Conditions: Gastrointestinal Cancer Biliary Tract Cancer
Allocation: NON_RANDOMIZED | Intervention model: SEQUENTIAL
Enrollment: 19
Healthy volunteers: no

Brief summary:
Part 1: Dose-Escalation Phase (Phase 1b) The primary objective is to assess the safety and tolerability of increasing doses of D07001 softgel in patients with unresectable locally advanced or metastatic gastrointestinal (GI) cancer.

Part 2: Dose-Expansion Phase (Phase 2) The primary objective is to assess the safety and tolerability of D07001 softgel in patients who have achieved stable disease or better following first line chemotherapy or combined chemoradiotherapy (CCRT) for unresectable metastatic or locally advanced biliary tract cancer (BTC)

Detailed description:
This open label, multicenter study will be conducted in 2 parts: a dose-escalation phase (Part 1) and a dose-expansion phase (Part 2).

In both Part 1 and Part 2, eligible patients will be assigned to receive oral D07001-softgel on Days 1, 3, 5, 8, 10, 12, 15, 17, and 19 of a 21-day cycle (9 doses per cycle).

Part 1: Dose Escalation Phase (Phase 1b) Part 1 of the study will follow a 3+3 dose escalation scheme at predefined dose levels. There will be sequential cohorts of 3 to 6 patients each with increasing doses of 40 mg, 60 mg, 80 mg, 120 mg, and 160 mg per cohort. There will be no intra patient dose escalation. Cycle 1 (21 days) is defined as the dose limiting toxicity (DLT) assessment period.

Part 2: Dose Expansion Phase (Phase 2) In Part 2 of the study, eligible patients will be randomized in a 1:1 ratio to receive D07001-softgel in an open label manner at 1 of the 2 dose levels selected for expansion. Twenty (20) patients will be enrolled to each dose expansion cohort. Patients will be treated until withdrawal from treatment due to disease progression according to RECIST v1.1, withdrawn consent, or when another treatment discontinuation criterion is met. Patients who are discontinued from study drug for reasons other than disease progression or toxicity in the first 2 cycles of Part 2 will be replaced.

Arms:
arm 1: 40 mg D07001-softgel capsules 60 mg D07001-softgel capsules 80 mg D07001-softgel capsules 120 mg D07001-softgel capsules 160 mg D07001-softgel capsules arm 2: higher dose-expansion of D07001-softgel capsules lower dose-expansion of D07001-softgel capsules

Interventions: intervention 1: D07001-softgel capsules
Intervention descriptions: intervention 1: Active Ingredient:Gemcitabine hydrochloride

Protocol document:
Prot_000.pdf: 
InnoPharmax Inc. 
Clinical Study Protocol 
Product: D07001-softgel 
Version: 4.0 
Protocol No.: Inno-GO-03 
Date: January 03, 2018 
Confidential 
Page 4 of 86 
 
 
Protocol Synopsis 
Sponsor: 
InnoPharmax Inc. 
Investigational Product: 
D07001-softgel capsules 
Active Ingredient: 
Gemcitabine hydrochloride 
Protocol No.: 
Inno-GO-03 
Study Title: 
Open-Label, Multicenter Study of D07001-Softgel Capsules 
(Oral Gemcitabine Hydrochloride) in Subjects with Unresectable, 
Metastatic or Locally Advanced Gastrointestinal Cancer in 
Dose-Escalation Phase and in Subjects with Advanced Biliary 
Tract Cancer Following Primary Chemotherapy or Combined 
Chemoradiotherapy in Dose-Expansion Phase 
Phase of Development: 
1b/2 
Study Site(s): 
The study will be conducted in Taiwan, Korea, and the United 
States of America (USA). 
Indication(s): 
Chemotherapy following primary chemotherapy or combined 
chemoradiotherapy (CCRT) for advanced biliary tract cancer 
(BTC) (cholangiocarcinoma or gallbladder cancer) 
Objectives: 
Part 1: Dose-Escalation Phase (Phase 1b) 
Primary Objective 
 
To assess the safety and tolerability of increasing doses of 
D07001-softgel in patients with unresectable locally 
advanced or metastatic gastrointestinal (GI) cancer 
Secondary Objectives 
 
To evaluate the pharmacokinetic (PK) profile of 
D07001-softgel 
Exploratory Objectives 
 
To evaluate the preliminary data on the efficacy of increasing 
doses of D07001-softgel 
 
To investigate the effect of increasing doses of 
D07001-softgel on additional parameters such as tumor 
markers 
 
To investigate the relationship between the efficacy, safety, 
and PK of D07001-softgel and pharmacogenomic, 
pharmacoproteomic, and cellular markers, including the 
levels of immune cells, circulating endothelial progenitor 
cells and circulating tumor cells 
Part 2: Dose-Expansion Phase (Phase 2) 
Primary Objectives 
 
To assess the safety and tolerability of D07001-softgel in 
patients who have achieved stable disease or better following 


InnoPharmax Inc. 
Clinical Study Protocol 
Product: D07001-softgel 
Version: 4.0 
Protocol No.: Inno-GO-03 
Date: January 03, 2018 
Confidential 
Page 5 of 86 
 
 
first-line chemotherapy or CCRT for unresectable metastatic 
or locally advanced BTC 
Secondary Objectives 
 
To assess the PK profile of D07001-softgel, including the 
effect of food 
Exploratory Objectives 
 
To evaluate the efficacy of D07001-softgel, as assessed by 
progression-free survival (PFS), overall survival (OS), time-
to-progression (TTP), and objective response rate (ORR; in 
patients with measurable disease at baseline only), in patients 
who have achieved stable disease or better following 
first-line chemotherapy or CCRT for unresectable metastatic 
or locally advanced BTC 
 
To investigate the effect of D07001-softgel on tumor markers 
 
To investigate the relationship between the efficacy, safety, 
and PK of D07001-softgel and pharmacogenomic, 
pharmacoproteomic, and cellular markers, including the 
levels of immune cells, circulating endothelial progenitor 
cells and circulating tumor cells 
Study Design: 
This open-label, multicenter study will be conducted in 2 parts: a 
dose-escalation phase (Part 1) and a dose-expansion phase 
(Part 2). 
In both Part 1 and Part 2, eligible patients will be assigned to 
receive oral D07001-softgel on Days 1, 3, 5, 8, 10, 12, 15, 17, 
and 19 of a 21-day cycle (9 doses per cycle).  There will be no 
gap between the cycles, e.g., the second cycle will commence 
immediately on the next day after Day 21 of the first cycle (Day 
22 overall).  Dosing on 2 consecutive days will not be allowed; 
there will be at least 1 day between 2 doses. 
Part 1: Dose-Escalation Phase (Phase 1b) 
Part 1 of the study will follow a 3+3 dose-escalation scheme 
at predefined dose levels.  There will be sequential cohorts of 
3 to 6 patients each with increasing doses of 40 mg, 60 mg, 
80 mg, 120 mg, and 160 mg per cohort.  There will be no 
intra-patient dose escalation.  Cycle 1 (21 days) is defined as the 
dose-limiting toxicity (DLT) assessment period. 
Each cohort will enroll 3 patients.  If none of the 3 patients in the 
cohort experiences a DLT, the study may proceed with dose 
escalation to the next cohort.  If 1 out of 3 patients experiences a 
DLT, 3 additional patients will be entered into that cohort for a 
total of 6 patients.  If none of the 3 additional patients in that 
cohort experiences a DLT, then the next ascending dose cohort 
may proceed.  Dose escalation to a subsequent cohort will only 
proceed after assessment of safety and tolerability data (and raw 
PK data, if available) of the preceding cohort at a review meeting 
of the Safety Board.  If ≥2 patients in a 3-6 patient cohort 
experience a DLT during Cycle 1, the maximum tolerated dose 


InnoPharmax Inc. 
Clinical Study Protocol 
Product: D07001-softgel 
Version: 4.0 
Protocol No.: Inno-GO-03 
Date: January 03, 2018 
Confidential 
Page 6 of 86 
 
 
(MTD) is considered to have been exceeded and no additional 
patients will be treated at the current or higher doses.  In this 
case, the Safety Board may decide to evaluate an intermediate 
dose level 20 mg lower than the dose level at which ≥2 patients 
in a cohort experienced a DLT.  If an MTD is not identified 
following dose escalation to 160 mg, the intermediate dose level 
of 140 mg may also be evaluated.  If an intermediate dose level is 
evaluated, up to 6 additional patients will be enrolled at that dose 
level. 
Patients in Part 1 will continue treatment until withdrawal due to 
a DLT (Cycle 1 only), disease progression according to Response 
Evaluation Criteria in Solid Tumours version 1.1 (RECIST v1.1), 
withdrawn consent, or when another treatment discontinuation 
criterion is met. 
PK samples will be obtained during Cycle 1. For Cycle 1 Day 1 
and Day 15 visits, overnight fasting, except for water and 
medications is required. 
Preliminary efficacy data will be obtained in Part 1.  Tumor 
assessments by RECIST v1.1 will be conducted every 6 weeks 
relative to the first dose of study medication for all patients until 
disease progression, death, or withdrawal of consent.  Disease 
progression will be based on the Investigator’s evaluation of 
tumor assessments.  Patients who are discontinued from study 
drug for a reason other than disease progression, death, or 
withdrawal of consent are to continue tumor assessments on the 
same schedule. 
Part 2: Dose-Expansion Phase (Phase 2) 
The highest dose level of D07001-softgel tested at which <2 of 
6 patients experience a DLT will be expanded in Part 2 of the 
study.  In addition, because the highest dose level selected in 
Part 1 could lead to cumulative toxicity during treatment, the 
next lowest dose level below the highest evaluated in Part 1 at 
which <2 of 6 patients experienced a DLT will also be expanded 
for assessment in Part 2.  If an MTD is not identified in Part 1 of 
the study, the 2 dose levels used in Part 2 will be 160 mg 
and 120 mg, or 160 mg and 140 mg if the intermediate dose of 
140 mg has been evaluated in Part 1. 
In Part 2 of the study, eligible patients will be randomized in a 
1:1 ratio to receive D07001-softgel in an open-label manner at 
1 of the 2 dose levels selected for expansion.  Twenty 
(20) patients will be enrolled to each dose-expansion cohort.  
Patients will be treated until withdrawal from treatment due to 
disease progression according to RECIST v1.1, withdrawn 
consent, or when another treatment discontinuation criterion is 
met.  Patients who are discontinued from study drug for reasons 
other than disease progression or toxicity in the first 2 cycles of 
Part 2 will be replaced. 
As for Part 1, tumor assessments by RECIST v1.1 in Part 2 will 
be conducted every 6 weeks relative to the first dose of study 


InnoPharmax Inc. 
Clinical Study Protocol 
Product: D07001-softgel 
Version: 4.0 
Protocol No.: Inno-GO-03 
Date: January 03, 2018 
Confidential 
Page 7 of 86 
 
 
medication for all patients until disease progression, death, or 
withdrawal of consent.  Disease progression will be based on the 
Investigator’s evaluation of tumor assessments.  Patients who are 
discontinued from study drug for a reason other than disease 
progression, death, or withdrawal of consent are to continue 
tumor assessments on the same schedule. 
PK samples will be obtained during Cycle 1.  For Cycle 1 Day 1 
visit, overnight fasting, except for water and medications is 
required. In addition, for all patients in the higher dose-expansion 
cohort only, the PK of D07001-softgel will be evaluated after a 
high-fat breakfast or after overnight fasting.  Patients will receive 
D07001-softgel on Cycle 1 Day 15 and Cycle 2 Day 1 in a high-
fat fed or a fasting state.  Patients will be randomized to the 
fed/fasted states in 1 of 2 sequences (Sequence 1: Cycle 1 Day 15 
high fat, Cycle 2 Day 1 fasting; Sequence 2: Cycle 1 Day 15 
fasting, Cycle 2 Day 1 high fat) with 10 patients per sequence in 
a cross-over design (20 patients overall).  PK samples will be 
taken over a period of 48 hours post-dose for the food-effect 
evaluation in this dose-expansion cohort. 
Safety, tolerability, efficacy, and PK data will be compared 
between the 2 dose-expansion cohorts to support selection of a 
recommended D07001-softgel dose for further clinical 
development as chemotherapy following primary chemotherapy 
or CCRT for unresectable metastatic or locally advanced BTC. 
Patients in Part 1 and Part 2 who are continuing to derive clinical 
benefit from study drug at the time of study closure, as assessed 
by the Investigator, will be provided access to the study drug via 
a mechanism to be determined by the Sponsor. 
Sample Size: 
No formal sample size calculations have been performed.  In 
Part 1 of the study, 3-6 patients will be enrolled to each of the 
predefined dose cohorts.  If an intermediate dose level is to be 
investigated, another 3-6 patients will be enrolled to that cohort.  
In Part 2, 40 patients will be enrolled to receive D07001-softgel 
at the 2 dose levels selected in Part 1 (20 patients at each dose 
level). 
Inclusion Criteria: 
Patients will be entered into this study only if they meet all of the 
following criteria: 
1. Provision of a signed and dated written Informed Consent 
Form (ICF) prior to any study-specific procedures 
2. Male or female patients aged 18 years or older at screening 
(aged 20 years or older in Taiwan) 
3. Histopathological or cytologic diagnosis of unresectable, 
metastatic or locally advanced GI cancer (Part 1) or 
unresectable metastatic or locally advanced BTC 
(cholangiocarcinoma or gallbladder cancer; Part 2) 
4. Part 1 only: Refractory to or have relapsed from all standard 
therapies of advanced GI malignancy  


InnoPharmax Inc. 
Clinical Study Protocol 
Product: D07001-softgel 
Version: 4.0 
Protocol No.: Inno-GO-03 
Date: January 03, 2018 
Confidential 
Page 8 of 86 
 
