Table 5, respectively. Recommended dose 
interruptions and modifications as well as management of toxicities are identified in Table 6, 
Table 7, Table 8, Table 9, Table 10, Table 11, Table 12, and Table 13. A Grade 1 or Grade 2 
AE will not require dose interruption or medication unless specified in these tables. 
The causal relationship of each AE should be assessed in relation to rebastinib and to 
carboplatin separately so that dose modifications can be made accordingly.  


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If a dose is interrupted, a patient should be monitored at least weekly prior to resumption of 
rebastinib or carboplatin.  
Upon resumption following a dose interruption of rebastinib, the Investigator must continue 
with the patient’s visit schedule calculated from Day 1 of the cycle that the dose interruption 
took place.  
Day 1 of each cycle (≥Cycle 2) can be delayed up to 14 days due to treatment-related AEs 
without dose reduction. If the dose delay is more than 14 days but less than 21 days, the dose 
of carboplatin will be reduced by one dose level. If carboplatin cannot be administered for 
21 days or more, then the patient will be discontinued unless the Investigator and the Sponsor 
agree that the patient is obtaining clinical benefit from study treatment. If carboplatin is not 
tolerated and a patient is determined to be obtaining clinical benefit from study treatment, 
rebastinib may be administered as a single agent after a patient completes 4 cycles of study 
treatment, provided that the patient received more than 80% of planned rebastinib doses per 
cycle. 
If an AE(s) that meet the criteria of a DLT(s) is experienced in ≥Cycle 2 in the dose 
escalation or in any cycle in the dose expansion, a dose of rebastinib, carboplatin, or both 
will be reduced by one dose level based on the causality relationship after recovery of the 
AE(s) to Grade <2 or a patient’s baseline. 
Permanent discontinuation of the study drugs should be considered for any severe or 
life-threatening event. Upon experiencing any of following conditions, a patient should 
discontinue study drug treatment permanently except when the Investigator determines that 
the patient is obtaining clinical benefit and has discussed this with the Sponsor: 
• Any Grade 4 AE excluding hematological AEs defined in Table 6. 
• Requirement for more than 2 dose reductions from the assigned dose at enrollment to 
manage AEs. 
• ALT or AST elevation >3X ULN and total bilirubin elevation >2X ULN if absent of 
initial findings of cholestasis such as alkaline phosphatase to <2X ULN. 
• Rebastinib: treatment interruption for >14 days due to treatment-related AEs. 
• Carboplatin, unless patients elect to receive rebastinib as a single agent after completion 
of Cycle 4: 
o Treatment interruption for 21 days or more due to treatment-related AEs. 
o Dose reduction from AUC4 
• Any DLT experienced in Cycle 1. 
Sites must follow-up with patients at least weekly during drug interruptions. 


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Table 4: 
Dose Reduction Steps for Rebastinib 
Starting Dose of Rebastinib 
1st Dose Reduction 
2nd Dose Reduction 
50 mg BID 
25 mg BID 
Discontinue 
75 mg BID 
50 mg BID 
25 mg BID 
100 mg BID 
75 mg BID 
50 mg BID 
150 mg BID 
100 mg BID 
75 mg BID 
Sites must follow-up with patients at least weekly during drug interruptions. 
 
Table 5: 
Dose Reduction Steps for Carboplatin 
Starting Dose of Carboplatin 
1st Dose Reduction 
2nd Dose Reduction 
AUC5  
AUC4 
Discontinue 
AUC6 
AUC5 
AUC4 
Sites must follow-up with patients at least weekly during drug interruptions. 
 
 
 


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Table 6: 
Recommended Dose Modification for Hematological Adverse Events 
Adverse Event 
Grade 
Occurrence 
Rebastinib 
Carboplatin 
Based on the causal relationship, dose interruptions/modification will apply to rebastinib only, carboplatin only, or both study drugs. 
Neutrophil 
Count 
Decrease 
≥3 
Any 
Continue the same dose unless the AE is deemed 
related to rebastinib. If related: 
• Hold rebastinib until ANC ≥1,000 /µL. 
• Restart at the same dose. 
Hold carboplatin. 
• Until ANC ≥1,000 /µL. 
• Restart at the same dose. 
• If Grade 4 neutropenia does not resolve within 
7 days, reduce one dose level. 
Platelet Count 
Decrease 
≥2 
Any 
Continue the same dose unless the AE is deemed 
related to rebastinib. If related: 
• Hold rebastinib until Platelets ≥75,000 /µL. 
• Restart at the same dose. 
Hold carboplatin. 
• Until Platelets ≥75,000 /µL. 
• Restart at the same dose. 
• If Grade 3 thrombocytopenia does not resolve 
within 7 days, reduce one dose level. 
• If AE is Grade 4, reduce one dose level. 
Anemia 
≥3 
Any 
Continue the same dose unless the AE is deemed 
related to rebastinib only. If related: 
• Hold rebastinib until Hgb ≥9.0 g/dL. 
• Restart at the same dose. 
Hold carboplatin until Hgb ≥9.0 g/dL. 
• If resolved in ≤7 days, maintain the same dose 
• If resolved in >7 days, reduce one dose level. 
• If AE is Grade 4, reduce one dose level. 
Febrile 
Neutropenia 
3 
1st and 2nd 
Hold rebastinib until ANC ≥1,500 /µL. 
• 
Restart the same dose unless AE is 
worsening. 
• 
If worsening, reduce one dose level. 
Hold carboplatin until ANC ≥1,500 /µL. 
• 
Reduce one dose level with G-CSF or GM-CSF.  
3rd 
• 
The same as above. 
• 
Permanently discontinue carboplatin treatment. 
Hgb = hemoglobin; G-CSF = granulocyte-colony stimulating factor; GM-CSF = granulocyte macrophage-colony stimulating factor. 
Sites must follow-up with patients at least weekly during drug interruptions. 


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Table 7: 
Recommended Dose Modification for Peripheral Sensory Neuropathy  
Adverse Event 
Grade 
Occurrence 
Rebastinib 
Carboplatin 
Based on the causal relationship, dose interruptions/modification will apply to rebastinib only, carboplatin only, or both study drugs. 
Peripheral 
Sensory 
Neuropathy 
2 
Any 
Continue the same dose unless AE is 
worsening. 
• 
If worsening, reduce one dose level. 
Hold carboplatin until AE resolves to Grade <2. 
• 
Resume at the same dose level. 
• 
If AE is worsening with each infusion, even if it 
remains Grade 1, reduce one more dose level.  
3 
Any 
Hold rebastinib until AE resolves to Grade <2. 
• Reduce one dose level. 
• Dose re-escalation by one dose level if 
deemed safe. 
Hold carboplatin until AE resolves to Grade <2. 
• 
Reduce one dose level. 
• 
If AE is worsening with each infusion, even if it 
remains Grade 1, reduce one more dose level. 
Sites must follow-up with patients at least weekly during drug interruptions. 
 
 


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Table 8: 
Recommended Dose Modification for Muscular Weakness  
Adverse Event 
Grade 
Occurrence 
Rebastinib 
Carboplatin 
Muscular 
Weakness 
2 
1st 
Hold rebastinib until toxicity resolves to Grade <2: 
• If AE persists at Grade 2 >5 days after dose 
interruption: 
• 
Refer patient to a neurologist for evaluation 
and management. 
• If AE resolves in ≤14 days, reduce dose level. 
• If AE does not resolve in >14 days, consider 
permanent discontinuation. 
No dose interruption is required. 
2nd 
Hold rebastinib until toxicity resolves to Grade <2: 
• Refer patient to a neurologist for evaluation and 
management. 
• If AE resolves in ≤14 days, reduce one dose level. 
• If AE does not resolve in >14 days, consider 
permanent discontinuation. 
No dose interruption is required. 
3rd 
Permanently discontinue study treatment. 
3 
1st 
Hold rebastinib until toxicity resolves to Grade <2: 
• Refer patient to a neurologist for evaluation and 
management. 
• If AE resolves in ≤14 days, reduce one dose level. 
• If AE does not resolve in >14 days, consider 
permanent discontinuation. 
No dose interruption is required. 
2nd 
Permanently discontinue study treatment. 
Sites must follow-up with patients at least weekly during drug interruptions. 


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Table 9: 
Recommended Dose Modification for Ocular Events (Except Asymptomatic Intraocular Pressure Increases) 
Adverse Event 
Grade 
Occurrence 
Rebastinib 
Carboplatin 
Based on the causal relationship, dose interruptions/modification will apply to rebastinib only, carboplatin only, or both study drugs. 
Ocular AEs and Visual 
AEsa 
2 
1st 
Implement close monitoring and hold rebastinib 
until AE resolves to Grade <2. 
• Reduce one dose level. 
Continue the same dose. 
• If AE is worsening with each infusion, reduce 
one dose level. 
2nd 
Hold rebastinib until AE resolves to Grade <2. 
• Reduce one dose level. 
• Perform ophthalmological assessment as 
clinically indicated.  
Continue the same dose. 
• If AE is worsening with each infusion, reduce 
one dose level. 
3rd 
A patient will be discontinued from the study. 
3 
1st 
Hold rebastinib until AE resolves to Grade <2 
• Reduce one dose level. 
• Perform ophthalmological assessment as 
clinically indicated. 
Hold carboplatin until AE resolves to Grade <2 
• Resume at the same dose. 
• If AE is worsening with each infusion, reduce 
one dose level. 
2nd 
A patient will be discontinued from the study. 
Retinal Vein Occlusion 
or Macular Edema 
Any 
1st  
A patient will be discontinued from the study. 
a. 
Includes optic nerve disorder, papilledema, vision blurred, visual field defect, visual impairment. Retinal vein occlusion and macular edema are described separately. 
Sites must follow-up with patients at least weekly during drug interruptions. 


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Table 10: 
Recommended Dose Modification and Management of Asymptomatic Intraocular Pressure Increases 
Condition 
Management 
Elevated intraocular pressure (IOP) and IOP of both 
eyes still ≤21 mmHg  
• Continue the same dose of rebastinib and carboplatin. 
• If the increase is >5 mmHg from baseline, monitor IOP as needed. 
IOP of either of eyes >21 mmHg but ≤30 mmHg  
• Continue the same dose of rebastinib and carboplatin. 
• Consult with an ophthalmologist: 
o 
May initiate treatment of elevated IOP with a medication(s): starting with a 
topical prostaglandin and then adding a medication(s) such as a beta blocker, 
alpha adrenergic agonist, and carbonic anhydrase inhibitor as appropriate to 
reduce and maintain IOP of both eyes ≤21 mmHg. 
o 
Perform monitoring of IOP as necessary to adjust medications (e.g., 
weekly). 
o 
If IOP cannot be reduced to ≤21 mmHg with 3 topical medications, 
 
Hold rebastinib until IOP of both eyes is reduced to ≤21 mmHg. 
• 
If does not recover within 14 days, then consider 
permanent discontinuation. 
 
Resume rebastinib by one dose level below. 
 
Carboplatin can be administered at the same dose per discretion of 
the Investigator. 
o After the first dose reduction, if IOP cannot be maintained at ≤21 mmHg 
with 3 topical medications, 
 
Hold rebastinib until IOP of both eyes is reduced to ≤21 mmHg. 
• 
If does not recover within 14 days, then consider 
permanent discontinuation.  
 
Resume rebastinib by one dose level below. 
 
Carboplatin can be administered at the same dose per discretion of 
the Investigator. 
o After the second dose reduction, if IOP cannot be reduced to ≤21 mmHg, 
then a patient will be discontinued from the study. 


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Condition 
Management 
IOP of either of eyes >30 mmHg  
• Hold rebastinib until IOP of both eyes is reduced to ≤21 mmHg. 
o 
Consult with an ophthalmologist. 
o 
Carboplatin can continue being administered at the same dose per discretion 
of the Investigator. 
o 
Resume rebastinib at one dose level lower. 
 
Consider prescribing a medication(s) as described above to prevent  
IOP (>21 mmHg). 
 
Perform monitoring of IOP (e.g., weekly) and adjust medications as 
necessary. 
o 
If IOP cannot be maintained at ≤21 mmHg with 3 topical medications, 
 
Hold rebastinib until IOP of both eyes is reduced to ≤21 mmHg. 
 
Reduce rebastinib by one dose level. 
o 
If dose reduction of rebastinib with medications cannot lower IOP ≤21 
mmHg, then a patient will be discontinued from the study. 
Sites must follow-up with patients at least weekly during drug interruptions. 


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Table 11: 
Recommended Dose Modification for Cardiovascular Events 
Adverse Event 
Grade 
Occurrence 
Rebastinib 
Carboplatin 
Based on the causal relationship, dose interruptions/modification will apply to rebastinib only, carboplatin only, or both study drugs. 
Cardiac 
Disordersa 
1 
Any 
Continue the same dose. 
Continue the same dose. 
Perform cardiovascular assessment and monitoring. 
2 
1st 
Continue the same dose unless AE is worsening. 
• If worsening, reduce one dose level. 
Hold carboplatin until AE resolves to Grade <2. 
• Reduce one dose level. 
• If AE is worsening with each infusion, even if 
it remains Grade 1, reduce one more dose 
level.  
Perform cardiovascular assessment and monitoring. 
2nd 
Hold rebastinib until AE resolves to Grade <2. 
• Reduce one dose level. 
Hold carboplatin until AE resolves to Grade <2. 
• Reduce one dose level. 
• If AE is worsening with each infusion, even if 
it remains Grade 1, hold carboplatin. 
Perform cardiovascular assessment and monitoring. 
3rd 
A patient will be discontinued from the study. 
3 
1st 
A patient will be discontinued from the study. 
Hypertension 
2 and systolic 
BP ≥150 or 
diastolic BP ≥95 
Any 
Hold rebastinib. 
• If BP is <150/95 mmHg, restart the same 
dose. 
Continue the same dose. 
3 
Any 
Hold rebastinib and initiate anti-hypertensive 
therapy. 
• If BP is <150/95 mmHg, restart the same 
dose.  
Hold carboplatin and initiate anti-hypertensive 
therapy. 
• If BP is <150/95 mmHg, restart the same 
dose. 
A patient will be discontinued from the study if hypertension is not controlled. 
a. 
Includes cardiac disorders system organ class. 
Sites must follow-up with patients at least weekly during drug interruptions.  


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Table 12: 
Recommended Dose Modification for Gastrointestinal Adverse Events 
Adverse Event 
Grade 
Occurrence 
Rebastinib 
Carboplatin 
Based on the causal relationship, dose interruptions/modification will apply to rebastinib only, carboplatin only, or both study drugs. 
Nauseaa, b 
Vomitinga, b 
3 
1st and 2nd  
Continue the same dose unless AE is 
worsening. 
• 
If worsening, hold rebastinib until AE 
resolves to Grade <2. 
Hold carboplatin until Grade 1 
• 
Reduce one dose level. 
• 
If AE is worsening with each infusion, even if 
it remains Grade 1, reduce one more dose level. 
• 
Dose re-escalation by one dose level at time if 
deemed safe. 
3rd and 
subsequent 
occurrence 
Hold rebastinib until AE resolves to Grade <2. 
• 
Reduce one dose level. 
• 
Dose re-escalation by one dose level at 
time if deemed safe. 
Permanently discontinue carboplatin treatment. 
Diarrheaa, c 
Mucositisa  
2 
Any 
Continue the same dose unless AE is 
worsening. 
• 
If worsening, reduce one dose level. 
Hold carboplatin until Grade <2 
• 
Resume carboplatin at the same dose. 
3 
1st and 2nd  
Continue the same dose unless AE is 
worsening. 
• 
If worsening, reducing one dose level. 
Hold carboplatin until Grade <2 
• 
Reduce one dose level. 
• 
If AE is worsening with each infusion, even if 
it remains Grade 1, reduce one more dose level. 
• 
Dose re-escalation by one dose level at time if 
deemed safe. 
3rd and 
subsequent 
occurrence 
Hold rebastinib until AE resolves to Grade <2. 
• 
Reduce dose by one dose level. 
• 
Dose re-escalation by one dose level at 
time if deemed safe. 
Permanently discontinue carboplatin treatment. 
a. 
Excludes Nausea, Vomiting, Diarrhea and Mucositis resolved to ≤Grade 1 or baseline within 1 week after optimal treatment.  
b. 
Prophylactic antiemetics should be used at the discretion of the Investigator. 
c. 
Anti-diarrheal agents are encouraged.  
Sites must follow-up with patients at least weekly during drug interruptions.  


