Clinical trial NCT03601897
Conditions: Locally Advanced or Metastatic Solid Tumor
Keywords: rebastinib paclitaxel breast cancer ovarian cancer endometrial cancer gynecological carcinosarcoma malignant mixed Mullerian tumor (MMMT)
Sponsor: Deciphera Pharmaceuticals, LLC
Start date: 2018-10-25; Completion date: 2022-05-23
Enrollment: 177 participants; Allocation: NON_RANDOMIZED; Intervention model: SEQUENTIAL

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

Detailed description:

Arms: arm 1: Dose escalation of rebastinib 50 milligram (mg) twice daily (BID) orally (PO) in combination with paclitaxel administered by intravenous (IV) infusion at 80 mg/meter squared (m\^2) on days 1, 8, and 15 of repeated 28-day cycles. arm 2: Dose escalation of rebastinib 100 mg BID PO in combination with paclitaxel IV infusion at 80 mg/m\^2 on days 1, 8, and 15 of repeated 28-day cycles. arm 3: Dose expansion in triple-negative breast cancer (TNBC). Rebastinib 50 mg BID PO in combination with paclitaxel IV infusion at 80 mg/m\^2 on days 1, 8, and 15 of repeated 28-day cycles. arm 4: Dose expansion in TNBC. Rebastinib 100 mg BID PO in combination with paclitaxel IV infusion at 80 mg/m\^2 on days 1, 8, and 15 of repeated 28-day cycles. arm 5: Dose expansion in inflammatory breast cancer. Rebastinib 50 mg BID PO in combination with paclitaxel IV infusion at 80 mg/m\^2 on days 1, 8, and 15 of repeated 28-day cycles. arm 6: Dose expansion in inflammatory breast cancer. Rebastinib 100 mg BID PO in combination with paclitaxel IV infusion at 80 mg/m\^2 on days 1, 8, and 15 of repeated 28-day cycles. arm 7: Dose expansion in ovarian cancer. Rebastinib 50 mg BID PO in combination with paclitaxel IV infusion at 80 mg/m\^2 on days 1, 8, and 15 repeated 28-day cycles. arm 8: Dose expansion in ovarian cancer. Rebastinib 100 mg BID PO in combination with paclitaxel IV infusion at 80 mg/m\^2 on days 1, 8, and 15 of repeated 28-day cycles. arm 9: Dose expansion in endometrial cancer. Rebastinib 50 mg BID PO in combination with paclitaxel IV infusion at 80 mg/m\^2 on days 1, 8, and 15 of repeated 28-day cycles. arm 10: Dose expansion in endometrial cancer. Rebastinib 100 mg BID PO in combination with paclitaxel IV infusion at 80 mg/m\^2 on days 1, 8, and 15 of repeated 28-day cycles. arm 11: Dose expansion in gynecological carcinosarcoma (GCS). Rebastinib 50 mg BID PO in combination with paclitaxel IV infusion at 80 mg/m\^2 on days 1, 8, and 15 of repeated 28-day cycles.
Interventions: intervention 1: Rebastinib intervention 2: Paclitaxel
Intervention descriptions: intervention 1: Administered orally intervention 2: Paclitaxel administered by IV infusion at 80 mg/m\^2

Protocol documents:
Prot_000.pdf: 
 
 
 
 
Protocol: DCC-2036-01-003 
Official Title: An Open-Label, Multicenter, Phase 1b/2 Study of Rebastinib (DCC-2036) in Combination 
with Paclitaxel to Assess Safety, Tolerability, and Pharmacokinetics in Patients with Advanced or 
Metastatic Solid Tumors 
NCT Number: NCT03601897 
Approval Date: 07 FEBRUARY 2020 
 


07 FEBRUARY 2020 
CONFIDENTIAL 
Deciphera Pharmaceuticals, LLC 
CLINICAL STUDY PROTOCOL 
Protocol DCC-2036-01-003 
An Open-Label, Multicenter, Phase 1b/2 Study of Rebastinib (DCC-2036) in 
Combination with Paclitaxel 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: 785-830-2100 
Fax: 781-701-8168 
Research Facility: 
643 Massachusetts St., Suite 200 
Lawrence, KS 66044 
Phone: 785-830-2100 
Fax: 785-830-2150 
IND Number: 
128702 
Document Version (Date): 
Version 1.0 (19 APR 2018) 
Amendment 1 (30 MAY 2018) 
Amendment 2 (7 SEP 2018) 
Amendment 3 (08 JAN 2019) 
Amendment 4 (20 JAN 2020) 
Amendment 5 (07 FEB 2020) 
NCT #: NCT03601897


Clinical Study Protocol DCC-2036-01-003, Amendment 5 
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SPONSOR SIGNATURE 
 
Date 
 
07 FEBRUARY 2020 
CONFIDENTIAL 
Deciphera Pharmaceuticals, LLC 
PPD
PPD
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 Paclitaxel to Assess Safety, Tolerability, and 
Pharmacokinetics in Patients with Advanced or Metastatic Solid Tumors 
Protocol Number: 
DCC-2036-01-003 
Study Phase: 
1b/2 
Study Centers: 
Part 1: Approximately 7 centers in the United States (US) 
Part 2: Approximately 20 centers in the US 
Number of 
Patients Planned: 
Part 1: Up to 36 evaluable patients to determine the recommended Phase 2 
dose (RP2D) 
Part 2: Up to 165 evaluable patients in indication-specific cohorts  
Objectives: 
Part 1: 
Primary Objectives: 
• To evaluate the safety and tolerability of 50 mg and 100 mg 
rebastinib twice daily (BID) when administered in combination with 
paclitaxel. 
• To determine the recommended phase 2 dose (RP2D) of rebastinib 
in combination with paclitaxel. 
Secondary Objectives: 
• To assess the preliminary efficacy of rebastinib administered in 
combination with paclitaxel by evaluating the objective response 
rate (ORR). 
• To assess the pharmacokinetics (PK) of rebastinib and paclitaxel 
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 paclitaxel. 
Part 2: 
Primary Objectives: 
• To evaluate the safety and tolerability of rebastinib at the RP2D in 
combination with paclitaxel. 
• To evaluate the ORR as the primary efficacy measure of rebastinib 
in combination with paclitaxel. 
Secondary Objectives: 
• To assess the PK of rebastinib and paclitaxel when administered in 
combination. 


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• To evaluate efficacy measures, such as PFS, CBR, response 
duration, time to response, TTP, and OS of rebastinib in 
combination with paclitaxel. 
Parts 1 and 2:  
Exploratory Objectives: 
• To evaluate changes in select blood and plasma biomarkers when 
rebastinib is administered in combination with paclitaxel. 
• 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 paclitaxel. 
• To assess polymorphisms in genes encoding drug metabolic 
enzymes and/or transporters involved in metabolism and disposition 
of rebastinib in combination with paclitaxel. 
• Assess the effects of rebastinib administered in combination with 
paclitaxel using patient reported outcome (PRO) measures. 
Study Design: 
This is an open-label Phase 1b/2 multicenter study in patients with 
advanced or metastatic solid tumors where paclitaxel may be considered 
appropriate treatment. Rebastinib will be administered in combination with 
paclitaxel in repeated 28-day cycles to primarily assess the safety, 
tolerability and preliminary efficacy of the combination. Adverse events 
(AEs) will be assessed, and laboratory values, vital sign measurements, 
electrocardiograms (ECGs), ophthalmologic examinations, physical 
examinations and Eastern Cooperative Oncology Group (ECOG) 
Performance Status (PS) will be obtained to evaluate the safety and 
tolerability of rebastinib when administered in combination with paclitaxel. 
Pharmacokinetic (PK) and pharmacodynamic (PD) samples will be 
collected at pre-specified time points. 
The study consists of two parts (Part 1 and Part 2). In Part 1, patients will be 
assigned to one of two pre-defined dose levels of rebastinib (50 or 100 mg 
BID) in combination with paclitaxel administered by intravenous (IV) 
infusion at 80 mg/m2. Each arm is planned to enroll at least 12 evaluable 
patients. Additionally, if rebastinib at 100 mg BID is deemed unsafe, an 
additional arm dosing at 75 mg BID of rebastinib in combination with 
paclitaxel may be initiated. Up to 36 evaluable patients will be dosed in Part 
1. Safety will be continuously monitored and any arm may be terminated 
early if deemed unsafe by the Sponsor. 
Safety and tolerability, PK and PD, and preliminary efficacy data obtained 
in Part 1 will be used to determine the RP2D. Data for at least 12 evaluable 
patients through Cycle 1 must be available in an arm to declare the dose as 
the RP2D. Patients must receive ≥80% of planned doses of rebastinib and 
paclitaxel in Cycle 1 to be considered as evaluable. Enrollment of patients 
will pause for determination of the RP2D prior to initiation of Part 2. An 
RP2D will be chosen by the Sponsor in consultation with the Investigators. 
Upon determination of the RP2D, Part 2 will be initiated to dose up to 
165 evaluable patients using the RP2D across five indication-specific 


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cohorts. A Simon two-stage design will be applied to Part 2 to further 
evaluate the safety, tolerability, and preliminary efficacy of rebastinib in 
combination with paclitaxel in triple-negative breast (Cohort 1), 
inflammatory breast (Cohort 2), ovarian (Cohort 3), endometrial (Cohort 4), 
and gynecological carcinosarcoma (Cohort 5) cancers. Each cohort will 
initially enroll up to 18 evaluable patients in the first stage. Patients must 
receive at least 1 dose of the combination and have 1 post-baseline 
assessment, or be discontinued prior to the post-baseline disease assessment 
due to an AE at least possibly related to rebastinib, to be considered 
evaluable. Tumor response will be assessed according to Response 
Evaluation Criteria in Solid Tumors (RECIST) Version 1.1. 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. Patients who meet criteria defined in 
Section 8.3 will be replaced and not be included in the responder analysis. If 
>4 responses (defined as partial response [PR] or complete response [CR]) 
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. Enrollment in 
each cohort will pause between the first and second stage of the Simon two-
stage for evaluation of response. 
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. Part 1, All Arms 
i. 
Histologically confirmed diagnosis of a locally advanced or 
metastatic solid tumor for which paclitaxel 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. Part 2  
A. Part 2, 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 
(Hammond et al, 2010). 
ii. 
Received at least one prior line but no more than three prior 
lines of systemic chemotherapy in the metastatic setting. 
iii. 
Has not received taxane-containing regimens within 
6 months prior to the first dose of study drug. 


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B. Part 2, Cohort 2: Inflammatory Breast Cancer (IBC) 
i. 
Histologically or cytologically confirmed stage IV breast 
carcinoma with a previous clinical diagnosis of IBC based on 
the presence of inflammatory changes in the involved breast, 
such as diffuse erythema and/or edema (peau d'orange), with 
or without an underlying palpable mass, and involving the 
majority of the skin of the breast; pathological evidence of 
dermal lymphatic invasion should be noted but is not 
required for diagnosis. 
ii. 
Received at least one prior line of systemic chemotherapy in 
the metastatic setting. 
iii. 
Has not received taxane-containing regimens within 6 
months prior to the first dose of study drug. 
C. Part 2, Cohort 3: 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. 
a. Progressed or relapsed within 6 months after the 
completion of a platinum-containing chemotherapy 
regimen. 
b. Patients that progressed during treatment or within 
1 month after the completion of the first 
platinum-containing chemotherapy regimen (primary 
platinum refractory) are excluded. 
ii. 
Received no more than five prior lines of systemic anticancer 
therapy. 
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 
regimen. 
iii. 
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. 
D. Part 2, Cohort 4: Endometrial Cancer 
i. 
Histologically confirmed adenocarcinoma of the 
endometrium. 
ii. 
Received at least one prior line of platinum-based therapy in 
the recurrent, metastatic, or high-risk disease setting.  


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a. For patients with known microsatellite instability-
high (MSI-H) or mismatch repair deficiency, 
progressed after a regimen including an anti-PD1 
agent. 
E. Part 2, Cohort 5: Gynecological Carcinosarcoma 
i. 
Histologically confirmed advanced (stage III or IV), 
persistent or recurrent gynecological carcinosarcoma. 
a. Homologous or heterologous type carcinosarcoma 
(malignant mixed Müllerian tumor [MMMT]) are 
allowed. 
ii. 
Must have received at least 1 prior chemotherapy regimen 
(including high-dose, consolidation, or extended therapy 
after surgical or nonsurgical assessment for gynecological 
carcinosarcoma). 
4. At least one measurable lesion according to RECIST Version 1.1.  
5. Eastern Cooperative Oncology Group (ECOG) performance status 
(PS) of ≤2. 
6. Able to provide a 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 
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: serum creatinine ≤1.5×ULN or creatinine 
clearance ≥30 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 


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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 6.8.9. 
9. If male, must agree to use two methods of contraception, with one of 
them being highly effective, 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 6.8.9. 
10. Patient must provide signed consent to participate in the study and is 
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).  
3. Peripheral neuropathy of any etiology > Grade 1. 
4. 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.  
5. 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. 
6. 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. See Section 
5.11.6 for further details. 
7. Known retinal neovascularization, macular edema or macular 
degeneration. 
8. 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. 
 


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9. 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. 
10. Left ventricular ejection fraction (LVEF) <50% at screening. 
11. Arterial thrombotic or embolic events such as cerebrovascular 
accident (including ischemic attacks) or moderate hemoptysis within 
6 months prior to the first dose of study drug. 
12. Venous thrombotic event (e.g., deep vein thrombosis) within the 
3 months prior to the first dose of study drug; following a 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.  
13. Active infection ≥ Grade 3 requiring IV anti-infective treatment 
within 7 days prior to the first dose of study drug.  
14. Known human immunodeficiency virus or hepatitis C infection only 
if the patient is taking medications that are excluded per protocol, 
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 the first dose of study drug; 
following major surgeries >4 weeks prior to the first dose of study 
drug, all surgical wounds must be healed and free of infection or 
dehiscence. 
18. 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. 