 
5. Part 2 only: 
a. Achieved stable disease or better, based on the 
Investigator’s assessment, in response to first-line 
systemic therapy or CCRT, with continued stable disease 
or better based on imaging studies obtained as part of 
screening  
b. Completed first-line systemic therapy (with 2-8 cycles of 
chemotherapy with a gemcitabine-based regimen) or 
CCRT, based on the local standard of care and 
preferences in the participating countries 
Note: No more than 30% of patients enrolled in Part 2 
will have received CCRT 
6. No more than 60 days have elapsed between completion of 
the prior line of chemotherapy or CCRT and enrollment 
7. Part 2 only: Patient has not received intervening systemic 
therapy since first-line treatment 
8. Eastern Cooperative Oncology Group (ECOG) Performance 
Status (PS) of 0-2 in Part 1 and 0-1 in Part 2 
9. Life expectancy is >12 weeks 
10. Adequate bone marrow function, demonstrated by: 
a. Absolute neutrophil count (ANC) ≥1,500 cell/mm3 
b. Platelet count ≥100,000 cells/mm3 
c. Hemoglobin ≥9 g/dL 
11. Adequate liver function, demonstrated by: 
a. Aspartate transaminase (AST) and alanine 
transaminase (ALT) ≤2.5 x upper limit of normal 
(ULN), or ≤5.0 x ULN in the case of liver metastases 
b. Total bilirubin ≤1.5 x ULN 
c. Albumin ≥3.0 g/dL 
d. International normalized ratio (INR) <1.5 
12. Adequate renal function, demonstrated by: 
a. Serum creatinine ≤1.5 x ULN 
b. Creatinine clearance ≥ 60mL/min calculated by 
Cockcroft-Gault formula or directly measured with 
24hr urine collection 
13. If a woman of childbearing potential, the patient has a 
negative serum pregnancy test at screening and is not 
breastfeeding 
14. If a woman of childbearing potential, patient must use a 
medically acceptable form of contraception as 2 barrier 
methods (e.g., combination of condom, diaphragm, or 
intrauterine device), hormonal contraception (estrogen or 
progesterone agents) or 1 barrier method in combination with 
spermicide.  Birth control is required 1 month prior to 
screening, for the duration of their study participation, and 
for 1 month after the end of the study; female partners of 
male patients must adhere to the same birth control methods. 


InnoPharmax Inc. 
Clinical Study Protocol 
Product: D07001-softgel 
Version: 4.0 
Protocol No.: Inno-GO-03 
Date: January 03, 2018 
Confidential 
Page 9 of 86 
 
 
15. Patient is willing to comply with protocol-required visit 
schedule and visit requirements 
Exclusion Criteria: 
Patients will not be entered into this study if they meet any of the 
following criteria: 
1. Part 2 only: More than one prior chemotherapy regimen for 
unresectable metastatic or locally advanced BTC 
Note: prior radiation (with or without radiosensitizing doses 
of chemotherapy) or fluoropyrimidine chemotherapy are 
allowed as postsurgical adjuvant therapy. 
2. Part 2 only: Received any systemic therapy (chemotherapy, 
biologics, immunotherapy, or investigational agents) for 
metastatic disease other than gemcitabine-based 
chemotherapy or CCRT for locally advanced BTC 
3. Diagnosis of active malignancy (other than GI cancer 
[Part 1] or BTC [Part 2]) within the past 2 years, except 
nonmelanoma skin carcinoma and carcinoma-in-situ of 
uterine cervix treated with curative intent 
4. Prior discontinuation of gemcitabine because of pulmonary 
or hepatic toxicity or hemolytic uremic syndrome (HUS) or 
hypersensitivity, allergic reaction, or intolerance 
5. Any GI disorder which would significantly impede 
absorption of an oral agent 
6. Known brain or leptomeningeal metastases 
7. Surgery or radiation therapy within the past 28 days 
8. Part 2 only: Evidence of disease progression, based on the 
Investigator’s assessment, on the screening computed 
tomography (CT) scan or magnetic resonance imaging (MRI) 
scan 
9. Any active disease or condition that would not permit 
compliance with the protocol 
10. Residual toxicity from prior chemotherapy or CCRT that is 
Grade ≥2 (residual Grade 2 neuropathy and alopecia are 
permitted) 
11. Clinically significant cardiovascular disease (e.g., 
uncontrolled hypertension, unstable angina, congestive heart 
failure, or New York Heart Association [NYHA] Grade 2 or 
greater), or uncontrolled serious cardiac arrhythmia 
12. Patient has a history of drug or alcohol abuse within last year 
13. Patient has documented cerebrovascular disease 
14. Patient has a seizure disorder not controlled on medication 
(based on decision of Investigator) 
15. Patient received an investigational agent within 28 days of 
enrollment 
16. Patients with uncontrolled active viral, bacterial, or systemic 


InnoPharmax Inc. 
Clinical Study Protocol 
Product: D07001-softgel 
Version: 4.0 
Protocol No.: Inno-GO-03 
Date: January 03, 2018 
Confidential 
Page 10 of 86 
 
 
fungal infection 
17. Patient has known human immunodeficiency virus (HIV) 
infection 
18. Patient has hepatitis B virus (HBV) and/or hepatitis C virus 
(HCV) infection in medical history.  If positive results are 
not indicative of true active or chronic infection, the patient 
can enter the study after discussion and agreement between 
the Investigator and the Clinical Research Organization 
(CRO) Medical Monitor 
19. Patient has received yellow fever vaccine or other live 
attenuated vaccine(s) within the 4 weeks prior to screening 
20. Patient has any other serious medical condition that, in the 
Investigator’s medical opinion, would preclude safe 
participation in, and compliance with, a clinical trial 
Primary Endpoints and 
Secondary Endpoints: 
Part 1: Dose-Escalation Phase (Phase 1b) 
Primary Endpoint: 
 
The safety endpoints are: 
o DLTs 
o MTD 
o Hematology, serum chemistry, coagulation 
parameters, and urinalysis laboratory data changes 
o Adverse event (AE)/serious adverse event (SAE) 
incidence 
o Incidence of patients experiencing toxicity Grade ≥3 
according to Common Terminology Criteria for 
Adverse Events (CTCAE) v4.03 
o Physical examination result changes 
o Vital signs changes 
o Electrocardiogram (ECG) results (including PR, 
QRS, QT, QTc, and RR intervals) 
Secondary Endpoints: 
 
PK parameters: maximum plasma concentration (Cmax), time 
to maximum plasma concentration (Tmax), area under the 
curve (AUC), elimination half-life (t1/2 el), apparent clearance 
(Cl/F), and apparent volume of distribution (Vz/F) 
Exploratory Endpoints: 
 
The efficacy endpoints are: 
o PFS 
o OS 
o TTP 
o ORR (in patients with measurable disease at baseline 
only) 
 
Tumor marker (cancer antigen [CA]-19-9, carcinoembryonic 
antigen [CEA]) levels at the times specified in the event 
schedule 


InnoPharmax Inc. 
Clinical Study Protocol 
Product: D07001-softgel 
Version: 4.0 
Protocol No.: Inno-GO-03 
Date: January 03, 2018 
Confidential 
Page 11 of 86 
 
 
 
Pharmacogenomic, pharmacoproteomic, and cellular analysis 
Part 2: Dose-Expansion Phase (Phase 2) 
Primary Endpoints: 
 
The safety endpoints are: 
o Dose interruptions/modifications 
o Hematology, serum chemistry, coagulation 
parameters, and urinalysis laboratory data changes 
o AE/SAE incidence 
o Incidence of patients experiencing toxicity Grade ≥3 
according to CTCAE v4.03 
o Physical examination result changes 
o Vital signs changes 
o ECG results (including PR, QRS, QT, QTc, and RR 
intervals) 
Secondary Endpoints: 
 
PK parameters: Cmax, Tmax, AUC, t1/2 el, Cl/F, and Vz/F 
Exploratory Endpoints: 
 
The efficacy endpoints are: 
o PFS 
o OS 
o TTP 
o ORR (in patients with measurable disease at baseline 
only) 
 
Tumor marker (CA-19-9, CEA) levels at the times specified 
in the event schedule 
 
Pharmacogenomic, pharmacoproteomic, and cellular analysis 
Drug Dosage/Cohorts: 
 
Part 1: Dose-Escalation Phase (Phase 1b)  
Patients will receive D07001-softgel in the fasted state 3 times 
per week in 21-day cycles.  Treatment will be administered in 
cohorts of 3-6 patients at dose levels of 40 mg, 60 mg, 80 mg, 
120 mg, and 160 mg.  Intermediate dose levels may be assessed 
in additional cohorts of 3-6 patients if considered necessary by 
the Safety Board. 
Part 2: Dose-Expansion Phase (Phase 2) 
Forty (40) patients will receive D07001-softgel in the fasted state 
3 times per week in 21-day cycles at the 2 dose levels selected in 
Part 1 of the study (20 patients per dose-expansion cohort). 
Route of Administration: 
Oral 
Study Duration: 
The study duration for an individual patient is estimated to be a 
total of approximately 17 months, including approximately 
8 months of treatment and an additional 9 months of follow up 
for survival. 
Efficacy Assessment: 
Tumor assessment will be made at screening using CT or MRI 
scans as measured by RECIST v1.1 guidelines.  Patients will be 
assessed every 2 cycles (6 weeks) starting from 0-7 calendar days 
before the start of Cycle 3 until disease progression, death, or 


InnoPharmax Inc. 
Clinical Study Protocol 
Product: D07001-softgel 
Version: 4.0 
Protocol No.: Inno-GO-03 
Date: January 03, 2018 
Confidential 
Page 12 of 86 
 
 
withdrawal of consent.  The imaging technique (CT or MRI) 
used at screening should be used throughout the study.  Patients 
will be considered evaluable for efficacy if they have at least 
1 disease assessment on study.  OS will be assessed by follow-up 
telephone contact every 6 weeks. 
Pharmacokinetic 
Assessment: 
In both Part 1 and 2 of the study, blood samples for PK will be 
collected at the following timepoints: 
 
Cycle 1 Days 1 and 15: pre-dose (within 0.5 hour before 
study drug administration) and 0.25, 0.5, 1, 2, 4, 6, 8, 24, and 
48 hours post-dose 
 
Cycle 1 Day 8: pre-dose (within 0.5 hour before study drug 
administration) 
In Part 2 only, blood samples for the food-effect evaluation will 
be collected at the following timepoints: 
 
Cycle 1 Day 15 and Cycle 2 Day 1: pre-dose (within 0.5 hour 
before study drug administration) and 0.25, 0.5, 1, 2, 4, 6, 8, 
24, and 48 hours post-dose. 
PK Evaluations: for both Part 1 and 2, plasma samples will be 
analyzed for gemcitabine (dFdC) and difluorodeoxyuridine 
(dFdU) concentrations using validated liquid chromatography 
(LC)/mass spectrometry (MS)/MS assays; resulting data for each 
analyte will be used to calculate the following PK parameters: 
 
dFdC and dFdU (Cycle 1 Days 1, 8, and 15): 
o AUC(0-t), AUC(0-24), AUC(0-48), AUC(0-inf), Cmax, Tmax, 
t1/2 el, %AUCextrapolated 
 
dFdC (Cycle 1 Days 1, 8, and 15): 
o Cl/F, Vz/F 
For Part 1 and 2, accumulation ratios for dFdC will be calculated 
for Day 15 relative to Day 1 for all AUC parameters and Cmax. 
For Part 1 and the lower dose-expansion cohort in Part 2, the 
accumulation ratios for dFdU will be calculated for Day 15 
relative to Day 1 for all AUC parameters and Cmax, and the 
metabolic ratio will be calculated for dFdU for Days 1 and 15 for 
all AUC parameters. 
For the food-effect evaluation in the higher dose-expansion 
cohort in Part 2, bioequivalence of the fed and fasted states will 
be evaluated for AUC(0-t) and Cmax (primary) and AUC(0-inf) 
(secondary), based on the 90% confidence interval (CI) within 
the predefined bioequivalence boundaries 0.8-1.25. 
DLT and Safety Assessment: Safety and tolerability will be assessed throughout treatment and 
the 30-day follow-up period.  National Cancer Institute (NCI) 
CTCAE v4.03 will be used for grading toxicities. 
DLT definition: In Part 1 of the study, any of the following AEs 
occurring during Cycle 1 will be classified as DLTs, if there is a 
reasonable possibility that it is related to the study drug 


InnoPharmax Inc. 
Clinical Study Protocol 
Product: D07001-softgel 
Version: 4.0 
Protocol No.: Inno-GO-03 
Date: January 03, 2018 
Confidential 
Page 13 of 86 
 
 
 
Hematologic: 
o Grade 4 neutropenia lasting >7 days 
o Febrile neutropenia (defined as neutropenia 
Grade ≥3 and a body temp ≥38.3°C) 
o Grade ≥3 neutropenic infection 
o Grade 4 anemia 
o Grade ≥3 thrombocytopenia with bleeding 
o Grade 4 thrombocytopenia  
 
any platelet count <10,000/µL 
 
platelet count 10,000-25,000/µL for >5 days 
 
Non-hematologic: 
o Grade ≥3 toxicities that are considered clinically 
significant, except those that have not been 
maximally treated (e.g., nausea, vomiting, diarrhea*) 
or can be easily treated (e.g., electrolyte 
abnormalities). 
[*Nausea, vomiting, and diarrhea: if Grade ≥3 
toxicities persist for more than 48 h despite 
maximum treatment (loperamide, ondansetron, etc.), 
they will be considered dose limiting] 
 
Delay by more than 7 days in receiving the next scheduled 
cycle due to persisting toxicities attributable to study drug. 
 
Failure to deliver at least 6 of the planned 9 doses during 
Cycle 1 due to treatment-related toxicities. 
 