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Table 13: 
Recommended Dose Modification for Other Grade 3 Non-Hematological Adverse Events 
Adverse Event 
Grade 
Occurrence 
Rebastinib 
Carboplatin 
Other Non-Hematologic 
3 
Any 
Based on the causal relationship, dose interruption/modification will apply to rebastinib only, 
carboplatin only or both study drugs. 
Hold rebastinib until AE resolves to Grade <2. 
• Reduce dose by one dose level. 
• Dose re-escalation by one dose level at a 
time if deemed safe.  
Hold carboplatin until the AE resolves to Grade <2. 
• 
Reduce on dose level dose. 
• 
If AE is worsening with each infusion, even if 
it remains Grade 1, reduce one more dose 
level. 
• 
Dose re-escalation by one dose level at a time 
if deemed safe. 
Sites must follow-up with patients at least weekly during drug interruptions. 


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5.4 
Packaging and Labeling 
Rebastinib will be supplied by the Sponsor as formulated drug in tablets for oral 
administration containing 25 mg and 75 mg of active rebastinib. The 75 mg tablets will be 
supplied in 30-count blister packs. The 25 mg tablets will be supplied in 28-count blister 
packs. Study drug labeling will be in accordance with applicable local and national 
regulations. 
Carboplatin will be supplied by the Sponsor. Carboplatin injection is a premixed aqueous 
solution of 10 mg/mL carboplatin (30). Carboplatin aqueous solution can be further diluted to 
concentrations as low as 0.5 mg/mL with 5% Dextrose in Water (D5W) or 0.9% Sodium 
Chloride Injection, USP. When prepared as directed, carboplatin aqueous solutions are stable 
for 8 hours at room temperature (25°C). Since no antibacterial preservative is contained in 
the formulation, it is recommended that carboplatin aqueous solutions be discarded 8 hours 
after dilution. 
Study drug will be provided and replenished via the IRT. Study drug dispensation 
instructions will be provided in the Pharmacy Manual.  
5.5 
Storage Conditions 
Instructions regarding the storage and handling will be on the label of the study drug as well 
as in the Pharmacy Manual.  
Rebastinib should be stored at controlled room temperature: 20°-25°C (68°-77°F). (Refer to 
United States Pharmacopeia [USP] Controlled Room Temperature.) The original packaging 
should be retained during storage to protect from light and moisture.  
Unopened vials of Carboplatin Injection are stable for the life indicated on the package when 
stored at 25°C (77°F) excursions permitted to 15°-30°C (59°-86°F) (See USP Controlled 
Room Temperature) and protected from light. 
5.6 
Study Drug Compliance 
To ensure study drug compliance, the Investigator or designee must supervise all study drug 
dosing that occurs at the site. At each visit, site personnel must review that the patient is 
compliant with at-home study drug dosing and remind the patient of study drug dosing 
requirements. Compliance must also be assessed by ongoing study drug count. 
If a patient demonstrates continued noncompliance of at-home study drug dosing despite 
educational efforts, the Investigator must contact the Sponsor to discuss discontinuation of 
the patient from the study.  
5.7 
Study Drug Accountability 
Accountability for the study drug at the study site is the responsibility of the Investigator. 
The Investigator must ensure that the study drug is used only in accordance with this 
protocol. Where allowed, the Investigator may choose to assign drug accountability 
responsibilities to a pharmacist or other appropriate individual. Drug accountability records 
indicating the study drug’s delivery date to the site, study drug inventory at the site, study 
drug dispensed to each patient, study drug returned by each patient, and study drug returned 
to the Sponsor or study drug destruction on site must be maintained by the clinical site. 
Accountability records must include dates, quantities, bottle numbers, and patient numbers. 
The study monitor must review drug accountability at the site on an ongoing basis during 


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monitoring visits. If any dispensing errors or discrepancies are discovered, the Sponsor must 
be notified immediately. 
Patients must be instructed to return all unused, partially used, and used study drug blister 
packs to the site at each visit. The Study Monitor must verify study drug records and 
inventory throughout the study. 
5.8 
Disposal, Return, or Retention of Unused Study Drug 
Patients must be instructed to return all used, partially used, and full rebastinib blister packs. 
The site staff or pharmacy personnel (as appropriate) must retain all rebastinib materials 
returned by the patients and carboplatin materials until returned to Sponsor/designee or 
destroyed by the study site. If the study drug will be destroyed at the study site, the 
Investigator or designee, must ensure that the materials are destroyed in compliance with 
applicable environmental regulations, institutional policy, and any special instructions 
provided by the Sponsor. Destruction must be adequately documented. 
5.9 
Method of Assigning Patients to Treatment 
In the Dose Escalation phase, patients will be assigned to the dose of rebastinib according to 
the cohort in which they are enrolled. In the Dose Expansion phase, patients will be assigned 
to an RP2D of rebastinib and enrolled into the appropriate indication-specific cohort.  
5.10 Blinding 
This is an open-label study.  
5.11 Prior and Concomitant Treatment and Procedures 
5.11.1 Prior Medications and Procedures 
Information regarding any medication including vitamin supplements, over-the-counter 
medications and oral herbal preparations or non-drug therapy or procedure taken or 
performed within 30 days prior to screening and before the first dose of study drug must be 
documented in the patient’s source documents and the electronic case report form (eCRF). 
5.11.2 Prior Anticancer Medications and Procedures 
Any prior anticancer medication or procedure must be documented in the patient’s source 
documents and the eCRF. 
5.11.3 Concomitant Medications  
All medications, including vitamin supplements, over-the-counter medications, and oral 
herbal preparations; non-drug therapies taken on or after the first day of study drug dose 
through and including Follow Up Safety Visit (FSV) or initiation of new anticancer therapy 
after the last dose of study drug must be documented in the patient’s source documents and 
the eCRF. In addition, any new medications taken after the last dose of study drug through 
FSV or initiation of new anticancer therapy after the last dose must be documented in the 
patient's source documents and the eCRF. 
5.11.4 Permitted Medication 
Medications that are required to manage AEs and control cancer symptoms are allowed 
based on standard clinical practice, unless specifically excluded. 


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Supportive care agents are permitted as needed per ASCO guidelines (1) or local institutional 
practices. However, supportive care agents, such as blood products, antiemetics, and pain 
medications are not allowed in Cycle 1 of the dose escalation as prophylaxis for management 
of toxicities. Premedication for carboplatin infusion is allowed in accordance with 
institutional practices.  
5.11.5 Medications to Avoid or Take with Caution 
The following medications must be avoided or taken with caution following discussion with 
the Sponsor: 
• Strong inhibitors or inducers of CYP3A4. 
o Non-Medication CYP inhibitors, including the following herbal supplements 
and foods, must be avoided or taken with caution: 
 Herbal supplements and foods that are strong inhibitors of CYP3A4 
including, but not limited to, grapefruit and grapefruit juice, Seville 
orange juice, or other products containing Seville oranges. 
 Strong inducers of CYP3A4 including, but not limited to, St. John’s 
wort 
 Patients taking any of the above listed supplements and foods must be 
closely monitored for any potential interactions with rebastinib. 
• Medications that are known substrates, inhibitors, or inducers of P-gp/multidrug 
resistance protein 1 (MDR1). 
• Medications that increase gastric pH (e.g., H2 receptor antagonists and antacids), with the 
exception of PPIs, may be taken provided they are not administered within 2 hours before 
or after administration of rebastinib. H2 receptor antagonists administered during the 
required premedication for each carboplatin infusion are allowed. 
• Warfarin and low molecular weight heparin. 
• Medications that are CYP2C19 and CYP2C9 substrates with a narrow therapeutic index 
(e.g., phenytoin, S-mephenytoin) should be used with caution. 
• Medications which are associated with conditional or possible risk of QT prolongation. 
Patients taking any of the above listed medications must be closely monitored for any 
potential drug-drug interactions. 
5.11.6 Prohibited Medications and Substances 
The following medications must be excluded during the study:  
• Anticancer therapies, including investigational therapy, concurrent radiation therapy 
(with the exception of radiotherapy for palliative radiation due to pre-existing bone 
metastases), or any other investigational therapy is not permitted during the study. 
o Palliative radiation during study treatment must be discussed with the Sponsor 
prior to implementation. 


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o Hormonal therapy including octreotide or bone targeted therapy such as 
bisphosphonates or a receptor activator of nuclear factor-kappa B (RANK) 
ligand inhibitor is not considered as anticancer therapy. 
• PPIs: PPIs must be discontinued within 4 days prior to the first dose of study drug. 
• Receipt of systemic corticosteroids or requirement chronically on study drugs, unless the 
dose is no more than the equivalent of prednisone 15 mg/day. 
• Dexamethasone may be used as pre-treatment for hypersensitivity prophylaxis 
prior to carboplatin administration. 
• Inhaled, intranasal, intraocular, topical, and intraarticular injections are allowed. 
• Medications that are known to prolong the QTc. Please refer to CredibleMeds website 
(https://www.crediblemeds.org/index.php/?cID=328), category “Known Risk of TdP 
[Torsades de Pointes]” for a list of medications that are known to prolong the QT interval 
and are clearly associated with a known risk of TdP. 
o Antiemetics, known to prolong the QTc interval such as ondansetron are 
permitted only as premedication for carboplatin infusion in accordance with 
institutional practice. 
5.11.7 Concomitant Procedures 
All procedures performed on or after the first dose of study drug through and including FSV 
or initiation of new anticancer therapy after the last dose of study drug must be documented 
in the patient’s source documents and the eCRF. 
Investigational procedures of any kind are excluded during the study. Surgical resection or 
palliative radiotherapy during study treatment must be discussed with the Sponsor prior to 
implementation. If the Investigator and Sponsor agree it is in the best interest of the patient, 
surgical resection or palliative radiotherapy may be performed; however, the patient must be 
censored in the PFS analysis from the time surgery or radiotherapy was performed. 
5.12 Other Precautions 
Formal photosensitivity studies have not been performed, however a potential for 
photoirritation/phototoxicity exists based absorption properties of rebastinib in the ultra 
violet visible range (above 290 nm). In order to mitigate the potential risk of 
photoirritation/phototoxicity, patients must be instructed to avoid strong sunlight, sunlamps, 
and other sources of ultraviolet radiation for the duration of the study. Prophylactic skin care 
recommendations for all patients on study drug include sunscreen with sun protection factor 
≥30, hypoallergenic moisturizing creams or ointments for dry skin, and gentle skincare with 
fragrance-free soaps and detergents. 


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6 
STUDY ASSESSMENTS 
The study specific assessments are detailed in this section and the Schedule of Assessments 
is outlined in Table 1 (Schedule of Assessments). Additional unscheduled safety or efficacy 
assessments may be performed at any time as clinically indicated to determine the relevance 
of specific findings and/or the duration of events. 
6.1 
Screening 
Screening must occur within 28 days prior to the first dose of study drug unless otherwise 
noted to confirm that patients meet the selection criteria for the study. The assessments to be 
conducted at screening are provided in Table 1 (Schedule of Assessments).  
6.2 
Treatment Period 
Patients will be registered in the study after confirmation of all eligibility criteria. The first 
dose of study drug must be administered in the clinic on Cycle 1 Day 1. Treatment period is 
defined as the time of first dose through the End of Treatment (EOT) visit. Study visits 
during the treatment period will occur as shown in Table 1 (Schedule of Assessments). All 
visits must occur within the windows specified. 
Patients who discontinue study drug must return for an EOT Visit within 14 days of the 
decision to stop study drug. 
6.3 
Follow Up Safety Visit 
Patients will be contacted for the FSV 30 (+/-7) days after receiving their last dose of study 
drug or before the start of new anticancer therapy, whichever occurs first. The assessments to 
be conducted at this visit are provided in Table 1 (Schedule of Assessments). This visit can 
be performed over the phone.  
6.4 
Lost to Follow Up 
A patient will be considered lost to follow up if both of the following occur: 
• Patient misses two consecutive study visits and is subsequently unable to be contacted by 
phone call (after three documented attempts by phone within 2 weeks following the 
second missed visit). 
• Patient does not respond within 2 weeks to a registered letter sent after the 
three attempted phone contacts. 
6.5 
Informed Consent Procedure 
Each patient must sign and date a study-specific informed consent form (ICF) before any 
study specific procedures can be performed. The ICF will comply with all applicable 
regulations governing the protection of patients. An ICF, approved by the Sponsor and the 
site's Institutional Review Board (IRB) must be used. The Investigator or designee must 
document the consenting process, including the date when the ICF was signed in the patient’s 
source document. 


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6.6 
Patient Identification and Registration 
A unique patient identification number (patient number) will be assigned to each patient once 
informed consent is obtained. If a patient is rescreened, the patient retains the original patient 
number.  
Patients who are candidates for enrollment into the study will be evaluated for eligibility by 
the Investigator to ensure that the inclusion and exclusion criteria (Section 4.1 and 
Section 4.2) have been satisfied and that the patient is eligible for participation in this clinical 
study. 
6.7 
Demographics and Medical History 
Demographic information must be collected at screening. 
Cancer history and prior treatment (including reason for discontinuation), must be obtained 
during screening. Cancer history will include: 
• Known histologic diagnosis 
• All prior cancer treatment regimens, including: 
• Surgery (include date(s), site(s), and extent of resection (e.g. R0, R1, or R2)  
• Systemic therapy: include dates of treatment, agents (including dosing regimen), 
reason for treatment (e.g., adjuvant therapy or for metastatic disease), best response, 
date of disease progression or date and reason for treatment discontinuation other than 
disease progression 
• Radiation therapy: include the site(s) treated, total dose(s), date(s) of treatment, and 
response(s) 
• Other procedures, such as radiofrequency ablation (if applicable) 
Medical history, including any significant conditions or diseases that stopped at or prior to 
informed consent, must be elicited from each patient during screening. Based on the medical 
history, the patient must be assessed for any disqualifying medical conditions as specified in 
the inclusion and exclusion criteria. The medical history shall include a complete review of 
systems, past medical and surgical histories, and any allergies. Ongoing conditions are 
considered concurrent medical conditions and should be graded per CTCAE; if possible, the 
start date for these comorbidities must be documented. 
6.8 
Safety 
The safety profile will be assessed based on physical examinations; ECOG PS; changes from 
baseline in vital signs, ECGs, LVEF based on echocardiogram/multigated acquisition 
(MUGA) scan, ophthalmologic examination, and clinical laboratory tests; and the reporting 
of AEs and AESIs. The investigators and Sponsor will review all relevant safety data by 
teleconference on a regular basis (at least monthly or more frequently if required to monitor 
emerging AEs). No external independent data safety monitoring board is planned for this 
study. 
6.8.1 
Dose-Limiting Toxicities 
A DLT will be defined as any one of the following AEs during the first cycle of treatment 
occurring during Dose Escalation of the study up until the time of establishing the MTD or 


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an RP2D, unless it is clearly and incontrovertibly due to disease progression or other 
identifiable extraneous causes. 
• Any AE preventing administration of ≥80% of planned doses of rebastinib during the 
first cycle 
• A delay in the initiation of Cycle 2 more than 2 weeks due to a lack of adequate 
recovery from toxicity (see Section 5.3) 
• Hematologic AEs: 
o Grade 4 neutropenia (<500/mm3; <0.5 x 10e9/L) ≥7 days  
o ≥Grade 3 febrile neutropenia 
o Grade 3 thrombocytopenia, associated with bleeding that requires transfusion 
therapy 
o Grade 4 thrombocytopenia 
• Non-hematologic AEs: 
o Any Grade ≥3 non-hematologic toxicity will be considered a DLT except: 
o 
Grade 3 nausea or vomiting lasting <7 days 
o 
Grade 3 diarrhea lasting <7 days 
o 
Grade 3 fatigue 
o 
Isolated, asymptomatic Grade 3 abnormalities in chemistry laboratory 
values that last for ≤7 days. This includes electrolyte abnormalities that 
respond to medical intervention 
o 
ALT or AST elevation of >3X ULN with total bilirubin elevation of >2X 
ULN will be considered as a DLT if absence of initial findings of cholestasis 
such as alkaline phosphatase elevation of <2X ULN and no other reason can 
be found to explain simultaneous elevation of ALT or AST and total 
bilirubin 
• Any AE at least possibly related to the study drugs rebastinib or carboplatin, 
regardless of NCI CTCAE grade, leading to dose modification of rebastinib or 
carboplatin in the first cycle 
6.8.2 
Physical Examinations and Height and Weight 
Physical examination will be performed at screening and the EOT visit according to the 
Schedule of Assessments in Table 1. A full physical examination includes a review of the 
following systems: head/neck/thyroid, eyes/ears/nose/throat, respiratory, cardiovascular, 
lymph nodes, abdomen, skin, musculoskeletal, and neurological. Breast, anorectal, and 
genital examinations will be performed when medically indicated. At all other visits, 
examinations will be driven by clinical findings and/or patient complaints. After screening, 
any clinically significant abnormal findings in physical examinations must be reported as 
AEs (Section 7).  
6.8.3 
Eastern Cooperative Oncology Group (ECOG) Performance Status  
Eastern Cooperative Oncology Group (ECOG) PS (31) will be assessed according to the 
Schedule of Assessments in Table 1. 