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Study Drug, 
Formulation, Dose 
and Route of 
Administration: 
Rebastinib will be administered in combination with paclitaxel. 
Rebastinib will be supplied by the Sponsor as 25 mg and 75 mg formulated 
tablets for oral administration. Paclitaxel will be supplied by the Sponsor. 
Patients will each receive an oral dose of rebastinib at 50 mg or 100 mg 
BID in 28-day cycles. In Part 1, if rebastinib at 100 mg BID is not tolerated, 
an additional arm dosing at 75 mg BID may be evaluated. In Part 2, 
rebastinib will be administered at the RP2D in combination with paclitaxel. 
Paclitaxel will be administered by IV infusion at 80 mg/m2 over 
approximately 60 minutes on Day 1, Day 8 and Day 15 of repeated 28-day 
cycles (see Section 5.2.2). All paclitaxel infusions will be administered at 
the study site. Rebastinib will be administered orally first, followed by 
paclitaxel IV infusion. Patients should be pre-medicated prior to paclitaxel 
administration in order to prevent severe hypersensitivity reactions per 
institutional practices. If the retreatment criteria as outlined in Section 5.2.2 
are not met and a dose of paclitaxel is missed, a single make-up dose of 
paclitaxel may be administered on Day 22 of a given cycle as long as the 
retreatment criteria are met. Assessments required on Day 8 of the 
corresponding cycle must be performed. For patients that are unable to 
tolerate paclitaxel, rebastinib may be administered as a single agent starting 
in Cycle 2. 
Study Endpoints: 
Parts 1 and 2 Endpoints: 
Safety: 
• Overall AEs 
• Serious adverse events (SAEs) 
• Adverse events of special interest (AESIs) 
• Dose reduction, dose interruptions, or discontinuation of study drug 
due to toxicity  
• Physical examinations  
• ECOG PS  
• Ophthalmic examinations  
• Changes from baseline in laboratory parameters  
• Electrocardiogram (ECGs)  
• Echocardiograms or multigated acquisition (MUGAs)  
• Vital signs 
Pharmacokinetics: 
Pharmacokinetic (PK) endpoints when rebastinib is administered in 
combination with paclitaxel and as a single agent include, but are not 
limited to: 
• Time to maximum observed concentration (Tmax: rebastinib only)  


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• 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) 
• Half-life (T1/2) 
• Volume of distribution (Vd) 
• Clearance (CL) 
Efficacy Endpoints: 
Radiographic tumor assessments (computed tomography [CT] or MRI) will 
be performed by RECIST Version 1.1. 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 (CR + PR) of the combination therapy. 
• Clinical benefit rate (CBR) (CBR = CR + PR + stable disease [SD]) 
at 8, 16, and 28 weeks of the combination therapy. 
• Time to response (defined as time from Cycle 1 Day 1 to PR or CR). 
• Progression-free-survival (PFS; defined as time from Cycle 1 Day 1 
to disease progression or death due to any cause). 
• Time to progression (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).  
• Overall survival (OS). 
Pharmacogenomics: 
The pharmacogenomics endpoints of the study include, but are not limited 
to:  
• Assessment of polymorphic variations in genes encoding drug 
metabolic enzymes and/or transporters involved in metabolism and 
disposition of rebastinib 
• Assessment of polymorphisms in genes that may be associated with 
clinical response and/or study drug-related toxicity. 


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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, 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: 
• Assess the safety profile of rebastinib in combination with paclitaxel 
using the National Cancer Institute’s Patient-Reported Outcomes 
Version of the Common Terminology Criteria for Adverse Events 
(NCI-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. Categorical variables will be summarized using frequency 
distributions and percentages. Data may be displayed by each of the Part 1 
arms and Part 2 indication-specific cohorts. 
This is a Phase 1b/2 study. Time‑to‑event data will be summarized via 
Kaplan-Meier (KM) using medians with associated 2-sided 90% confidence 
intervals (CIs). Proportions, when appropriate, will be reported with exact 
2-sided 90% confidence intervals. 
Analysis Populations 
Safety Population: All patients who are exposed to any amount of either 
study drug. This population will be used for analysis of safety data. 
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 one post-baseline 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. This 
population will be used for analysis of efficacy data. 
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 


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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 
The point estimate and 2-sided 90% exact CIs will be presented for all the 
response endpoints and by the arm or indication-specific cohort. For time-
to-event endpoints, Kaplan-Meier method will be used. Point estimates and 
90% CIs will be presented. 
ORR (defined as CR+PR) is the primary efficacy endpoint of Part 2 and a 
secondary endpoint of Part 1. The secondary endpoints of both parts include 
CBR, time to response, PFS, TTP, DOR, and OS. All response endpoints 
will be defined based on RECIST Version 1.1. 
Sample Size Justification 
Part 1: Part 1 will primarily be used to evaluate the safety and tolerability of 
the combination. At least 12 evaluable patients will be enrolled in each arm, 
which is considered appropriate for analysis of safety and tolerability to 
determine the RP2D. 
Part 2: A Simon two-stage design will apply to Part 2 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 in order to demonstrate the 
target efficacy of >10 responses in a total of 33 patients. Thus, this part of 
study may enroll up to 165 (33 patients per indication-specific cohort) 
evaluable patients. If a non-evaluable rate of 10% is considered, 
approximately 183 patients may be enrolled. 
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 for 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; RP2D = recommended Phase 2 dose. 
PART 1: Histologically confirmed diagnosis of a locally advanced or 
metastatic solid tumor where paclitaxel is considered appropriate 
treatment.  Patients must have progressed despite standard therapies, or 
for whom conventional therapy is not considered effective or tolerable, as 
judged appropriate by the Investigator.*  
* In Part 1, an arm may be discontinued anytime upon discussion between 
the Sponsor and Investigator. 
RP2D Determination 
N = 12 
If tolerated N = 12 
Rebastinib 
50 mg BID + 
Paclitaxel 
If 100 mg not tolerated (N = 12) 
Rebastinib 
100 mg BID + 
Paclitaxel 
Rebastinib 
75 mg BID + 
Paclitaxel 
PART 2: Dosing of patients using the RP2D across 
five indication-specific cohorts.  A Simon two-stage 
design will be applied. 
N ≤ 18 
Cohort 1: 
Triple-Negative 
Breast Cancer 
Cohort 2: 
Inflammatory 
Breast Cancer 
Cohort 3: 
Ovarian 
Cancer 
Cohort 4: 
Endometrial 
Cancers 
Cohort 5: 
Gynecological  
Carcinosarcoma 
If >4 responses, 
enroll additional 
patients in the 
cohort for a total of 
up to 33 patients 
If ≤4 responses, 
discontinue the 
cohort 


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Table 1: 
Schedule of Assessments 
Assessments / Proceduresa 
Screeningc 
Cycle 1 
Cycles ≥2 
EOT 
Visitf 
30 Day 
Safety 
Follow-upf 
Cycle Day 
-28 to -1 
1 
(Baseline) 
8 
(±2 days) 
15 
(±2 days) 
1 
(±2 days) 
8e 
(±2 days) 
15e 
(±2 days) 
1 
(+7 days) 
1 
(+7 days) 
Informed Consent 
X 
 
 
 
 
 
 
 
 
Inclusion/Exclusion Criteria 
X 
 
 
 
 
 
 
 
 
Medical and Cancer History 
X 
 
 
 
 
 
 
 
 
Prior Medications/Proceduresg 
X 
 
 
 
 
 
 
 
 
Pregnancy Testh 
X 
X 
 
 
X 
 
 
X 
 
Hematologyb 
Xc 
Xd 
X 
X 
X 
X 
X 
X 
 
Serum Chemistriesb 
Xc 
Xd 
X 
X 
X 
X 
X 
X 
 
Coagulationi 
Xc 
Xd 
 
 
X 
 
 
X 
 
Urinalysisj 
Xc 
Xd 
 
 
X 
 
 
X 
 
Physical Examinationk 
X 
Examinations will be driven by clinical findings and/or patient 
complaints 
X 
 
ECOG PS 
X 
X 
X 
X 
X 
 
 
X 
 
Vital Signs and Weightl 
X 
X 
X 
X 
X 
X 
X 
X 
 
Height 
X 
 
 
 
 
 
 
 
 
12-lead ECGm 
X 
X 
 
X 
X 
 
 
X 
 
Echocardiogram/MUGAn 
X 
 
 
 
Xn 
 
 
X 
 
Ophthalmologic Examination0 
X 
 
 
 
X 
 
 
X 
 
Adverse Event Reporting 
Continuous from signing informed consent through 30 Day Safety Follow-up 
Concomitant Medications/ Procedures 
Continuous from signing informed consent through 30 Day Safety Follow-up 
NCI-PRO-CTCAEp 
 
X 
X 
X 
X 
 
X 
X 
 
EORTC-QLQ-C30p 
 
X 
X 
X 
X 
 
X 
X 
 
GP-5 from FACT-Gp 
 
X 
X 
X 
X 
 
X 
X 
 
Dosing of Rebastinibq 
 
Continuous from Cycle 1 Day 1 (Baseline) through end of treatment 
 
Dosing of Paclitaxelr 
 
Xr 
X 
Xr 
X 
X 
X 
 
 
Radiologic Imagings 
X 
 
 
 
Xs 
 
 
X 
 
Tumor Markerst 
Xc 
X 
 
 
X 
 
 
X 
 
PK Samplingu, v 
 
X 
 
X 
X 
 
 
X 
 


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Assessments / Proceduresa 
Screeningc 
Cycle 1 
Cycles ≥2 
EOT 
Visitf 
30 Day 
Safety 
Follow-upf 
Cycle Day 
-28 to -1 
1 
(Baseline) 
8 
(±2 days) 
15 
(±2 days) 
1 
(±2 days) 
8e 
(±2 days) 
15e 
(±2 days) 
1 
(+7 days) 
1 
(+7 days) 
Archived Tissue Samplew 
X 
 
 
 
 
 
 
 
 
Fresh Tumor Biopsyx 
Xx 
 
 
 
Xx 
 
 
X 
 
Pharmacogenomics 
 
X 
 
 
 
 
 
 
 
Whole Blood Immunophenotyping 
Biomarker Samplesy 
 
X 
 
X 
Xy 
 
 
X 
 
Plasma Biomarker Samplesy 
 
X 
 
X 
Xy 
 
 
X 
 
AE = adverse event; BID = twice daily; CA-125 = cancer antigen 125; CNS = central nervous system; CT = computed tomography; ECG = electrocardiogram; 
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; MRI = magnetic resonance imaging; MUGA = multigated acquisition; 
NCI-PRO-CTCAE = National Cancer Institute’s Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events; 
PK = pharmacokinetic(s); RECIST = Response Evaluation Criteria in Solid Tumors. 
a. 
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 AEs. 
b. Hematology and serum chemistries may be performed up to 3 days prior to the corresponding study visit. 
c. 
Screening must occur within 28 days prior to the first dose of study drug. Screening samples for hematology, serum chemistries, coagulation, urinalysis, and 
tumor markers, if applicable, must be drawn within 14 days prior to the first dose of study drug. Standard procedures performed as part of the practice of 
medicine prior to consent (e.g., imaging, physical exam) may be used to determine eligibility if completed within 28 days prior to the initial dose of study 
drug (see Sections 6.1 and 6.8).  
d. If the screening clinical laboratory testing and ECOG PS were performed within 3 days before the Cycle 1 Day 1 dose, the assessments 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 
e. 
For patients that are unable to tolerate paclitaxel, rebastinib may be administered as a single agent starting in Cycle 2. If paclitaxel is discontinued, visits on 
Day 8 and Day 15 are not required in Cycle 2 and beyond. 
f. 
The EOT Visit should be performed within 7 days after a patient’s last dose of study drug. Patients should be contacted between 30 and 37 days after the last 
dose of study drug, or prior to initiation of new anticancer therapy, to collect new information pertaining to AEs and concomitant medications/procedures. 
These assessments can be performed over the phone. 
g. 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.  
h. A serum pregnancy test will be performed at screening for female patients of childbearing potential (see Section 6.8.8). A negative result must be 
documented within 3 days prior to the first dose of study drug; results from screening tests performed within this timeframe may be used in lieu of a Cycle 1 
Day 1 (Baseline) result. 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.  


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i. 
Coagulation testing will be performed on Day 1 of each cycle and at the time of a tumor biopsy. 
j. 
If urinalysis testing is abnormal, a microscopic analysis will be performed. 
k. A full physical examination will be performed at screening and the EOT Visit. At all other visits, examinations will be driven by clinical findings and/or 
patient complaints. If the patient reports a complaint or a possible AE, a targeted physical examination may be conducted at the Investigator’s discretion. 
After consent, any clinically significant abnormal findings in physical examinations must be reported as AEs (see Sections 6.8.1 and 7).  
l. 
Vital sign measurements will be collected after the patient has been at rest (seated or supine position) for at least 5 minutes. Measurements will include 
sitting blood pressure, heart rate, respiratory rate, and temperature. On days when PK sampling is performed, vital sign measurements should be obtained as 
indicated in Table 2 and Table 3. In addition, weight will be obtained at each study visit.  
m. 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 6.8.4). 
n. An echocardiogram or multigated acquisition (MUGA) scan will be performed during screening, Day 1 of Cycles 2, 3, 6, every third cycle thereafter (i.e., 
Day 1 of Cycles 9, 12, 15, etc.), and at the EOT Visit. The same modality (echocardiogram or MUGA scan) used during screening should be used for all 
subsequent assessments (see Section 6.8.5). Assessments may be performed up to 7 days prior to the corresponding study visit or post-dose at the 
corresponding study visit. Additionally, an assessment done within 14 days prior to the last dose of study drug can be used in lieu of an EOT assessment. 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. 
o. Ophthalmologic examinations will be performed during screening, Day 1 (±7 days) of every third cycle thereafter (i.e., Cycles 3, 6, 9, etc.), and EOT Visit 
(±7 days). 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. Additionally, an examination done within 30 days prior 
to the last dose of study drug can be used in lieu of an EOT exam (see Section 6.8.6). 
p. The NCI-PRO-CTCAE (Part 1 and 2), EORTC-QLQ-C30 (Part 2 only) and GP5 from FACT-G (Part 2 only) questionnaires should be completed prior to 
any other study assessments when possible (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.  
q. Rebastinib will be administered orally per assigned dose level (either 50 mg BID, 100 mg BID, or 75 mg BID) in 28-day cycles. On days of planned study 
visits, patients will take the study drug at the study site after pre-dose assessments are completed and prior to the paclitaxel infusion. 
r. 
Paclitaxel will be administered on Day 1, Day 8 and Day 15 of repeated 28-day cycles. Rebastinib will be administered orally prior to the paclitaxel infusion. 
All paclitaxel infusions may be administered ±2 days to the corresponding study visit however each infusion much be at least 5 days apart. All paclitaxel 
infusions will be administered at the study site. If the retreatment criteria are not met and a dose of paclitaxel is missed, a single make-up dose of paclitaxel 
may be administered on Day 22. Assessments required on Day 8 of the corresponding cycle must be performed (see Section 5.2.2). 
s. 
CT or MRI scans of the pelvis, abdomen, and chest 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. Imaging may be performed up to 7 days prior to the corresponding study visit. EOT 
imaging will only be performed on patients if imaging was not performed within the previous 12 weeks. Determination of objective tumor response will be 
performed by the Investigator according to the RECIST Version 1.1. 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.  MRI or CT scans without contrast can be used for patients who are allergic to radiographic 
contrast media. Throughout the study, the same assessment technique must be used (see Section 6.10.1).  


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t. 
CA-125 should be measured for ovarian cancer patients at screening and Day 1 of each cycle or approximately at the same time they undergo radiologic 
assessment. Ovarian cancer patients experiencing CA-125 response must have a confirmatory test performed at least 28 days after the initial response is 
documented. For other disease indications, results of tumor marker(s) performed based on standard of cares will be collected.  
u. PK sampling will be performed as described in Table 2 and Table 3. An unscheduled PK sample may be taken at the time of the onset of a new suspected, 
treatment-related adverse event.  
v. On serial PK sampling days (i.e., Cycle 1 Day 1 and Cycle 1 Day 15), paclitaxel will be administered following the completion of the required PK sampling 
as indicated in Table 2 and Table 3. If the associated paclitaxel infusion cannot be administered at the study visit (e.g., due to scheduling conflicts), it may be 
performed on the following day (i.e., Day 2, Day 16). Pre-infusion and end-of-infusion ECGs, vitals and PK samples would be required.  
w. Tumor tissue samples will be collected for all patients enrolled onto the study. If an archived tumor tissue sample is unavailable, a fresh tumor biopsy is 
required prior to the first dose of study drug. 
x. A fresh tumor biopsy will be collected at screening (if archival tumor tissue is unavailable) and Cycle 3 Day 1 (±7 days). An optional tumor biopsy may be 
performed upon progression if a patient responded to treatment (CR/PR) and then progressed, and has provided consent for undergoing tumor biopsy. Fresh 
biopsies will be performed for patients whose tumor is safe and 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. 
y. Whole Blood Immunophenotyping Biomarker and Plasma samples will be collected pre-dose at Cycle 1 Day 1 (Baseline), Cycle 1 Day 15, Cycle 2 Day 1, 
Cycle 3 Day 1 and every three cycles thereafter (i.e., Cycle 6, 9, 12, etc.), and at the EOT Visit. Details on sample collection, processing, and shipping of the 
samples, including any exceptions, will be provided in a separate Laboratory Manual. 
 