Upon the second occurrence of a toxicity leading to a dose 
hold. 
Safety will be further assessed throughout the study by: 
 
The incidence of AEs, SAEs, deaths on study, and dose 
modifications (dose reduction, interruption, or 
discontinuation of study drug) 
 
The change from baseline in clinical laboratory test results, 
(hematology, serum chemistry, coagulation parameters, 
urinalysis), and tumor markers 
 
Vital signs measurements 
 
Physical examination findings 
 
ECOG PS results 
 
ECG findings (including PR, QRS, QT, QTc, and RR 
intervals) 
Statistical Considerations: 
Analysis Sets 
Part 1 
The following analysis sets will be defined for Part 1 of the 
study: 
 
Enrolled Set (ENS): all patients who provided informed 
consent 


InnoPharmax Inc. 
Clinical Study Protocol 
Product: D07001-softgel 
Version: 4.0 
Protocol No.: Inno-GO-03 
Date: January 03, 2018 
Confidential 
Page 14 of 86 
 
 
 
Safety Analysis Set (SAF): all patients who received at 
least 1 dose of D07001-softgel 
 
PK Analysis Set (PKS): all patients in the SAF with 
evaluable PK data, and who have no major protocol 
deviations considered to impact the analysis of the PK 
data 
Part 2 
The following analysis sets will be defined for Part 2 of the 
study: 
 
ENS: all patients who provided informed consent 
 
SAF: all patients who received at least 1 dose of study 
drug, classified by actual dose received 
 
Modified Intent-to-Treat Set (mITT): all randomized 
patients who received at least 1 dose of study drug and 
with at least 1 post-baseline disease assessment 
Note: Patients who do not meet eligibility criteria prior to 
randomization but are still randomized into the study will 
be excluded from the mITT Set. 
 
Per-Protocol Set: a subset of the mITT Set that excludes 
patients who have major protocol deviations 
 
PKS: all patients in the SAF with evaluable PK data, and 
who have no major protocol deviations considered to 
impact the analysis of the PK data 
Data for patients in Part 1 and Part 2 will be analyzed separately.   
Data for Part 1 and Part 2 will be summarized using descriptive 
statistics (number of patients, mean, median, standard deviation 
(StD), minimum, and maximum) for continuous variables and 
using frequency and percentages for discrete variables.  Unless 
otherwise specified, the baseline assessment will be the latest, 
valid pre-dose assessment available. 
Safety and PK analyses will be performed using descriptive 
statistics. 
Exploratory analyses for efficacy of each dose-expansion cohort 
in Part 2, time-to-event estimates and survival curves will be 
generated using Kaplan-Meier methodology for PFS and other 
survival outcomes. 

SAP_001.pdf: 
 
Statistical Analysis Plan 
InnoPharmax Inc. 
 
Final 1.0 
Protocol No.: Inno-GO-03 
 
21/Jan/2019 
  
Page 1 of 63 
STATISTICAL ANALYSIS PLAN 
 
 
 
 
Inno-GO-03 
 
Open-Label, Multicenter Study of D07001-Softgel Capsules (Oral Gemcitabine Hydrochloride) in 
Subjects with Unresectable, Metastatic or Locally Advanced Gastrointestinal Cancer in Dose-
Escalation Phase and in Subjects with Advanced Biliary Tract Cancer Following Primary 
Chemotherapy or Combined Chemoradiotherapy in Dose-Expansion Phase 
 
 
 
 
Version:  Final 1.0 
Date: 21/Jan/2019 
REVISION HISTORY 
Version 
Version Date 
Author 
Summary of Changes Made 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 


 
Statistical Analysis Plan 
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Final 1.0 
Protocol No.: Inno-GO-03 
 
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SIGNATURE PAGE – INNOPHARMAX INC.  
Declaration 
The undersigned has/have reviewed and agree to the statistical analyses and procedures of this clinical study, as 
presented in this document. 
 
 
 
 
 
 
Kelly Lin 
 
Date (DD Mmm YY) 
Director, Medical Affairs 
 
 
 
 
 
 




 
Statistical Analysis Plan 
InnoPharmax Inc. 
 
Final 1.0 
Protocol No.: Inno-GO-03 
 
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TABLE OF CONTENTS 
REVISION HISTORY .................................................................................................................... 1 
SIGNATURE PAGE – INNOPHARMAX INC.  ........................................................................... 2 
SIGNATURE PAGE – (CRO) ........................................................................................................ 3 
TABLE OF CONTENTS ................................................................................................................ 4 
LIST OF TABLES .......................................................................................................................... 6 
ABBREVIATION AND ACRONYM LIST .................................................................................. 7 
STATISTICAL ANALYSIS PLAN ............................................................................................. 10 
1. 
STUDY OBJECTIVES.................................................................................................... 10 
1.1 
Primary Objectives ............................................................................................. 10 
1.2 
Secondary Objectives ......................................................................................... 10 
1.3 
Exploratory Objectives ....................................................................................... 11 
2. 
STUDY DESIGN ............................................................................................................ 11 
3. 
STUDY POPULATION .................................................................................................. 14 
4. 
STATISTICAL BASIS FOR SAMPLE SIZE ................................................................. 14 
5. 
RANDOMIZATION ....................................................................................................... 14 
6. 
STATISTICAL ANALYSIS CONVENTIONS .............................................................. 15 
6.1 
Analysis Variables .............................................................................................. 15 
6.1.1 
Demographic and Background Variables ........................................................15 
6.1.2 
Safety Variables ..............................................................................................16 
6.1.2.1 
Adverse Events ................................................................................16 
6.1.2.2 
Clinical Laboratory Tests .................................................................18 
6.1.2.3 
Vital Signs ........................................................................................19 
6.1.2.4 
Electrocardiogram ............................................................................19 
6.1.2.5 
Echocardiogram/MUGA ..................................................................20 
6.1.2.6 
Physical Examination .......................................................................20 
6.1.2.7 
Pregnancy .........................................................................................20 
6.1.2.8 
Prior and Concomitant Medication ..................................................20 
6.1.3 
Pharmacokinetic Variables ..............................................................................20 
6.1.3.1 
Pharmacokinetic Parameter Calculation Methods ...........................23 
6.1.4 
Efficacy Variables ...........................................................................................30 
6.1.4.1 
Tumor assessments ..........................................................................30 
6.1.4.2 
Other efficacy endpoints ..................................................................30 
6.1.5 
Tumor Marker .................................................................................................31 
6.1.6 
Pharmacogenomic, Pharmacoproteomic, and Cellular Marker .......................31 
6.2 
Analysis Populations .......................................................................................... 31 
6.2.1 
Enrolled Set .....................................................................................................31 
6.2.2 
Safety Analysis Set ..........................................................................................31 
6.2.3 
Pharmacokinetic Set ........................................................................................32 


 
Statistical Analysis Plan 
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Final 1.0 
Protocol No.: Inno-GO-03 
 
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6.2.4 
Efficacy Analysis Set ......................................................................................32 
6.2.4.1 
Modified Intent-to-Treat (mITT) Set ...............................................32 
6.2.4.2 
Per-Protocol (PP) Set .......................................................................32 
6.3 
Statistical Analysis Methods .............................................................................. 32 
6.3.1 
Listings and Descriptive Statistics ..................................................................33 
6.3.2 
Statistical Significance Level ..........................................................................33 
6.3.3 
Software ..........................................................................................................33 
6.3.4 
Missing Data ...................................................................................................33 
6.3.5 
Interim Analysis and Early Stopping Guidelines ............................................33 
6.3.6 
Protocol Deviations .........................................................................................34 
6.3.7 
Disposition of Patients.....................................................................................34 
6.3.8 
Demographic Data and Baseline Characteristics ............................................34 
6.3.9 
Medical History ...............................................................................................34 
6.3.10 
Prior and Concomitant Medication .................................................................35 
6.3.11 
Exposure to the Investigational Medicinal Product .........................................35 
6.3.12 
Pharmacokinetic Concentrations and Variables ..............................................35 
6.3.12.1 Handling of Values Below the Limit of Quantification (BLQ) in 
Concentration Summaries and Listings ...........................................36 
6.3.12.2 Assessment of Food Effect ..............................................................37 
6.3.12.3 Assessment of Dose Proportionality ................................................37 
6.3.13 
Safety Analysis ................................................................................................38 
6.3.13.1 Adverse Events ................................................................................38 
6.3.13.2 Clinical 
Safety 
Laboratory 
Tests 
(hematology, 
biochemistry, 
coagulation parameters and urinalysis) ............................................39 
6.3.13.3 Vital Signs, Weight, BMI and ECOG PS ........................................40 
6.3.13.4 Twelve-Lead Electrocardiogram ......................................................40 
6.3.13.5 Echocardiogram/MUGA ..................................................................40 
6.3.13.6 Physical Examination and Pregnancy ..............................................41 
6.3.14 
Efficacy Analysis ............................................................................................41 
6.3.15 
Tumor Marker and Others ...............................................................................42 
7. 
REFFRENCES................................................................................................................. 43 
8. 
TABLES AND LISTINGS TO BE INCLUDED IN SETION 14 OF THE CLINICAL 
STUDY REPORT ............................................................................................................ 44 
9. 
FIGURES ......................................................................................................................... 52 
10. 
LISTINGS TO BE INCLUDED IN SECTION 16 OF THE CLINICAL STUDY REPORT
 ......................................................................................................................................... 53 
11. 
DOCUMENTATION OF STATISTICAL METHODS.................................................. 59 
12. 
EVENT SCHEDULES .................................................................................................... 60 
 
 
 


 
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Protocol No.: Inno-GO-03 
 
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LIST OF TABLES 
Table 1 Pharmacokinetic Parameters For Part 1, Dose Escalation Phase - Cycle 1, Days 1, 2, 3, 8, 
15, 16 and 17 .................................................................................................................... 21 
Table 2 Pharmacokinetic Parameters for Part 2 Dose Expansion Phase - Cycle 1, Days 1, 2, 3, 8, 15, 
16 and 17 and  Cycle 2, Days 1, 2 and 3.......................................................................... 22 
Table 3 Summary of Required Pharmacokinetic Parameters for Dose Escalation ....................... 25 
Table 4 Summary of Required Pharmacokinetic Parameters for Dose Expansion ...................... 27 
Table 5 Part 1 Event Schedule (Dose Escalation) ........................................................................ 60 
Table 6 Part 2 Event Schedule (Dose Expansion) ........................................................................ 62 
 
 
 


 
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Final 1.0 
Protocol No.: Inno-GO-03 
 
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ABBREVIATION AND ACRONYM LIST  
Abbreviation / Acronym 
Definition / Expansion 
AE 
Adverse event 
ALT 
Alanine transaminase 
ALP 
Alkaline phosphatase 
ANC 
Absolute neutrophil count 
aPTT 
Activated partial thromboplastin time 
AST 
Aspartate transaminase 
ATC 
Anatomical therapeutic chemical 
AUC 
Area under the concentration-time curve 
%AUCextrapolated 
Percentage area under the plasma concentration-time curve 
extrapolated 
AUC(0-inf)  
AUC from time zero extrapolated to infinity 
AUClast  
AUC from time zero to the last quantifiable concentration 
BOR 
Best Overall Response 
BLQ 
Below the lower limit of quantification 
BMI 
Body Mass Index 
Bpm 
Beats per minute 
BTC 
Biliary tract cancer 
BUN 
Blood urea nitrogen 
CA 
Cancer antigen 
CCRT 
Combined chemoradiotherapy 
CEA 
Carcinoembryonic antigen 
CI 
Confidence interval 
CL/F 
Apparent clearance  
CSP 
Clinical Study Protocol 
Cmax 
Maximum observed concentration 
Cmin 
Minimum observed concentration in the dosing interval 
Ctrough 
Trough concentration  
CR 
Complete response 
CS 
Clinically significant 
CSR 
Clinical Study Report 


 
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Final 1.0 
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Abbreviation / Acronym 
Definition / Expansion 
CTCAE 
Common Terminology Criteria for Adverse Events 
CV 
Coefficient of variation 
dFdC 
Gemcitabine 
dFdU 
Difluorodeoxyuridine 
DLT 
Dose-limiting toxicity 
DBP 
Diastolic blood pressure 
ECG 
Electrocardiogram 
ECOG 
Eastern Cooperative Oncology Group 
ENS 
Enrolled set 
EOT 
End of treatment 
gCV 
geometric coefficient of variation 
γ-GT 
γ-glutamyl transpeptidase 
GI 
Gastrointestinal 
HR 
Heart rate 
IMP 
Investigational Medicinal Product 
IV 
Intravenous 
IVRS 
Interactive voice response system 
LDH 
Lactate dehydrogenase 
LLOQ 
Lower limit of quantification 
MCH 
Mean corpuscular hemoglobin 
MCHC 
Mean corpuscular hemoglobin concentration 
MCV 
Mean corpuscular volume 
MedDRA 
Medical Dictionary for Regulatory Activities 
mITT 
Modified Intent-to-Treat 
MTD 
Maximum tolerated dose 
MUGA 
Multigated acquisition 
NCS 
Not clinically significant 
NK 
Not known 
ORR 
Objective response rate 
OS 
Overall survival 
PD 
Progressive disease 
PFS 
Progression-free survival 


 
Statistical Analysis Plan 
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Abbreviation / Acronym 
Definition / Expansion 
PK 
Pharmacokinetic 
PKS 
Pharmacokinetic analysis set 
PP 
Per-Protocol 
PR 
Partial response 
PS 
Performance status 
RBC 
Red blood cell 
RDI 
Relative dose intensity 
RECIST 
Response Evaluation Criteria in Solid Tumors 
SAE 
Serious adverse event 
SAF 
Safety Analysis Set 
SAP 
Statistical Analysis Plan 
SBP 
Systolic blood pressure 
SD 
Standard deviation/ Stable disease 
SE 
Standard error of the mean 
SOC 
System Organ Class 
t½ 
Apparent terminal elimination half-life  
tlast 
Time of last quantifiable concentration 
TEAE 
Treatment-emergent adverse event 
tmax 
Time corresponding to occurrence of Cmax 
TTP 
Time-to-progression 
ULN 
Upper limit of normal 
VAS 
Visual analogue scale 
Vz/F 
Apparent volume of distribution 
WBC 
White blood cell 
WHO-DD 
World Health Organization - Drug Dictionary 
λz 
Terminal elimination rate constant 
 
 


 
Statistical Analysis Plan 
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Protocol No.: Inno-GO-03 
 
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STATISTICAL ANALYSIS PLAN 
The Statistical Analysis Plan (SAP) details the statistical methodology to be used in analyzing study data and 
outlines the statistical programming specifications for the Tables, Listings and Figures (TLFs). It describes the 
variables and populations, anticipated data transformations and manipulations and other details of the analyses 
not provided in the Clinical Study Protocol (CSP). 
The analyses described are based on the final CSP Version 4.0, dated 03 January 2018 The SAP will be finalized 
prior to database lock and describes the statistical analysis as it is foreseen when the study is being planned. If 
circumstances should arise during the study rendering this analysis inappropriate, or if improved methods of 
analysis should arise, updates to the analyses may be made. If after data base lock there are important changes in 
the data analysis relative to what is described in the final SAP, those new analysis methods and the reasons for 
such new analysis will be described in a separate SAP Addendum. 
 