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6.8.4 
Vital Signs, Weight, and Height 
Vital sign measurements, height, and weight will be performed according to the Schedule of 
Assessments in Table 1. Vital sign measurements will consist of sitting blood pressure, heart 
rate, respiratory rate, and body temperature. These should be assessed following a 5-minute 
rest (seated or supine position). Assessment of vital signs will be conducted according to the 
schedule shown in Table 2. Vital signs assessments for study treatment will be completed 
before the collection of the corresponding PK sample and only performed at visits when 
carboplatin is infused.  
6.8.5 
Electrocardiograms 
Digital, 12-lead ECGs will be performed with central over-reading according to the Schedule 
of Assessments in Table 1. All sites will be provided with an ECG machine and associated 
materials by the central ECG diagnostic service. All ECG assessments for study treatment 
will be conducted according to the schedule shown in Table 2. Assessment of ECG will be 
performed prior to PK sampling and only at visits when carboplatin is infused. 
Performance of all ECGs must adhere to the following guidelines: 
• All standard digital ECGs must be performed after the patient has been in the supine or 
semi-recumbent position for at least 5 minutes. The resting period begins once the leads 
are placed. 
• A hard copy of both the initial ECG tracing as well as the central over-read copy must be 
printed and signed by the Investigator at the site. 
ECG data will be transmitted to the central ECG diagnostic service and all interval 
measurements will be reviewed and adjusted using the central ECG core labs methodology 
by a trained ECG analyst. A cardiologist at the central ECG diagnostic service will then 
review each ECG to confirm if intervals were calculated correctly and to provide an 
interpretation. A report containing this information will be provided to the site for review and 
signature by the Investigator. This report will be filed with the machine ECG report for each 
visit in the patient’s source documents. The values reported by the central ECG diagnostic 
service will be used for data analysis. 
Heart rate and the following ECG intervals will be captured: 
• PR interval 
• QT and QTcF (QTcF = QT/RR1/3) intervals 
• QRS duration 
• RR interval 
The central ECG diagnostic service's standard reference ranges will be used throughout the 
study.  
For safety purposes, standard 12-lead ECGs may be obtained by the site as needed. 
6.8.6 
Echocardiograms/Multigated Acquisition Scans 
Echocardiograms or MUGA scans will be performed according to the Schedule of 
Assessments in Table 1. The same modality (echocardiogram or MUGA scan) must be used 


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throughout the study. Left ventricular ejection fraction must be documented in the patient’s 
source documents and eCRF. 
6.8.7 
Ophthalmologic Examination 
Ophthalmologic examinations will be performed by a licensed ophthalmologist according to 
the Schedule of Assessments in Table 1. This examination does not have to be repeated 
during screening if there is documentation of an examination that met protocol criteria within 
60 days prior to the first dose of study drug and after the last dose of previous anticancer 
treatment. An examination is not required at the EOT visit if one has been done within 
30 days prior to the last dose of all study drug. 
Examinations will include slit lamp examination of anterior structures (including eyelids, 
conjunctiva, cornea, anterior chamber, iris, and lens) and posterior structures (including optic 
nerve, macula, retinal vessels, the retinal periphery, and the vitreous); assessment of 
intraocular pressure by Goldmann Applanation Tonometry in each eye; assessment of visual 
acuity in each eye using best spectacle-corrected Snellen visual acuity at distance; assessment 
of visual fields based on Humphrey automated visual field testing using static automated 
perimetry testing with a 24-2 test pattern, but if not available a 30-2 test pattern; color vision 
testing using the Ishihara test; and, fundoscopy with mandatory digital photography of the 
fundus. Copies of digital photography of the fundus will be collected by the Sponsor. In 
addition to the scheduled examinations, ophthalmologic examinations will be performed as 
clinically indicated based on the occurrence of visual symptoms. Examinations prompted by 
visual disturbances should include fundus fluorescein angiography, optical coherence 
tomography, and electroretinogram as clinically indicated.  
6.8.8 
Clinical Laboratory Tests 
Blood and urine samples will be collected according to the Schedule of Assessments in 
Table 1 and analyzed at the site's local laboratory. All blood samples must be collected while 
patients are in a seated or supine position. Laboratory test results that are abnormal and 
considered clinically significant must be reported as AEs (Section 7). Screening laboratory 
results must be available before the first dose of study drug. All samples must be collected in 
accordance with acceptable laboratory procedures, and graded for toxicity as defined by the 
NCI CTCAE Version 5.0. In the case of clinically-significant Grade 3 or 4 laboratory 
abnormalities, the laboratory test should be repeated at appropriate intervals until recovery to 
Grade 0 or 1 and results recorded on the unscheduled CRF. The need for repeat testing, as 
well as the appropriate intervals for re-testing will be determined by the Investigator or sub-
Investigator. 
The safety laboratory tests are provided in Table 14. 


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Table 14: 
Safety Laboratory Tests 
Serum Chemistry 
Hematology 
Urinalysisb 
Alanine aminotransferase 
Albumin 
Alkaline phosphatase 
Aspartate aminotransferase  
Bicarbonate 
Blood urea nitrogen 
Calcium 
Chloride 
Creatinine 
Creatine Phosphokinase 
Follicle-stimulating hormonea 
Glucose 
Lactate dehydrogenase 
Magnesium 
Phosphorus  
Potassium 
Sodium 
Total and direct bilirubinc 
Total protein 
Hemoglobin 
Hematocrit 
Platelets 
Leukocytes 
Differential (absolute): 
• Eosinophils 
• Basophils 
• Neutrophils 
• Lymphocytes  
• Monocytes 
Coagulation Studies 
Activated partial thromboplastin time  
Prothrombin time 
International Normalized Ratio 
Urine protein  
Urine blood 
Specific gravity  
Urine ketones  
Urine glucose  
a. This may be required to demonstrate a patient is non-childbearing potential as defined in Section 6.8.9. 
b. If any result is abnormal, a microscopic analysis must be performed by the local laboratory. 
c. Indirect bilirubin should be calculated. 
6.8.9 
Pregnancy Test 
A serum β-hCG test to rule out pregnancy in women of childbearing potential will be 
obtained at screening. A urine or serum pregnancy test will be performed pre-dose on Day 1 
of every cycle and at the EOT visit as outlined in Table 1 (Schedule of Assessments). 
Pregnancy testing will not be required for patients who are non-childbearing females; defined 
as one who is post-menopausal (amenorrhoeic for ≥12 months with a follicle-stimulating 
hormone (FSH) ≥40 mIU/mL) or is surgically sterile (has documented bilateral 
oophorectomy or hysterectomy). 
6.8.10 Contraception and Pregnancy Avoidance Measures 
6.8.10.1 
Contraception 
The effects of rebastinib on sperm, conception, pregnancy, and lactation are not known. 
Participation in this study requires female patients to agree to use two methods of 
contraception, with one of the methods being highly effective, and male patients to agree to 
practice effective barrier contraception. Methods of contraception must be in successful use 
prior to the first dose of study drug and until 120 days following the last dose of study drug. 
Contraception for the patient is waived for the following: 
True abstinence for the patient, when this is in line with the preferred and usual lifestyle of 
the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, and postovulation 
methods) and withdrawal are not acceptable methods of contraception. 
If the male has documented bilateral orchiectomy or is considered infertile as documented 
through examination of a semen specimen or by demonstration of the absence of the vas 
deferens by ultrasound before the first dose of the study drug. 


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If the female is of non-childbearing potential, per the following: 
• Postmenopausal: spontaneous amenorrhea for at least 12 consecutive months and have a 
serum FSH level ≥40 mIU/mL. 
• Surgically sterile defined as documented bilateral oophorectomy or hysterectomy. 
NOTE: All other female patients (including patients with tubal ligations and patients who do 
not have a documented hysterectomy) will be considered to be of childbearing potential. 
Acceptable highly effective methods of contraception: 
• Vasectomy 6 months or more previously, with a negative post-vasectomy semen analysis 
for sperm. 
• Bilateral tubal ligation performed at least 6 months previously. 
• Continuous use of an intrauterine device. 
• Combined (estrogen and progestogen containing) or progestogen-only hormonal 
contraception associated with inhibition of ovulation: 
• oral 
• intravaginal 
• transdermal 
• injectable 
• implantable 
Acceptable methods of contraception: 
• Male and female condom with or without spermicide. 
• Barrier contraception (such as diaphragm, cervical cap, or sponge) and spermicide. 
Additional notes: 
• Acceptable methods of contraception listed above are examples. Please contact the 
Sponsor with any questions. 
• Female condom cannot be used with male condom (as a double method of contraception) 
due to risk of tearing. 
• Male and female patients who are not sexually active at the time of screening must agree 
to follow the contraceptive requirements of this study if they become sexually active with 
a partner of the opposite sex. 
• Male patients must not donate sperm after the first dose of study drug, throughout the 
study, and for 120 days following the last dose of study drug. 
• Female patients and female partners of male patients must not plan to become pregnant 
during the study through 120 days following the last dose of study drug. 
• Male patients whose female partner becomes pregnant through well-documented in vitro 
fertilization (donated sperm) or banked sperm (collected before the patient received study 
drug), must be compliant with the contraception requirements. In this scenario, the male 


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patient must commit to using acceptable methods of contraception (to ensure there is no 
exposure of the fetus to study drug) for the duration of the study and until 120 days after 
the last dose of study drug. 
Unique situations that may not fall within the above specifications must be discussed with the 
Sponsor. 
If there is any question that a woman of childbearing potential or male patient will not 
reliably comply with the requirements for contraception, that patient must not be entered into 
the study. 
6.8.10.2 
Pregnancy 
Patients must be counseled to inform the Investigator of any pregnancy that occurs during 
study treatment and for 120 days after the last dose of the study drug.  
If a female patient becomes pregnant while participating in the study, study drug must be 
permanently discontinued immediately. If the female partner of a male patient becomes 
pregnant while participating in the study, the patient must notify the Investigator 
immediately. The Investigator must notify the Sponsor and Pharmacovigilance within 
24 hours from the point in time when the Investigator becomes aware of the patient’s (or 
partner's) pregnancy. 
The patient or partner must be followed until the end of the pregnancy and the infant must be 
followed for 1 year after the birth, provided informed consent is obtained. A separate ICF 
must be provided to explain these follow up activities. Pregnancy itself does not constitute an 
AE. 
6.8.11 Adverse Events 
All AEs will be assessed, documented, and reported in accordance with 
ICH Good Clinical Practice (GCP) guidelines. Section 7 outlines the definitions, collection 
periods, criteria and procedures for documenting, grading, and reporting AEs.  
6.8.12 Safety Follow Up  
All patients must be followed for AEs; medications, including any anticancer treatments; and 
procedures from last dose of study drug through the FSV (30 [+/-7] days after last dose of 
study drug) or before the start of new anticancer therapy, whichever occurs first.  
6.9 
Pharmacokinetics  
6.9.1 
Sample Collection 
At the visits indicated in Table 1 (Schedule of Assessments) and Table 2, blood samples will 
be collected for the determination of the concentrations of rebastinib and carboplatin. 
Details on sample collection, processing, and shipping will be provided in a separate 
protocol-specific Laboratory Manual. 
For each visit with a PK blood draw, a record of study drug administration must be collected 
as described in Section 5.2. The collection date and time that each PK blood sample is drawn 
must also be recorded. 