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Table 2: 
Schedule of Electrocardiograms, Vitals and Pharmacokinetic Sampling (Part 1) 
Study Visita 
Cycle 1, Day 1 (Baseline) 
Cycle 1, Day 15 
≥Cycle 2, Day 1 
EOT Visit 
Time Point (hours) 
ECGb 
Vitalsb 
PK 
ECGb 
Vitalsb 
PKd 
ECGb 
Vitalsb 
PKd 
ECGb 
Vitalsb 
PKd  
Pre-rebastinib dosec 
X 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
1 hour post-rebastinib dose (± 10 min) 
X 
X 
X 
X 
X 
X 
 
 
 
 
 
 
2 hours post-rebastinib dose (± 10 min) 
 
 
X 
 
 
X 
 
 
 
 
 
 
4 hours post-rebastinib dose (± 30 min) 
 
 
X 
 
 
X 
 
 
 
 
 
 
6 hours post-rebastinib dose (± 30 min) 
X 
X 
X 
X 
X 
X 
 
 
 
 
 
 
Pre-paclitaxel infusion (within 
10 minutes of start) 
 
 
X 
 
 
X 
 
 
 
 
 
 
End of paclitaxel infusion (within 
10 minutes of end) 
X 
X 
X 
X 
X 
X 
 
 
 
 
 
 
AE = adverse event; ECG = electrocardiogram; min = minute; PK = pharmacokinetic. 
a. 
If PK sampling cannot be performed at a study visit (e.g., due to scheduling conflicts, AE, etc.), it may be performed on another study day, provided that the 
patient, Investigator, and Sponsor are in agreement. Additionally, if the associated paclitaxel infusion cannot be administered at the study visit (e.g., due to 
scheduling conflicts), it may be performed on the following day. Pre-infusion and end-of-infusion ECGs, vitals and PK samples would be required.  
b. ECG and vital sign assessments will be completed before the collection of the corresponding PK sample. 
c. 
Pre-dose assessments should be obtained within 60 minutes prior to rebastinib administration.  
d. An unscheduled PK sample may be taken at the time of the onset of a new suspected, treatment-related AE.  


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Table 3: 
Schedule of Electrocardiograms, Vitals and Pharmacokinetic Sampling (Part 2) 
Study Visita 
Cycle 1, Day 1 (Baseline) 
Cycle 1, Day 15 
≥Cycle 2, Day 1 
EOT Visit 
Time Point (hours) 
ECGb 
Vitalsb 
PK 
ECGb 
Vitalsb 
PKd 
ECGb 
Vitalsb 
PKd 
ECGb 
Vitalsb 
PKd 
Pre-rebastinib dosec 
X 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Pre-paclitaxel infusion 
(within 10 minutes prior to 
start)e 
 
 
X 
 
 
X 
 
 
 
 
 
 
End of paclitaxel infusion 
(within 10 minutes after the 
end)e 
X 
X 
X 
X 
X 
X 
 
 
 
 
 
 
AE = adverse event; ECG = electrocardiogram; min = minute; PK = pharmacokinetic. 
a. 
If PK sampling cannot be performed at a study visit (e.g., due to scheduling conflicts, AE, etc.), it may be performed on another study day, provided that the 
patient, Investigator, and Sponsor are in agreement. Additionally, if the associated paclitaxel infusion cannot be administered at the study visit (e.g. due to 
scheduling conflicts), it may be performed on the following day. Pre-infusion and end-of-infusion ECGs, vitals and PK samples would be required.  
b. ECG and vital sign assessments will be completed before the collection of the corresponding PK sample. 
c. Pre-dose assessments should be obtained within 60 minutes prior to rebastinib administration.  
d. An unscheduled PK sample may be taken at the time of the onset of a new suspected, treatment-related AE.  
e. 
A PK sample should be obtained within 10 minutes prior to the start and after the end-of-infusion. 
 
 


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LIST OF ABBREVIATIONS  
Abbreviation 
Definition 
β-hCG 
Beta-human chorionic gonadotropin 
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 
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  
CYP 
Cytochrome P450  
DFS 
Disease-free survival  
DLT 
Dose-limiting toxicity 
DOR 
Duration of response 
ECG 
Electrocardiogram 
ECOG PS 
Eastern Cooperative Oncology Group Performance Status 


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Abbreviation 
Definition 
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 
FACIT 
Functional Assessment of Chronic Illness Therapies 
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 
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  
IBC 
Inflammatory breast cancer 
IEC 
Independent Ethics Committee  
ICF 
Informed consent form  
ICH 
International Conference on Harmonization 
IHC 
Immunohistochemical  
IOP 
Intraocular pressure 
IP 
Investigational product 
IRB 
Institutional Review Board  
IRT 
Interactive response technology 
ISH 
In situ hybridization 
ITD 
Internal tandem duplication 
IV 
Intravenous 
KM 
Kaplan-Meier 
LVEF  
Left ventricular ejection fraction  


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Abbreviation 
Definition 
MDR1 
Multidrug resistance protein 1 
MedDRA 
Medical Dictionary for Regulatory Activities 
mITT 
Modified Intent to Treat 
MMMT 
Malignant mixed Müllerian tumor 
mPFS 
Median progression free survival  
MTD 
Maximum-tolerated dose  
MRI 
Magnetic resonance imaging 
MSI-H 
Microsatellite Instability - High 
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  
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 
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 
SOP 
Standard operating procedure 
SUSAR 
Serious Unexpected Suspected Adverse Reaction 
T1/2 
Half-life 
T315I 
Threonine 315 to isoleucine  
TAMs 
Tumor-associated macrophages  
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 
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 
WCT PVG 
Worldwide Clinical Trials Pharmacovigilance 


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TABLE OF CONTENTS 
1. 
INTRODUCTION AND RATIONALE .....................................................................33 
1.1. 
Introduction .................................................................................................................33 
1.2. 
Clinical Indications .....................................................................................................33 
1.2.1. 
Breast Cancer ..............................................................................................................34 
1.2.2. 
Inflammatory Breast Cancer .......................................................................................34 
1.2.3. 
Ovarian Cancer ...........................................................................................................35 
1.2.4. 
Endometrial Cancer ....................................................................................................35 
1.2.5. 
Gynecological Carcinosarcomas ................................................................................35 
1.3. 
Overview of Rebastinib ..............................................................................................36 
1.3.1. 
Nonclinical Experience ...............................................................................................36 
1.3.2. 
Clinical Experience .....................................................................................................38 
1.4. 
Overview of Paclitaxel ...............................................................................................39 
1.5. 
Rationale .....................................................................................................................39 
1.5.1. 
Study Rationale ...........................................................................................................39 
1.5.2. 
Dose, Regimen, and Treatment Duration Rationale ...................................................40 
2. 
STUDY OBJECTIVES ..............................................................................................44 
2.1. 
Part 1 ...........................................................................................................................44 
2.1.1. 
Primary Objectives .....................................................................................................44 
2.1.2. 
Secondary Objectives .................................................................................................44 
2.2. 
Part 2 ...........................................................................................................................44 
2.2.1. 
Primary Objectives .....................................................................................................44 
2.2.2. 
Secondary Objectives .................................................................................................44 
2.3. 
Parts 1 and 2 ................................................................................................................44 
2.3.1. 
Exploratory Objectives ...............................................................................................44 
3. 
STUDY DESIGN .......................................................................................................45 
3.1. 
Overview of Study Design ..........................................................................................45 
3.2. 
Number of Patients .....................................................................................................46 
3.3. 
Duration of Study .......................................................................................................46 
4. 
STUDY POPULATION .............................................................................................47 
4.1. 
Inclusion Criteria ........................................................................................................47 
4.2. 
Exclusion Criteria .......................................................................................................49 


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5. 
STUDY DRUG ADMINISTRATION AND MANAGEMENT ...............................51 
5.1. 
Study Drug Description ..............................................................................................51 
5.1.1. 
Rebastinib ...................................................................................................................51 
5.1.2. 
Paclitaxel .....................................................................................................................51 
5.2. 
Study Drug Dose and Administration .........................................................................51 
5.2.1. 
Rebastinib ...................................................................................................................51 
5.2.2. 
Paclitaxel .....................................................................................................................52 
5.3. 
Dose Interruption/Modification and Management of Toxicities ................................52 
5.4. 
Packaging and Labeling ..............................................................................................63 
5.5. 
Storage Conditions ......................................................................................................63 
5.6. 
Study Drug Compliance .............................................................................................63 
5.7. 
Study Drug Accountability .........................................................................................63 
5.8. 
Disposal, Return, or Retention of Unused Study Drug ..............................................64 
5.9. 
Method of Assigning Patients to Treatment ...............................................................64 
5.10. 
Blinding ......................................................................................................................64 
5.11. 
Prior and Concomitant Treatment and Procedures .....................................................64 
5.11.1. 
Prior Medications and Procedures ..............................................................................64 
5.11.2. 
Prior Anticancer Medications and Procedures ...........................................................64 
5.11.3. 
Concomitant Medications ...........................................................................................64 
5.11.4. 
Permitted Medication ..................................................................................................65 
5.11.5. 
Medications to Avoid or Take with Caution ..............................................................65 
5.11.6. 
Prohibited Medications and Substances .....................................................................66 
5.11.7. 
Concomitant Procedures .............................................................................................66 
5.12. 
Other Precautions ........................................................................................................66 
6. 
STUDY ASSESSMENTS ..........................................................................................67 
6.1. 
Screening ....................................................................................................................67 
6.2. 
Treatment Period ........................................................................................................67 
6.3. 
Safety Follow-Up ........................................................................................................67 
6.4. 
Lost to Follow-Up.......................................................................................................67 
6.5. 
Informed Consent Procedure ......................................................................................67 
6.6. 
Patient Identification and Registration .......................................................................68 
6.7. 
Demographics and Medical History ...........................................................................68 
6.8. 
Safety ..........................................................................................................................68 


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6.8.1. 
Physical Examinations and Height and Weight ..........................................................69 
6.8.2. 
Eastern Cooperative Oncology Group (ECOG) Performance Status .........................69 
6.8.3. 
Vital Signs, Weight, and Height .................................................................................69 
6.8.4. 
Electrocardiograms .....................................................................................................69 
6.8.5. 
Echocardiograms/Multigated Acquisition Scans ........................................................70 
6.8.6. 
Ophthalmologic Examination .....................................................................................70 
6.8.7. 
Clinical Laboratory Tests ...........................................................................................70 
6.8.8. 
Pregnancy Test ............................................................................................................71 
6.8.9. 
Contraception and Pregnancy Avoidance Measures ..................................................71 
6.8.10. 
Adverse Events ...........................................................................................................73 
6.8.11. 
30 Day Safety Follow-Up ...........................................................................................73 
6.9. 
Pharmacokinetics ........................................................................................................74 
6.9.1. 
Sample Collection .......................................................................................................74 
6.9.2. 
Sample Assessment ....................................................................................................74 
6.10. 
Efficacy .......................................................................................................................74 
6.10.1. 
Radiologic Imaging ....................................................................................................74 
6.10.2. 
Response Assessment Using CA-125 (Ovarian Cancer Patients Only) .....................74 
6.10.3. 
Relevant Tumor Markers ............................................................................................75 
6.11. 
Biomarker Research Studies .......................................................................................75 
6.11.1. 
Sample Collection .......................................................................................................75 
6.11.2. 
Sample Assessments ...................................................................................................75 
6.12. 
Pharmacogenomic Measurements ..............................................................................76 
6.12.1. 
Sample Collection .......................................................................................................76 
6.12.2. 
Sample Assessment ....................................................................................................76 
6.13. 
Patient Reported Outcome Measurements..................................................................76 
6.13.1. 
National Cancer Institute’s Patient-Reported Outcomes Version of the 
Common Terminology Criteria for Adverse Events (NCI-PRO-CTCAE) ................76 
6.13.2. 
European Organisation for Research and Treatment of Cancer Quality of 
Life Questionnaire for Cancer 30-item (EORTC QLQ-C30) .....................................76 
6.13.3. 
Functional Assessment of Cancer Therapy – General (FACT-G) ..............................77 
7. 
ADVERSE EVENT AND SERIOUS ADVERSE EVENT 
DOCUMENTATION, SEVERITY GRADING, AND REPORTING ......................78 
7.1. 
Adverse Events ...........................................................................................................78 


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7.2. 
Severity Assessment ...................................................................................................78 
7.3. 
Causality Assessment .................................................................................................79 
7.4. 
Study Drug Action Taken ...........................................................................................80 
7.5. 
Adverse Event Outcome .............................................................................................80 
7.6. 
Treatment Given .........................................................................................................80 
7.7. 
Additional Points to Consider for Adverse Events .....................................................81 
7.7.1. 
Clinically Significant Assessments .............................................................................81 
7.8. 
Serious Adverse Events ..............................................................................................81 
7.9. 
Adverse Event of Special Interest for Study Drug .....................................................82 
7.10. 
Adverse Event Reporting Periods ...............................................................................82 
7.11. 
Adverse Event and Serious Adverse Event Reporting Requirements ........................82 
7.12. 
Abuse, Misuse, Overdose, and Medication Error .......................................................83 
8. 
DISCONTINUATION AND REPLACEMENT OF PATIENTS ..............................84 
8.1. 
Discontinuation of Treatment .....................................................................................84 
8.2. 
End of Study ...............................................................................................................84 
8.3. 
Replacement of Patients .............................................................................................85 
9. 
STATISTICAL CONSIDERATIONS .......................................................................86 
9.1. 
Determination of Sample Size ....................................................................................86 
9.2. 
Analysis Endpoints .....................................................................................................86 
9.2.1. 
Safety ..........................................................................................................................86 
9.2.2. 
Pharmacokinetics ........................................................................................................86 
9.2.3. 
Efficacy .......................................................................................................................87 
9.2.4. 
Pharmacogenomics .....................................................................................................87 
9.2.5. 
Pharmacodynamics .....................................................................................................87 
9.2.6. 
Patient Reported Outcomes ........................................................................................88 
9.3. 
Populations for Analysis .............................................................................................88 
9.4. 
Procedures for Handling Missing, Unused, and Spurious Data .................................88 
9.5. 
Interim Analyses .........................................................................................................89 
9.6. 
Adjustment for Multiple Comparisons .......................................................................89 
9.7. 
Blinding ......................................................................................................................89 
9.8. 
Statistical Methods ......................................................................................................89 
9.8.1. 
General Methods .........................................................................................................89 
9.8.2. 
Disposition of Patients ................................................................................................90 