1. 
STUDY OBJECTIVES 
This open-label, multicenter Phase 1b/2 study will be conducted in 2 parts: a dose-escalation phase (Part 1) and a 
dose-expansion phase (Part 2). 
1.1 
Primary Objectives 
Part 1: Dose-Escalation Phase (Phase 1b) 
 
To assess the safety and tolerability of increasing doses of D07001-softgel in patients with 
unresectable locally advanced or metastatic gastrointestinal (GI) cancer 
Part 2: Dose-Expansion Phase (Phase 2) 
 
To assess the safety and tolerability of D07001-softgel in patients who have achieved stable 
disease or better following first-line chemotherapy or combined chemoradiotherapy (CCRT) for 
unresectable metastatic or locally advanced biliary tract cancer (BTC) 
1.2 
Secondary Objectives 
Part 1: Dose-Escalation Phase (Phase 1b) 
 
To evaluate the pharmacokinetic (PK) profile of D07001-softgel 


 
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Part 2: Dose-Expansion Phase (Phase 2) 
 
To assess the PK profile of D07001-softgel, including the effect of food 
1.3 
Exploratory Objectives 
Part 1: Dose-Escalation Phase (Phase 1b) 
 
To evaluate the preliminary data on the efficacy of increasing doses of D07001-softgel 
 
To investigate the effect of increasing doses of D07001-softgel on additional parameters such as 
tumor markers 
 
To investigate the relationship between the efficacy, safety, and PK of D07001-softgel and 
pharmacogenomic, pharmacoproteomic, and cellular markers, including the levels of immune 
cells, circulating endothelial progenitor cells and circulating tumor cells 
Part 2: Dose-Expansion Phase (Phase 2) 
 
To evaluate the efficacy of D07001-softgel, as assessed by progression-free survival (PFS), 
overall survival (OS), time to progression (TTP), and objective response rate (ORR; in patients 
with measurable disease at baseline only), in patients who have achieved stable disease or better 
following first-line chemotherapy or CCRT for unresectable metastatic or locally advanced BTC 
 
To investigate the effect of D07001-softgel on tumor markers 
 
To investigate the relationship between the efficacy, safety, and PK of D07001-softgel and 
pharmacogenomic, pharmacoproteomic, and cellular markers, including the levels of immune 
cells, circulating endothelial progenitor cells and circulating tumor cells 
 
2. 
STUDY DESIGN 
This open label, multicenter study will be conducted in 2 parts: a dose escalation phase (Part 1) and a dose 
expansion phase (Part 2). 
In both Part 1 and Part 2, eligible patients will be assigned to receive oral D07001-softgel on Days 1, 3, 5, 8, 10, 
12, 15, 17, and 19 of a 21 day cycle (9 doses per cycle).  There will be no gap between the cycles, e.g., the 


 
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InnoPharmax Inc. 
 
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Protocol No.: Inno-GO-03 
 
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second cycle will commence immediately on the next day after Day 21 of the first cycle (Day 22 overall).  
Dosing on 2 consecutive days will not be allowed; there will be at least 1 day between 2 doses. 
Part 1: Dose Escalation Phase (Phase 1b) 
Part 1 of the study will follow a 3+3 dose escalation scheme at predefined dose levels.  There will be sequential 
cohorts of 3 to 6 patients each with increasing doses of 40 mg, 60 mg, 80 mg, 120 mg, and 160 mg per cohort.  
There will be no intra patient dose escalation.  Cycle 1 (21 days) is defined as the dose limiting toxicity (DLT) 
assessment period. 
Each cohort will enroll 3 patients.  If none of the 3 patients in the cohort experiences a DLT, the study may 
proceed with dose escalation to the next cohort.  If 1 out of 3 patients experience a DLT, 3 additional patients 
will be entered into that cohort for a total of 6 patients.  If none of the 3 additional patients in that cohort 
experiences a DLT, then the next ascending dose cohort may proceed.  Dose escalation to a subsequent cohort 
will only proceed after assessment of safety and tolerability data (and raw PK data, if available) of the preceding 
cohort at a review meeting of the Safety Board.  If ≥2 patients in a 3-6 patient cohort experience a DLT during 
Cycle 1, the maximum tolerated dose (MTD) is considered to have been exceeded and no additional patients will 
be treated at the current or higher doses.  In this case, the Safety Board may decide to evaluate an intermediate 
dose level 20 mg lower than the dose level at which ≥2 patients in a cohort experienced a DLT.  If an MTD is 
not identified following dose escalation to 160 mg, the intermediate dose level of 140 mg may also be evaluated.  
If an intermediate dose level is evaluated, up to 6 additional patients will be enrolled at that dose level. 
Patients in Part 1 will continue treatment until withdrawal due to a DLT (Cycle 1 only), disease progression 
according to Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1), withdrawn consent, or 
when another treatment discontinuation criterion is met. 
PK samples will be obtained during Cycle 1. For Cycle 1 Day 1 and Day 15 visits, overnight fasting, except for 
water and medications are required. 
Preliminary efficacy data will be obtained in Part 1.  Tumor assessments by RECIST v1.1 will be conducted 
every 6 weeks relative to the first dose of study medication for all patients until disease progression, death, or 
withdrawal of consent.  Disease progression will be based on the Investigator’s evaluation of tumor assessments.  
Patients who are discontinued from study drug for a reason other than disease progression, death, or withdrawal 
of consent are to continue tumor assessments on the same schedule. 
Part 2: Dose Expansion Phase (Phase 2) 


 
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The highest dose level of D07001-softgel tested at which <2 of 6 patients experience a DLT will be expanded in 
Part 2 of the study. In addition, because the highest dose level selected in Part 1 could lead to cumulative toxicity 
during treatment, the next lowest dose level below the highest evaluated in Part 1 at which <2 of 6 patients 
experienced a DLT will also be expanded for assessment in Part 2.  If an MTD is not identified in Part 1 of the 
study, the 2 dose levels used in Part 2 will be 160 mg and 120 mg, or 160 mg and 140 mg if the intermediate 
dose of 140 mg has been evaluated in Part 1. 
In Part 2 of the study, eligible patients will be randomized in a 1:1 ratio to receive D07001-softgel in an open 
label manner at 1 of the 2 dose levels selected for expansion.  20 patients will be enrolled to each dose expansion 
cohort.  Patients will be treated until withdrawal from treatment due to disease progression according to RECIST 
v1.1, withdrawn consent, or when another treatment discontinuation criterion is met. Patients who are 
discontinued from study drug for reasons other than disease progression or toxicity in the first 2 cycles of Part 2 
will be replaced. 
As for Part 1, tumor assessments by RECIST v1.1 in Part 2 will be conducted every 6 weeks relative to the first 
dose of study medication for all patients until disease progression, death, or withdrawal of consent. Disease 
progression will be based on the Investigator’s evaluation of tumor assessments.  Patients who are discontinued 
from study drug for a reason other than disease progression, death, or withdrawal of consent are to continue 
tumor assessments on the same schedule. 
PK samples will be obtained during Cycle 1.  For Cycle 1 Day 1 visit, overnight fasting, except for water and 
medications are required. In addition, for all patients in the higher dose expansion cohort only, the PK of 
D07001-softgel will be evaluated after a high-fat breakfast or after overnight fasting.  Patients will receive 
D07001-softgel on Cycle 1 Day 15 and Cycle 2 Day 1 in a high-fat fed or a fasting state.  Patients will be 
randomized to the fed/fasted states in 1 of 2 sequences (Sequence 1: Cycle 1 Day 15 high fat, Cycle 2 Day 1 
fasting; Sequence 2: Cycle 1 Day 15 fasting, Cycle 2 Day 1 high fat) with 10 patients per sequence in a cross 
over design (20 patients overall).  PK samples will be taken over a period of 48 hours post-dose for the food 
effect evaluation in this dose expansion cohort. 
Safety, tolerability, efficacy, and PK data will be compared between the 2 dose expansion cohorts to support 
selection of a recommended D07001-softgel dose for further clinical development as chemotherapy following 
primary chemotherapy or CCRT for unresectable metastatic or locally advanced BTC. 
Patients in Part 1 and Part 2 who are continuing to derive clinical benefit from study drug at the time of study 
closure, as assessed by the Investigator, will be provided access to the study drug via a mechanism to be 
determined by the Sponsor. 


 
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Protocol No.: Inno-GO-03 
 
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Event Schedules for Part 1 and Part 2 are outlined in Table 5 and Table 6. 
3. 
STUDY POPULATION 
Part 1 of the study will identify dose-limiting toxicity (DLT) and establish the maximum tolerated dose (MTD), 
if any, of D07001-softgel in patients with unresectable locally advanced or metastatic GI cancer. There will be 
sequential cohorts of 3 to 6 patients each with increasing doses of 40 mg, 60 mg, 80 mg, 120 mg, and 160 mg 
per cohort. If an MTD is not identified following dose escalation to 160 mg, the intermediate dose level of 140 
mg may also be evaluated. 
In Part 2, 40 patients who have achieved stable disease or better following first-line chemotherapy or CCRT for 
unresectable metastatic or locally advanced BTC will be enrolled to receive D07001-softgel at the 2 dose levels 
selected in Part 1 (20 patients at each dose level). No more than 30% of patients enrolled in Part 2 will have 
received CCRT. 
Detailed lists of inclusion and exclusion criteria are shown in Sections 4.1 and 4.2 of the CSP. 
 
4. 
STATISTICAL BASIS FOR SAMPLE SIZE 
No formal sample size calculations have been performed. 
In Part 1 of the study, 3 to 6 patients will be enrolled to each of the predefined dose cohorts. If an intermediate 
dose level (140 mg) is to be investigated, another 3 to 6 patients will be enrolled to that cohort. 
In Part 2, 40 patients will be enrolled to receive D07001-softgel at the 2 dose levels selected in Part 1. 
 
5. 
RANDOMIZATION 
No randomization will be performed in Part 1 of the study.  
In Part 2, enrolled patients will be randomized in a 1:1 ratio to one of the 2 dose levels selected for expansion 
using a computer-generated block randomization scheme with stratification based on whether or not the patient 
received CCRT as first-line treatment. No more than 30% of patients enrolled in Part 2 will have received prior 
CCRT. In addition, patients randomized to the higher dose-expansion cohort in Part 2 will be randomized in a 
1:1 ratio to 1 of the 2 sequences for the food-effect evaluation using a computer-generated block randomization 


 
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scheme. Computer-generated randomization will be performed centrally through an interactive voice response 
system (IVRS) service provider. The IVRS service provider will hold the randomization code throughout the 
conduct of the study. 
 
6. 
STATISTICAL ANALYSIS CONVENTIONS 
6.1 
Analysis Variables 
6.1.1 
Demographic and Background Variables 
The following demographic and anthropometric information will be recorded: 
 
Date of informed consent 
 
Inclusion/Exclusion criteria 
 
Medical history (including previous and current medical conditions and medications) 
 
Drug abuse and alcohol history 
 
Age 
 
Sex 
 
Ethnic 
 
Race 
 
Height (cm) 
 
Body weight (kg) 
 
Body mass index (BMI) calculated as [weight/height2] (kg/m2) 
 
Diagnosis 
All medical history will be coded using Version 20.0 or higher of the Medical Dictionary for Regulatory 
Activities (MedDRA). 
 


 
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6.1.2 
Safety Variables 
Safety will be assessed based on DLTs, adverse events (AEs), physical examination, vital signs, weight, BMI, 
Eastern Cooperative Oncology Group (ECOG) performance status (PS), electrocardiogram (ECG), and 
laboratory assessments (hematology, serum chemistry, coagulation parameters and urinalysis). 
 