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Samples from the PK sampling will be kept frozen by the Sponsor or its designee until all 
analyses have been completed and then disposed of according to the Sponsor or designee 
standard operating procedures. 
6.9.2 
Sample Assessment 
The following PK parameters will be calculated using non-compartmental methods of plasma 
rebastinib and carboplatin, obtained after single and repeated dose administration. The 
parameters will include, but may not be limited to Cmax, Time to maximum observed 
concentration (Tmax), AUC, and T1/2. 
6.10 Efficacy 
6.10.1 Radiologic Imaging 
All patients will have radiographic tumor evaluation by CT scans of the chest, abdomen, and 
pelvis according to the Schedule of Assessments in Table 1. Magnetic resonance imaging 
(MRI) or CT scans without contrast may be used for patients who are allergic to radiographic 
contrast media. Throughout the study, the same assessment technique should be used.  
Determination of objective tumor response will be performed by the Investigator according 
to the RECIST Version 1.1 or mRECIST (pleural mesothelioma only). Assessments should 
be completed regardless of dosing delays or additional unscheduled imaging assessments. An 
MRI of the brain will be performed at screening and subsequent imaging time points if CNS 
metastases are present at screening or signs and symptoms suggests CNS metastases.  
Imaging may be performed up to 14 days prior to the corresponding study visit or post-dose 
at the corresponding study visit.  
6.10.2 Response Assessment Using CA-125 (Ovarian Cancer Patients Only) 
CA-125 will be collected for all patients with ovarian cancer according to the Schedule of 
Assessments in Table 1. Ovarian cancer patients experiencing CA-125 response must have a 
confirmatory test performed at least 28 days after initial response is documented. Response 
assessment using RECIST Version 1.1 and CA-125 response criteria will be performed 
according to the Gynecological Cancer Intergroup (GCIG) guidelines (1).  
6.10.3 Relevant Tumor Markers 
Data of relevant tumor markers (examples: cancer antigen (CA)-15.3, CA-19-9, CA-27.29, 
prostate-specific antigen, and carcinoembryonic antigen) performed as standard of care will 
be collected. 
6.11 Biomarker Research Studies 
6.11.1 Sample Collection 
Biomarker samples will be collected according to the schedule in Table 1 (Schedule of 
Assessments). Sampling may be discontinued at any time by the Sponsor contingent on data. 
A Laboratory Manual describing the details of obtaining, storing, and shipping the samples 
will be provided. 
Remaining biomarker samples, including archival samples, will be stored for up to 15 years. 
These samples will be used for further analysis intended to address scientific questions 


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related to study drug and/or cancer. A decision to perform such exploratory biomarker 
research studies will be based on data obtained from rebastinib clinical studies, new scientific 
findings related to the drug class or disease, and/or reagent and assay availability. 
6.11.2 Sample Assessments 
6.11.2.1 
Tumor Tissue Samples 
At screening, an archival tumor tissue sample is required for all patients enrolled onto the 
study. If archival tumor tissue is unavailable, a fresh tumor biopsy will be collected prior to 
the first dose of study drug. Additionally, a fresh tumor biopsy will be collected at Cycle 3 
Day 1 (within 14 days). An optional tumor biopsy may be performed at EOT upon 
progression if a patient had previously responded to treatment and has provided consent for 
undergoing this optional tumor biopsy. Fresh biopsies are only required for patients whose 
tumor is anatomically accessible and should only be collected if a patient qualifies for the 
study based on all other entry criteria. Aspiration cytology samples, such as fine-needle 
aspirates, are not acceptable.  
Tissue samples will be used to assess changes in the tumor microenvironment (e.g., changes 
in the composition of infiltrating mononuclear cells including tumor-associated 
macrophages) when rebastinib is administered in combination with carboplatin. 
6.11.2.2 
Whole Blood and Plasma Samples 
Whole blood samples will be collected for immunophenotyping of peripheral blood 
mononuclear cells according to the Schedule of Assessments in Table 1. Plasma samples will 
be collected to measure circulating levels of chemokines/cytokines. Changes in 
chemokines/cytokines will be monitored to investigate the impact of rebastinib in 
combination with carboplatin on systemic immune response.  
Additional testing of the samples may be performed to assess the pharmacodynamic effects 
of rebastinib in combination with carboplatin based on data obtained from rebastinib studies, 
new scientific findings related to the drug class or disease, and/or reagent and assay 
availability. 
6.12 Pharmacogenomic Measurements 
6.12.1 Sample Collection 
A pharmacogenomic sample will be collected according to the schedule of study assessments 
in Table 1 (Schedule of Assessments). A Laboratory Manual describing the details of 
obtaining, storing, and shipping the sample will be provided. 
6.12.2 Sample Assessment 
Pharmacogenomic samples will be used to assess polymorphisms in genes encoding drug 
metabolic enzymes and/or transporters involved in metabolism and disposition of rebastinib 
in combination with carboplatin. Additionally, polymorphisms in genes that may be 
associated with clinical response and/or study drug-related toxicity will be assessed. 
Pharmacogenomic samples may be stored for up to 15 years. 


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6.13 Patient Reported Outcome (PRO) Measurements 
Patients will be asked to complete PRO assessments in the form of questionnaires using an 
electronic data capture system (electronic PRO [ePRO] device) according to the Schedule of 
Assessments in Table 1. Only English-speaking patients will complete the questionnaires.  
Patient entered data will not be modified by the Investigator or site staff, ePRO vendor, CRO, 
or Sponsor. 
6.13.1 National Cancer Institute (NCI) Patient-Reported Outcomes Version of the 
Common Terminology Criteria for Adverse Events (PRO-CTCAE) 
The NCI PRO-CTCAE measurement system was developed to gather symptomatic AEs by 
patient self-reporting (33). The PRO-CTCAE library is comprised of 124 items representing 
78 symptomatic AEs. The PRO-CTCAE items evaluate symptom attributes such as symptom 
occurrence, frequency, severity, and interference with daily activities, and are intended to be 
complementary to the items in the NCI’s CTCAE. Patients will fill out a subset of the library 
to report on their symptom experience over the preceding 7 days, using specific descriptor 
terms for each attribute. 
In this study, a subset of PRO-CTCAE items has been selected from the PRO-CTCAE item 
library. The time required for completion is approximately 20 minutes. Patients will be asked 
to complete questions from the PRO-CTCAE item library according to the Schedule of 
Assessments in Table 1. 
6.13.2 European Organisation for Research and Treatment of Cancer Quality of Life 
Questionnaire for Cancer 30-item (EORTC QLQ-C30) 
The EORTC QLQ-C30 is a validated, standardized, patient-completed questionnaire used 
extensively in international clinical studies. It was developed to assess health-related QOL in 
patients with cancer (34). The time required for completion is approximately 12 minutes. The 
questionnaire is composed of multi-item and single-item scales. These include 5 functional 
scales (physical functioning, role functioning, emotional functioning, social functioning, 
cognitive functioning), 9 symptom scales (fatigue, nausea and vomiting, pain, dyspnea, 
insomnia, appetite loss, constipation, diarrhea, and financial difficulties), and a global health 
status/QOL scale. 
The physical functioning and role functioning scales are completed by the patient’s response 
to Likert scale response options of “not at all,” “a little,” “quite a bit,” or “very much.” The 
2 items comprising the global health status scale also evaluate the patient’s experience over 
the past week using numerical rating scales with 1 representing “very poor” and 
7 representing “excellent”. 
Patients will be asked to complete EORTC QLQ-C30 according to the Schedule of 
Assessments in Table 1.  
6.13.3 GP5 from Functional Assessment of Cancer Therapy – General (FACT-G) 
The GP5 burden-of-side-effects, a part of the 
Functional Assessment of Chronic Illness Therapy (FACIT) FACT-G questionnaire, is a 
validated, standardized, patient-completed question used extensively in international clinical 
studies (35). It was developed to assess health-related QOL in patients with cancer. The time 
required for completion is less than 1 minute. The question is completed by the patient’s 


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selection of one of the following options of “not at all,” “a little bit,” “somewhat,” “quite a 
bit,” and “very much.”  
Patients will be asked to complete the GP5 from FACT-G according to the Schedule of 
Assessments in Table 1. 


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7 
ADVERSE EVENT AND SERIOUS ADVERSE EVENT DOCUMENTATION, 
SEVERITY GRADING, AND REPORTING 
7.1 
Adverse Events 
An AE is defined as any untoward medical occurrence in a patient administered a 
pharmaceutical product during the study, which does not necessarily have a causal 
relationship with the study drug. An AE can be any unfavorable and unintended sign 
(e.g., including an abnormal laboratory finding), symptom, or disease temporally associated 
with the use of the study drug, whether or not it is considered to be study drug related. This 
includes any newly occurring event or previous condition that has increased in severity or 
frequency after the ICF is signed. When there is a change in severity of an existing AE, 
including improvement or worsening of an event, a new AE should be reported. 
Planned hospital admissions or surgical procedures for an illness or disease that existed 
before the patient was screened in the study and progression of underlying disease are not to 
be considered AEs unless the condition deteriorated in an unexpected manner during the 
study (e.g., surgery was performed earlier than planned). 
Diagnostic and therapeutic non-invasive and invasive procedures, such as surgery, must not 
be reported as AEs. Elective surgeries or procedures must not be reported as AEs. All 
diagnostic and therapeutic non-invasive and invasive procedures and elective surgeries must 
be documented on the appropriate eCRF page. However, the medical condition for which the 
procedure was performed must be reported if it meets the definition of an AE. 
Each AE must be assessed immediately to determine if it meets the definition of serious 
(Section 7.8). If an SAE occurs, expedited reporting must follow local regulations, as 
appropriate. 
7.2 
Severity Assessment 
The Investigator must determine and record the severity of all serious and non-serious AEs. 
The NCI CTCAE Version 5.0 must be used for grading the severity of AEs (Cancer Therapy 
Evaluation Program website).  
The severity of an AE that does not appear in the CTCAE scale must be determined 
according to Table 15 (36). 
Table 15: 
National Cancer Institute (NCI) Common Terminology Criteria for 
Adverse Events (CTCAE) Version 5.0 Severity Grading Scale 
Grade 1 
Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; 
intervention not indicated. 
Grade 2  
Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate 
instrumental ADL*. 
Grade 3  
Severe or medically significant but not immediately life-threatening; hospitalization or 
prolongation of hospitalization indicated; disabling; limiting self care ADL**. 
Grade 4  
Life-threatening consequences; urgent intervention indicated. 
Grade 5  
Death related to AE. 
ADL = activities of daily living  
*Instrumental ADL refer to preparing meals, shopping for groceries or clothes, using the telephone, managing money, etc. 
** Self care ADL refer to bathing, dressing and undressing, feeding self, using the toilet, taking medications, and not 
bedridden. 


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7.3 
Causality Assessment 
The Investigator’s assessment of relationship of the AE, if any to the study drug must be 
provided for all AEs. An Investigator’s causality assessment is the determination of whether 
there is reasonable possibility that the study drug caused or contributed to an AE. 
Relationship to each study drug must be determined by the Investigator according to the 
following criteria in Table 16.  
Table 16: 
Relationship to Study Drug Criteria 
Related 
There is an association between the event and the administration of study drug, a 
plausible mechanism for the event to be related to the study drug and causes other 
than the study drug has been ruled out, and/or the event re-appeared on re-exposure to 
the study drug. 
Possibly Related 
There is an association between the event and the administration of the study drug 
and there is a plausible mechanism for the event to be related to study drug, but there 
may also be alternative etiology, such as characteristics of the patient's clinical status 
or underlying disease. 
Unlikely Related 
The event is unlikely to be related to the study drug and likely to be related to factors 
other than the study drug. 
Not Related 
The event is related to an etiology other than the study drug (the alternative etiology 
must be documented in the study patient’s medical record). 
 
For the purpose of the safety analyses, all AEs that are classified at least as possibly related 
will be considered treatment-related events. 
7.4 
Study Drug Action Taken 
The Investigator must classify the study drug action taken with regard to the AE. The action 
taken must be classified according to the categories shown in Table 17. 
Table 17: 
Classification for Study Drug Action Taken with Regard to an Adverse 
Event 
Classification 
Definition 
Dose Not Changed 
Study drug dose not changed in response to an AE. 
Dose Reduced 
Study drug dose reduced in response to an AE. 
Drug Interrupted 
Study drug administration interrupted in response to an AE. 
Drug Withdrawn 
Study drug administration permanently discontinued in response to an AE. 
Not Applicable 
Action taken regarding study drug administration does not apply. 
"Not applicable" must be used in circumstances such as when the study drug had 
been completed before the AE began, or during dose hold, and no opportunity to 
decide whether to continue, interrupt, or withdraw study drug is possible. 
 


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7.5 
Adverse Event Outcome 
An AE must be followed until the Investigator has determined and provided the final 
outcome. The outcome must be classified according to the categories shown in Table 18. 
Table 18: 
Classifications for Outcome of an Adverse Event 
Classification 
Definition 
Recovered/Resolved 
Resolution of an AE with no residual signs or symptoms. 
Recovered/Resolved with Sequelae 
Resolution of an AE with residual signs or symptoms. 
Recovering/Resolving 
Improvement of an AE 
Not Recovered/Not Resolved 
(Continuing) 
Either incomplete improvement or no improvement of an AE, such 
that it remains ongoing. 
Fatal 
Outcome of an AE is death. "Fatal" must be used when death is at 
least possibly related to the AE. 
Unknown 
Outcome of an AE is not known (e.g., a patient lost to follow-up). 
7.6 
Treatment Given 
The Investigator must ensure adequate medical care is provided to patients for any AEs. In 
addition, the Investigator must describe whether any treatment was given for the AE. "Yes" 
is used if any treatment was given in response to an AE and may include treatments such as 
other medications, hospitalization, surgery, physical therapy, etc. "No" indicates the absence 
of any kind of treatment for an AE. 
7.7 
Additional Points to Consider for Adverse Events 
7.7.1 
Clinically Significant Assessments 
Study assessments including laboratory tests, ECGs, physical examinations, and vital signs 
must be assessed, and those deemed as clinically significant must be documented as an AE. 
When possible, a clinical diagnosis for the study assessment must be provided rather than the 
abnormal test result alone (e.g., urinary tract infection, anemia). In the absence of a 
diagnosis, the abnormal study assessment itself may be listed as the AE (e.g., bacteria in 
urine or decreased hemoglobin). 
An abnormal study assessment is considered clinically significant if the patient has one or 
more of the following: 
• Worsening, from baseline, concomitant signs or symptoms related to the abnormal study 
assessment. 
• Further diagnostic testing or medical/surgical intervention is required. 
• A change in the dose of study drug, if study drug is withheld, or discontinuation from 
study drug occurs. 
Repeat testing to determine whether the result is abnormal, in the absence of any of the above 
criteria, does not necessarily meet clinically significant criteria. The determination of 
whether the study assessment results are clinically significant must be made by the 
Investigator. 
Symptoms of the disease under study must not be recorded as AEs as long as they are within 
the normal day-to-day fluctuation or expected progression of the disease, including 


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significant worsening unless the deterioration was unexpected, and are part of the efficacy 
data to be collected in the study. 
7.8 
Serious Adverse Events 
An AE is considered serious if it meets any of the following: 
• Results in death (regardless of cause, that occurs during participation in the study or 
occurs after participation in the study and is suspected of being a delayed toxicity due to 
administration of the study drug). 
• Is life threatening (an event/reaction in which the patient was at risk of death at the time 
of the event/reaction; it does not refer to an event/reaction which hypothetically might 
have caused death if it were more severe). 
• Requires inpatient hospitalization or prolongation of existing hospitalization. 
• Results in persistent or significant disability/incapacity. 
• Results in congenital anomaly/birth defect. 
• Important medical events that may not result in death, are life-threatening, or require 
hospitalization may be considered serious when, based upon appropriate medical 
judgment, they may jeopardize the patient and may require medical or surgical 
intervention to prevent one of the outcomes (i.e., allergic bronchospasm requiring 
intensive treatment in an emergency room or at home, blood dyscrasias or convulsions 
that do not result in inpatient hospitalization, or the development of drug dependency or 
drug abuse). 
Clinical outcomes or symptoms related to progressive disease need to be reported as an SAE 
if they meet SAE criteria and occur within 30 days of the last study drug administration. 
They must be reported according to the diagnosis or symptom of event and not by the term 
“disease progression,” unless the disease progression is considered atypical, accelerated, or 
caused by study drug. 
Clarification must be made between the terms "serious" and "severe," because they are not 
synonymous. The term "severe" is often used to describe the intensity (severity) of a specific 
event, as in mild, moderate, or severe myocardial infarction. The event itself, however, may 
be of relatively minor medical significance, such as a severe headache. This is not the same 
as "serious," which is based on patient/event outcome or action criteria described above, and 
is usually associated with events that pose a threat to a patient's life or functioning. 
Seriousness, not severity, serves as a guide for defining regulatory reporting obligations. For 
example, a laboratory abnormality judged to be Grade 4, in itself, may not constitute an SAE 
unless the clinical status of the patient indicates a life-threatening AE. 
7.9 
Adverse Events of Special Interest for Study Drug 
An AESI is an AE (serious or nonserious) of scientific and medical concern specific to study 
drug, for which ongoing monitoring and immediate notification by the Investigator to the 
Sponsor is required. Such AEs may require further investigation to characterize and 
understand them. AESIs may be added or removed during a study by a protocol amendment. 