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9.8.3. 
Demographic and Baseline Characteristics ................................................................90 
9.8.4. 
Extent of Exposure .....................................................................................................90 
9.9. 
Efficacy Analysis ........................................................................................................90 
9.9.1. 
Primary Endpoint ........................................................................................................90 
9.10. 
Secondary Endpoints ..................................................................................................90 
9.10.1. 
Progression-free Survival ...........................................................................................90 
9.10.2. 
Time to Progression ....................................................................................................91 
9.10.3. 
Overall Survival ..........................................................................................................91 
9.10.4. 
Time to Response .......................................................................................................91 
9.10.5. 
Clinical Benefit Rate ...................................................................................................91 
9.10.6. 
Duration of Response .................................................................................................91 
9.11. 
Safety Analysis ...........................................................................................................92 
9.11.1. 
Adverse Events ...........................................................................................................92 
9.11.2. 
Eastern Cooperative Oncology Group Performance Status ........................................92 
9.11.3. 
Vital Signs ..................................................................................................................92 
9.11.4. 
Echocardiogram/Multigated Acquisition Scans .........................................................92 
9.11.5. 
Ophthalmologic Assessments .....................................................................................92 
9.11.6. 
Clinical Laboratory Parameters ..................................................................................92 
9.12. 
Pharmacokinetic Analysis ..........................................................................................93 
9.13. 
Tumor Marker Analysis ..............................................................................................93 
9.13.1. 
Biomarker and Pharmacodynamic Analysis ...............................................................93 
9.14. 
Pharmacogenomic Analysis........................................................................................93 
9.15. 
Procedures for Reporting Deviations to Original Statistical Analysis Plan ...............93 
10. 
QUALITY CONTROL AND QUALITY ASSURANCE .........................................94 
10.1. 
Study Site Monitoring Visits ......................................................................................94 
10.2. 
Protocol Compliance ..................................................................................................94 
11. 
DATA HANDLING AND RECORD KEEPING ......................................................95 
11.1. 
Electronic Case Report Form ......................................................................................95 
11.2. 
Record Retention ........................................................................................................95 
12. 
ETHICS ......................................................................................................................96 
12.1. 
Ethical Conduct of the Study ......................................................................................96 
12.2. 
Patient Information and Consent ................................................................................96 
12.3. 
IRB ..............................................................................................................................96 


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12.4. 
Patient Confidentiality ................................................................................................96 
12.5. 
Reporting of Safety Issues or Serious Breaches of the Protocol or 
International Conference on Harmonization Good Clinical Practice .........................97 
12.6. 
Liability and Insurance ...............................................................................................97 
13. 
STUDY TERMINATION ..........................................................................................98 
13.1. 
Criteria for Suspension or Premature Termination of the Study ................................98 
13.2. 
Criteria for Premature Termination or Suspension of Investigational Sites ...............98 
13.3. 
Procedures for Premature Termination or Suspension of the Study or the 
Participation of Investigational Site(s) .......................................................................98 
14. 
PUBLICATION OF STUDY RESULTS ...................................................................99 
15. 
REFERENCES .........................................................................................................100 
 
LIST OF TABLES IN THE TEXT 
Table 1: 
Schedule of Assessments ............................................................................................16 
Table 2: 
Schedule of Electrocardiograms, Vitals and Pharmacokinetic Sampling (Part 
1) .................................................................................................................................20 
Table 3: 
Schedule of Electrocardiograms, Vitals and Pharmacokinetic Sampling (Part 
2) .................................................................................................................................21 
Table 4: 
Treatment-emergent Adverse Events of Special Interest by Worst CTCAE 
Grade and Rebastinib Cmax and AUC0-4h Subgroup during Cycles 1 and 2 ................42 
Table 5: 
Dose Reduction Steps for Rebastinib .........................................................................54 
Table 6: 
Dose Reduction Steps for Paclitaxel...........................................................................54 
Table 7: 
Recommended Dose Modification for Hematological Adverse Events .....................55 
Table 8: 
Recommended Dose Modification for Peripheral Sensory Neuropathy ....................56 
Table 9: 
Recommended Dose Modification for Muscular Weakness ......................................57 
Table 10: Recommended Dose Modification for Ocular Events (Except Asymptomatic 
Intraocular Pressure Increases) ...................................................................................58 
Table 11: Recommended Dose Modification and Management of Asymptomatic 
Intraocular Pressure Increases ....................................................................................59 
Table 12: Recommended Dose Modification for Cardiovascular Events ..................................60 
Table 13: Recommended Dose Modification for Gastrointestinal Adverse Events ...................61 
Table 14: Recommended Dose Modification for Other Grade 3 Non-Hematological 
Adverse Events ...........................................................................................................62 
Table 15: Safety Laboratory Tests ..............................................................................................71 


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Table 16: National Cancer Institute Common Terminology Criteria for Adverse Events 
(NCI-CTCAE) Version 5.0 Severity Grading Scale ...................................................79 
Table 17: Relationship to Study Drug Criteria ...........................................................................79 
Table 18: Classification for Study Drug Action Taken with Regard to an Adverse 
Event ...........................................................................................................................80 
Table 19: Classifications for Outcome of an Adverse Event ......................................................80 
Table 20: Partial or Missing Date Algorithms ............................................................................89 
 
LIST OF FIGURES IN THE TEXT 
Figure 1: 
Study Schema .............................................................................................................15 


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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 (Coffelt et al, 2011; 
Condeelis et al, 2006; De Palma et al, 2005; Harney et al, 2015; Ibberson et al, 2013; Mazzieri et 
al, 2011; Robinson et al, 2009; Rohan et al, 2014; Wyckoff et al, 2007). Since TIE2 expression is 
restricted to endothelial cells and highly protumoral TEMs, TIE2 is an attractive target for 
interrupting tumor cell/microenvironment interactions (Hanahan et al, 2012; Lewis et al, 2011). 
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 
(Mazzieri et al, 2011; Rigamonti et al, 2014). Proangiogenic TEMs have also been shown to 
limit therapeutic effectiveness of vascular disrupting agents (Rigamonti et al, 2014; Welford et 
al, 2011).  
It has been demonstrated that TEMs mediate invasion and metastasis in the polyoma middle T 
antigen syngeneic breast cancer model (PyMT) (Mazzieri et al, 2011), and that TIE2 expression 
correlates with poor overall survival (OS) and a high risk of metastasis in breast cancer patients 
(Rohan et al, 2014). 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 (Harney et al, 2015; Robinson et al, 2009). Moreover, 
chemotherapy increases the density and activity of TMEM sites and promotes distant metastasis 
in the PyMT and patient-derived xenografts (Karagiannis et al, 2017). 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 (Welford et al, 2011). 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 paclitaxel. In Part 1, patients with histologically 
confirmed, locally advanced or metastatic solid tumor where paclitaxel may be considered 
appropriate treatment, and which is refractory to standard therapies such as breast, ovarian, 
endometrial, non-small cell lung, and pancreatic cancer will be enrolled. In Part 2, five 
indication-specific cohorts in triple-negative breast, inflammatory breast, ovarian, and 
endometrial cancers, as well as gynecological carcinosarcoma will be open once the 
recommended Phase 2 dose (RP2D) of rebastinib in combination with weekly paclitaxel is 


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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 
Part 2 have been selected mainly based on use of paclitaxel and treatment options available 
currently. 
1.2.1. 
Breast Cancer 
Paclitaxel is commonly used in combination or as single agent in the neoadjuvant, adjuvant, and 
metastatic setting for treatment of breast cancer patients. Approved dosages and administration 
are 175 or 135 mg/m2 given every 3 weeks. However, weekly administration at lower doses has 
been tested and demonstrated better efficacy and/or safety. Several Phase 2 studies in metastatic 
breast cancer have shown that weekly paclitaxel produced significant response rates (Gori et al, 
2002; Green et al, 2005; Lombardi et al, 2004; Sato et al, 2003; Seidman et al, 1998). The largest 
of these reported a 21.5% response rate and a 41.8% overall clinical benefit (Perez et al, 2001). 
Studies in early breast cancer have also demonstrated the superiority of weekly paclitaxel 
compared to administration every 3 weeks. In the adjuvant setting, a Phase 3 study of 4950 
patients with node-positive or high-risk node-negative breast cancer after adjuvant therapy with 
doxorubicin-cyclophosphamide then randomized to either paclitaxel or docetaxel given weekly 
or every 3 weeks was conducted. This Intergroup E-1199 Investigators showed disease-free 
survival (DFS) significantly improved and OS marginally improved only for the weekly 
paclitaxel (hazard ratio [HR] = 0.84; p-value = 0.011 and HR = 0.87; p-value = 0.09, 
respectively) and every-3-week docetaxel arms (HR = 0.79; p-value = 0.001 and HR, 0.86; p-
value = 0.054, respectively). Weekly paclitaxel improved DFS and OS (HR = 0.69; p-
value = 0.010 and HR = 0.69; p-value = 0.019, respectively) in triple-negative breast cancer 
(Sparano et al, 2015). In patients with metastatic breast cancer, Seidman and the Cancer and 
Leukemia Group B of 9840 Investigators, showed that weekly paclitaxel was superior to 
paclitaxel given every 3 weeks for the treatment of metastatic breast cancer. In fact, weekly 
paclitaxel doubled the time to progression and increased the response rate from 29% to 42% 
compared to paclitaxel given every 3 weeks without affecting patients’ quality of life (QOL) 
(Seidman et al, 2008). Taken together, these results suggest that weekly paclitaxel is the optimal 
way of treating breast cancer patients in a variety of settings. 
1.2.2. 
Inflammatory Breast Cancer 
Inflammatory breast cancer (IBC) is rare. It accounts approximately for 0.5 to 2 percent of 
invasive breast cancers (Anderson et al, 2005; Hance et al, 2005). In the United States (US), its 
incidence appears to be increasing, particularly among white women (Anderson et al, 2005; 
Hance et al, 2005; Tai et al, 2005). As compared with locally advanced breast cancer, IBC is 
diagnosed at an earlier age (a median of 59 versus 66 years of age). The incidence of IBC is 
higher in black Americans compared with whites, and black women are diagnosed at a younger 
age. IBC is an aggressive form of locally advanced breast cancer. In general, women with IBC 
without distant metastatic disease are approached similarly to those with non-inflammatory 
locally advanced breast cancer. Metastatic IBC is also managed similarly to metastatic non-IBC. 


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1.2.3. 
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 bevacizumab, an anti-
VEGF antibody. If PFI is less than 6 months (platinum-resistant), patients are recommended to 
be treated with paclitaxel with or without bevacizumab. The weekly administration of paclitaxel 
has been investigated as treatment of platinum-resistant ovarian cancer by several groups with 
reports suggesting that approximately 10–20% of patients will achieve an objective response to 
the regimen (Alvarez et al, 2003; Ozols, 1999; Tiersten et al, 2004). 
1.2.4. 
Endometrial Cancer 
For women who develop metastatic endometrial cancer, platinum-based combination regimens 
are used. The two most commonly used regimens to treat metastatic endometrial cancer are 
carboplatin plus paclitaxel or the triple drug combination of cisplatin, doxorubicin, plus 
paclitaxel (Harris et al, 2007). Upon progression, paclitaxel (175 mg/m2 every 3 weeks) is an 
option in the second-line setting, particularly in patients who were not previously treated with 
this agent in the first-line setting. In one study, paclitaxel resulted in a 25% objective response 
rate (ORR) among 48 paclitaxel-naive patients (Lincoln et al, 2003). However, weekly 
administration (80 mg/m2) is also a reasonable option, particularly in patients who were 
previously treated with paclitaxel. This is based on data showing activity with weekly treatment 
in women with platinum and paclitaxel-resistant ovarian cancer (Markman et al, 2006). 
1.2.5. 
Gynecological Carcinosarcomas 
Gynecological carcinosarcomas (previously called malignant mixed Müllerian tumors 
[MMMTs]) are rare and aggressive cancers that are dedifferentiated (metaplastic) carcinomas 
comprised of carcinomatous and sarcomatous elements arising from a single malignant epithelial 
clone. For women with previously untreated metastatic carcinosarcoma, carboplatin and 
paclitaxel have activity in carcinosarcoma and cause less toxicity than ifosfamide-based 
regimens (Homesley et al, 2007; Sutton et al, 2000). 
For women who experience disease progression following receipt of adjuvant or first-line 
chemotherapy, single-agent chemotherapy is preferred. A choice between agents should be 
individualized, taking into account the prior toxicities the patient has experienced and the 
toxicities associated with an alternative agent. However, in light of the limited evidence of 
survival benefit with treatment, hospice care is also reasonable, particularly for patients at risk 
for significant treatment-related toxicity (Gynecologic Oncology Group et al, 2006; Sigurdsson 
et al, 1986).  


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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 
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 (Harney et al, 
2017). 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 (Harney et al, 2017). 
Rebastinib was also evaluated in combination with eribulin, another marketed inhibitor of 
microtubules. Rebastinib in combination with eribulin led to a significant increase in OS 
compared with single agent eribulin therapy in the PyMT implant breast tumor model (Harney et 
al, 2017). 
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).  


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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 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.  
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. 


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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. 
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 
(Anampa et al, 2018).  


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1.4. 
Overview of Paclitaxel 
Paclitaxel is the first member of the taxane family (a drug class that acts by disrupting the 
microtubular network essential for cell division) to receive marketing approval for cancer 
treatment. 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, non-small cell lung cancer (NSCLC), ovarian cancer, endometrial cancer and pancreatic 
cancer. In metastatic breast cancer, paclitaxel has demonstrated ORRs of 19 and 25% in two 
Phase 3 studies (Gradishar et al, 2005; Jones et al, 2005). ORRs of paclitaxel in platinum-
resistant, recurrent ovarian cancers have been reported as approximately 20% in multiple studies 
(Kumar et al, 2010). As the second line treatment of endometrial cancer, paclitaxel has reached 
an ORR of 27% (Lincoln et al, 2003). Paclitaxel can be administered weekly (80 to 100 mg/m2 
on days 1, 8, and 15 of a 28-day cycle) or every three weeks (135 to 175 mg/m2).  
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 plasma 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. 
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 


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obtained from neo-adjuvant treatment with paclitaxel, doxorubicin, or cyclophosphamide shows 
that TMEM density increased by treatment (Rigamonti et al, 2014). 
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, further evaluation of safety, efficacy, PK and pharmacodynamics (PD) is warranted for 
the combination with paclitaxel in various cancer types where paclitaxel-based therapies are 
used. 
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 approximately 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 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 (AESI). Exposure-response relationship analysis for rebastinib and selected 
AESIs listed below were conducted (Table 4). 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 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 
paclitaxel 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. In addition, 
50 mg BID will be explored simultaneously in Part 1 for determining the RP2D. Both dose levels 
have been tested and tolerated in combination with paclitaxel in 3 breast cancer patients each. In 


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the case where 100 mg BID rebastinib in combination with paclitaxel were not tolerated, 75 mg 
BID may be tested. 
 


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Table 4: 
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%) 
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%) 


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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 (%) 
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 Toxicity Criteria 
Adverse Event; SOC = system organ class; CTCAE Version 3.0 
 


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2. 
STUDY OBJECTIVES 
2.1. 
Part 1 
2.1.1. 
Primary Objectives  
• To evaluate the safety and tolerability of 50 mg and 100 mg rebastinib BID when 
administered in combination with paclitaxel. 
• To determine the RP2D of rebastinib in combination with paclitaxel. 
2.1.2. 
Secondary Objectives  
• To assess the preliminary efficacy of rebastinib administered in combination with paclitaxel 
by evaluating the ORR. 
• To assess the PK of rebastinib and paclitaxel 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 OS of rebastinib 
in combination with paclitaxel. 
2.2. 
Part 2 
2.2.1. 
Primary Objectives  
• To evaluate the safety and tolerability of rebastinib at the RP2D in combination with 
paclitaxel. 
• To evaluate the ORR as the primary efficacy measure of rebastinib in combination with 
paclitaxel.  
2.2.2. 
Secondary Objectives  
• To assess the PK of rebastinib and paclitaxel when administered in combination.  
• To evaluate efficacy measures, such as PFS, CBR, response duration, time to response, TTP, 
and OS of rebastinib in combination with paclitaxel. 
2.3. 
Parts 1 and 2 
2.3.1. 
Exploratory Objectives  
• To evaluate changes in select blood and plasma biomarkers when rebastinib is administered 
in combination with paclitaxel. 
• 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 paclitaxel.  
• To assess polymorphisms in genes encoding drug metabolic enzymes and/or transporters 
involved in metabolism and disposition of rebastinib in combination with paclitaxel. 
• Assess the effects of rebastinib administered in combination with paclitaxel using patient 
reported outcome (PRO) measures. 
 