6.1.2.1 
Adverse Events 
An AE is any new, undesirable medical occurrence or change (worsening) of an existing condition in a subject 
that occurs from date of informed consent to 30-day follow up visit, whether or not considered to be product 
related.  Therefore, AEs are treatment-emergent signs or symptoms.  Elective hospitalizations for pre-treatment 
conditions (e.g., elective cosmetic procedures) and hospitalization to facilitate study assessments are not AEs.  
Abnormal laboratory values that are considered clinically significant should be reported as AEs.  Disease 
progression as measured by tumor assessment should not be reported as an AE.  Concomitant illness, which 
existed before entry into the study, will not be considered AEs unless they worsen during the treatment or safety 
follow-up period.  Pre-existing conditions will be recorded on the CRF as medical history.  
All AEs will be coded using the Version 20.0 or higher of the Medical Dictionary for Regulatory Activities 
(MedDRA). 
Regarding DLT definition, in Part 1 of the study, any of the following AEs occurring during Cycle 1 will be 
classified as DLTs, if there is a reasonable possibility that it is related to the study drug: 
 
Hematologic: 
o 
Grade 4 neutropenia lasting >7 days 
o 
Febrile neutropenia (defined as neutropenia Grade ≥3 and a body temperature ≥38.3°C) 
o 
Grade ≥3 neutropenic infection 
o 
Grade 4 anemia 
o 
Grade ≥3 thrombocytopenia with bleeding 
o 
Grade 4 thrombocytopenia 
 
any platelet count <10,000/μL 
 
platelet count 10,000-25,000/μL for >5 days 


 
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 
Non-hematologic: 
o 
Grade ≥3 toxicities that are considered clinically significant, except those that have not been 
maximally treated (e.g., nausea, vomiting, diarrhea*) or can be easily treated (e.g., electrolyte 
abnormalities). 
[*Nausea, vomiting, and diarrhea: if Grade ≥3 toxicities persist for more than 48 h despite maximum 
treatment (loperamide, ondansetron, etc.), they will be considered DLTs] 
 
Delay by more than 7 days in receiving the next scheduled cycle due to persisting toxicities attributable 
to study drug. 
 
Failure to deliver at least 6 of the planned 9 doses during Cycle 1 due to treatment-related toxicities. 
 
Upon the second occurrence of a toxicity leading to a dose hold. 
Any AEs with incomplete start and end dates/times will be shown in listing as follows: 
 
AEs with unknown start and/or end times (but where the date is known) will be shown as DDMMYYYY 
 
AEs with unknown start and/or dates will be shown as Unknown 
 
Any AEs with incomplete start and end dates/times will be imputed as follows: 
 
Missing day and month 
o If the year of the incomplete start date is the same as the year of the date of the first dose of 
investigational product, then the day and month of the date of the first dose of investigational product 
will be assigned to the missing fields 
o If the year of the incomplete start date is before the year of the date of the first dose of investigational 
product, then December 31 will be assigned to the missing fields 
o If the year of the incomplete start date is after the year of the date of the first dose of investigational 
product, then 01 January will be assigned to the missing fields. 
 
Missing month only 
o 
The day will be treated as missing and both month and day will be replaced according to the above 
procedure. 


 
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 
Missing day only 
o 
If the month and year of the incomplete start date are the same as the month and year of the date of the 
first dose of investigational product, then the day of the date of the first dose of investigational product 
will be assigned to the missing day 
o 
If either the year is before the year of the date of the first dose of investigational product or if both 
years are the same but the month is before the month of the date of the first dose of investigational 
product, then the last day of the month will be assigned to the missing day 
o 
If either the year is after the year of the date of the first dose of investigational product or if both years 
are the same but the month is after the month of the date of the first dose of investigational product, 
then the first day of the month will be assigned to the missing day. 
 
If the stop date is complete and the imputed start date is after the stop date, then the start date will be 
imputed using the stop date. 
Adverse events will be regarded as Treatment Emergent Adverse Event (TEAE) if they started on or after the 
date and time of administration of the first dose of study drug, or if they were presented prior to the 
administration of the first dose of study drug and increased in severity during the study. Therefore, TEAE 
summaries will include all TEAEs starting on or after the day of the first intake of study treatment and starting 
no later than 30 days after the last study treatment date. 
6.1.2.2 
Clinical Laboratory Tests 
Safety laboratory assessments in Part 1 and Part 2 will be assessed at screening; Day 1, Day 8, and Day 15 of 
cycles 1 and 2; Day 1 of cycle 3 onwards; EOT; and at the 30-day follow up visit. The following safety 
laboratory parameters will be measured: 
 
Hematology: RBCs, Hematocrit, Hemoglobin, MCH, MCHC, MCV, Platelets, WBCs (with differential), 
Neutrophils, Lymphocytes, Monocytes, Basophils, Eosinophils, ANC 
 
Serum chemistry: Albumin, Glucose, BUN, Bicarbonate, Creatinine, Total protein, Uric acid, Magnesium, 
Phosphorus, ALP, ALT, AST, Total bilirubin (direct and indirect), LDH, γ-GT, Electrolytes (sodium, 
potassium, chloride, calcium) 
 
Coagulation parameters: Prothrombin time, aPTT, Fibrinogen, D-dimer, INR 
 
Urinalysis: Bilirubin, Blood, Glucose, Ketones, Leukocytes, Epithelial cells, Casts, Crystals, Bacteria, 
Nitrite, pH, Protein, Specific gravity  


 
Statistical Analysis Plan 
InnoPharmax Inc. 
 
Final 1.0 
Protocol No.: Inno-GO-03 
 
21/Jan/2019 
  
Page 19 of 63 
Abbreviations: ALP=alkaline phosphatase; ALT=alanine aminotransferase; ANC=absolute neutrophil count; 
aPTT= activated partial thromboplastin time; AST=aspartate aminotransferase; ANC=absolute neutrophil count; 
BUN=blood urea nitrogen; γ-GT=γ-glutamyl transpeptidase; INR=international normalized ratio; LDH=lactate 
dehydrogenase; MCH=mean corpuscular hemoglobin; MCHC= mean corpuscular hemoglobin concentration; 
MCV=mean corpuscular volume; RBC=red blood cell; WBC=white blood cell. 
 
6.1.2.3 
Vital Signs 
Vital signs assessments in Part 1 and Part 2 will be assessed at screening; Day 1, Day 8, and Day 15 of cycles 1 
and 2; Day 1 of cycle 3 onwards; EOT; and at the 30-day follow up visit. The following vital signs 
measurements will be obtained: 
 
Sitting Systolic blood pressure (SBP) [mmHg] 
 
Sitting Diastolic blood pressure (DBP) [mmHg] 
 
Pulse rate (beats per minute [bpm]) 
 
Respiratory rate (breaths per minute [brpm]) 
 
Body temperature (oral) [°C] 
 
6.1.2.4 
Electrocardiogram  
ECG assessments in Part 1 and Part 2 will be assessed at screening; Day 1, Day 8, and Day 15 of cycle 1; Day 1 
of cycle 2; Day 1 of cycle 3 onwards; EOT; and at the 30-day follow up visit. The following 12-lead resting 
ECG parameters will be recorded: 
 
RR-interval (msec) 
 
PR-interval (msec) 
 
QRS-duration (msec) 
 
QT-interval (msec) 
 
QT-interval corrected (QTc) (msec) 
 
Heart rate (HR) (beats per minute [bpm]) 
The ECG will be evaluated by the Investigator as ‘Normal’, ‘Abnormal, NCS’ or ‘Abnormal, CS’. 


 
Statistical Analysis Plan 
InnoPharmax Inc. 
 
Final 1.0 
Protocol No.: Inno-GO-03 
 
21/Jan/2019 
  
Page 20 of 63 
 
6.1.2.5 
Echocardiogram/MUGA 
Echocardiogram/MUGA in Part 1 and Part 2 will be assessed at screening; Day1 of Cycles 3 and 5. The 
assessment will be evaluated by the Investigator as ‘Normal’, ‘Abnormal, NCS’ or ‘Abnormal, CS’. 
 
6.1.2.6 
Physical Examination 
A physical examination for Part 1 and Part 2 will be performed at screening; Day 1, Day 8, and Day 15 of cycles 
1 and 2; Day 1 of cycle 3 onwards; EOT; and at the 30-day follow up visit. 
The Physical Examination will be evaluated by the Investigator as ‘Normal’, ‘Abnormal, NCS’ or ‘Abnormal, 
CS’. 
All clinically significant treatment-emergent findings that were not present at baseline or described in the past 
medical history will be recorded as AEs. 
 
6.1.2.7 
Pregnancy 
Serum pregnancy tests will be conducted at Screening and on Cycle 1 Day 1. Urine pregnancy tests will be 
conducted on Day 1 of all subsequent cycles, at the EOT visit, and at the 30-day safety follow-up visit. 
 
6.1.2.8 
Prior and Concomitant Medication 
Prior and Concomitant medication will be coded using the World Health Organization-Drug Dictionary (WHO-
DD) (Version March 2017 or higher) and will be classified by Anatomical Therapeutic Chemical (ATC) 
categories. 
If the stop date is missing, ongoing or after the date and time of administration of the first dose of study drug, the 
medication will be considered as Concomitant medication. If the stop date is before the date and time of 
administration of the first dose of study drug, the medication will be considered as Prior medication. 
 
6.1.3 
Pharmacokinetic Variables 
The following PK parameters will be determined for gemcitabine (dFdC) and dFdU in plasma: 


 
Statistical Analysis Plan 
InnoPharmax Inc. 
 
Final 1.0 
Protocol No.: Inno-GO-03 
 
21/Jan/2019 
  
Page 21 of 63 
Table 1 
Pharmacokinetic Parameters For Part 1 Dose Escalation Phase - Cycle 1, Days 1, 2, 
3, 8, 15, 16 and 17  
Parameter 
Definition 
Cmax 
Maximum observed concentration 
tmax 
Time corresponding to occurrence of Cmax 
t½ 
Apparent terminal elimination half life 
AUC(0-t) 
AUC from time zero to the last quantifiable concentration 
AUC(0-24) 
AUC from time zero to 24 hours post-dose 
AUC(0-48) 
AUC from time zero to 48 hours post-dose 
AUC(0-inf) 
AUC from time zero extrapolated to infinity 
%AUCex 
Percentage of AUC(0-inf) obtained by extrapolation 
CL/F* 
Apparent clearance following oral administration 
Vz/F* 
Apparent volume of distribution during terminal phase 
Ctrough 
Pre-dose concentration** 
Rac(AUC(0-t)) 
Accumulation Ratio, calculated as (Day 15 AUC(0-t) / Day 1 AUC(0-t)) 
Rac(AUC(0-24)) 
Accumulation Ratio, calculated as (Day 15 AUC(0-24) / Day 1 AUC(0-24)) 
Rac(AUC(0-48)) 
Accumulation Ratio, calculated as (Day 15 AUC(0-48) / Day 1 AUC(0-48)) 
Rac(AUC(0-inf)) 
Accumulation Ratio, calculated as (Day 15 AUC(0-inf) / Day 1 AUC(0-inf)) 
Rac(Cmax) 
Accumulation Ratio, calculated as (Day 15 Cmax / Day 1 Cmax) 
MR(AUC(0-t))1 
Metabolic Ratio for (AUC(0-t)), calculated as Day 1 dFdC (AUC(0-t)) / Day 1 dFdU 
(AUC(0-t)) 
MR(AUC(0-24))1 
Metabolic Ratio for (AUC(0-24)), calculated as Day 1 dFdC (AUC(0-24)) / Day 1 dFdU 
(AUC(0-24)) 
MR(AUC(0-48))1 
Metabolic Ratio for (AUC(0-48)), calculated as Day 1 dFdC (AUC(0-48)) / Day 1 dFdU 
(AUC(0-48)) 
MR(AUC(0-inf))1 
Metabolic Ratio for (AUC(0-inf)), calculated as Day 1 dFdC (AUC(0-inf)) / Day 1 dFdU 
(AUC(0-inf)) 
MR(AUC(0-t))15 
Metabolic Ratio for (AUC(0-t)), calculated as Day 15 dFdC (AUC(0-t)) / Day 15 dFdU 
(AUC(0-t)) 
MR(AUC(0-24))15 
Metabolic Ratio for (AUC(0-24)), calculated as Day 15 dFdC (AUC(0-24)) / Day 15 
dFdU (AUC(0-24)) 
MR(AUC(0-48))15 
Metabolic Ratio for (AUC(0-48)), calculated as Day 15 dFdC (AUC(0-48)) / Day 15 
dFdU (AUC(0-48)) 
MR(AUC(0-inf))15 
Metabolic Ratio for (AUC(0-inf)), calculated as Day 15 dFdC (AUC(0-inf)) / Day 
15 dFdU (AUC(0-inf)) 
*dFdC only; all others for both dFdU and dFdC, or as defined.  
** Pre-dose on Cycle 1 Day 1, 3, 8, 15 and 17 
 


 
Statistical Analysis Plan 
InnoPharmax Inc. 
 