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The following AEs are considered AESIs:  
• Muscular weakness (Grade 3) 
• Central retinal vein occlusion 
7.10 Adverse Event Reporting Periods 
The AE (including SAEs and AESIs) reporting period begins from the time that the patient 
provides informed consent through and including 30 days after the last administration of the 
study drug for all enrolled patients. Any SAE or AESI occurring after the reporting period 
must be promptly reported if a causal relationship to study drug is suspected.  
If a patient begins a new anticancer therapy, the safety reporting period ends at the time the 
new treatment is started; however, death must always be reported when it occurs during the 
safety reporting period irrespective of intervening treatment. 
7.11 Adverse Event and Serious Adverse Event Reporting Requirements 
Each patient must be carefully monitored for the development of any AEs. This information 
must be obtained in the form of non-leading questions (e.g., “How are you feeling?”) and 
from signs and symptoms detected during each examination, observations of study personnel, 
and spontaneous reports from patients.  
All AEs (serious and non-serious) spontaneously reported by the patient and/or in response to 
an open question from study personnel or revealed by observation, physical examination or 
other diagnostic procedures must be recorded. When possible, signs and symptoms indicating 
a common underlying pathology must be noted as one comprehensive event. Accompanying 
signs or symptoms (e.g., abnormal laboratory values) must not be reported as additional AEs. 
If a diagnosis is unknown, one or more symptoms may be reported as separate AEs. 
All SAEs and AESIs that occur within the reporting period, regardless of causality, must be 
reported by the Investigator to IQVIA Pharmacovigilance within 24 hours from the point in 
time when the Investigator becomes aware of the SAE or AESI. SAEs and AESIs must be 
followed until resolution, the condition stabilizes, or the Investigator and Sponsor agree that 
follow up is not required. For the purposes of study analysis, if the event has not resolved at 
the end of the study reporting period, it must be documented as ongoing. For purposes of 
regulatory safety monitoring, the Investigator is required to follow the event to resolution and 
report to the Sponsor the outcome of the event.   
If there are serious, unexpected (defined as not reported in the IB), suspected adverse drug 
reactions (SUSARs) associated with the use of the study drug, the Sponsor or authorized 
designee will ensure that the appropriate regulatory agency(ies) and all participating 
Investigators are notified on an expedited basis. It is the responsibility of the Investigator to 
promptly notify the local IRB of SUSARs according to the institutional policy.  
7.12 Abuse, Misuse, Overdose, and Medication Error 
Occurrences of events of overdose, drug misuse, drug abuse and medication error must be 
reported to the Sponsor.  
Abuse of a medicinal product: Persistent or sporadic, intentional excessive use of medicinal 
products, which is accompanied by harmful physical or psychological effects [DIR 
2001/83/EC Art 1(16)]. 


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Misuse: Intentional and inappropriate use of a medicinal product not in accordance with the 
prescribed or authorized dose, route of administration, and/or the indication(s) or not within 
the legal status of its supply. 
Overdose: Administration of a quantity of study drug given per administration or per day, 
which is above the assigned dose. 
Medication Error: An error made in prescribing, dispensing, administration, and/or use of 
the study drug. Medication errors are reportable to the Sponsor as defined below. 
• The dispensing, administration and/or use of the unassigned study drug. 
• The administration and/or use of an expired study drug. 
Note: cases of patients missing doses of the study drug are not considered reportable as 
medication errors. 
AEs or SAEs associated with drug abuse, misuse, overdose, or medication error must be 
reported as appropriate (Section 7.1 and Section 7.8). 


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8 
DISCONTINUATION AND REPLACEMENT OF PATIENTS 
8.1 
Discontinuation of Treatment 
A patient is free to withdraw from the study drug treatment for any reason and at any time 
without giving reason for doing so and without penalty or prejudice. The Investigator is also 
free to terminate a patient's study drug treatment at any time if the patient's clinical condition 
warrants it. The primary reason for discontinuation or withdrawal of a patient from study 
treatment must be determined using the following categories: 
• Progressive Disease (clinical or radiological) 
• AE 
• Withdrawal by patient from treatment 
• Death 
• Lost to follow-up 
• Non-compliance with study drug 
• Physician decision 
• Pregnancy 
• Termination of study by Sponsor 
• Any other reason that in the opinion of the Investigator, would justify removing the 
patient from study treatment, based on the best interest of the patient. 
8.2 
Follow Up Safety Visit / End of Study 
The end of study is defined as the date when the patient has withdrawn or completed through 
the FSV. 
If the patient is unable to complete the FSV, the reason will be captured in the clinical 
database using the following categories: 
• New Anticancer Treatment 
• Death 
• Lost to Follow Up 
• Withdrawal by patient from Study 
• Termination of Study by Sponsor 
• Other reason 
If a patient voluntarily withdraws from the study, the Investigator should attempt to contact 
the patient to determine the reason(s) for discontinuation and request the patient return for an 
EOT Visit and FSV. Patients must return all used, partially used, and unused study drug 
blister packs. 


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8.3 
Replacement of Patients 
In the Dose Escalation phase, patients must receive ≥80% of planned doses of rebastinib and 
one dose of carboplatin in Cycle 1 to be considered for determination of RP2D unless 
patients experienced a DLT(s). Patients who do not receive the requisite amount of the 
combination will be replaced.  
In the Dose Expansion phase, patients who do not receive at least one dose of the 
combination will be replaced. In addition, patients may be replaced if they discontinue the 
study drug treatment prior to the scheduled, first post-dose tumor assessment (Cycle 3 Day 1) 
due to reasons other than disease progression (clinical or radiological) or an AE(s) at least 
possibly related to rebastinib. 


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9 
STATISTICAL CONSIDERATIONS 
9.1 
Determination of Sample Size 
Dose Escalation: The Dose Escalation phase will primarily be used to determine the MTD or 
an RP2D and evaluate the safety and tolerability of the combination using modified 3+3 dose 
escalation rules. Three dose escalation cohorts are planned, and an additional cohort may be 
added. Approximately 18 patients will be enrolled in the Dose Escalation phase. 
Dose Expansion: A Simon’s two-stage design will apply to this phase of the study. The 
number of patients required for each cohort was calculated to demonstrate 20% improvement 
in ORR (from 20% historical ORR in the setting to 40% for the combination) under 
80% power and one-sided alpha of 0.05. In the initial stage, up to 18 patients will be 
evaluated. Greater than 4 responses will be required to enroll additional patients to 
demonstrate the target efficacy of >10 responses in a total of 33 patients. Thus, this part of 
the study may enroll approximately 99 patients (33 patients per indication-specific cohort). 
Patients who meet criteria defined in Section 8.3 will be replaced and not be included in the 
responder analysis. For example, if 10% of patients will be replaced, approximately 
110 patients may be enrolled. 
Given that this is a Phase 1b/2 study, no formal hypothesis testing will be performed.  
All relevant study data will be presented in listings.  
9.2 
Analysis Endpoints 
9.2.1 
Safety 
Safety endpoints include: 
• DLTs in the Dose Escalation Phase 
• AEs 
• SAEs 
• AESIs 
• Dose reduction or discontinuation of study drug due to toxicity 
• Physical examinations 
• ECOG PS 
• Ophthalmic examinations 
• Changes from baseline in laboratory parameters 
• ECGs 
• Echocardiograms/MUGAs 
• Vital signs 


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9.2.2 
Pharmacokinetics 
Pharmacokinetic endpoints when rebastinib is administered in combination with carboplatin 
and as a single agent include, but are not limited to: 
• Tmax (rebastinib only) 
• Time to maximum observed concentration at steady state (Tmax,ss: rebastinib only) 
• Maximum observed concentration (Cmax) 
• Maximum observed concentration at steady state (Cmax,ss) 
• Concentration observed at the end of the dosing interval (Cmin, trough concentration) 
• Concentration observed at the end of the dosing interval at steady state (Cmin,ss) 
• Area under the concentration-time curve (AUC) 
• T1/2 
• Volume of distribution (Vd) 
• 
CL 
9.2.3 
Efficacy 
Radiographic tumor assessments (computed tomography [CT] or MRI) will be performed by 
RECIST Version 1.1 or mRECIST (pleural mesothelioma only). For ovarian patients, 
additional response assessments will be performed using RECIST Version 1.1 and CA-125 
response criteria by the GCIG guideline (31). The endpoints for preliminary assessment of 
antitumor activity include: 
• Objective response rate (CR + PR) 
• CBR (CBR = CR + PR + SD) at 6, 12, and 18 weeks of the combination therapy 
• Best overall response  
• Time to response (defined as time from Cycle 1 Day 1 to PR or CR) 
• PFS (defined as time from Cycle 1 Day 1 to disease progression or death due to any 
cause) 
• TTP (defined as time from Cycle 1 Day 1 to the first documentation of progressive 
disease) 
• Duration of response (DOR; time from first PR, CR to disease progression or death due 
to any cause). This endpoint will be determined for responses only, defined as time of 
first documentation of response to first documentation of disease progression or death of 
any cause 
• 
OS 
9.2.4 
Pharmacogenomics 
The pharmacogenomics endpoints of the study include, but are not limited to:  
• 
Assessment of polymorphisms in genes that may be associated with clinical response 
and/or study drug-related toxicity 


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9.2.5 
Pharmacodynamics 
The pharmacodynamic endpoints of the study include, but are not limited to: 
• Assess changes of plasma chemokines/cytokines upon treatment  
• Assess changes in monocyte population in peripheral blood 
• 
Evaluate changes in tumor microenvironment using tumor tissue, if obtained, including 
but not limited to tumor associated macrophage, tumor infiltrating lymphocytes using 
IHC, ISH or other fit-for-purpose assays 
9.2.6 
Patient Reported Outcomes 
Assess the safety profile of rebastinib in combination with carboplatin using certain questions 
from the NCI PRO-CTCAE, a treatment-bother question (GP5) from FACIT’s FACT-G, as 
well as the EORTC QLQ-C30. 
9.3 
Populations for Analysis 
Enrolled Population: The Enrolled Population contains all patients who signed the ICF.  
Safety Population: The Safety Population is defined as all patients who have received at 
least one dose of either study drug. Safety Population will be used for all safety analysis. 
Modified intent-to-treat (mITT) Population: All patients who had at least one full dose of 
the combined study drugs, had measurable disease at baseline, and had at least 1 postbaseline 
disease assessment unless the patient discontinued prior to the post-baseline disease 
assessment due to an AE at least possibly related to rebastinib or due to clinical progression. 
PK Population: The PK population will include all patients who received at least one dose 
of either study drug and had at least one measurable concentration in plasma for either study 
drug. Additionally, this population will be used for analysis of PD data, if post-dose PD data 
is available. 
9.4 
Procedures for Handling Missing, Unused, and Spurious Data 
Unless specified in the individual endpoint analysis, missing data will not be imputed in 
except for identification of TEAEs and none study drug medication with missing start or end 
time. All available data will be presented on the data listings as collected.  
A TEAE is defined as an AE that occurred on or after the time of initial study drug and 
within 30 days after the date of last dose of study drug.  
Algorithms for imputing partial or missing dates of AEs are shown in Table 19. Same 
algorithm will be used for date of non-study drug medication.  


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Table 19: 
Partial or Missing Date Algorithms 
Variable 
Missing Day 
Missing Day, Month 
Missing Day, Month, 
Year 
Date of Last 
Therapy/Date of 
Initial Diagnosis 
Assign 1 
Assign January 1 if prior 
to date of informed 
consent, otherwise use 
date of informed consent 
Missing (do not impute) 
Adverse Event/Start 
Date 
Assign first day of month 
unless it is the month of first 
dose of study medication. 
Otherwise, assign date of 
first dose of study 
medication.  
Assign January 1 unless 
the year is year of first 
dose of study medication 
Otherwise, assign date of 
first dose of study 
medication. 
Assign date first dose of 
study medication. 
Adverse Event End 
Date 
Assign the last day of the 
month or end of study date, 
whichever is earlier.  
Assign December 31 or 
end of study date, 
whichever is earlier. 
If ongoing, end date is 
missing. Otherwise, 
assign end of study date. 
9.5 
Interim Analyses 
No interim analysis for efficacy will be performed for this study.  
9.6 
Adjustment for Multiple Comparisons 
Due to the exploratory nature of the study, adjustments for multiplicity will not be made. 
9.7 
Blinding 
This is an open-label study. 
9.8 
Statistical Methods 
9.8.1 
General Methods 
Data collected in this study will be documented using summary tables and patient data 
listings. Continuous variables will be summarized using descriptive statistics (number of 
patients, mean, median, standard deviation, minimum, and maximum). Categorical variables 
will be summarized using frequencies and proportions. Time-to-event data will be 
summarized via Kaplan-Meier (KM) using medians with associated 2-sided 90% CIs. 
Proportions, when appropriate, will be reported with exact 2-sided 90% CIs. 
Unless specified otherwise, baseline measurements must be the most recent value prior to 
receiving the first dose of study medication. If an assessment is not available, then the last 
assessment prior to that visit would be used.  
Medical history, AEs, and concurrent procedures will be coded using 
Medical Dictionary for Regulatory Activities (MedDRA). Prior and concomitant medications 
will be coded using the World Health Organization Drug Dictionary. 
Unless specified, the mITT Population is used for efficacy analysis and the Safety Population 
is used for the safety analysis. 


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9.8.2 
Disposition of Patients 
Patient disposition will be summarized overall for all patients who entered the study 
(i.e., signed the informed consent for the study). In addition, the number of patients in each 
population (Enrolled, Safety, and mITT) and patients that were removed from a population 
will be summarized. The number and proportion of patients who complete the study, as well 
as those who discontinue the study will be summarized along with the reason for 
discontinuation.  
9.8.3 
Demographic and Baseline Characteristics 
Demographic and baseline characteristics at study entry will be summarized by for the Safety 
and mITT Populations for each study cohort. 
9.8.4 
Extent of Exposure 
The total number of patients who received either study medication will be summarized by n 
and percentage. In addition, the number of cycles received will be displayed using 
continuous descriptive statistics. These analyses will be performed for the Safety Population. 
9.9 
Efficacy Analysis 
Efficacy analysis for the dose escalation and expansion cohorts will be performed separately. 
For the dose expansion, efficacy analysis will be done by cohort. Additionally, analysis may 
be done by including dose-escalation patients who met the inclusion criteria and received the 
same dose level as an RP2D selected for a given cohort. 
9.9.1 
Primary Endpoint  
The primary endpoint, ORR, defined as the proportion of patients with a CR or PR, will be 
analyzed in the mITT population as the primary analysis. Patients with unknown or missing 
response will be treated as non-responders, that is, they will be included in the denominator 
when calculating the proportion. Results will be summarized for each cohort with the 
proportion and exact 2-sided 90% CIs. 
Time to response (CR or PR) (reported in weeks) is defined as the interval between the date 
of first dose of study medication and the earliest date of first documented CR or PR. Patients 
who do not have a PR or CR will be censored at the date of the last adequate assessment. 
Summaries for each cohort will be provided using the methods of KM, and will include 
medians with 2-sided 90% CIs. 
9.10 Secondary Endpoints 
9.10.1 Progression-free Survival  
The PFS (reported in weeks) is defined as the interval between the date of initial dose and the 
earliest documented evidence of disease progression based on Investigator review, or death 
due to any cause. Patients who undergo surgical resection of target or non-target lesions, who 
have received other anticancer treatments, or patients who do not have a documented date of 
progression or death due to any cause will be censored at the date of the last assessment. For 
analysis of PFS, summaries for each cohort will be provided using the methods of KM, and 
will include medians with 2-sided 90% CIs.  