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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 where paclitaxel is considered appropriate treatment. Rebastinib will be administered in 
combination with paclitaxel in repeated 28-day cycles to primarily assess the safety, tolerability 
and preliminary efficacy of the combination. Adverse events will be assessed, and laboratory 
values, vital sign measurements, electrocardiograms (ECGs), ophthalmologic examinations, 
physical examinations and Eastern Cooperative Oncology Group performance status (ECOG PS) 
will be obtained to evaluate the safety and tolerability of rebastinib when administered in 
combination with paclitaxel. Pharmacokinetic and PD samples will be collected at pre-specified 
time points. 
The study consists of two parts (Part 1 and Part 2). In Part 1, patients will be assigned to one of 
two pre-defined dose levels of rebastinib (50 or 100 mg BID) in combination with paclitaxel 
administered by intravenous (IV) infusion at 80 mg/m2. Each arm is planned to enroll at least 12 
evaluable patients. Additionally, if rebastinib at 100 mg BID is deemed unsafe, an additional arm 
dosing at 75 mg BID of rebastinib in combination with paclitaxel may be initiated. Up to 36 
evaluable patients will be dosed in Part 1. Safety will be continuously monitored and any arm 
may be terminated early if deemed intolerable by the Sponsor.  
Safety and tolerability, PK and PD, and preliminary efficacy data obtained in Part 1 will be used 
to determine the RP2D. Data for at least 12 evaluable patients through Cycle 1 must be available 
in an arm to declare the dose as the RP2D. Patients must receive ≥80% of planned doses of 
rebastinib and paclitaxel in Cycle 1 to be considered evaluable. Enrollment of patients will pause 
for determination of the RP2D prior to initiation of Part 2. An RP2D will be chosen by the 
Sponsor in consultation with the Investigators. 
Upon determination of the RP2D, Part 2 will be initiated to dose up to 165 evaluable patients 
using the RP2D across five indication-specific cohorts. A Simon two-stage design will be 
applied to Part 2 to further evaluate the safety, tolerability, and preliminary efficacy of rebastinib 
in combination with paclitaxel in triple-negative breast (Cohort 1), inflammatory breast 
(Cohort 2), ovarian (Cohort 3), endometrial (Cohort 4), and gynecological carcinosarcoma 
(Cohort 5) cancers. 
Each cohort will initially enroll up to 18 evaluable patients in the first stage. Patients must 
receive at least 1 dose of the combination and have 1 post-baseline assessment, or be 
discontinued prior to the post-baseline disease assessment due to an AE at least possibly related 
to rebastinib, to be considered evaluable. Tumor response will be assessed according to 
Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1. 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. Patients who 
meet criteria defined in Section 8.3 will be replaced and not be included in the responder 
analysis. If >4 responses (defined as PR or complete response [CR]) 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 


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in the first stage, expanding the cohort may be triggered earlier. Enrollment in each cohort will 
pause between the first and second stage of the Simon two-stage for evaluation of response. 
3.2. 
Number of Patients 
A total of approximately 201 patients (up to 36 evaluable patients in Part 1 and up to 
165 evaluable patients in Part 2) may be enrolled in this study. The study will be conducted at 
approximately 7 centers in the US during Part 1 and approximately 20 centers in the US during 
Part 2. 
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 for 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. Part 1, All Arms 
i. 
Histologically confirmed diagnosis of a locally advanced or metastatic solid 
tumor for which paclitaxel 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. Part 2 
A. Part 2, 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 (Hammond et al, 2010) 
ii. 
Received at least one prior line but no more than three prior lines of systemic 
chemotherapy in the metastatic setting. 
iii. 
Has not received taxane-containing regimens within 6 months prior to the first 
dose of study drug. 
B. Part 2, Cohort 2: Inflammatory Breast Cancer (IBC) 
i. 
Histologically or cytologically confirmed stage IV breast carcinoma with a 
previous clinical diagnosis of IBC based on the presence of inflammatory 
changes in the involved breast, such as diffuse erythema and/or edema (peau 
d'orange), with or without an underlying palpable mass and involving the 
majority of the skin of the breast; pathological evidence of dermal lymphatic 
invasion should be noted but is not required for diagnosis. 
ii. 
Received at least one prior line of systemic chemotherapy in the metastatic 
setting. 
iii. 
Has not received taxane-containing regimens within 6 months prior to the first 
dose of study drug. 
C. Part 2, Cohort 3: 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.  
a. Progressed or relapsed within 6 months after the completion of a 
platinum-containing chemotherapy regimen. 
b. Patients that progressed during treatment or within 1 month after the 
completion of the first platinum-containing chemotherapy regimen 
(primary platinum refractory) are excluded. 


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ii. 
Received no more than five prior lines of systemic anticancer therapy. 
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 regimen. 
iii. 
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. 
D. Part 2, Cohort 4: Endometrial Cancer 
i. 
Histologically confirmed adenocarcinoma of the endometrium. 
ii. 
Received at least one prior line of platinum-based therapy in the recurrent, 
metastatic, or high-risk disease setting.  
a. For patients with known microsatellite instability-high (MSI-H) or 
mismatch repair deficiency, progressed after a regimen including an 
anti-PD1 agent. 
E. Part 2, Cohort 5: Gynecological Carcinosarcoma 
i. 
Histologically confirmed advanced (stage III or IV), persistent, or recurrent 
gynecological carcinosarcoma. 
a. Homologous or heterologous type carcinosarcoma (MMMT) are 
allowed. 
ii. 
Must have received at least 1 prior chemotherapy regimen (including high-
dose, consolidation, or extended therapy after surgical or nonsurgical 
assessment for gynecological carcinosarcoma). 
4. At least one measurable lesion according to RECIST Version 1.1. 
5. ECOG PS of ≤2. 
6. Able to provide a 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 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: serum creatinine ≤1.5×ULN or creatinine clearance 
≥30 mL/min based either on urine collection or Cockcroft Gault estimation. 


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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 6.8.9. 
9. If male, must agree to use two methods of contraception, with one of them being 
highly effective, 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 6.8.9. 
10. Patient must provide signed consent to participate in the study and is 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. 
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. Peripheral neuropathy of any etiology >Grade 1. 
4. 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. 
5. 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. 
6. 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. See 
Section 5.11.6 for further details. 
7. Known retinal neovascularization, macular edema or macular degeneration. 


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8. 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. 
9. 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. 
10. Left ventricular ejection fraction (LVEF) <50% at screening. 
11. Arterial thrombotic or embolic events such as cerebrovascular accident (including 
ischemic attacks) or moderate hemoptysis within 6 months prior to the first dose of 
study drug. 
12. Venous thrombotic event (e.g., deep vein thrombosis) within the 3 months prior to the 
first dose of study drug; following a 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.  
13. Active infection ≥Grade 3 requiring IV anti-infective treatment within 7 days prior to 
the first dose of study drug.  
14. Known human immunodeficiency virus or hepatitis C infection only if the patient is 
taking medications that are excluded per protocol, 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 the first dose of study drug; following major surgeries 
>4 weeks prior to the first dose of study drug, all surgical wounds must be healed and 
free of infection or dehiscence. 
18.  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. 
 


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5. 
STUDY DRUG ADMINISTRATION AND MANAGEMENT 
5.1. 
Study Drug Description 
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. 
Paclitaxel 
Paclitaxel will be provided by the Sponsor. Please refer to the paclitaxel label or prescribing 
information (US Prescribing Information) for more details. 
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. In Part 1, patients will be enrolled to 
receive 50 mg BID or 100 mg BID of rebastinib orally (PO) in combination with paclitaxel in 
repeated 28-day cycles. If 100 mg BID is deemed intolerable, a cohort of 75 mg BID in 
combination with paclitaxel will be initiated. In Part 2, rebastinib will be administered at the 
RP2D in combination with paclitaxel. For patients that are unable to tolerate paclitaxel, 
rebastinib may be administered as a single agent starting in Cycle 2. 
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 (minimum of 6 hours apart). 
• 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.  
• On days of PK sample collection, 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 diary.  
• On days of paclitaxel administration, patients must take the dose of rebastinib prior to 
infusion of paclitaxel. Premedication will be administered prior to or after the rebastinib 
dose.  
• If a patient forgets to take a dose at the scheduled time, the patient can 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 omitted and the patients must 
continue treatment with the next scheduled dose. 


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• 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 paclitaxel 
infusion are allowed. 
5.2.2. 
Paclitaxel 
Paclitaxel will be administered by IV infusion at 80 mg/m2 over approximately 60 minutes on 
Day 1, Day 8 and Day 15 of repeated 28-day cycles. Rebastinib will be administered orally prior 
to the paclitaxel infusion. All paclitaxel infusions may be administered ±2 days to the 
corresponding study visit however each infusion much be at least 5 days apart. All paclitaxel 
infusions will be administered at the study site. 
Patients should be pre-medicated prior to paclitaxel administration in order to prevent severe 
hypersensitivity reactions per institutional practices. On serial PK sampling days during Part 1, 
paclitaxel will be infused after the required PK sampling (see Table 2).  
To initiate each weekly treatment, the following is needed: 
• ANC ≥500/µL 
• Platelets ≥75,000/µL 
If the retreatment criteria are not met and a dose of paclitaxel is missed, a single make-up dose of 
paclitaxel may be administered on Day 22 of a given cycle as long as retreatment criteria are 
met. Assessments required on Day 8 of the corresponding cycle must be performed. 
For patients that are unable to tolerate paclitaxel, rebastinib may be administered as a single 
agent starting in Cycle 2. 
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 paclitaxel will be 
completed according to Table 5 and Table 6, respectively. Recommended dose interruptions and 
modifications as well as management of toxicities are identified in Table 7, Table 8, Table 9, 
Table 10, Table 11, Table 12, Table 13, and Table 14. 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 paclitaxel 
separately so that dose modifications can be made accordingly. Upon resumption following a 
dose interruption, the Investigator must continue with the patient’s original visit schedule 
calculated from Cycle 1 Day 1 (Baseline). Re-escalation of rebastinib or paclitaxel will be done 
by one dose-level at time upon agreement between the Sponsor and Investigator. 
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 


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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 7. 
• Requirement for more than 2 dose reductions from the assigned dose at enrollment to manage 
AEs. 
• Rebastinib: treatment interruption for >14 days due to treatment-related AEs. 
• Paclitaxel: treatment interruption for >21 days due to treatment-related AEs. 
Sites must follow up with patients at least weekly during drug interruptions. 
 


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Table 5: 
Dose Reduction Steps for Rebastinib 
Starting Dose of Rebastinib 
1st Dose Reduction 
2nd Dose Reduction 
50 mg BID 
25 mg BID 
Discontinue 
100 mg BID 
75 mg BID 
50 mg BID 
75 mg BID 
50 mg BID 
25 mg BID 
Sites must follow-up with patients at least weekly during drug interruptions. 
 
Table 6: 
Dose Reduction Steps for Paclitaxel 
Starting Dose of Paclitaxel 
1st Dose Reduction 
2nd Dose Reduction 
80 mg/m2 
70 mg/m2 
60 mg/m2 
Sites must follow-up with patients at least weekly during drug interruptions. 


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Table 7: 
Recommended Dose Modification for Hematological Adverse Events 
Adverse Event 
Grade 
Occurrence 
Rebastinib 
Paclitaxel 
Based on the causal relationship, dose interruption/modification will apply to rebastinib only, paclitaxel only or both study drugs. 
Neutrophil 
Count 
Decrease 
4 
Any 
Continue the same dose unless the AE is deemed 
related to rebastinib. If related: 
• Hold rebastinib until ANC≥500 /µL. 
• Restart at the same dose. 
Hold paclitaxel. 
• Until ANC ≥500 /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 paclitaxel. 
• 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 paclitaxel 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 paclitaxel until ANC ≥1,500/µL. 
• Reduce one dose level with G-CSF or GM-CSF.  
3rd 
• The same as above. 
• Permanently discontinue paclitaxel 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 8: 
Recommended Dose Modification for Peripheral Sensory Neuropathy  
Adverse Event 
Grade 
Occurrence 
Rebastinib 
Paclitaxel 
Based on the causal relationship, dose interruption/modification will apply to rebastinib only, paclitaxel only or both study drugs. 
Peripheral 
Sensory 
Neuropathy 
2 
1st  
Continue the same dose unless AE is 
worsening. 
• If worsening, reduce one dose level. 
Hold paclitaxel 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.  
2nd and subsequent 
occurrence(s) 
Continue the same dose unless AE is 
worsening. 
• If worsening, reduce one dose level.  
Permanently discontinue paclitaxel.  
3 
1st 
Hold rebastinib until AE resolves to 
Grade <2. 
• Reduce one dose level. 
Hold paclitaxel 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. 
2nd and subsequent 
occurrence 
Hold rebastinib until AE resolves to 
Grade <2. 
• Reduce one dose level. 
Permanently discontinue paclitaxel. 
Sites must follow-up with patients at least weekly during drug interruptions. 
 
 


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Table 9: 
Recommended Dose Modification for Muscular Weakness 
Adverse Event 
Grade 
Occurrence 
Rebastinib 
Paclitaxel 
Based on the causal relationship, dose interruption/modification will apply to rebastinib only, paclitaxel only or both study drugs. 
Muscular 
Weakness 
2 
1st 
Hold rebastinib until toxicity resolves to Grade <2: 
• If AE persists at Grade 2 >5 days after dose 
interruption: 
o 
Refer patient to a neurologist for evaluation 
and management 
• If AE resolves in ≤14 days, continue the same 
dose level 
• If AE does not resolve in >14 days, consider 
permanent discontinuation 
Hold paclitaxel until toxicity resolves to Grade <2: 
• If AE persists at Grade 2 >5 days after dose 
interruption: 
o 
Refer patient to a neurologist for 
evaluation and management 
• If AE resolves in ≤14 days, continue the same 
dose level 
• If AE does not resolve in >14 days, consider 
permanent discontinuation 
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 1 dose level 
• If AE does not resolve in >14 days, consider 
permanent discontinuation 
Hold paclitaxel until toxicity resolves to Grade <2: 
• Refer patient to a neurologist for evaluation and 
management 
• If AE resolves in ≤14 days, reduce 1 dose level 
• If AE does not resolve in >14 days, consider 
permanent discontinuation 
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 1 dose level 
• If AE does not resolve in >14 days, consider 
permanent discontinuation 
Hold paclitaxel until toxicity resolves to Grade <2: 
• Refer patient to a neurologist for evaluation and 
management 
• If AE resolves in ≤14 days, reduce 1 dose level 
• If AE does not resolve in >14 days, consider 
permanent discontinuation 
2nd  
Permanently discontinue study treatment 
Sites must follow-up with patients at least weekly during drug interruptions. 