Final 1.0 
Protocol No.: Inno-GO-03 
 
21/Jan/2019 
  
Page 22 of 63 
Table 2 
Pharmacokinetic Parameters for Part 2 Dose Expansion Phase - Cycle 1, Days 1, 2, 
3, 8, 15, 16 and 17 and  Cycle 2, Days 1, 2 and 3 
Parameter 
Definition 
Cmax 
Maximum observed concentration 
tmax 
Time corresponding to occurrence of Cmax 
t½ 
Apparent terminal elimination half life 
AUC(0-t) 
AUC from time zero to the last quantifiable concentration 
AUC(0-24) 
AUC from time zero to 24 hours post-dose 
AUC(0-48) 
AUC from time zero to 48 hours post-dose 
AUC(0-inf) 
AUC from time zero extrapolated to infinity 
%AUCex 
Percentage of AUC(0-inf) obtained by extrapolation 
CL/F* 
Apparent clearance following oral administration 
Vz/F* 
Apparent volume of distribution during terminal phase 
Ctrough 
Pre-dose concentration** 
Rac(AUC(0-t))# 
Accumulation Ratio, calculated as (Day 15 AUC(0-t) / Day 1 AUC(0-t)) 
Rac(AUC(0-24)) # 
Accumulation Ratio, calculated as (Day 15 AUC(0-24) / Day 1 AUC(0-24)) 
Rac(AUC(0-48)) # 
Accumulation Ratio, calculated as (Day 15 AUC(0-48) / Day 1 AUC(0-48)) 
Rac(AUC(0-inf)) # 
Accumulation Ratio, calculated as (Day 15 AUC(0-inf) / Day 1 AUC(0-inf)) 
Rac(Cmax) # 
Accumulation Ratio, calculated as (Day 15 Cmax / Day 1 Cmax) 
MR(AUC(0-t))1# 
Metabolic Ratio for (AUC(0-t)), calculated as Day 1 dFdC (AUC(0-t)) / Day 1 dFdU 
(AUC(0-t)) 
MR(AUC(0-24))1# 
Metabolic Ratio for (AUC(0-24)), calculated as Day 1 dFdC (AUC(0-24)) / Day 1 dFdU 
(AUC(0-24)) 
MR(AUC(0-48))1# 
Metabolic Ratio for (AUC(0-48)), calculated as Day 1 dFdC (AUC(0-48)) / Day 1 dFdU 
(AUC(0-48)) 
MR(AUC(0-inf))1# 
Metabolic Ratio for (AUC(0-inf)), calculated as Day 1 dFdC (AUC(0-inf)) / Day 1 dFdU 
(AUC(0-inf)) 
MR(AUC(0-t))15# 
Metabolic Ratio for (AUC(0-t)), calculated as Day 15 dFdC (AUC(0-t)) / Day 15 dFdU 
(AUC(0-t)) 
MR(AUC(0-24))15# 
Metabolic Ratio for (AUC(0-24)), calculated as Day 15 dFdC (AUC(0-24)) / Day 15 
dFdU (AUC(0-24)) 
MR(AUC(0-48))15# 
Metabolic Ratio for (AUC(0-48)), calculated as Day 15 dFdC (AUC(0-48)) / Day 15 
dFdU (AUC(0-48)) 
MR(AUC(0-inf))15# 
Metabolic Ratio for (AUC(0-inf)), calculated as Day 15 dFdC (AUC(0-inf)) / Day 
15 dFdU (AUC(0-inf)) 
*dFdC only; all others for both dFdU and dFdC, or as defined.  
** Pre-dose on Cycle 1 Day 1, 3, 8, 15 and 17 and Cycle 2 Day 1 and 3. 
# For Part 2, accumulation ratios for dFdC will be calculated for Day 15 relative to Day 1 for all AUC 
parameters and Cmax. For the lower dose-expansion cohort in Part 2, the accumulation ratios for dFdU will be 
calculated for Day 15 relative to Day 1 for all AUC parameters and Cmax, and the metabolic ratio will be 
calculated for dFdU for Days 1 and 15 for all AUC parameters. 


 
Statistical Analysis Plan 
InnoPharmax Inc. 
 
Final 1.0 
Protocol No.: Inno-GO-03 
 
21/Jan/2019 
  
Page 23 of 63 
6.1.3.1 
Pharmacokinetic Parameter Calculation Methods 
PK parameters will be calculated by non-compartmental analysis methods from the concentration-time data 
using WinNonlin (WNL) Professional (Version 6.3) following these guidelines: 
 
Actual sampling times relative to dosing rather than nominal times will be used in the calculation of all 
derived PK parameters. 
 
There will be no imputation of missing data.  
 
All below the limit of quantification (BLQ) values pre-dose and in the absorption phase prior to the first 
quantifiable concentration will be substituted by zeros. Thereafter BLQ values between evaluable 
concentrations will be substituted by missing, before the calculation of the PK variables. Terminal BLQ 
values will be disregarded. 
 
For Day 1, all BLQ values pre-dose and in the absorption phase prior to the first quantifiable 
concentration will be substituted by zeros. For subsequent dosing days not separated by a washout, 
BLQ values pre-dose, in the absorption phase, and between evaluable concentrations will be substituted 
by missing, before the calculation of the PK variables. Terminal BLQ values will be disregarded. 
PK parameters will be estimated according to the following guidelines: 
 
Cmax will be obtained directly from the concentration-time data. 
 
tmax is the time at which Cmax is observed. 
 
λZ will be estimated at terminal phase by linear regression after log-transformation of the concentrations: 
 Only those data points that are judged to describe the terminal log-linear decline will be used in the 
regression.  
 A minimum number of three data points in the terminal phase will be used in calculating λz with the line 
of regression starting at any post-Cmax data point (Cmax should not be part of the regression slope) and 
including Clast, tlast. 
 The adjusted correlation coefficient (R2 adjusted) in general should be greater than 0.90. Any value less 
than 0.90 may be used at the PK Scientist’s best knowledge and judgment. 
 An appropriate number of decimal places should be used for λz to enable the reported value of t½ to be 
calculated. 
 
t1/2 will be calculated as ln2/λZ. 


 
Statistical Analysis Plan 
InnoPharmax Inc. 
 
Final 1.0 
Protocol No.: Inno-GO-03 
 
21/Jan/2019 
  
Page 24 of 63 
 
AUC is calculated as follows: 
 
The linear trapezoidal method will be employed for all incremental trapezoids arising from increasing 
concentrations and the logarithmic trapezoidal method will be used for those arising from decreasing 
concentrations. 
 AUC(0-t)=
 t
0
dt
t
C
)
(
. 
 AUC(0-inf) =
 t
0
dt
t
C
)
(
＋

t
dt
t
C
)
(
= AUC(0-t)＋Ct/λz. 
 Ct is last observed quantifiable concentration. 
 
%AUCex will be calculated as (1－[AUC(0-t)/AUC(0-inf)])×100. 
 
CL/F will be calculated as dose/AUC(0-inf), parent drug only. 
 
Vz/F will be calculated as CL/F/λZ, parent drug only. 
The following PK parameters will also be derived using SAS (version x.x); (See Table 1 and Table 2 for more 
detail): 
 
Rac 
 
Accumulation ratio calculated as: 
Cmax (last dose interval)/ Cmax (first dose interval). 
AUC(0-τ),ss (last dose interval)/ AUC(0-τ),ss (first dose interval). 
 
MR 
Metabolite ratio calculated as AUC (parent)/ AUC (metabolite).


 
Statistical Analysis Plan 
InnoPharmax Inc. 
 
Final 1.0 
Protocol No.: Inno-GO-03 
 
21/Jan/2019 
  
                                                                             Page 25 of 63 
Table 3 
Summary of Required Pharmacokinetic Parameters for Dose Escalation Phase  
 
  
  
DOSE ESCALATION 
dFdC 
 
dFdU 
Cycle 1 
 
Cycle 1 
  
  
Day 1 
Day 8 
Day 15 
 
Day 1 
Day 8 
Day 15 
Cmax 
Maximum observed concentration 
yes 
N/A 
yes 
 
yes 
N/A 
yes 
Ctrough 
Pre-dose concentration 
yes 
yes 
yes 
 
yes 
yes 
yes 
tmax 
Time corresponding to occurrence of Cmax 
yes 
N/A 
yes 
 
yes 
N/A 
yes 
t½ 
Apparent terminal elimination half life 
yes 
N/A 
yes 
 
yes 
N/A 
yes 
AUC(0-t) 
AUC from time zero to the last quantifiable concentration 
yes 
N/A 
yes 
 
yes 
N/A 
yes 
AUC(0-24) 
AUC from time zero to 24 hours post-dose 
yes 
N/A 
yes 
 
yes 
N/A 
yes 
AUC(0-48) 
AUC from time zero to 48 hours post-dose 
yes 
N/A 
yes 
 
yes 
N/A 
yes 
AUC(0-inf) 
AUC from time zero extrapolated to infinity 
yes 
N/A 
yes 
 
yes 
N/A 
yes 
%AUCex 
Percentage of AUC(0-inf) obtained by extrapolation 
yes 
N/A 
yes 
 
yes 
N/A 
yes 
CL/F 
Apparent clearance following oral administration 
yes 
N/A 
yes 
 
no 
N/A 
no 
Vz/F 
Apparent volume of distribution during terminal phase 
yes 
N/A 
yes 
 
no 
N/A 
no 
Rac(AUC(0-t)) 
Accumulation Ratio, calculated as (Day 15 AUC(0-t) / Day 1 AUC(0-t)) 
N/A 
N/A 
yes 
 
N/A 
N/A 
yes 
Rac(AUC(0-24)) 
Accumulation Ratio, calculated as (Day 15 AUC(0-24) / Day 1 
AUC(0-24)) 
N/A 
N/A 
yes 
 
N/A 
N/A 
yes 


 
Statistical Analysis Plan 
InnoPharmax Inc. 
 
Final 1.0 
Protocol No.: Inno-GO-03 
 
21/Jan/2019 
  
                                                                             Page 26 of 63 
  
  
DOSE ESCALATION 
dFdC 
 
dFdU 
Cycle 1 
 
Cycle 1 
  
  
Day 1 
Day 8 
Day 15 
 
Day 1 
Day 8 
Day 15 
Rac(AUC(0-48)) 
Accumulation Ratio, calculated as (Day 15 AUC(0-48) / Day 1 
AUC(0-48)) 
N/A 
N/A 
yes 
 
N/A 
N/A 
yes 
Rac(AUC(0-inf)) 
Accumulation Ratio, calculated as (Day 15 AUC(0-inf) / Day 1 
AUC(0-inf)) 
N/A 
N/A 
yes 
 
N/A 
N/A 
yes 
Rac(Cmax) 
Accumulation Ratio, calculated as (Day 15 Cmax / Day 1 Cmax) 
N/A 
N/A 
yes 
 
N/A 
N/A 
yes 
MR(AUC(0-t))1 
Metabolic Ratio for (AUC(0-t)), calculated as Day 1 dFdC (AUC(0-t)) / 
Day 1 dFdU (AUC(0-t)) 
N/A 
N/A 
N/A 
 
yes 
N/A 
N/A 
MR(AUC(0-24))1 
Metabolic Ratio for (AUC(0-24)), calculated as Day 1 dFdC (AUC(0-24)) 
/ Day 1 dFdU (AUC(0-24)) 
N/A 
N/A 
N/A 
 
yes 
N/A 
N/A 
MR(AUC(0-48))1 
Metabolic Ratio for (AUC(0-48)), calculated as Day 1 dFdC (AUC(0-48)) 
/ Day 1 dFdU (AUC(0-48)) 
N/A 
N/A 
N/A 
 
yes 
N/A 
N/A 
MR(AUC(0-inf))1 
Metabolic Ratio for (AUC(0-inf)), calculated as Day 1 dFdC 
(AUC(0-inf)) / Day 1 dFdU (AUC(0-inf)) 
N/A 
N/A 
N/A 
 
yes 
N/A 
N/A 
MR(AUC(0-t))15 
Metabolic Ratio for (AUC(0-t)), calculated as Day 15 dFdC (AUC(0-t)) / 
Day 15 dFdU (AUC(0-t)) 
N/A 
N/A 
N/A 
 
N/A 
N/A 
yes 
MR(AUC(0-24))15 
Metabolic Ratio for (AUC(0-24)), calculated as Day 15 dFdC 
(AUC(0-24)) / Day 15 dFdU (AUC(0-24)) 
N/A 
N/A 
N/A 
 
N/A 
N/A 
yes 
MR(AUC(0-48))15 
Metabolic Ratio for (AUC(0-48)), calculated as Day 15 dFdC 
(AUC(0-48)) / Day 15 dFdU (AUC(0-48)) 
N/A 
N/A 
N/A 
 
N/A 
N/A 
yes 
MR(AUC(0-inf))15 Metabolic Ratio for (AUC(0-inf)), calculated as Day 15 dFdC 
(AUC(0-inf)) / Day 15 dFdU (AUC(0-inf)) 
N/A 
N/A 
N/A 
 
N/A 
N/A 
yes 
 
 
 


 
Statistical Analysis Plan 
InnoPharmax Inc. 
 
Final 1.0 
Protocol No.: Inno-GO-03 
 
21/Jan/2019 
  
                                                                             Page 27 of 63 
Table 4 
Summary of Required Pharmacokinetic Parameters for Dose Expansion Phase 
 
  
  
DOSE EXPANSION 
dFdC 
 
dFdU 
Cycle 1 
Cycle 2 
 
Cycle 1 
Cycle 2 
  
  
Day 1 
Day 8 
Day 15 
Day 1 
 
Day 1 
Day 8 
Day 15 
Day 1 
Cmax 
Maximum observed concentration 
yes 
N/A 
yes 
yes 
 
yes 
N/A 
yes 
yes 
Ctrough 
Pre-dose concentration 
yes 
yes 
yes 
yes 
 
yes 
yes 
yes 
yes 
tmax 
Time corresponding to occurrence of Cmax 
yes 
N/A 
yes 
yes 
 
yes 
N/A 
yes 
yes 
t½ 
Apparent terminal elimination half life 
yes 
N/A 
yes 
yes 
 
yes 
N/A 
yes 
yes 
AUC(0-t) 
AUC from time zero to the last quantifiable 
concentration 
yes 
N/A 
yes 
yes 
 
yes 
N/A 
yes 
yes 
AUC(0-24) 
AUC from time zero to 24 hours post-dose 
yes 
N/A 
yes 
yes 
 
yes 
N/A 
yes 
yes 
AUC(0-48) 
AUC from time zero to 48 hours post-dose 
yes 
N/A 
yes 
yes 
 
yes 
N/A 
yes 
yes 
AUC(0-inf) 
AUC from time zero extrapolated to infinity 
yes 
N/A 
yes 
yes 
 
yes 
N/A 
yes 
yes 
%AUCex 
Percentage of AUC(0-inf) obtained by 
extrapolation 
yes 
N/A 
yes 
yes 
 
yes 
N/A 
yes 
yes 
CL/F 
Apparent clearance following oral 
administration 
yes 
N/A 
yes 
yes 
 
no 
N/A 
no 
no 
Vz/F 
Apparent volume of distribution during terminal 
phase 
yes 
N/A 
yes 
yes 
 
no 
N/A 
no 
no 
Rac(AUC(0-t)) 
Accumulation Ratio, calculated as (Day 15 
AUC(0-t) / Day 1 AUC(0-t)) 
N/A 
N/A 
yes 
N/A 
 
N/A 
N/A 
yes# 
N/A 


 
Statistical Analysis Plan 
InnoPharmax Inc. 
 