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9.10.2 Time to Tumor Progression  
The secondary endpoint of TTP (reported in weeks) is defined as the interval between the 
date of initial dose and the earliest documented evidence of disease progression based on 
Investigator review. Patients who undergo surgical resection of target or non-target lesions, 
who have received other anticancer treatments, who do not have a documented date of 
progression or death due to any cause, or who die prior to tumor progression will be censored 
at the date of the last assessment. Summaries for each cohort will be provided using the 
methods of KM, and will include medians with 2-sided 90% CIs. 
9.10.3 Overall Survival  
Overall survival (reported in weeks) is defined as the interval between the date of initial dose 
and date of death from any cause. Patients who are still alive or who are lost to follow-up 
will be censored at the date of last contact. Summaries for each cohort will be provided using 
the methods of KM, and will include medians with 2-sided 90% CIs. 
9.10.4 Time to Response  
The time to response, as defined by the Investigator review, will be summarized and 
displayed using the methods of KM, and will include medians with 2-sided 90% CIs. 
9.10.5 Clinical Benefit Rate  
Clinical benefit rate (CBR) will be calculated and summarized with n and percentage at 6, 
12, and 18 weeks. Clinical benefit will be defined as having a response (complete or partial) 
or SD. Proportions with exact 2-sided 90% CIs will be reported for each cohort. 
9.10.6 Duration of Response  
Duration of response, defined as the time from first PR or CR to disease progression or death, 
will be calculated for patients who have PR or CR. DOR will be presented using descriptive 
statistics. Summaries for each cohort will be provided using the methods of KM, and will 
include medians with 2-sided 90% CIs. 
9.11 Safety Analysis 
9.11.1 Adverse Events 
AEs will be summarized utilizing the number and proportion of patients by system organ 
class and preferred term for the Safety Population. All tables will only include TEAEs, where 
treatment emergent is defined as any AE that occurs after administration of the first dose of 
study drug and through 30 days after the last dose of study drug. 
AE toxicity grade will be classified using NCI CTCAE Version 5.0 criteria (See Table 15). If 
a patient has multiple occurrences of the same system organ class (SOC) or preferred term, 
then only the most severe event will be summarized in the tables for that SOC and preferred 
term. AEs of ≥Grade 3 will also be summarized. A missing toxicity grade will not be 
imputed. 
The AE analysis will be repeated for SAEs and AEs leading to dose reduction or 
discontinuation, and AESIs.  
No formal hypothesis-testing analysis of AE incidence rates will be performed.  


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9.11.2 Eastern Cooperative Oncology Group Performance Status 
Assessments will be summarized overall by time point utilizing continuous descriptive 
statistics. In addition, the change from baseline will be summarized for continuous 
parameters. For categorical parameters, the n and percentage will be displayed for each 
cohort. 
9.11.3 Vital Signs 
Assessments will be summarized overall by time point utilizing continuous descriptive 
statistics. In addition, the change from baseline will be summarized for continuous 
parameters.  
9.11.4 Echocardiogram/Multigated Acquisition Scans 
Assessments will be summarized overall by time point utilizing continuous descriptive 
statistics. In addition, the change from baseline will be summarized for continuous 
parameters. For categorical parameters, the n and percentage will be displayed for each 
cohort. 
9.11.5 Ophthalmologic Assessments  
Ophthalmologic assessments will be summarized by n and percent for each cohort. 
9.11.6 Clinical Laboratory Parameters 
Assessments will be summarized overall by time point utilizing continuous descriptive 
statistics. In addition, the change from baseline will be summarized for continuous 
parameters. For categorical parameters, the n and percentage will be displayed. Shift tables 
for labs with NCI-CTCAE grades will be presented. 
9.12 Pharmacokinetic Analysis 
Pharmacokinetic concentrations will be summarized utilizing continuous descriptive 
statistics: n, median, mean, standard deviation, minimum, and maximum. Cmax, Cmin, and 
AUC will also be summarized using geometric mean. 
9.12.1 Tumor Markers Analysis  
Tumor markers will be summarized overall by time points using descriptive statistics for 
each cohort. In addition, changes from baseline will be summarized.  
9.12.2 Biomarker and Pharmacodynamic Analysis 
Biomarker and pharmacodynamic parameters will be summarized graphically and with 
descriptive statistics (mean, SD, median, min, max). 
9.13 Pharmacogenomic Analysis 
Pharmacogenomic analysis will explore the impact of variations in genes encoding for drug 
metabolism enzymes and drug transporters on patient’s response to study drug. 
9.14 Procedures for Reporting Deviations to Original Statistical Analysis Plan 
All deviations from the original SAP will be provided in the final clinical study report. 


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10 QUALITY CONTROL AND QUALITY ASSURANCE 
10.1 Study Site Monitoring Visits 
During study conduct, the Sponsor or its agent will conduct periodic monitoring visits to 
ensure that the protocol and GCPs are being followed. The monitors will review source 
documents to confirm that the data recorded on eCRFs is accurate. The Investigator and 
institution will allow the Sponsor’s monitors or designees and appropriate regulatory 
authorities direct access to source documents to perform this verification. 
The study site may be subject to review by the IRB, and/or to quality assurance audits 
performed by the Sponsor, or companies working with or on behalf of the Sponsor, and/or to 
inspection by appropriate regulatory authorities. 
It is important that the Investigator and their relevant personnel are available during the 
monitoring visits and possible audits or inspections and that sufficient time is devoted to the 
process. 
10.2 Protocol Compliance 
The Investigator must conduct the study in compliance with the protocol provided by the 
Sponsor, and given approval/favorable opinion by the IRB and the appropriate regulatory 
authority(ies). Modifications to the protocol must not be made without agreement between 
both the Investigator and the Sponsor. Changes to the protocol will require written IRB and 
the appropriate regulatory authority(ies) approval/favorable opinion prior to implementation, 
except when the modification is needed to eliminate an immediate hazard(s) to patients. The 
IRB may provide, if applicable regulatory authority(ies) permit, expedited review and 
approval/favorable opinion for minor change(s) in ongoing studies that have the 
approval/favorable opinion of the IRB. The Sponsor must ensure that all protocol 
modifications are submitted to the regulatory authority(ies) in accordance with the governing 
regulations.  
If other unexpected circumstances arise that require deviation from protocol-specified 
procedures, the Investigator must consult with the Sponsor (and IRB, as required) to 
determine the appropriate course of action. 
The site must document all protocol deviations in the patient’s source documents. In the 
event of a significant deviation, the site must notify the Sponsor (and IRB, as required). 
Significant deviations include, but are not limited to, those that involve fraud or misconduct, 
increase the health risk to the patient, or confound interpretation of primary study 
assessments. 


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11 DATA HANDLING AND RECORD KEEPING 
11.1 Electronic Case Report Form 
The Sponsor or designee will provide the study sites with secure access to and training on the 
electronic data capture application sufficient to permit site personnel to enter or correct 
information in the eCRFs on the patients for which they are responsible. 
An eCRF is required and must be completed for each enrolled patient. The Investigator has 
ultimate responsibility for the accuracy, authenticity, and timely collection and reporting of 
all clinical, safety, and laboratory data entered on the eCRFs and any other data collection 
forms. Source documentation supporting the eCRF data must indicate the patient’s 
participation in the study and must document the dates and details of study procedures, AEs, 
other observations, and patient status. 
The Investigator, or designated representative, must complete the eCRF as soon as possible 
after information is collected. 
The audit trail will show the user’s identification information and the date and time of the 
any correction. The eCRFs must be signed electronically by the Investigator to attest that the 
data contained on the eCRFs, including any changes made to the eCRFs, is correct and 
endorse the final submitted data for the patients for whom the Investigator is responsible. 
The completed eCRFs are the sole property of the Sponsor and must not be made available in 
any form to third parties, except for authorized representatives of the Sponsor or appropriate 
regulatory authorities, without written permission from the Sponsor. 
The Sponsor will retain the eCRF data and corresponding audit trails. A copy of the final 
archival eCRF in the form of a compact disc or other electronic media will be provided to the 
site for placement in the Investigator’s study file. 
11.2 Record Retention 
To enable evaluations and/or audits from regulatory authorities or the Sponsor, the 
Investigator agrees to keep records, including the identity of all participating patients 
(sufficient information to link records, e.g., eCRFs and hospital records), all signed ICFs, 
SAE forms, source documents, detailed records of treatment disposition, and adequate 
documentation of relevant correspondence (e.g., letters, meeting minutes, telephone calls 
reports). The records must be retained by the Investigator according to the ICH, local 
regulations, or as specified in the Clinical Study Agreement, whichever is longer. 
If the Investigator becomes unable for any reason to continue to retain study records for the 
required period (e.g., retirement, relocation), the Sponsor must be prospectively notified. The 
study records must be transferred to a designee acceptable to the Sponsor, such as another 
Investigator, another institution, or to the Sponsor. The Investigator must obtain the 
Sponsor's written permission before disposing of any records, even if retention requirements 
have been met. 


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12 ETHICS 
12.1 Ethical Conduct of the Study 
The study will be conducted in accordance with the Declaration of Helsinki on Ethical 
Principles for Medical Research Involving Human Subjects, adopted by the General 
Assembly of the World Medical Association. 
In addition, the study will be conducted in accordance with the protocol, ICH GCP, and 
applicable local regulatory requirements and laws. 
The Investigator must ensure that this study is conducted in full conformity with Regulations 
for the Protection of Human Subjects of Research codified in 21 CFR Part 50, 21 CFR 
Part 54, 21 CFR Part 56, 21 CFR Part 312, 21 CFR Part 314 and ICH GCP E6. 
12.2 Patient Information and Consent 
All parties must ensure protection of patient personal data and must not include patient 
names on any Sponsor forms, reports, publications, or in any other disclosures, except where 
required by laws. In case of data transfer, the Sponsor must maintain high standards of 
confidentiality and protection of patient personal data. 
The ICF must be in compliance with ICH GCP, local regulatory requirements, and legal 
requirements. 
The ICF used in this study, and any changes made during the course of the study, must be 
prospectively approved by both the IRB and the Sponsor before use. 
The Investigator must ensure that each study patient is fully informed about the nature and 
objectives of the study and possible risks associated with participation. The Investigator, or a 
person designated by the Investigator, must obtain written informed consent from each 
patient or the patient's legally acceptable representative before any study-specific activity is 
performed.  
12.3 IRB 
It is the responsibility of the Investigator to have prospective approval of the study protocol, 
protocol amendments, ICFs, and other relevant documents from the IRB. All correspondence 
with the IRB must be retained in the Investigator Site File. 
The only circumstance in which an amendment may be initiated prior to IRB approval is 
where the change is necessary to eliminate apparent immediate hazards to the patients. In that 
event, the Investigator must notify the IRB and the Sponsor in writing immediately after the 
implementation. 
12.4 Patient Confidentiality 
The Sponsor and designees affirm and uphold the principle of the patient’s right to protection 
against invasion of privacy. Throughout this study, a patient’s source data must only be 
linked to the Sponsor’s clinical study database or documentation via a unique identification 
number. As permitted by all applicable laws and regulations, limited patient attributes, such 
as sex, age, or date of birth may be used to verify the patient and accuracy of the patient’s 
unique identification number. 
To comply with ICH GCP and to verify compliance with this protocol, the Sponsor requires 
the Investigator to permit its monitor or designee’s monitor, representatives from any 


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regulatory authority (e.g., FDA), the Sponsor’s designated auditors, and the appropriate IRBs 
to review the patient’s original medical records (source data or documents), including, but 
not limited to, any genetic/genomic data the patient might have from testing done prior to 
entering the study, laboratory test result reports, ECG reports, admission and discharge 
summaries for hospital admissions occurring during a patient’s study participation, and 
autopsy reports. Access to a patient’s original medical records requires the specific 
authorization of the patient as part of the informed consent process (Section 12.2). 
Copies of any patient source documents that are provided to the Sponsor must have certain 
personally identifiable information removed (i.e., patient name, address, and other identifier 
fields not collected on the patient’s eCRF). 
12.5 Reporting of Safety Issues or Serious Breaches of the Protocol or International 
Conference on Harmonization Good Clinical Practice 
In the event of any prohibition or restriction imposed (i.e., clinical hold) by an applicable 
Competent Authority, or if the Investigator is aware of any new information which might 
influence the evaluation of the benefits and risks of the study drug, the Sponsor must be 
informed immediately.  
In addition, the Investigator must inform the Sponsor immediately of any urgent safety 
measures taken by the Investigator to protect the study patients against any immediate 
hazard, and of any serious breaches of this protocol or of ICH GCP that comes to the 
attention of the Investigator. 
12.6 Liability and Insurance 
The Sponsor has subscribed to an insurance policy covering, in its terms and provisions, its 
legal liability for injuries caused to participating persons and arising out of this research 
performed strictly in accordance with the scientific protocol as well as with applicable law 
and professional standards. 


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13 STUDY TERMINATION 
When the Sponsor is aware of information on matters concerning the quality, efficacy, and 
safety of the study drug, as well as other important information that may affect proper 
conduct of the clinical study, the Sponsor may discontinue the clinical study and send a 
written notice of the discontinuation along with the reason to the Investigator.  
If an Investigator intends to discontinue participation in the study, the Investigator must 
immediately inform the Sponsor of the discontinuation and the reason for it. 
13.1 Criteria for Suspension or Premature Termination of the Study 
Criteria for either temporary suspension or premature termination of the study include: 
1. New information regarding the safety or efficacy of the study drug that indicates a change 
in the known risk/benefit profile for the compound, such that the risk/benefit is no longer 
acceptable for patients participating in the study. 
2. Significant violation of GCP that compromises the ability to achieve the primary study 
objectives or compromises patient safety. 
3. The Sponsor may suspend or prematurely terminate the study for reasons not related to 
the conduct of the study. 
13.2 Criteria for Premature Termination or Suspension of Investigational Sites 
A study site may be terminated prematurely or suspended if the site (including the 
Investigator) is found to be in significant violation of GCP, protocol, contractual agreement, 
or is unable to ensure adequate performance of the study. 
13.3 Procedures for Premature Termination or Suspension of the Study or the 
Participation of Investigational Site(s) 
In the event that the Sponsor elects to terminate or suspend the study or the participation of 
an investigational site, a study-specific procedure for early termination or suspension will be 
provided by the Sponsor. The procedure will be followed by applicable investigational sites 
during the course of termination or study suspension. 


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14 PUBLICATION OF STUDY RESULTS 
Any and all scientific, commercial, and technical information disclosed by the Sponsor in 
this protocol or elsewhere must be considered the confidential and proprietary property of the 
Sponsor. The Investigator shall hold such information in confidence and shall not disclose 
the information to any third party except to such of the Investigator's employees and staff as 
have been made aware that the information is confidential and who are bound to treat it as 
such and to whom disclosure is necessary to evaluate that information. The Investigator shall 
not use such information for any purpose other than determining mutual interest in 
performing the study and, if the parties decide to proceed with the study, for the purpose of 
conducting the study. 
The Investigator understands that the information developed from this clinical study will be 
used by the Sponsor in connection with the development of the study drug and therefore may 
be disclosed as required to other clinical Investigators, business partners and associates, the 
FDA, and other government agencies. The Investigator also understands that, to allow for the 
use of the information derived from the clinical study, the Investigator has the obligation to 
provide the Sponsor with complete test results and all data developed in the study. 
No publication or disclosure of study results will be permitted except under the terms and 
conditions of a separate written agreement between the Sponsor and the Investigator and/or 
the Investigator's institution.  