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Table 10: 
Recommended Dose Modification for Ocular Events (Except Asymptomatic Intraocular Pressure Increases) 
Adverse Event 
Grade 
Occurrence 
Rebastinib 
Paclitaxel 
Based on the causal relationship, dose interruption/modification will apply to rebastinib only, paclitaxel only or both study drugs. 
Ocular AEs and Visual 
AEsa 
2 
1st 
Implement close monitoring and hold 
rebastinib until AE resolves to Grade <2. 
• Resume at the same dose. 
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 paclitaxel 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 11: 
Recommended Dose Modification and Management of Asymptomatic Intraocular Pressure Increases 
Sites must follow up with patients at least weekly during drug interruptions. 
Condition 
Management 
Elevated intraocular pressure (IOP) and IOP of both eyes 
still ≤21 mmHg  
• Continue the same dose of rebastinib and paclitaxel. 
• 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 paclitaxel. 
• 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 not recovered within 14 days, consider permanent 
discontinuation.  
• 
If recovered, reduce rebastinib by one dose level. 
 
Paclitaxel can be administered at the same dose per discretion of 
the Investigator. 
o 
If dose reduction of rebastinib with medications cannot lower 
IOP ≤21 mmHg, then a patient will be discontinued from the study. 
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 
Paclitaxel can continue being administered at the same dose per discretion 
of the Investigator. 
o 
Resume rebastinib at the same dose. 
 
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, 
 
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. 


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Table 12: 
Recommended Dose Modification for Cardiovascular Events 
Adverse Event 
Grade 
Occurrence 
Rebastinib 
Paclitaxel 
Based on the causal relationship, dose interruption/modification will apply to rebastinib only, paclitaxel 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 paclitaxel 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 paclitaxel until AE resolves to Grade <2. 
• Reduce one dose level. 
• If AE is worsening with each infusion, even 
if it remains Grade 1, hold paclitaxel. 
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 paclitaxel 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 13: 
Recommended Dose Modification for Gastrointestinal Adverse Events 
Adverse Event 
Grade 
Occurrence 
Rebastinib 
Paclitaxel 
Based on the causal relationship, dose interruption/modification will apply to rebastinib only, paclitaxel 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 paclitaxel 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 paclitaxel treatment. 
Diarrheaa, c 
Mucositisa  
2 
Any 
Continue the same dose unless AE is 
worsening. 
• 
If worsening, reduce one dose level. 
Hold paclitaxel until Grade <2 
• 
Resume paclitaxel at the same dose. 
3 
1st and 2nd  
Continue the same dose unless AE is 
worsening. 
• 
If worsening, reducing one dose level. 
Hold paclitaxel 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 paclitaxel 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 14: 
Recommended Dose Modification for Other Grade 3 Non-Hematological Adverse Events 
Adverse Event 
Grade 
Occurrence 
Rebastinib 
Paclitaxel 
Based on the causal relationship, dose interruption/modification will apply to rebastinib only, paclitaxel only or both study drugs. 
Other 
Non-Hematologica 
3 
Any 
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 paclitaxel 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. 
a. 
The following AEs are exceptions to the above:  
• 
Grade 3 nausea, vomiting, and diarrhea resolved to Grade 1 or baseline within 1 week with optimal treatment,  
• 
Transient Grade 3 fatigue (lasting <1 week) and, 
• 
Any other Grade 3 non-hematologic AE that can be controlled to Grade 1 or baseline within 1 week with appropriate treatment. 
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. 
Paclitaxel 100 mg (16.7 mL) multidose vials will be supplied by the Sponsor. Paclitaxel is a clear 
colorless to slightly yellow viscous solution. It is supplied as a nonaqueous solution intended for 
dilution with a suitable parenteral fluid prior to IV infusion. Each mL of sterile nonpyrogenic 
solution contains 6 mg paclitaxel, 527 mg of Polyoxyl 35 Castor Oil, NF, 49.7% (v/v) 
Dehydrated Alcohol, USP and 2 mg Citric Acid, USP.  
Study drug will be provided and replaced via the Interactive Response Technology (IRT). Study 
drug dispensation instructions will be provided in the Pharmacy Manual.  
5.5. 
Storage Conditions 
Instructions regarding the storage, handling and administration will be on the label and/or in the 
Pharmacy Manual.  
Rebastinib: Store at controlled room temperature between 20° to 25°C (68° to 77°F). Retain in 
the original package to protect from light and moisture.  
Paclitaxel: Store in original cartons at controlled room temperature between 20° to 25°C 
(68° to 77°F) at clinical sites. The original packaging should be retained during storage and the 
vials should be protected from light.  
For both study drugs, refer to United States Pharmacopeia [USP] Controlled Room Temperature. 
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 


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dates, quantities, bottle numbers, and patient numbers. The Sponsor or its designee must review 
drug accountability at the site on an ongoing basis during 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 study drug blister packs. 
The site staff or pharmacy personnel (as appropriate) must retain all materials returned by the 
patients until returned to Sponsor/designee or destroyed by the study site. If the study drug will 
be destroyed at the study site, the site must have a governing standard operating procedure (SOP) 
documenting the process to be followed when destroying investigational product (IP) on site. 
The Sponsor must review this SOP and give approval prior to destruction of IP at the 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 Part 1, patients will be assigned to the dose of rebastinib according to the arm in which they 
are enrolled. In Part 2, patients will be assigned to the 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 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 the 
30 Day Safety Follow-up Visit, or initiation of new anticancer therapy must be documented in 
the patient’s source documents and the eCRF. In addition, any new medications taken after the 


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last dose of study drug through the 30 Day Safety Follow-up Visit, or initiation of new 
anticancer therapy, 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. 
Supportive care agents, such as blood products, antiemetics, and pain medications are permitted 
as needed per ASCO guidelines (Hammond et al, 2010) or local institutional practices.  
Medications used only as premedication for paclitaxel infusion per institutional practices are 
permitted even if listed as to avoid or take with caution, or prohibited. 
5.11.5. 
Medications to Avoid or Take with Caution 
The following medications must be avoided or taken with caution: 
• Medications that are strong inhibitors, and inducers of CYP3A4 and CYP2C8. 
o For patients that have permanently discontinue paclitaxel and are being treated 
with rebastinib as a single agent, substrates, inhibitors and inducers of CYP2C8 
may be taken without restriction. 
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 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 paclitaxel infusion are allowed. 
• Warfarin and low molecular weight heparin. 
• Medications that are CYP2C19 and CYP2C9 substrates with a narrow therapeutic index 
• 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. 


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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. 
o Hormonal therapy including octreotide or bone targeted therapy such as 
bisphosphonates or a receptor activator of nuclear factor-kappa B (RNAK) ligand 
inhibitor is not considered as anticancer therapy.  
• Any other investigational therapy 
• Proton pump inhibitors (PPIs): PPIs must be discontinued 4 days prior to the first dose of 
study drug.  
• Medications that are known to prolong the QTc interval. 
o Antiemetics, known to prolong the QTc interval such as ondansetron are 
permitted only as premedication for paclitaxel infusion in accordance with 
institutional practice. 
• Systemic corticosteroids or requirement chronically on study drugs, unless the dose is no 
more than the equivalent of prednisone 15 mg/day.  
o Inhaled, intranasal, intra-ocular, topical, and intra-articular injections are allowed.  
o Premedication with steroids, in accordance with institutional practice (including 
dexamethasone) is permitted prior to paclitaxel dosing. 
5.11.7. 
Concomitant Procedures 
All procedures performed on or after the first dose of study drug through the 30 Day Safety 
Follow-up Visit, or initiation of new anticancer therapy, must be documented in the patient’s 
source documents and the eCRF. 
Investigational procedures of any kind are excluded during the study. Surgical resection during 
study treatment must be discussed with the Sponsor prior to implementation. 
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 ultraviolet 
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 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 (Baseline). The treatment 
period is defined as the time of first dose of study 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 prematurely discontinue study drug must return for an EOT Visit within 7 days 
after the last dose of study drug. 
6.3. 
Safety Follow-Up 
Patients should be contacted between 30 and 37 days after the last dose of study drug, or prior to 
initiation of new anticancer therapy, to collect new information pertaining to AEs and 
concomitant medications and procedures as outlined in Table 1 (Schedule of Assessments). 
These assessments 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., tumor tissue sample only, 
R0, R1, or R2). 
• Systemic therapy: include dates of treatment, agents (including dose and dosing 
regimen), reason for treatment (e.g., adjuvant therapy or for metastatic disease), 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; if possible, the start date for these comorbidities must be 
documented. 
6.8. 
Safety 
The safety profile will be assessed based on:  
• Overall AEs  
• SAEs  
• AESIs  
• Dose reduction or discontinuation of study drug due to toxicity  
• Physical examinations  


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• ECOG PS  
• Ophthalmic examinations  
• Changes from baseline in laboratory parameters  
• Electrocardiogram (ECGs)  
• Echocardiograms or multigated acquisition (MUGAs)  
• Vital signs 
6.8.1. 
Physical Examinations and Height and Weight 
A full physical examination will be performed at screening, and the EOT Visit. 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. If the patient reports a complaint or a possible AE, a targeted physical examination 
may be conducted at the Investigator’s discretion. After consent, any clinically significant 
abnormal findings in physical examinations must be reported as AEs (Section 7).  
6.8.2. 
Eastern Cooperative Oncology Group (ECOG) Performance Status  
Eastern Cooperative Oncology Group (ECOG) PS (Oken et al, 1982) will be assessed according 
to the Schedule of Assessments in Table 1. 
6.8.3. 
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 will be assessed following a 5-minute rest 
(seated or supine position). On study visits when PK blood samples will be collected, assessment 
of vital signs will be conducted according to the schedule shown in Table 2 (Part 1) and Table 3 
(Part 2). Vital sign assessments will be completed before the collection of the corresponding PK 
sample.  
6.8.4. 
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. On study visits when PK blood samples will be 
collected, assessment of ECG will be conducted according to the schedule shown in Table 2 
(Part 1) and Table 3 (Part 2). Assessment of ECG will be performed prior to PK sampling. 
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.  
• An ECG must be performed before the collection of corresponding PK samples and prior to 
the dose of study drug. 


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• A hard copy of both the initial ECG tracing and central over-read copy must be printed and 
signed by the Investigator at the site.  
6.8.5. 
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 throughout the 
study. Left ventricular ejection fraction (LVEF) must be documented in the patient’s source 
documents and eCRF. 
6.8.6. 
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. 
Additionally, an examination done within 30 days prior to the last dose of study drug can 
substitute one required on the EOT Visit. 
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 and stored centrally. 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.  
6.8.7. 
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 National Cancer Institute 
Common Terminology Criteria for Adverse Events (NCI-CTCAE), Version 5.0. (CTCAE 2017). 
The safety laboratory tests are provided in Table 15. 


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Table 15: 
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  
• 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 6.8.8. 
b. If any result is abnormal, a microscopic analysis must be performed by the local laboratory. 
c. Indirect bilirubin must be calculated and documented at the site. 
6.8.8. 
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 at all other visits 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). 
6.8.9. 
Contraception and Pregnancy Avoidance Measures 
6.8.9.1. 
Contraception 
The effects of rebastinib on sperm, conception, pregnancy, and lactation are not known. 
Participation in this study requires patients to agree to use two methods of contraception, with 
one of the methods being highly effective. 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. 


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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. 
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. Local requirements may 
prohibit the use of some of these 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. 


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• 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 
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. 
6.8.9.2. 
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 IQVIA 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. 
6.8.10. 
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.  
6.8.11. 
30 Day 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 30 Day Safety Follow-up Visit (30 [+7] days 
after last dose of study drug) or prior to the initiation of a new anticancer therapy, whichever 
occurs first. 


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6.9. 
Pharmacokinetics  
6.9.1. 
Sample Collection 
At the visits indicated in Table 1 (Schedule of Assessments), Table 2 (Part 1) and Table 3 
(Part 2), blood samples will be collected for the determination of the concentrations of rebastinib 
and paclitaxel.  
Details on sample collection, processing, and shipping of the samples, including any exceptions, 
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. 
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 SOPs. 
6.9.2. 
Sample Assessment 
The following PK parameters will be calculated using non-compartmental methods of plasma 
rebastinib and paclitaxel, obtained after single and repeated dose administration. The parameters 
will include, but may not be limited to Cmax, Tmax, AUC, and T1/2. 
6.10. 
Efficacy 
6.10.1. 
Radiologic Imaging 
All patients will have radiographic tumor evaluation by CT or MRI scans of the chest, abdomen, 
and pelvis according to the Schedule of Assessments in Table 1. MRI or CT scans without 
contrast can 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. 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 7 days prior to the corresponding study visit. EOT imaging will 
only be performed on patients if imaging was not performed within the previous 12 weeks. 
6.10.2. 
Response Assessment Using CA-125 (Ovarian Cancer Patients Only) 
CA-125 should be collected for all patients with ovarian cancer according to the Schedule of 
Assessments in Table 1. 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 by the Investigator 
according to the Gynecological Cancer Intergroup (GCIG) guidelines (Rustin et al, 2011). 


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6.10.3. 
Relevant Tumor Markers 
Data of relevant tumor markers such as CA-15.3, CA-125, CA-19-9, CA-27.29, prostate-specific 
antigen, and carcinoembryonic antigen will be collected as appropriate for disease assessment 
and if performed as a part of the standard of care. 
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 collecting, storing, and shipping of the samples, 
including any exceptions, 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 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. An 
optional tumor biopsy may be performed upon progression if a patient responded to treatment 
(CR/PR) and then progressed, and has provided consent for undergoing tumor biopsy. Fresh 
biopsies are only required for patients whose tumor is safe and accessible as judged by the 
Investigator 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 paclitaxel. 
6.11.2.2. Whole Blood and Plasma Samples 
Whole blood samples will be collected for immunophenotyping of peripheral blood mononuclear 
cells. 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 of paclitaxel on systemic immune response.  


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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 collecting, 
storing, and shipping the sample, including any exceptions, will be provided. The 
pharmacogenomic sample will be stored and analyzed by a central laboratory, and may be stored 
for up to 15 years. 
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 paclitaxel. Additionally, polymorphisms in genes that may be associated with 
clinical response and/or study drug-related toxicity will be assessed. 
6.13. 
Patient Reported Outcome Measurements 
Patients will be asked to complete PRO assessments in the form of questionnaires at the 
investigative site prior to any other study assessments (when possible) according to the Schedule 
of Assessments in Table 1.  
Questionnaires will only be available in English, therefore, only English-speaking patients may 
complete the questionnaire. Patient-entered data will not be modified by the Investigator or site 
staff, ePRO vendor, CRO, or Sponsor. 
6.13.1. 
National Cancer Institute’s Patient-Reported Outcomes Version of the Common 
Terminology Criteria for Adverse Events (NCI-PRO-CTCAE) 
The NCI-PRO-CTCAE measurement system was developed to gather symptomatic AEs by 
patient self-reporting (Kim et al, 2017). The NCI-PRO-CTCAE library is comprised of 124 items 
representing 78 symptomatic AEs. The NCI-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 NCI-PRO-CTCAE items has been selected from the NCI-PRO-CTCAE 
item library. The time required for completion is approximately 20 minutes. Patients will be 
asked to complete NCI-PRO-CTCAE (using patient reported outcome software, if possible) 
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 (EORTC, 2018). The time required for completion is approximately 
4 minutes. The questionnaire is composed of multi-item and single-item scales. These include 5 


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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 (using patient reported outcome software, 
if possible) according to the Schedule of Assessments in Table 1.  
6.13.3. 
Functional Assessment of Cancer Therapy – General (FACT-G) 
The Functional Assessment of Chronic Illness Therapy (FACIT) FACT-G questionnaire, is a 
validated, standardized, patient completed question used extensively in international clinical 
studies (FACIT, 2018). It was developed to assess health related QOL in patients with cancer. 
The time required for completion is less than 1 minute. The questionnaire will be composed of 1 
question (GP5 from FACT-G) to measure the burden of side effects experienced by the patient. It 
is completed by the patient’s 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 (using patient reported outcome 
software, if possible) 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, but 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 a 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 16 (CTCAE, 2017). 