Final 1.0 
Protocol No.: Inno-GO-03 
 
21/Jan/2019 
  
                                                                             Page 28 of 63 
  
  
DOSE EXPANSION 
dFdC 
 
dFdU 
Cycle 1 
Cycle 2 
 
Cycle 1 
Cycle 2 
  
  
Day 1 
Day 8 
Day 15 
Day 1 
 
Day 1 
Day 8 
Day 15 
Day 1 
Rac(AUC(0-24)) 
Accumulation Ratio, calculated as (Day 15 
AUC(0-24) / Day 1 AUC(0-24)) 
N/A 
N/A 
yes 
N/A 
 
N/A 
N/A 
yes# 
N/A 
Rac(AUC(0-48)) 
Accumulation Ratio, calculated as (Day 15 
AUC(0-48) / Day 1 AUC(0-48)) 
N/A 
N/A 
yes 
N/A 
 
N/A 
N/A 
yes# 
N/A 
Rac(AUC(0-inf)) 
Accumulation Ratio, calculated as (Day 15 
AUC(0-inf) / Day 1 AUC(0-inf)) 
N/A 
N/A 
yes 
N/A 
 
N/A 
N/A 
yes# 
N/A 
Rac(Cmax) 
Accumulation Ratio, calculated as (Day 15 Cmax 
/ Day 1 Cmax) 
N/A 
N/A 
yes 
N/A 
 
N/A 
N/A 
yes# 
N/A 
MR(AUC(0-t))1 
Metabolic Ratio for (AUC(0-t)), calculated as Day 
1 dFdC (AUC(0-t)) / Day 1 dFdU (AUC(0-t)) 
N/A 
N/A 
N/A 
N/A 
 
yes# 
N/A 
N/A 
yes# 
MR(AUC(0-24))1 
Metabolic Ratio for (AUC(0-24)), calculated as 
Day 1 dFdC (AUC(0-24)) / Day 1 dFdU 
(AUC(0-24)) 
N/A 
N/A 
N/A 
N/A 
 
yes# 
N/A 
N/A 
yes# 
MR(AUC(0-48))1 
Metabolic Ratio for (AUC(0-48)), calculated as 
Day 1 dFdC (AUC(0-48)) / Day 1 dFdU 
(AUC(0-48)) 
N/A 
N/A 
N/A 
N/A 
 
yes# 
N/A 
N/A 
yes# 
MR(AUC(0-inf))1 
Metabolic Ratio for (AUC(0-inf)), calculated as 
Day 1 dFdC (AUC(0-inf)) / Day 1 dFdU (AUC(0-
inf)) 
N/A 
N/A 
N/A 
N/A 
 
yes# 
N/A 
N/A 
yes# 
MR(AUC(0-t))15 
Metabolic Ratio for (AUC(0-t)), calculated as Day 
15 dFdC (AUC(0-t)) / Day 15 dFdU (AUC(0-t)) 
N/A 
N/A 
N/A 
N/A 
 
N/A 
N/A 
yes# 
N/A 
MR(AUC(0-24))15 
Metabolic Ratio for (AUC(0-24)), calculated as 
Day 15 dFdC (AUC(0-24)) / Day 15 dFdU 
(AUC(0-24)) 
N/A 
N/A 
N/A 
N/A 
 
N/A 
N/A 
yes# 
N/A 


 
Statistical Analysis Plan 
InnoPharmax Inc. 
 
Final 1.0 
Protocol No.: Inno-GO-03 
 
21/Jan/2019 
  
                                                                             Page 29 of 63 
  
  
DOSE EXPANSION 
dFdC 
 
dFdU 
Cycle 1 
Cycle 2 
 
Cycle 1 
Cycle 2 
  
  
Day 1 
Day 8 
Day 15 
Day 1 
 
Day 1 
Day 8 
Day 15 
Day 1 
MR(AUC(0-48))15 
Metabolic Ratio for (AUC(0-48)), calculated as 
Day 15 dFdC (AUC(0-48)) / Day 15 dFdU 
(AUC(0-48)) 
N/A 
N/A 
N/A 
N/A 
 
N/A 
N/A 
yes# 
N/A 
MR(AUC(0-inf))1
5 
Metabolic Ratio for (AUC(0-inf)), calculated as 
Day 15 dFdC (AUC(0-inf)) / Day 15 dFdU 
(AUC(0-inf)) 
N/A 
N/A 
N/A 
N/A 
 
N/A 
N/A 
yes# 
N/A 
# For the lower dose-expansion cohort in Part 2. 


 
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6.1.4 
Efficacy Variables 
In Part 1 of the study, Tumor assessments by RECIST v1.1 will be conducted every 6 weeks relative to the first 
dose of study medication for all patients until disease progression, death, or withdrawal of consent. Disease 
progression will be based on the Investigator’s evaluation of tumor assessments. Patients who are discontinued 
from study drug for a reason other than disease progression, death, or withdrawal of consent are to continue 
tumor assessments on the same schedule. 
As for Part 1, tumor assessments by RECIST v1.1 in Part 2 will be conducted every 6 weeks relative to the first 
dose of study medication for all patients until disease progression, death, or withdrawal of consent. Disease 
progression will be based on the Investigator’s evaluation of tumor assessments. Patients who are discontinued 
from study drug for a reason other than disease progression, death, or withdrawal of consent are to continue 
tumor assessments on the same schedule. 
 
6.1.4.1 
Tumor assessments  
The classification of the objective tumor response is: 
 
Complete response (CR) 
 
Partial response (PR) 
 
Progressive disease (PD) 
 
Stable disease (SD) 
Best overall response (BOR) will be assessed for patients with measurable disease at baseline. BOR is defined as 
a patient’s best objective response since treatment started.  
Patients with a best response of CR or PR will be classified as responders for the evaluation of the objective 
response rate (ORR). All other patients with measurable disease at baseline, including patients who do not have 
tumor response assessment due to rapid progression or toxicity, will be considered as non-responders and will be 
included in the denominator for the response rate. 
 
6.1.4.2 
Other efficacy endpoints 
Besides ORR, the following exploratory efficacy endpoints will also be assessed in this study: 


 
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 
PFS: The time from treatment assignment /randomization until PD or death. 
 
OS: The time from treatment assignment/randomization until death from any cause. 
 
TTP: The time from treatment assignment until PD. 
 
6.1.5 
Tumor Marker 
Cancer antigen [CA]-19-9 and carcinoembryonic antigen [CEA] levels will be measured per the assessment 
schedules for Part 1 and Part 2. 
 
6.1.6 
Pharmacogenomic, Pharmacoproteomic, and Cellular Marker 
Samples for pharmacogenomic, pharmacoproteomic and cellular markers will be collected if the patient provides 
additional consent.  
Cellular markers will include the levels of immune cells, circulating endothelial progenitor cells and circulating 
tumor cells. 
 
6.2 
Analysis Populations 
6.2.1 
Enrolled Set 
The Enrolled Set (ENS) will include all patients who provided informed consent. 
 
6.2.2 
Safety Analysis Set 
The Safety Analysis Set (SAF) will include all patients who received at least 1 dose of D07001-softgel, 
classified by actual dose received. 
 


 
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6.2.3 
Pharmacokinetic Set 
The Pharmacokinetic Set (PKS) will include all patients in the SAF with evaluable PK data, and who have no 
major protocol deviations considered to impact the analysis of the PK data. 
Data may be excluded from PK analysis (concentrations listed only) if any of the following criteria are fulfilled: 
 Subject received a concomitant medication that could render the plasma concentration-time profile 
unreliable as judged by <insert name of roles/functions that will make the decision> 
 The pre-dose concentration is greater than 5% of the corresponding Cmax in any given treatment period. 
 Subject vomits within 2 x the reported median tmax for the analyte. 
 Subject has moderate or severe diarrhea within 2 x the reported median tmax for the analyte. 
Any data excluded will be reported in the CSR.  
 
6.2.4 
Efficacy Analysis Set 
The following analysis sets will be defined only for Part 2 of the study. 
6.2.4.1 
Modified Intent-to-Treat (mITT) Set 
mITT Set will include all randomized patients who received at least 1 dose of study drug and with at least 1 post-
baseline disease assessment. 
Note: Patients who do not meet eligibility criteria prior to randomization but are still randomized into the study 
will be excluded from the mITT Set. 
 
6.2.4.2 
Per-Protocol (PP) Set  
PP Set will be a subset of the mITT Set that excludes patients who have major protocol deviations. 
 
6.3 
Statistical Analysis Methods 
Data for patients in Part 1 and Part 2 will be analyzed separately. 


 
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6.3.1 
Listings and Descriptive Statistics 
All original and derived parameters as well as population characteristics will be listed and described using 
summary statistics. Frequency counts (number of patients [n] and percentages) will be made for each qualitative 
variable. Descriptive statistics (n, mean, standard deviation [SD], median, minimum and maximum) will be 
calculated for each quantitative variable (unless otherwise stated). Additional descriptive statistics will be 
reported for PK concentrations and parameters. All listings will include repeated and unscheduled measurements. 
All descriptive statistics will be presented by dose level and study part. The baseline for all measurements 
(where applicable) will be the last pre-dose measurement. 
All descriptive statistics will be presented by dose level for measurements obtained during the treatment period. 
Descriptive statistics for all data obtained at Screening and follow-up will be presented separately. 
 
6.3.2 
Statistical Significance Level 
All statistical tests will be two-sided and will be performed at the 5% level of significance, unless otherwise 
stated. 
 
6.3.3 
Software 
All statistical analyses will be performed using Statistical Analysis Software (SAS®) Version 9.2 or later. The 
PK analysis will be performed using WinNonlin Professional Software Version 6.3 or later. 
 
6.3.4 
Missing Data 
Unless otherwise stated, there will be no special imputation of missing data.  
 
6.3.5 
Interim Analysis and Early Stopping Guidelines  
Study enrollment may be suspended by the Safety Board if ≥2 patients in the 20 mg cohort (dose level -1) 
experience a DLT or upon the identification or discovery of an unexpected, significant, or unacceptable risk to 
the patients enrolled in the study. 


 
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A safety data review will be performed in the interim of the dose-expansion phase, i.e. when 10 patients in each 
of the 2 dose-expansion cohorts have completed at least 2 cycles of treatment. 
 
6.3.6 
Protocol Deviations 
All protocol deviations will be recorded by the Investigator and will be listed by patient. All protocol deviations 
will be discussed between CRO (Medical Monitor, Data Manager, Biostatistician) and the Sponsor and classified 
as “minor” or “major”. Major protocol deviations will lead to the exclusion of a patient from the PP Set. Protocol 
Deviations will be assessed prior to database lock. 
 
6.3.7 
Disposition of Patients 
Patients screened, randomized, treated with study drug and who have discontinued the treatment (i.e. end of 
treatment (EOT)) or discontinued the study (i.e. end of study (EOS)) will be presented by dose level and overall 
for each study part. If patients who discontinue from the treatment should, if possible, have an EOT visit. This 
visit should take place as soon as possible after the patient stops taking study drug or as soon as possible after it 
was learned that the patient will not be able to complete follow up. The reason for discontinuation from the 
treatment or study (EOT/ EOS) and for screen failure will also be summarized and listed by patient. 
 
6.3.8 
Demographic Data and Baseline Characteristics  
All demographic data and baseline characteristics will be listed on the ENS and will be summarized using the 
SAF, PKS, mITT, and PP Sets. 
 
6.3.9 
Medical History 
Medical history will be recorded at Screening. All of the medical history will be listed for each patient by dose 
level using the SAF. All of the medical history are also summarized and listed by SOC and PT terms.  
 


 
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6.3.10 
Prior and Concomitant Medication 
All prior medications will be coded using the WHO-DD and will be classified by ATC categories. All prior 
medications will be listed for each patient by dose level using the SAF. Frequency count and associated 
percentages of prior medications will be summarized by ATC Level 2, ATC Level 4 and Preferred Term. 
All concomitant medications and therapeutic interventions (e.g., drug therapy, surgery, etc.) taken from 
screening and ongoing or those medications that started after first dosing start will be coded using the WHO-DD 
and will be classified by ATC categories. 
All concomitant medications will be listed for each patient by dose level using the SAF. Frequency count and 
associated percentages of concomitant medications will be summarized by ATC Level 2, ATC Level 4 and 
Preferred Term. 
 
6.3.11 
Exposure to the Investigational Medicinal Product 
Per ICH E3: When the dose received by each subject can vary, the actual dose received should be shown and 
individual subject's doses should be presented in a data listing.  
The actual doses received will be presented in the Exposure to Investigational Medicinal Product listing for the 
SAF. Study dose administration will include dose reduction, hold and dose compliance. The patient’s drug 
compliance (i.e. amount used/amount expected to be used in interval between visits) will be recorded on the drug 
administration CRF. 
Total actual dose of study drug, total treatment duration, actual treatment duration and Relative Dose Intensity 
(RDI) will be summarized by study part and dose level. 
• 
Total treatment duration = last dose date - first dose date +1 
• 
Actual treatment duration = total treatment duration, excluding dose interruptions and delay periods   
• 
RDI (%) = percentage of the actual dose intensity delivered relative to the intended dose intensity  
The number of treatment cycles, dose modification (reduction/ interruption/ delay), and drug compliance will 
also be summarized by study part and dose level. 
6.3.12 
Pharmacokinetic Concentrations and Variables 
The analysis of the PK data will be based on the PKS. 