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34. EORTC Quality of Life http://groups.eortc.be/qol/eortc-qlq-c30 [accessed 
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k_Reference_8.5x11.pdf 

SAP_001.pdf: 
 
  
 
Protocol: DCC-2036-01-004  
 
Official Title: An Open-Label, Multicenter, Phase 1b/2 Study of Rebastinib (DCC-2036) in Combination 
with Carboplatin to Assess Safety, Tolerability, and Pharmacokinetics in Patients with Advanced or 
Metastatic Solid Tumors 
 
NCT Number:  NCT03717415 
 
Approval Date: 12 APR 2022 
 


Deciphera Pharmaceuticals, LLC 
DCC-2036-01-004
Statistical Analysis Plan, Version 1.0 
April 12, 2022
1 
STATISTICAL ANALYSIS PLAN 
PHASE 1B/2 
DATE OF PLAN: 
April 12, 2022 
BASED ON: 
Protocol DCC-2036-01-004, Amendment 4 (February 24, 2020) 
STUDY TITLE: 
An Open-Label, Multicenter, Phase 1b/2 Study of Rebastinib (DCC-2036) in Combination with 
Carboplatin to Assess Safety, Tolerability, and Pharmacokinetics in Patients with Advanced or 
Metastatic Solid Tumors 
SPONSOR: 
Deciphera Pharmaceuticals, LLC 
200 Smith Street 
Waltham, MA 02451 
This study is being conducted in compliance with good clinical practice, including the archiving 
of essential documents. 
NCT #: NCT03717415
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-004 
Statistical Analysis Plan, Version 1.0 
 
April 12, 2022 
 
2 
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DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-004 
Statistical Analysis Plan, Version 1.0 
 
April 12, 2022 
 
3 
TABLE OF CONTENTS 
LIST OF ABBREVIATIONS ..........................................................................................................6 
1. 
INTRODUCTION ........................................................................................................8 
2. 
STUDY OBJECTIVES ................................................................................................9 
2.1. 
Dose Escalation ............................................................................................................9 
2.1.1. 
Primary Objectives .......................................................................................................9 
2.1.2. 
Secondary Objectives ...................................................................................................9 
2.2. 
Dose Expansion ............................................................................................................9 
2.2.1. 
Primary Objectives .......................................................................................................9 
2.2.2. 
Secondary Objectives ...................................................................................................9 
2.3. 
Exploratory Objectives (Dose Escalation and Dose Expansion) ..................................9 
3. 
STUDY DESIGN OVERVIEW .................................................................................10 
3.1. 
Overall Study Design ..................................................................................................10 
3.1.1. 
Dose Escalation Phase ................................................................................................10 
3.1.2. 
Dose Expansion Phase ................................................................................................11 
3.2. 
Duration of Participation ............................................................................................12 
3.3. 
Study Treatments ........................................................................................................12 
3.4. 
Planned Analyses ........................................................................................................12 
3.4.1. 
Interim Analyses .........................................................................................................12 
3.4.2. 
Final Analyses ............................................................................................................12 
3.5. 
Blinding ......................................................................................................................13 
3.6. 
Sample Size Considerations .......................................................................................13 
4. 
STUDY ENDPOINTS ................................................................................................14 
4.1. 
Efficacy Endpoints ......................................................................................................14 
4.1.1. 
Objective Response Rate ............................................................................................14 
4.1.2. 
Clinical Benefit Rate ...................................................................................................14 
4.1.3. 
Duration of Response .................................................................................................14 
4.1.4. 
Time to Response .......................................................................................................14 
4.1.5. 
Progression-Free Survival ..........................................................................................14 
4.1.6. 
Time to Progression ....................................................................................................14 
4.1.7. 
Overall Survival ..........................................................................................................14 
4.1.8. 
Cancer-Antigen 125 Response (ovarian cancer expansion cohort only) ....................15 
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-004 
Statistical Analysis Plan, Version 1.0 
 
April 12, 2022 
 
4 
4.2. 
Safety Endpoints .........................................................................................................15 
4.3. 
Patient-Reported Outcomes ........................................................................................15 
4.4. 
Pharmacokinetic Endpoints ........................................................................................15 
4.5. 
Pharmacogenomic Endpoints .....................................................................................16 
4.6. 
Biomarker and Pharmacodynamic Endpoints ............................................................16 
5. 
DEFINITIONS AND CONVENTIONS FOR DATA HANDLING .........................17 
5.1. 
Definition of Baseline .................................................................................................17 
5.2. 
Handling of Missing Data ...........................................................................................17 
5.3. 
Study Visits .................................................................................................................18 
5.4. 
Study Day ...................................................................................................................18 
5.5. 
Coding Dictionaries ....................................................................................................18 
6. 
ANALYSIS POPULATIONS ....................................................................................19 
6.1. 
Enrolled Population ....................................................................................................19 
6.2. 
Safety Population ........................................................................................................19 
6.3. 
Modified Intent-to-Treat Population ...........................................................................19 
6.4. 
Pharmacokinetic Population .......................................................................................19 
7. 
ANALYSES AND SUMMARIES .............................................................................20 
7.1. 
General Considerations ...............................................................................................20 
7.2. 
Participant Disposition ................................................................................................20 
7.3. 
Protocol Deviations ....................................................................................................21 
7.4. 
Demographics and Baseline Characteristics ...............................................................22 
7.4.1. 
Demographic Characteristics ......................................................................................22 
7.4.2. 
Medical History ..........................................................................................................22 
7.4.3. 
Cancer History ............................................................................................................22 
7.4.4. 
Prior Anti-Cancer Therapy and Procedures ................................................................23 
7.4.5. 
Prior Medications and Procedures ..............................................................................23 
7.4.6. 
Concomitant Medications and Procedures .................................................................23 
7.5. 
Safety Analysis ...........................................................................................................24 
7.5.1. 
Study Drug Exposure ..................................................................................................24 
7.5.2. 
Adverse Events ...........................................................................................................24 
7.5.3. 
Dose-Limiting Toxicities ............................................................................................27 
7.5.4. 
Clinical Laboratory Parameters ..................................................................................28 
7.5.5. 
Vital Signs, Weight, and Height .................................................................................29 
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-004 
Statistical Analysis Plan, Version 1.0 
 
April 12, 2022 
 
5 
7.5.6. 
ECOG Performance Status .........................................................................................29 
7.5.7. 
Electrocardiograms .....................................................................................................29 
7.5.8. 
Echocardiogram/Multigated Acquisition Scans .........................................................30 
7.5.9. 
Ophthalmologic Examinations ...................................................................................30 
7.6. 
Efficacy Analysis ........................................................................................................30 
7.6.1. 
Objective Response Rate ............................................................................................31 
7.6.2. 
Clinical Benefit Rate ...................................................................................................32 
7.6.3. 
Duration of Response .................................................................................................33 
7.6.4. 
Time to Response .......................................................................................................33 
7.6.5. 
Progression-Free Survival ..........................................................................................33 
7.6.6. 
Time to Progression ....................................................................................................34 
7.6.7. 
Overall Survival ..........................................................................................................34 
7.6.8. 
CA-125 Response (ovarian cancer expansion cohort only) .......................................34 
7.7. 
Patient-Reported Outcomes Analysis .........................................................................34 
7.8. 
Pharmacokinetic Analysis ..........................................................................................34 
7.9. 
Biomarker and Pharmacodynamic Analysis ...............................................................34 
7.10. 
Pharmacogenomic Analysis........................................................................................34 
8. 
REFERENCES ...........................................................................................................35 
 
LIST OF TABLES 
Table 1: 
Partial or Missing Date Imputation Rules ..................................................................17 
Table 2: 
Safety Laboratory Tests ..............................................................................................29 
Table 3: 
Summary of the Best Overall Response Status when Confirmation of 
Response is Required ..................................................................................................32 
 
LIST OF FIGURES 
Figure 1: 
Study Schema .............................................................................................................11 
 
 
 
 
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-004 
Statistical Analysis Plan, Version 1.0 
 
April 12, 2022 
 
6 
LIST OF ABBREVIATIONS 
 
Below is a list of the abbreviation that will be used throughout this document. 
Abbreviation 
Definition 
AE 
Adverse event 
AESI 
Adverse events of special interest 
AUC 
Area under the plasma concentration-time curve 
BID 
Twice daily 
CA-125 
Cancer antigen-125 
CBR 
Clinical Benefit Rate 
CI 
Confidence interval 
CR 
Complete response 
CSR 
Clinical study report 
CT 
Computed tomography 
CTCAE 
Common Terminology Criteria for Adverse Events 
DLT 
Dose-limiting toxicity 
DOR 
Duration of response 
ECG 
Electrocardiogram 
ECOG 
Eastern Cooperative Oncology Group 
EORTC-QLQ-C30 
European Organisation for Research and Treatment of Cancer Quality 
of Life Questionnaire for Cancer 30 item 
FACT-G 
Functional Assessment of Cancer Therapy - General 
ICH 
International Conference on Harmonization 
IHC 
Immunohistochemical 
ISH 
In situ hybridization 
IV 
Intravenous 
LVEF 
Left ventricular ejection fraction 
MedDRA 
Medical Dictionary for Regulatory Activities 
mITT 
Modified Intent-to-Treat 
mRECIST 
Modified Response Evaluation Criteria in Solid Tumors 
MTD 
Maximum tolerated dose 
MUGA 
Multigated acquisition 
NCI-CTCAE 
National Cancer Institute Common Terminology Criteria for Adverse 
Events 
NCI-PRO-CTCAE 
National Cancer Institute Patient Reported Outcomes Common 
Toxicity Criteria for Adverse Events 
NE 
Not evaluable 
ORR 
Objective response rate 
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-004 
Statistical Analysis Plan, Version 1.0 
 
April 12, 2022 
 
7 
OS 
Overall survival 
PD 
Progressive disease 
PFS 
Progression-free survival 
PK 
Pharmacokinetic 
PO 
Orally 
PR 
Partial response 
PRO 
Patient-reported outcome 
PT 
Preferred term 
RECIST 
Response Evaluation Criteria in Solid Tumors 
RP2D 
Recommended Phase 2 dose 
SAE 
Serious adverse event 
SAP  
Statistical analysis plan 
SD 
Stable disease 
SOC 
System organ class 
TEAE 
Treatment-emergent adverse event 
TTP 
Time to progression 
US 
United States 
 
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-004 
Statistical Analysis Plan, Version 1.0 
 
April 12, 2022 
 
8 
1. 
INTRODUCTION 
This statistical analysis plan (SAP) describes the methods to be used in the analysis of safety and 
tolerability data from clinical study protocol DCC-2036-01-004 entitled “An Open-Label, 
Multicenter, Phase 1b/2 Study of Rebastinib (DCC-2036) in Combination with Carboplatin to 
Assess Safety, Tolerability, and Pharmacokinetics in Patients with Advanced or Metastatic Solid 
Tumors” in order to answer the study objectives, which is based on Protocol Amendment 4 of 
the clinical study protocol, dated February 24, 2020. This study consists of two parts. The Dose 
Escalation phase will be conducted in approximately 4 centers in the United States (US) and the 
Dose Expansion phase will be conducted in up to 18 centers in the US. 
The analyses in this SAP will be used to support an abbreviated clinical study report (CSR) that 
provides a comprehensive summary of safety. Efficacy will be summarized for participants in the 
Dose Expansion phase only using ORR only. All other efficacy parameters will be listed. 
Analyses of pharmacokinetic (PK), pharmacodynamic, pharmacogenomic, and patient-reported 
outcomes (PRO) data, if conducted, will be described in separate analysis plans. 
Populations for analysis, data handling rules, statistical methods, and formats for data 
presentation are described within this document.  The statistical analyses and summary 
tabulations described in this SAP will provide the basis for the results sections of the abbreviated 
CSR for this trial. The SAP outlines any differences in data analysis methods relative to those 
planned in the study protocol. Any changes to the data analysis methods after the SAP is 
finalized will be described in the abbreviated CSR. 
Formats for tables, listings, and figures are specified in a separate document. 
 
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


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DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-004 
Statistical Analysis Plan, Version 1.0 
 
April 12, 2022 
 
10 
3. 
STUDY DESIGN OVERVIEW 
3.1. 
Overall Study Design 
This is an open-label Phase 1b/2 multicenter study in participants with advanced or metastatic 
solid tumors who have exhausted available, approved therapies and for which carboplatin is 
considered appropriate treatment. Adverse events (AEs) will be assessed, and laboratory values, 
vital sign measurements, and electrocardiograms (ECGs) will be obtained to evaluate the safety 
and tolerability of rebastinib when administered in combination with carboplatin.  
Rebastinib will be administered orally (PO) twice daily (BID), and carboplatin will be infused 
once every 3 weeks. A dose of carboplatin will be calculated using the Calvert formula based on 
the target area under the plasma concentration time curve (AUC) on Day 1 of each cycle. Each 
cycle is 21 days, however, initiation of subsequent cycles after Cycle 1 is dependent upon 
carboplatin dosing. 
The study consists of two parts: Dose Escalation and Dose Expansion. 
3.1.1. 
Dose Escalation Phase  
In the Dose Escalation phase, doses of rebastinib and carboplatin will be escalated using 
modified 3 + 3 dose escalation rules starting with rebastinib at 50 mg BID in combination with 
AUC5 of carboplatin. The next cohort is rebastinib at 100 mg BID with AUC5 of carboplatin. 
The third planned cohort will be rebastinib at 100 mg BID with AUC6 of carboplatin. Based on 
the safety and tolerability, other combinations of doses such as rebastinib at 50 mg BID with 
AUC6 of carboplatin may be evaluated if, at least, rebastinib at 50 mg BID with AUC5 of 
carboplatin is deemed safe. In addition, the rebastinib dose may be increased to 150 mg BID with 
AUC5 and/or AUC6 of carboplatin if 100 mg BID of rebastinib with AUC5 and AUC6 of 
carboplatin is tolerated. Alternatively, 75 mg BID of rebastinib may be evaluated if 100 mg BID 
is not tolerated and 50 mg BID is deemed as safe.  
Dose escalation will proceed to the next dose level if no dose-limiting toxicity (DLT) is observed 
in a minimum of 3 participants completing 1 cycle. If a DLT is observed in only 1 participant in 
a cohort of 3 participants, an additional 3 participants will be enrolled up to a total of at least 6 
participants at this dose level. Dose escalation will then only proceed if no more than 1 
participant in the cohort of 6 participants has experienced a DLT during the first cycle of 
treatment in the Dose Escalation phase. If a DLT is observed in 2 or more participants in a cohort 
of 3 to 6 participants, dose escalation will stop.  
The determination of the maximum tolerated dose (MTD) or recommended Phase 2 dose (RP2D) 
will be based on the safety and tolerability of at least 6 participants at a dose level of the 
combination. As necessary for evaluation of safety and tolerability, up to 12 participants may be 
enrolled per cohort. Enrollment of additional participants to further explore safety and 
tolerability may take place simultaneously while a cohort at the next dose level is enrolling 
participants in the Dose Escalation phase. 
The MTD is defined as the highest dose level of rebastinib and carboplatin at which no more 
than 1 of 6 participants experiences a DLT during the first cycle. The RP2D will be a dose level 
of rebastinib and carboplatin deemed safe and tolerable on the basis of the totality of safety, 
tolerability, PK, and preliminary efficacy data. The RP2D will not exceed the MTD. 
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-004 
Statistical Analysis Plan, Version 1.0 
 
April 12, 2022 
 
11 
3.1.2. 
Dose Expansion Phase 
The Dose Expansion phase will be initiated upon determination of the MTD or an RP2D in the 
Dose Escalation phase. In this phase, approximately 99 participants in three indication-specific 
cohorts will be enrolled with the RP2D. A Simon’s two-stage design will be applied to further 
evaluate the safety, tolerability, and preliminary efficacy of rebastinib in combination with 
carboplatin in triple negative breast cancer (Cohort 1), ovarian cancer (Cohort 2), and 
mesothelioma (Cohort 3) participants. An RP2D used for each cohort will be chosen based on 
agreement between the Investigators and the Sponsor. 
Each cohort in the Dose Expansion phase will initially enroll up to 18 participants in the first 
stage. The decision to enroll participants beyond the first stage will be based on response 
assessments obtained after the first post-dose response assessment of the last participant enrolled 
in the first stage of a cohort. Up to a total of 33 participants per cohort may be enrolled based on 
response assessments. If >4 responses (defined as unconfirmed partial response [PR] or complete 
response [CR] as best response) are seen in a cohort, additional participants will be enrolled for a 
total of up to 33 participants. If ≤4 responses are seen in a cohort, the cohort will be terminated. 
Participants who do not receive at least one dose of the combination will be replaced. In addition, 
if participants discontinued the study drug treatment prior to the scheduled, first post-dose tumor 
assessment (Cycle 3 Day 1) due to reasons other than disease progression (clinical or 
radiological) or an AE(s) at least possibly related to rebastinib, they may be replaced. Replaced 
participants will not be included in the responder analysis. There will be an enrollment pause 
between the first and second stage for evaluation of response. 
The Study Schema for Dose Escalation and Dose Expansion are presented below: 
Figure 1: 
Study Schema 
 