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Table 16: 
National Cancer Institute Common Terminology Criteria for Adverse Events 
(NCI-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; AE = adverse event.  
* 
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. 
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 17.  
Table 17: 
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 as at least possibly related will 
be considered treatment-related events. 


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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 18. 
Table 18: 
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 during a dose hold or in circumstances such as 
when the study drug had been completed before the AE began and no 
opportunity to decide whether to continue, interrupt, or withdraw study drug is 
possible. 
AE = adverse event  
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 19. 
Table 19: 
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). 
AE = adverse event  
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, or physical therapy. "No" indicates the absence of any kind 
of treatment for an AE. 


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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 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 


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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 Event 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. 
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.  


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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. If an underlying 
diagnosis is subsequently determined for the reported symptom(s), then the reported symptom(s) 
term(s) must be revised to be “attributed” or “due” to the diagnosis. 
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. Further instructions on reporting of SAEs and AESIs will be 
provided on the reporting form.  
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)]. 
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 discontinue from the study drug and/or study 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 involvement in the study 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) 
• Adverse event 
• 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. 
End of Study 
The end of study is defined as the date when the patient has withdrawn or completed all 
necessary follow-up assessments through the 30 Day Safety Follow-up Visit. 
If the patient is unable to complete the 30 Day Safety Follow-up Visit, the reason will be 
captured in the clinical database using the following categories: 
• New anticancer therapy 
• Death 
• Lost to follow-up 
• Withdrawal by patient from study 
• Termination of study by Sponsor 
• Other  
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 30 Day Safety Follow-up Visit. Patients must return all used, partially used, and unused 
study drug blister packs. 


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8.3. 
Replacement of Patients 
In Part 1, patients must receive ≥80% of planned doses of rebastinib and paclitaxel in Cycle 1 to 
be considered for determination of the RP2D. Patients who do not receive the requisite amount 
of the combination may be replaced.  
In Part 2, patients who do not receive at least one dose of the combination will be replaced. 
Patients must receive at least 1 dose of the combination and have 1 post-baseline assessment, or 
be discontinued prior to the post-baseline disease assessment due to an AE at least possibly 
related to rebastinib, to be considered evaluable. 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 AE(s) at least possibly related 
to rebastinib, patients may be replaced. Patients who meet these criteria will be replaced and not 
be included in the responder analysis.  
 


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9. 
STATISTICAL CONSIDERATIONS 
9.1. 
Determination of Sample Size 
Part 1 will primarily be used to evaluate the safety and tolerability of the combination. Up to 
36 evaluable patients will be enrolled in total with at least 12 evaluable patients in each arm. This 
is considered appropriate for analysis of safety and tolerability to determine the RP2D. 
A Simon two-stage design will apply to Part 2 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 in order to demonstrate the target efficacy of >10 responses in a total of 
33 patients. Thus, this part of study may enroll up to 165 (33 patients per indication-specific 
cohort) evaluable patients. If a non-evaluable rate of 10% is considered, approximately 
183 patients may be enrolled. 
9.2. 
Analysis Endpoints 
9.2.1. 
Safety 
Safety endpoints include: 
• Overall AEs 
• SAEs 
• AESIs 
• Dose reduction, dose interruptions, or discontinuation of study drug due to toxicity  
• Physical examinations  
• ECOG PS  
• Ophthalmic examinations  
• Changes from baseline in laboratory parameters  
• Electrocardiogram (ECGs)  
• Echocardiograms or multigated acquisition (MUGAs)  
• Vital signs 
9.2.2. 
Pharmacokinetics 
Pharmacokinetic endpoints when rebastinib is administered in combination with paclitaxel 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). 


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• 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). 
• Half-life (T1/2). 
• Volume of distribution (Vd). 
• Clearance (CL). 
9.2.3. 
Efficacy 
Radiographic tumor assessments (CT or MRI) will be performed by RECIST Version 1.1. For 
ovarian patients, additional response assessments will be performed using RECIST Version 1.1 
and CA-125 response criteria by the GCIG guideline (Oken et al, 1982). The endpoints for 
preliminary assessment of antitumor activity include: 
• Objective response rate (CR + PR) of the combination therapy. 
• Clinical benefit rate (CBR) (CBR = CR + PR + stable disease [SD]) at 8, 16, and 28 weeks of 
the combination therapy. 
• Time to response (defined as time from Cycle 1 Day 1 to PR or CR). 
• Progression-free-survival (PFS; defined as time from Cycle 1 Day 1 to disease progression or 
death due to any cause). 
• Time to progression (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).  
• Overall survival (OS). 
9.2.4. 
Pharmacogenomics 
The pharmacogenomics endpoints of the study include, but are not limited to:  
• Assessment of polymorphic variations in genes encoding drug metabolic enzymes and/or 
transporters involved in metabolism and disposition of rebastinib. 
• Assessment of polymorphisms in genes that may be associated with clinical response and/or 
study drug-related toxicity. 
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. 


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• Evaluate changes in tumor microenvironment, 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 paclitaxel using the NCI-PRO-
CTCAE, a "treatment-bother" question (GP5) from Functional Assessment of Chronic Illness 
Therapy's (FACIT 2018), 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). 
9.3. 
Populations for Analysis 
Safety Population: The Safety Population is defined as all patients who have received at least 
one dose of either study drug. This population will be used for analysis of safety data. 
Modified intent-to-treat (mITT) Population: mITT Population includes all patients 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 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. This population will be used for analysis of efficacy data. 
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 except for 
identification of TEAEs and 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 20. The same 
algorithm will be used for date of non-study drug medication.  


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Table 20: 
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. 
Data may be displayed by each of the Part 1 arms and Part 2 indication-specific cohorts. 
Continuous variables will be summarized using descriptive statistics (number of patients, mean, 
median, standard deviation, minimum, and maximum). Categorical variables will be summarized 
using frequency distributions and proportions. Time‑to‑event data will be summarized via 
Kaplan Meier (KM) using medians with associated 2-sided 90% confidence intervals (CIs). 
Proportions, when appropriate, will be reported with exact 2-sided 90% confidence intervals. 
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. 


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Unless specified, the mITT Population is used for efficacy analysis and the Safety Population is 
used for the safety analysis. 
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 
(Safety, PK, 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 for the Safety and 
mITT Populations. 
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 each of the Part 1 arms and Part 2 indication-specific cohorts will be 
performed separately. Additionally, analysis may be done by including Part 1 patients who met 
the inclusion criteria and received the same dose level as an RP2D selected for a given 
indication-specific cohort. Note that ORR is a secondary endpoint in Part 1 and a primary 
endpoint in Part 2. 
9.9.1. 
Primary Endpoint  
The primary endpoint in Part 2, 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. 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. Results will be summarized for each cohort with the proportion and 
exact 2-sided 90% confidence intervals.  
9.10. 
Secondary Endpoints 
9.10.1. 
Progression-free Survival  
The PFS (reported in weeks) is defined as the interval between Cycle 1 Day 1 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, including surgical resection or radiation (other than palliative 
radiation to pre-existing bone metastases), or who do not have a documented date of progression 


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or death due to any cause will be censored at the date of the last assessment. Summaries for each 
cohort will be provided using the methods of Kaplan-Meier, and will include medians with 
2-sided 90% confidence intervals. 
9.10.2. 
Time to Progression  
The secondary endpoint of TTP (reported in weeks) is defined as the interval between Cycle 1 
Day 1 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.  
9.10.3. 
Overall Survival  
Overall survival (reported in weeks) is defined as the interval between Cycle 1 Day 1 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 
Kaplan-Meier, and will include medians with 2-sided 90% confidence intervals. Summaries for 
each cohort will be provided using the methods of Kaplan-Meier, and will include medians with 
2-sided 90% confidence intervals. 
9.10.4. 
Time to Response  
The time to response, as defined only for patients with PR or CR as the time from Cycle 1 Day 1 
to the first PR or CR, will be summarized and displayed using the methods of Kaplan-Meier, and 
will include medians with 2-sided 90% confidence intervals.  
9.10.5. 
Clinical Benefit Rate  
Clinical benefit rate (CBR) will be calculated and summarized with n and percentage at 8, 16, 
and 28 weeks, and in the event of disease progression. Clinical benefit will be defined as having 
a response (complete or partial) or stable disease. Proportions with exact 2-sided 90% confidence 
intervals will be reported for each arm or indication-specific cohort.  
9.10.6. 
Duration of Response  
Duration of response, defined as the time from first PR/CR to disease progression or death due to 
any cause, will be calculated for patients who have a PR or CR. Duration of response will be 
summarized and displayed using the methods of Kaplan-Meier and will include medians with 
2-sided 90% confidence intervals. Duration of response will be summarized only for patients 
who have a CR or PR. 


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9.11. 
Safety Analysis 
9.11.1. 
Adverse Events 
Adverse events 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.  
Adverse event toxicity grade will be classified using NCI-CTCAE Version 5.0 criteria (See 
Table 16). 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. Adverse events 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.  
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 arm or indication-specific 
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. For 
categorical parameters, the n and percentage will be displayed for each arm or indication-specific 
cohort. 
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. 
9.11.5. 
Ophthalmologic Assessments  
Ophthalmologic assessments will be summarized by n and percent.  
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 for each arm or indication-specific 
cohort. Shift tables for labs with NCI-CTCAE grades will be presented.  


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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. Geometric means are standard for AUC and Cmax. 
9.13. 
Tumor Marker Analysis 
Assessment of CA-125 in ovarian cancer will be done using GCIG criteria described in (Section 
6.10.2). Otherwise, levels of tumor markers will be summarized overall by time points using 
descriptive statistics. In addition, changes from baseline will be summarized for each arm or 
indication-specific cohort.  
9.13.1. 
Biomarker and Pharmacodynamic Analysis 
Biomarkers and pharmacodynamic parameters will be summarized graphically and with 
descriptive statistics (mean, SD, median, min, max). 
9.14. 
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. 
9.15. 
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, 21CFR 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 


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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 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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SAP_001.pdf: 
 
  
 
Protocol: DCC-2036-01-003  
 
Official Title: An Open-Label, Multicenter, Phase 1b/2 Study of Rebastinib (DCC-2036) in Combination 
with Paclitaxel to Assess Safety, Tolerability, and Pharmacokinetics in Patients with Advanced or 
Metastatic Solid Tumors  
 
NCT Number: NCT03601897  
 
Approval Date: 02 May 2022 


Deciphera Pharmaceuticals, LLC 
DCC-2036-01-003
Statistical Analysis Plan, Version 1.0 
May 2, 2022
1 
STATISTICAL ANALYSIS PLAN 
PHASE 1B/2
DATE OF PLAN: 
May 2, 2022 
BASED ON: 
Protocol DCC-2036-01-003, Amendment 5 (February 7, 2020) 
STUDY TITLE: 
An Open-Label, Multicenter, Phase 1b/2 Study of Rebastinib (DCC-2036) in Combination with 
Paclitaxel 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 #: NCT03601897
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DCC-2036-01-003
Statistical Analysis Plan, Version 1.0 
May 2, 2022
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SIGNATURE PAGE 
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This document has been reviewed and accepted by: 
 
 
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Deciphera Pharmaceuticals, LLC 
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Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-003 
Statistical Analysis Plan, Version 1.0 
 
May 2, 2022 
 
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TABLE OF CONTENTS 
LIST OF ABBREVIATIONS ..........................................................................................................7 
1. 
INTRODUCTION ........................................................................................................9 
2. 
STUDY OBJECTIVES ..............................................................................................10 
2.1. 
Part 1 ...........................................................................................................................10 
2.1.1. 
Primary Objectives .....................................................................................................10 
2.1.2. 
Secondary Objectives .................................................................................................10 
2.2. 
Part 2 ...........................................................................................................................10 
2.2.1. 
Primary Objectives (Part 2) ........................................................................................10 
2.2.2. 
Secondary Objectives (Part 2) ....................................................................................10 
2.3. 
Exploratory Objectives (Parts 1 and 2) .......................................................................10 
3. 
STUDY DESIGN OVERVIEW .................................................................................12 
3.1. 
Overall Study Design ..................................................................................................12 
3.1.1. 
Part 1 ...........................................................................................................................12 
3.1.2. 
Part 2 ...........................................................................................................................12 
3.2. 
Duration of Participation ............................................................................................13 
3.3. 
Study Treatments ........................................................................................................14 
3.4. 
Planned Analyses ........................................................................................................14 
3.4.1. 
Interim Analyses .........................................................................................................14 
3.4.2. 
Final Analyses ............................................................................................................14 
3.5. 
Blinding ......................................................................................................................15 
3.6. 
Sample Size Calculation .............................................................................................15 
4. 
STUDY ENDPOINTS ................................................................................................16 
4.1. 
Efficacy Endpoints ......................................................................................................16 
4.1.1. 
Objective Response Rate ............................................................................................16 
4.1.2. 
Clinical Benefit Rate ...................................................................................................16 
4.1.3. 
Duration of Response .................................................................................................16 
4.1.4. 
Time to Response .......................................................................................................16 
4.1.5. 
Progression-Free Survival ..........................................................................................16 
4.1.6. 
Time to Progression ....................................................................................................16 
4.1.7. 
Overall Survival ..........................................................................................................17 
4.1.8. 
Cancer-Antigen 125 Response (ovarian cancer expansion cohort only) ....................17 
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4.2. 
Safety Endpoints .........................................................................................................17 
4.3. 
Patient-Reported Outcomes ........................................................................................17 
4.4. 
Pharmacokinetic Endpoints ........................................................................................18 
4.5. 
Pharmacogenomic Endpoints .....................................................................................18 
4.6. 
Biomarker and Pharmacodynamic Endpoints ............................................................18 
5. 
DEFINITIONS AND CONVENTIONS FOR DATA HANDLING .........................19 
5.1. 
Definition of Baseline .................................................................................................19 
5.2. 
Handling of Missing Data ...........................................................................................19 
5.3. 
Study Visits .................................................................................................................20 
5.4. 
Study Day ...................................................................................................................20 
5.5. 
Coding Dictionaries ....................................................................................................20 
6. 
ANALYSIS POPULATIONS ....................................................................................21 
6.1. 
Enrolled Population ....................................................................................................21 
6.2. 
Safety Population ........................................................................................................21 
6.3. 
Modified Intent-to-Treat Population ...........................................................................21 
6.4. 
Pharmacokinetic Population .......................................................................................21 
7. 
ANALYSES AND SUMMARIES .............................................................................22 
7.1. 
General Considerations ...............................................................................................22 
7.2. 
Participant Disposition ................................................................................................22 
7.3. 
Protocol Deviations ....................................................................................................24 
7.4. 
Demographics and Baseline Characteristics ...............................................................24 
7.4.1. 
Demographic Characteristics ......................................................................................24 
7.4.2. 
Medical History ..........................................................................................................25 
7.4.3. 
Cancer History ............................................................................................................25 
7.4.4. 
Prior Anti-Cancer Therapy and Procedures ................................................................25 
7.4.5. 
Prior Medications and Procedures ..............................................................................25 
7.4.6. 
Concomitant Medications and Procedures .................................................................26 
7.5. 
Safety Analysis ...........................................................................................................26 
7.5.1. 
Study Drug Exposure ..................................................................................................26 
7.5.2. 
Adverse Events ...........................................................................................................27 
7.5.3. 
Clinical Laboratory Parameters ..................................................................................29 
7.5.4. 
Vital Signs, Weight, and Height .................................................................................31 
7.5.5. 
ECOG Performance Status .........................................................................................31 
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DCC-2036-01-003 
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May 2, 2022 
 
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7.5.6. 
Electrocardiograms .....................................................................................................31 
7.5.7. 
Echocardiogram/Multigated Acquisition Scans .........................................................31 
7.5.8. 
Ophthalmologic Examinations ...................................................................................32 
7.6. 
Efficacy Analysis ........................................................................................................32 
7.6.1. 
Objective Response Rate ............................................................................................32 
7.6.2. 
Clinical Benefit Rate ...................................................................................................33 
7.6.3. 
Duration of Response .................................................................................................34 
7.6.4. 
Time to Response .......................................................................................................34 
7.6.5. 
Progression-Free Survival ..........................................................................................34 
7.6.6. 
Time to Progression ....................................................................................................35 
7.6.7. 
Overall Survival ..........................................................................................................35 
7.6.8. 
CA-125 Response (ovarian cancer expansion cohort only) .......................................35 
7.7. 
Patient-Reported Outcomes Analysis .........................................................................35 
7.8. 
Pharmacokinetic Analysis ..........................................................................................35 
7.9. 
Biomarker and Pharmacodynamic Analysis ...............................................................36 
7.10. 
Pharmacogenomic Analysis........................................................................................36 
8. 
REFERENCES ...........................................................................................................37 
 
 
 
 
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DCC-2036-01-003 
Statistical Analysis Plan, Version 1.0 
 
May 2, 2022 
 
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LIST OF TABLES 
Table 1: 
Partial or Missing Date Imputation Rules ..................................................................19 
Table 2: 
Safety Laboratory Tests ..............................................................................................30 
Table 3: 
Summary of the Best Overall Response Status when Confirmation of 
Response is Required ..................................................................................................33 
 
 
LIST OF FIGURES 
Figure 1: 
Study Schema .............................................................................................................13 
 
 
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DCC-2036-01-003 
Statistical Analysis Plan, Version 1.0 
 
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LIST OF ABBREVIATIONS 
Below is a list of the abbreviation that will be used throughout this document. 
Abbreviation 
Definition 
AE 
Adverse event 
AESI 
Adverse event 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 
CTCAE 
Common Terminology Criteria for Adverse Events 
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 
EOT 
End of Treatment 
FACT-G 
Functional Assessment of Cancer Therapy - General 
IHC 
Immunohistochemical 
ISH 
In situ hybridization 
IV 
Intravenous 
LVEF 
Left ventricular ejection fraction 
MedDRA 
Medical Dictionary for Regulatory Activities 
mITT 
Modified Intent-to-Treat 
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 
ORR 
Objective response rate 
OS 
Overall survival 
PD 
Progressive disease 
PFS 
Progression-free survival 
PK 
Pharmacokinetic 
PR 
Partial response 
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DCC-2036-01-003 
Statistical Analysis Plan, Version 1.0 
 
May 2, 2022 
 
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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 
 
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DCC-2036-01-003 
Statistical Analysis Plan, Version 1.0 
 
May 2, 2022 
 
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1. 
INTRODUCTION 
This statistical analysis plan (SAP) describes the methods to be used in the analysis of efficacy, 
safety, and tolerability data from clinical protocol DCC-2036-01-003 entitled “An Open-Label, 
Multicenter, Phase 1b/2 Study of Rebastinib (DCC-2036) in Combination with Paclitaxel to 
Assess Safety, Tolerability, and Pharmacokinetics in Patients with Advanced or Metastatic Solid 
Tumors” in order to answer the study objectives, and is based on Protocol Amendment 5 of the 
study protocol, dated February 7, 2020. This study comprises of two parts: Part 1 will be 
conducted in approximately seven centers in the United States (US) and Part 2 will be conducted 
in approximately 20 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 
Part 2 only using objective response rate (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. 
 
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Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-003 
Statistical Analysis Plan, Version 1.0 
 
May 2, 2022 
 
12 
3. 
STUDY DESIGN OVERVIEW 
3.1. 
Overall Study Design 
This is an open-label Phase 1b/2 multicenter study in patients with advanced or metastatic solid 
tumors where paclitaxel may be considered appropriate treatment. Adverse events (AEs) will be 
assessed, and laboratory values, vital signs measurements, electrocardiograms (ECGs), 
ophthalmologic examinations, physical examinations, and Eastern Cooperative Oncology Group 
(ECOG) Performance Status will be obtained to evaluate the safety and tolerability of rebastinib 
when administered in combination with paclitaxel. 
Rebastinib will be administered in combination with paclitaxel in repeated 28-day cycles to 
primarily assess the safety, tolerability, and preliminary efficacy of the combination. PK and 
pharmacodynamic samples will be collected at pre-specified time points. 
The study consists of two parts (Part 1 and Part 2).  
3.1.1. 
Part 1 
In Part 1, patients will be assigned to one of two pre-defined dose levels of rebastinib (50 or 100 
mg BID) in combination with paclitaxel administered by intravenous (IV) infusion at 80 mg/m2. 
Each arm is planned to enroll at least 12 evaluable patients. Additionally, if rebastinib at 100 mg 
BID is deemed unsafe, an additional arm dosing at 75 mg BID of rebastinib in combination with 
paclitaxel may be initiated. Up to 36 evaluable patients will be dosed in Part 1. Safety will be 
continuously monitored, and any arm may be terminated early if deemed unsafe by the Sponsor. 
Safety and tolerability, PK and pharmacodynamic, and preliminary efficacy data obtained in Part 
1 will be used to determine the RP2D. Data for at least 12 evaluable patients through Cycle 1 
must be available in an arm to declare the dose as the RP2D. Patients must receive ≥80% of 
planned doses of rebastinib and paclitaxel in Cycle 1 to be considered as evaluable. Enrollment of 
patients will pause for determination of the RP2D prior to initiation of Part 2. An RP2D will be 
chosen by the Sponsor in consultation with the Investigators. 
3.1.2. 
Part 2 
Upon determination of the RP2D, Part 2 will be initiated to dose up to 165 evaluable patients 
using the RP2D across four indication-specific cohorts. A Simon’s two-stage design will be 
applied to Part 2 to further evaluate the safety, tolerability, and preliminary efficacy of rebastinib 
in combination with paclitaxel in triple-negative breast (Cohort 1), inflammatory breast (Cohort 
2), ovarian (Cohort 3), endometrial (Cohort 4), and gynecological carcinosarcoma (Cohort 5) 
cancers.  
Each cohort will initially enroll up to 18 evaluable patients in the first stage. Patients must 
receive at least one dose of the combination and either 1) have at least one post-baseline 
assessment, or 2) be discontinued prior to the first post-baseline disease assessment due to 
disease progression (clinical or radiological) or AE(s) at least possibly related to rebastinib, to be 
considered evaluable. Tumor response will be assessed according to Response Evaluation 
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Criteria in Solid Tumors (RECIST) v1.1. 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. Non-evaluable patients will be replaced and 
not be included in the responder analysis. If >4 responses (defined as partial response [PR] or 
complete response [CR]) 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. Enrollment in each cohort will pause between the first and second stage of the 
Simon two-stage for evaluation of response. The Study Schema for Part 1 and Part 2 is presented 
below (Figure 1). 
Figure 1: 
Study Schema 
 
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. 
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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 administered prior to the paclitaxel infusion. Patients will 
take their assigned dose at the same time each day, approximately 12 hours apart. 
Patients are pre-medicated prior to paclitaxel administration in order to prevent severe 
hypersensitivity reactions per sponsor’s recommendations. Paclitaxel will be administered by IV 
infusion at 80 mg/m2 over approximately 60 minutes on Day 1, Day 8, and Day 15 of repeated 
28-day cycles.  
In Part 1, patients will be assigned to the dose of rebastinib according to the arm in which they 
are enrolled. Patients will be enrolled to receive 50 mg BID or 100 mg BID of rebastinib orally 
in combination with paclitaxel in repeated 28-day cycles. If 100 mg BID is deemed intolerable, a 
cohort of 75 mg BID in combination with paclitaxel will be initiated. 
In Part 2, patients will be assigned to the RP2D of rebastinib and enrolled into the appropriate 
indication-specific cohort. Rebastinib will be administered at the RP2D in combination with 
paclitaxel. For patients that are unable to tolerate paclitaxel, rebastinib may be administered as a 
single agent starting in Cycle 2. 
If the retreatment criteria are not met and a dose of paclitaxel is missed, a single make-up dose of 
paclitaxel may be administered on Day 22 of a given cycle as long as retreatment criteria are 
met. Assessments required on Day 8 of the corresponding cycle must be performed. 
Dose interruptions and modifications may be initiated 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. 
3.4. 
Planned Analyses 
3.4.1. 
Interim Analyses 
No formal interim analyses leading to an interim CSR are planned for this study.  
Informal interim analyses are planned for each Expansion Cohort in Part 2 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 RECIST v1.1, 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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3.5. 
Blinding 
This is an open-label study. 
3.6. 
Sample Size Calculation 
Part 1 will primarily be used to evaluate the safety and tolerability of the combination. Up to 36 
evaluable patients will be enrolled in total with at least 12 evaluable patients in each arm. This is 
considered appropriate for analysis of safety and tolerability to determine the RP2D. 
A Simon’s two-stage design will apply to Part 2 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 in order to demonstrate the target efficacy of >10 responses in a total of 
33 patients. Thus, this part of study may enroll up to 165 (33 patients per indication-specific 
cohort). If a non-evaluable rate of 10% is considered, approximately 182 patients may be 
enrolled. 
 
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Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-003 
Statistical Analysis Plan, Version 1.0 
 
May 2, 2022 
 
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4. 
STUDY ENDPOINTS 
The following study endpoints are for Part 1 and Part 2 of the study. 
4.1. 
Efficacy Endpoints 
For efficacy endpoints based on imaging, RECIST v1.1 (Eisenhauer, et al., 2009) will be used 
for all summaries. 
The primary endpoint in Part 2 is the ORR, which is defined as the proportion of participants 
with a best overall response of CR or PR. per RECIST v1.1. 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. Participants without a best overall response CR or PR will be 
considered as non-responders. 
4.1.2. 
Clinical Benefit Rate  
CBR at 8, 16, and 28 weeks is defined as proportion of participants with overall response of CR, 
PR, or stable disease (SD) at 8, 16, and 28 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.  
DocuSign Envelope ID: 9E5CC4F0-D81E-473F-88F6-A854597554B4


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DocuSign Envelope ID: 9E5CC4F0-D81E-473F-88F6-A854597554B4


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DocuSign Envelope ID: 9E5CC4F0-D81E-473F-88F6-A854597554B4
DocuSign Envelope ID: 9E5CC4F0-D81E-473F-88F6-A854597554B4


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-003 
Statistical Analysis Plan, Version 1.0 
 
May 2, 2022 
 
20 
Medication/Procedure Start 
Date 
Assign 1 unless it is the 
month of first dose of 
study drug. Otherwise, 
assign date of first dose of 
study drug or imputed 
medication end date, 
whichever is earlier. 
Assign January 1 
unless the year is 
year of first dose of 
study drug. 
Otherwise, assign 
date of first dose of 
study drug or 
imputed medication 
end date, whichever 
is earlier. 
Assign date first 
dose of study. 
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: 9E5CC4F0-D81E-473F-88F6-A854597554B4


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-003 
Statistical Analysis Plan, Version 1.0 
 
May 2, 2022 
 
21 
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 patients who are exposed to any dose 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 patients who had at least one full dose 
of the combined study drugs, had measurable disease at baseline, and had at least one 
postbaseline 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 patients 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. Additionally, this population will be used for analysis of pharmacodynamic 
data, if post-dose pharmacodynamic data are available. Analysis of PK data will be described in a 
separate document.  
 
DocuSign Envelope ID: 9E5CC4F0-D81E-473F-88F6-A854597554B4


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DocuSign Envelope ID: 9E5CC4F0-D81E-473F-88F6-A854597554B4


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DocuSign Envelope ID: 9E5CC4F0-D81E-473F-88F6-A854597554B4
DocuSign Envelope ID: 9E5CC4F0-D81E-473F-88F6-A854597554B4


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-003 
Statistical Analysis Plan, Version 1.0 
 
May 2, 2022 
 
32 
Echocardiogram or MUGA assessments will also be presented in data listings. 
7.5.8. 
Ophthalmologic Examinations 
Ophthalmologic examinations will be performed at Screening, Day 1 of Cycle 3, every third 
cycle thereafter (i.e., Cycles 6, 9, 12, etc.), and the EOT visit.  
Participant level analysis of intraocular pressure will also be performed using the maximum 
value from the visit. Categories of intraocular pressure will be defined as follows: ≤21 mmHg, 
>21 mmHg, >21 but ≤30 mmHg, and >30 mmHg. The number and percentage of participants in 
each category will be summarized at baseline, at each visit, and for the maximum post-baseline 
value. The number and percentage of participants with an increase from baseline of at least 5 
mmHg in either eye will also be summarized.  
Ophthalmologic examinations will also be presented in data listings. 
7.6. 
Efficacy Analysis 
All efficacy analyses will be performed in the mITT population for Part 2 only. Tabular 
summaries for best overall response and ORR will be presented by the expansion cohort.  
Response assessments are based on RECIST v1.1 (Eisenhauer, et al., 2009). ORR will be 
analyzed in the mITT population as the primary analysis. ORR with and without confirmation 
will be summarized. 
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 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: 9E5CC4F0-D81E-473F-88F6-A854597554B4


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-003 
Statistical Analysis Plan, Version 1.0 
 
May 2, 2022 
 
33 
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 6 weeks (8 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 8, 16, and 28 weeks, defined as the as proportion of participants with overall 
response of CR, PR, or SD at 8, 16, and 28 weeks, will be derived for each participant. Per 
protocol, participants could have a disease assessment 7 days prior to the 8-, 16-, and 28-week 
DocuSign Envelope ID: 9E5CC4F0-D81E-473F-88F6-A854597554B4


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DocuSign Envelope ID: 9E5CC4F0-D81E-473F-88F6-A854597554B4


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DocuSign Envelope ID: 9E5CC4F0-D81E-473F-88F6-A854597554B4
DocuSign Envelope ID: 9E5CC4F0-D81E-473F-88F6-A854597554B4


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-003 
Statistical Analysis Plan, Version 1.0 
 
May 2, 2022 
 
36 
7.9. 
Biomarker and Pharmacodynamic Analysis 
Biomarker and pharmacodynamic analyses, if conducted, are outside the scope of this SAP. 
7.10. 
Pharmacogenomic Analysis 
Pharmacogenomic analyses, if conducted, are outside the scope of this SAP. 
 
DocuSign Envelope ID: 9E5CC4F0-D81E-473F-88F6-A854597554B4


Deciphera Pharmaceuticals, LLC 
 
DCC-2036-01-003 
Statistical Analysis Plan, Version 1.0 
 
May 2, 2022 
 
37 
8. 
REFERENCES 
Eisenhauer, E. A., Therasse, P., Bogaerts, J., Schwartz, L. H., Sargent, D., Ford, R., . . . Verweij, 
J. (2009, January). New response evaluation criteria in solid tumours: revised RECIST 
guideline (version 1.1). European Journal of Cancer, 45(2), 228-247. 
 
DocuSign Envelope ID: 9E5CC4F0-D81E-473F-88F6-A854597554B4