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Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-004 
Statistical Analysis Plan, Version 1.0 
 
April 12, 2022 
 
12 
3.2. 
Duration of Participation 
Participants will receive study treatment until they develop progressive disease (PD), experience 
unacceptable toxicity, or withdraw consent. Participants will be eligible to receive study 
treatment as long as the Investigator and the Sponsor agree that the participant is showing 
clinical benefit, and for as long as rebastinib is being developed to support the indication, and 
continuation of treatment does not conflict with the Sponsor’s right to terminate the study. After 
the participant discontinues treatment, the participant will be contacted via phone for safety 
follow-up. The study will end following the last participant's last visit. 
3.3. 
Study Treatments 
Rebastinib will be provided as tablets for oral administration containing 25 mg and 75 mg of 
active rebastinib. Rebastinib will be dosed BID continuously throughout each cycle. In the Dose 
Escalation phase, rebastinib will be escalated from 50 mg to 100 mg in combination with 
carboplatin. In addition, doses of 150 mg and 75 mg may also be explored. 
Carboplatin will be administered by IV infusion over approximately 60 minutes on Day 1 of 
each cycle and at least 21 days apart. A carboplatin dose will be calculated using the Calvert 
formula. The maximum carboplatin dose should not exceed AUC (mg x min/mL) x 150 mL/min. 
Rebastinib will be administered prior to IV infusion of carboplatin. On Day 1 of Cycles 1 and 2 
when serial PK samples will be collected, participants must take the dose of carboplatin 2 hours 
(+/-30 minutes) after rebastinib dosing.  
In the Dose Escalation phase, carboplatin will be dose escalated from AUC5 to AUC6 to 
determine an RP2D of rebastinib and carboplatin combination. In the Dose Expansion phase, the 
MTD or an RP2D of carboplatin in combination with rebastinib will be administered. A different 
dose level not exceeding the MTD may be chosen for each cohort.  
3.4. 
Planned Analyses 
3.4.1. 
Interim Analyses 
No formal interim analyses leading to an interim clinical study report are planned for this study.  
Informal interim analyses are planned for each Expansion Cohort in the Dose Expansion phase 
after the first 18 evaluable participants have been enrolled and been followed for at least one 
post-baseline assessment. If 5 or more objective responses are observed according to Response 
Evaluation Criteria in Solid Tumors (RECIST v1.1) or modified RECIST (mRECIST) for pleural 
mesothelioma participants, then 15 additional participants will be enrolled into the expansion 
cohort. If 5 or more objective responses are observed prior to the enrollment of the 18th 
participant, then continued enrollment can proceed without a pause. Otherwise, a pause in an 
enrollment will occur until the 5th objective response is observed or the Expansion Cohort will 
be closed if less than 5 objective responses are observed. 
3.4.2. 
Final Analyses 
The final analysis will be conducted once all participants are off study. 
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Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-004 
Statistical Analysis Plan, Version 1.0 
 
April 12, 2022 
 
13 
3.5. 
Blinding 
This is an open-label study. 
3.6. 
Sample Size Considerations 
Dose Escalation: The Dose Escalation phase will primarily be used to determine the MTD or an 
RP2D and evaluate the safety and tolerability of the combination using modified 3+3 dose 
escalation rules. Three dose escalation cohorts are planned, and an additional cohort may be 
added. Approximately 18 participants will be enrolled in the Dose Escalation phase. 
Dose Expansion: A Simon’s two-stage design will apply to this phase of the study. The number 
of participants required for each cohort was calculated to demonstrate 20% improvement in 
objective response rate (ORR) (from 20% historical ORR in the setting to 40% for the 
combination) under 80% power and one-sided alpha of 0.05. In the initial stage, up to 18 
participants will be evaluated. Greater than 4 responses will be required to enroll additional 
participants to demonstrate the target efficacy of >10 responses in a total of 33 participants. 
Thus, this part of study may enroll approximately 99 participants (33 participants per indication-
specific cohort). Participants who do not receive at least one dose of the combination will be 
replaced. In addition, if the study drug treatment is discontinued prior to the scheduled, first post-
dose tumor assessment (Cycle 3 Day 1) due to reasons other than disease progression (clinical or 
radiological) or an AE(s) at least possibly related to rebastinib, participants may be replaced. 
Replaced participants will not be included in the responder analysis. For example, if 10% of 
participants will be replaced, approximately 110 participants may be enrolled. 
 
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-004 
Statistical Analysis Plan, Version 1.0 
 
April 12, 2022 
 
14 
4. 
STUDY ENDPOINTS 
The following study endpoints are for the Dose Escalation and Dose Expansion phases. 
4.1. 
Efficacy Endpoints 
For efficacy endpoints based on imaging, RECIST v1.1 (Eisenhauer, Therasse and Bogaerts) will 
be used for all summaries except for participants with pleural mesothelioma. For participants 
with pleural mesothelioma, mRECIST will be used instead of RECIST v1.1 (Armato III and 
Nowak).  
The primary endpoint in the Dose Expansion phase is the ORR, which is defined as the 
proportion of participants with a best overall response of CR or PR. Below is a list of the 
efficacy endpoints to be analyzed in this study. 
4.1.1. 
Objective Response Rate  
ORR is defined as the proportion of participants with best overall response of CR or PR 
according to RECIST v1.1 (or mRECIST for participants with pleural mesothelioma). 
Participants without a best overall response CR or PR will be considered as non-responders. 
4.1.2. 
Clinical Benefit Rate  
CBR at 6, 12, and 21 weeks is defined as proportion of participants with overall response of CR, 
PR, or stable disease (SD) at 6, 12, and 21 weeks. Participants without a CR, PR, or SD at the 
time point of interest (or a later time point) will be considered as not having clinical benefit at 
that time point. 
4.1.3. 
Duration of Response  
Duration of response (DOR) is defined as time from first PR or CR until the earliest documented 
evidence of PD or death due to any cause, whichever occurs first.  
4.1.4. 
Time to Response  
Time to response is defined as the time from first dose of study drug until to the first assessment 
demonstrating PR or CR.  
4.1.5. 
Progression-Free Survival  
PFS is defined as the time from first dose of study drug until the earliest documented evidence of 
PD or death due to any cause, whichever occurs first. 
4.1.6. 
Time to Progression  
TTP is defined as the time from first dose of study drug until the earliest documented evidence of 
PD.  
4.1.7. 
Overall Survival  
Overall survival (OS) is defined as the time from first dose of study drug until the date of death 
from any cause.  
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DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-004 
Statistical Analysis Plan, Version 1.0 
 
April 12, 2022 
 
18 
 
5.3. 
Study Visits 
For safety parameters, unscheduled visits will be mapped to a scheduled visit if possible, using a 
window based on all the available actual visit dates for the scheduled visit. Data that are 
collected from unscheduled visits and cannot be mapped to a scheduled visit will not be included 
in the by-visit summary tables but will be presented in the listings. Early termination visits for 
safety measurements will not be mapped to any scheduled post-baseline visit but will be used as 
the last assessment during treatment period. 
5.4. 
Study Day 
If the date of interest occurs on or after the date of first dose of study drug, then study day will be 
calculated as (date of interest – date of first dose) + 1.  
If the date of interest occurs prior to the date of first dose of study drug, then study day will be 
calculated as (date of interest – date of first dose/randomization). There is no study day 0 
5.5. 
Coding Dictionaries 
Medical history, AEs, and concurrent procedures will be coded using Medical Dictionary for 
Regulatory Activities (MedDRA) version 24.1. Prior and concomitant medications will be coded 
using the World Health Organization Drug Dictionary version B3 (September 2021).  
 
 
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-004 
Statistical Analysis Plan, Version 1.0 
 
April 12, 2022 
 
19 
6. 
ANALYSIS POPULATIONS 
6.1. 
Enrolled Population 
Enrolled Population includes all participants who signed the informed consent form. 
6.2. 
Safety Population 
Safety Population includes all participants who are exposed to any amount of either study drug. 
This population will be used for analysis of safety data. 
6.3. 
Modified Intent-to-Treat Population 
Modified Intent-to-Treat Population (mITT) includes all participants who had at least one full 
dose of the combined study drugs, had measurable disease at baseline, and had at least one post-
baseline assessment. Participants without a post-baseline assessment who discontinued study 
treatment due clinical progression, death, or a related AE will also be included in the mITT 
population. Participants without measurable disease at baseline or who do not have the disease of 
interest in the Dose Expansion phase will be excluded. This population will be used for analysis 
of efficacy data. 
6.4. 
Pharmacokinetic Population 
PK Population includes all participants who received at least 1 dose of rebastinib and had at least 
1 non-missing PK concentration in plasma reported for rebastinib. This population will be used 
for analysis of PK data. Analysis of PK data will be described in a separate document.  
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


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DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


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DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-004 
Statistical Analysis Plan, Version 1.0 
 
April 12, 2022 
 
29 
Table 2: 
Safety Laboratory Tests 
Serum Chemistry 
Hematology 
Urinalysisb 
Alanine aminotransferase 
Albumin 
Alkaline phosphatase 
Aspartate aminotransferase  
Bicarbonate 
Blood urea nitrogen 
Calcium  
Chloride  
Creatinine 
Creatine Phosphokinase  
Follicle-stimulating hormonea  
Glucose 
Lactate dehydrogenase  
Magnesium  
Phosphorus 
Potassium 
Sodium 
Total and direct bilirubinc 
Total protein 
Hemoglobin 
Hematocrit  
Platelets  
Leukocytes   
Differential (absolute): 
•  Eosinophils 
•  Basophils 
•  Neutrophils 
•  Lymphocytes 
•  Monocytes 
Coagulation Studies 
Activated partial thromboplastin time 
Prothrombin time 
International Normalized Ratio 
Urine protein  
Urine blood  
Specific gravity  
Urine ketones 
Urine glucose 
a. This may be required to demonstrate a participant is non-childbearing potential 
b. If any result is abnormal, a microscopic analysis must be performed by the local laboratory. 
c. Indirect bilirubin should be calculated. 
Shift from baseline to the worst post-baseline grade according to NCI-CTCAE v5.0 will be 
summarized for all applicable laboratory parameters. Frequencies and percentages of any 
worsening and worsening to Grade 3 or 4 will be presented. 
Laboratory parameters will also be presented in data listings. 
7.5.5. 
Vital Signs, Weight, and Height 
Vital sign measurements will consist of sitting blood pressure, heart rate, respiratory rate, and 
body temperature assessed at Screening, Cycle 1 Day 1, Cycle 1 Day 8 (±1 day), Day 1 of Cycles 
2 and above, and the EOT visit (within 14 days of the decision to stop study drug). These should 
be assessed following a 5-minute rest (seated or supine position). Height will only be taken at 
Screening. 
Vital signs and weight will be summarized using continuous descriptive statistics and will be 
presented by study visit and time point for the actual value and change from baseline. 
Vital signs, weight, and height will also be presented in data listings. 
7.5.6. 
ECOG Performance Status 
ECOG performance status will be presented in data listings. 
7.5.7. 
Electrocardiograms  
Single 12-lead ECGs will be performed at Screening, Cycle 1 Day 1, Day 1 of Cycles 2 and 
above, and the EOT visit. ECG data will be transmitted to the central ECG diagnostic service and 
all interval measurements will be reviewed and adjusted using the central ECG core labs 
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


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DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-004 
Statistical Analysis Plan, Version 1.0 
 
April 12, 2022 
 
31 
Efficacy parameters will also be presented in data listings. 
7.6.1. 
Objective Response Rate 
The best response (CR, PR, SD, PD, or not evaluable [NE]) according to RECIST v1.1 (or 
mRECIST for participants with pleural mesothelioma) will be derived for each participant. 
Participants with no adequate post-baseline disease assessment who otherwise qualify for the 
mITT population will have a best response of NE and included as non-responders in the analysis 
of ORR. Response assessed after disease progression will not be considered in determination of 
the best overall response. The ORR is calculated as the proportion of participants who have 
achieved best overall response of CR or PR after the initiation of study treatment. Best overall 
response and ORR with and without confirmation will be summarized. Confirmed responses are 
those responses that persist on repeat imaging at least 28 days after the initial response 
assessment. The derivation for best overall response with confirmation is summarized in the 
table below. 
 
 
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-004 
Statistical Analysis Plan, Version 1.0 
 
April 12, 2022 
 
32 
Table 3: 
Summary of the Best Overall Response Status when Confirmation of 
Response is Required 
Overall Response at 
First Time Point 
Overall Response at 
Subsequent Time Point 
Best Overall Response with 
Confirmation 
CR 
CR 
CR 
CR 
PR 
SD, PD, or PRa 
CR 
SD or PD 
SD provided minimum criteriab for SD 
duration met, otherwise PD 
CR 
NE 
SD provided minimum criteria for SD 
duration met, otherwise NE 
PR 
CR or PR 
PR 
PR 
SD 
SD 
PR 
PD 
SD provided minimum criteria for SD 
duration met, otherwise PD 
PR 
NE 
SD provided minimum criteria for SD 
duration met, otherwise NE 
CR = complete response; PR = partial response; SD = stable disease, PD = progressive disease; NE = not evaluable. 
a If a CR is truly met at first time point, then any disease seen at a subsequent time point, even disease meeting PR 
criteria relative to baseline, makes the disease PD at that point (since disease must have reappeared after CR). Best 
response would depend on whether minimum duration for SD was met. However, sometimes ‘CR’ may be claimed 
when subsequent scans suggest small lesions were likely still present and in fact the patient had PR, not CR at the 
first time point. Under these circumstances, the original CR should be changed to PR and the best confirmed 
response is PR. 
b Minimum criteria for SD duration is 4 weeks (6 weeks for first assessment with a -2 week window). 
The ORR with and without confirmation will be summarized as a binary endpoint with exact 2-
sided 90% CIs estimated using the Clopper-Pearson method. 
The frequency and percentage of participants with a best overall response of CR, PR, SD, PD, or 
NE with and without confirmation will also be summarized.  
7.6.2. 
Clinical Benefit Rate 
The CBR at 6, 12, and 21 weeks, defined as the as proportion of participants with overall 
response of CR, PR, or SD at 6, 12, and 21 weeks, will be derived for each participant. Per 
protocol, participants could have a disease assessment 14 days prior to the 6-, 12-, and 21-week 
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


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DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31


Deciphera Pharmaceuticals, LLC 
DCC-2036-01-004
Statistical Analysis Plan, Version 1.0 
April 12, 2022
35 
8.
REFERENCES
Armato III, SG and AK Nowak. "Revised Modified Response Evaluation Criteria in Solid 
Tumors for Assessment of Response in Malignant Pleural Mesothelioma (Version 1.1)." 
Journal of Thoracic Oncology 13.7 (2018): 1012-1021. 
Eisenhauer, E A, et al. "New response evaluation criteria in solid tumours: revised RECIST 
guideline (version 1.1)." European Journal of Cancer 45.2 (2009): 228-247. 
DocuSign Envelope ID: 1FA4BF86-761A-4D0A-9E08-B8D5B12C8A31