 
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Concentrations below the lower limit of quantification (LLOQ) will be indicated as BLQ in the listings.  
Pharmacokinetic concentration data will be listed by patient including actual sampling times relative to dosing. 
Plasma concentrations will be summarized by study part and dose level. The following descriptive statistics will 
be presented for plasma concentrations obtained at each nominal time point: n, arithmetic mean, SD, coefficient 
of 
variation 
(CV%), 
geometric 
mean, 
geometric 
SD, 
geometric 
CV% 
(calculated 
as:  
gCV% =SQRT(es²-1)*100; where s is the standard deviation of the log-transformed values), median, minimum 
and maximum values. 
Pharmacokinetic parameters will be listed by patient and summarized by study part and by dose level. 
Descriptive statistics for calculated PK parameters will include: n, arithmetic mean, SD, CV%, geometric mean, 
median, minimum and maximum values. For tmax, only median, minimum and maximum values will be 
presented. For each PK parameter, descriptive statistics will not be determined if fewer than three patients report 
a calculated result for a given parameter. 
Individual plasma concentration versus actual times will be plotted by dose level for each analyte using linear 
and semi-logarithmic scales. Mean plasma concentrations versus nominal times will also be presented using 
linear and semi-logarithmic scales. All dose levels will be overlaid on the same plot. 
 
6.3.12.1 
Handling of Values Below the Limit of Quantification (BLQ) in Concentration Summaries 
and Listings 
Graphical presentation: 
For graphs of arithmetic means, all BLQ mean concentrations will be substituted by zeros.  Graphs of geometric 
means include only time points with minimum concentration greater than zero. Any arithmetic mean that is BLQ 
will be excluded from log/linear presentation of arithmetic means. Any BLQ values prior to the last quantifiable 
concentration will be plotted at zero for individual linear/linear graphs and excluded from log/linear graphs. All 
BLQ values after the last quantifiable concentration will be excluded from individual linear/linear and log/linear 
graphs.  
Handling of values below the limit of quantification (BLQ) in listings and for the calculation of descriptive 
statistics at each time point: 
All concentrations below the limit of quantification (BLQ) or missing data will be labeled as such in the 
concentration data listings. Missing samples will be reported as no sample ("NS") and excluded from analysis. 


 
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Values that are BLQ will be substituted with zero for the calculation of descriptive statistics of concentration by 
time point and will be displayed as “not calculable” or NC if no descriptive statistics will be determined. 
 
6.3.12.2 
Assessment of Food Effect 
For the food-effect evaluation in the higher dose-expansion cohort in Part 2, bioequivalence of the fed and fasted 
states will be evaluated for AUC(0-t) and Cmax (primary) and AUC(0-inf) (secondary), based on the 90% confidence 
interval (CI) using the PKS. 
Individual PK parameter values will be used to fit a mixed linear model. Food-effect, sequence group and dosing 
period will be fixed effects while subject will be taken as random effect. The response variable ‘log_pkvar’ 
represents the natural log transformed Cmax, AUC(0-t) or AUC(0 inf). Food-effect can be estimated by the model. 
Ratio of Fed/Fasted can be obtained by the back log transformed of estimated difference. 
The following SAS code will be used as reference: 
 
PROC MIXED DATA=DATA; 
 
   CLASS fed_status period sequence subject; 
 
   MODEL log_pkvar = fed_status sequence period ; 
                RAMDOM  subject (sequence); 
 
       /*where "log_pkvar"= log transformed Cmax, AUC(0-t) and  AUC(0-inf)*/ 
 
 
LSMEANS fed_status / CL ALPHA=0.05; 
 
 
ESTIMATE 'Fed versus Fasted’ food   1 -1   / CL ALPHA=0.1; 
 
 
ODS OUTPUT ESTIMATES=estim  LSMEANS=ls_means; 
QUIT; 
Before the data will be analyzed the fixed effect fed_status will be numerically coded; Fed=1 and Fasted=2. 
When the back-transformed estimated difference lies between 0.8 and 1.25 then then this suggests evidence of 
no effect of food. 
 
6.3.12.3 
Assessment of Dose Proportionality  
Dose proportionality will be assessed for Cmax, Ctrough, AUC(0-t) and AUC(0-inf) using the PKS. 


 
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Individual concentration values will be used to perform a least-squares linear regression analysis, using the 
formula log_pkvar=A log_dose + B, where ‘log_pkvar’ represents the natural log transformed Cmax, AUC(0-t) or 
AUC(0-inf) and ‘log_dose’ represents the natural log transformed dose. An estimate of the slope of the regression 
line and corresponding 95% confidence interval (CI) will be obtained. 
The following SAS code will be used: 
PROC REG DATA=pkparam alpha=0.05; 
MODEL log_pkvar = log_dose / CLB; 
RUN; 
For each of the parameters Cmax, Ctrough, AUC(0-t) and AUC(0-inf) a plot of the log-transformed concentration 
against the log-transformed dose will be constructed including the fitted line from the linear regression and the 
line of unity. 
 
6.3.13 
Safety Analysis 
The analysis of the safety variables will be based on the SAF. 
6.3.13.1 
Adverse Events 
The following listings will be provided: 
 
All pre-treatment AEs and TEAEs 
 
TEAEs leading to study discontinuation 
 
TEAEs leading to treatment discontinuation 
 
TEAEs leading to dose modification (reduction/ interruption/ delay) 
 
TEAEs leading to death 
 
SAEs 
 
Patients experiencing toxicity Grade ≥3 according to CTCAE v4.03  
 
DLTs, MTD (Part 1 only) 
The following information will be included in the AE listings: AE description, MedDRA PT, MedDRA SOC, 
Start date/time, End date/time/ongoing, Frequency, Serious AE (Yes/No), Severity, Causal relationship to IMP, 


 
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Action taken with study drug, Action taken to treat Adverse Event, Outcome, Seriousness, CTCAE Grade, 
Treatment emergent (Yes/No), Dose limiting toxicity event (Yes/No). The earliest date will be regarded as start 
date of the event and the latest date/time will be regarded as stop date of the event within the assigned study 
period. 
Adverse events will be summarized on a per-subject basis and per-event. A per-subject basis means that even if a 
subject reported the same event repeatedly (i.e., events mapped to the same PT) during the study period, the 
event will be counted only once. A per-event basis the number of events will be reported. 
Treatment-related 
TEAEs, 
severity 
of 
TEAEs, 
causality 
of 
TEAEs, 
TEAEs 
leading 
to 
dose 
reduction/interruption, , serious TEAEs , serious TEAEs with causality, TEAEs with Grade 3, DLTs will also be 
summarized. In the case of multiple occurrences of the same TEAEs (at the same SOC or PT level) in an 
individual subject, the AE with the strongest assessment of relationship to study and the AE with the worst 
severity will be reported.  
 
6.3.13.2 
Clinical Safety Laboratory Tests (hematology, biochemistry, coagulation parameters and 
urinalysis) 
Laboratory values (hematology, biochemistry, coagulation parameters and urinalysis) will be listed by patient 
and study time point including changes from baseline (with the exception of urinalysis). The baseline for the 
laboratory values will be the last pre-dose measurement. 
All values outside the clinical reference ranges will be flagged in the data listings. The abnormal values will be 
flagged with ‘L’ for values below the lower limit of the clinical reference range and ‘H’ for values above the 
upper limit of the clinical reference range and included in the listings. The Investigator will assess whether the 
values outside the clinical reference range are clinically significant and these will be reported as abnormal not 
clinically significant (NCS) or abnormal clinically significant (CS). Clinically significant laboratory values will 
be recorded by the Investigator as AEs. 
For all hematology, biochemistry and coagulation data with numerical response, descriptive statistics will be 
presented by study part, dose level and time point (N, mean, SD, median, minimum, maximum). Summary tables 
will be provided for observed and changes from baseline values. 
For all hematology, biochemistry, coagulation and urinalysis, shift tables for important qualitative items (number, 
% in each category) will be presented by study part, dose level and time point. 


 
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6.3.13.3 
Vital Signs, Weight, BMI and ECOG PS 
Vital signs, weight, BMI and ECOG PS data will be listed by patient including changes from baseline. The 
baseline for the vital signs measurements will be the last pre-dose measurement. 
Descriptive statistics (n, mean, SD, median, minimum, maximum) for both observed values and changes from 
baseline will be presented by study part, dose level and time point. 
ECOG PS shift tables for important qualitative items (number, % in each category) will be presented by study 
part, dose level and time point. 
 
6.3.13.4 
Twelve-Lead Electrocardiogram 
All ECG parameters obtained from the ECG measurement will be listed by patient for each dose level and time 
point including changes from baseline. The baseline for the ECG measurements will be the last pre-dose 
measurement. 
Descriptive statistics (n, mean, SD, median, minimum, maximum) for both observed values and changes from 
baseline will be presented by study part, dose level and time point. 
The ECG will be summarized by the Investigator as ‘Normal’, ‘Abnormal, NCS’ or ‘Abnormal, CS’. 
Shift tables for important qualitative items (number, % in each category) will be presented by study part, dose 
level and time point. 
 
6.3.13.5 
Echocardiogram/MUGA 
Echocardiogram/MUGA will be summarized by the Investigator as ‘Normal’, ‘Abnormal, NCS’ or ‘Abnormal, 
CS’. 
Shift tables for important qualitative items (number, % in each category) will be presented by study part, dose 
level and time point. 
 


 
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6.3.13.6 
Physical Examination and Pregnancy 
The results of the physical examination and pregnancy (only female) will be listed by patient and time point. 
Physical examination will be summarized by the Investigator as ‘Normal’, ‘Abnormal, NCS’ or ‘Abnormal, CS’. 
Physical examination shift tables for important qualitative items (number, % in each category) will be presented 
by study part, dose level and time point. 
 
6.3.14 
Efficacy Analysis 
The analysis of the efficacy variables will be based on the SAF in Part 1, and on mITT and PP Set in Part 2. The 
total duration of the study from the start of randomization to the analysis of PFS is expected to be 29 months (12 
months of accrual + 17 months of the study duration for an individual patient). 
Data for target lesions and the non-target lesions along with the tumor evaluation according to RECIST v1.1 will 
be listed and BOR will be listed by study part, dose level and visit. Summary table for ORR (number, % in all 
patients with measurable disease at baseline) will be presented with the corresponding exact 95% CI.  
In Part 1, a by-patient listing will be generated for the survival outcomes. time-to-event estimates (including 
median event times and 95% CI) will be generated. PFS, OS and TTP will be estimated using Kaplan-Meier 
method and summarized. Kaplan-Meier plots will also be produced.  
In Part 2, time-to-event estimates (including median event times and 95% CI) will be generated. PFS, OS and 
TTP will be estimated using Kaplan-Meier method and summarized. Kaplan-Meier plots will also be produced. 
If patients are excluded from the mITT Set, a sensitivity analysis will be performed by repeating the analyses 
without excluding patients from the mITT Set. 
PFS 
Time for PFS (Days) = event date (progression or death date) or censor date – date of first time taking study 
drugs 
Censoring rules 
• 
Without an event: Censored at the date of last valid tumor assessment; if no post-dosing tumor 
assessment, censored at the date of first dosing. 


 
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• 
With an event (progression or death): If an event occurs after 2 subsequent missed tumor assessments, 
censored at the date of last valid tumor assessment.  
• 
Taking new anti-tumor treatment: censored at the last valid tumor assessment before taking new anti-
tumor treatment.  
OS  
Time for OS (Days) = event date (death from any reason) or censor date – date of first time taking study drugs 
Censoring rules 
• 
Without an event: Censored at the date of last valid tumor assessment/ last survival confirmation date; if 
no post-dosing tumor assessment, censored at the date of first dosing. 
TTP 
Time for TTP (Days) = event date (progression) or censor date – date of first time taking study drugs 
Censoring rules 
• 
Without an event: Censored at the date of last valid tumor assessment; if no post-dosing tumor 
assessment, censored at the date of first dosing. 
• 
With an event (progression): If an event occurs after 2 subsequent missed tumor assessments, censored 
at the date of last valid tumor assessment.  
• 
Taking new anti-tumor treatment: censored at the last valid tumor assessment before taking new anti-
tumor treatment. 
 
6.3.15 
Tumor Marker and Others 
All tumor marker measured will be listed by patient for study part and each dose level. Descriptive statistics will 
also be presented. 
Pharmacogenomic, pharmacoproteomic, and cellular markers (including the levels of immune cells, circulating 
endothelial progenitor cells and circulating tumor cells) obtained will be listed. 
 


 
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7. 
REFFRENCES 
1. SAS® Version 9.2 of the SAS System for Personal Computers. Copyright ©2002-2003. SAS Institute Inc. 
SAS and all other SAS Institute Inc. product or service names are registered trademarks or trademarks of 
SAS Institute Inc., Cary, NC, USA. 
2. WinNonlin Professional Software Version 6.3. http://www.pharsight.com 
3. CPMP/EWP/QWP/1401/98 Rev. 1/ Corr, Guideline on the investigation of bioequivalence, London, 20 
January 2010.  
 
 


 
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8. 
TABLES AND LISTINGS TO BE INCLUDED IN SETION 14 OF THE 
CLINICAL STUDY REPORT 
Subject Disposition 
Table 14.1.1.1 
Summary of Analysis Sets by Dose Level in Part 1 (Enrolled Set) 
Table 14.1.1.2 
Summary of Analysis Sets by Dose Level in Part 2 (Enrolled Set) 
Table 14.1.2.1 
Subject Disposition Details by Dose Level in Part 1 (Enrolled Set) 
Table 14.1.2.2 
Subject Disposition Details by Dose Level in Part 2 (Enrolled Set) 
 
 
Baseline and Demographic Data 
Table 14.1.3.1.1 
Subject Demographics by Dose Level in Part 1 (Enrolled Set) 
Table 14.1.3.1.1 
Subject Demographics by Dose Level in Part 2 (Enrolled Set ) 
Table 14.1.3.2.1 
Primary Diagnosis and Duration by Dose Level in Part 1 (Safety Analysis Set) 
Table 14.1.3.2.2 
Primary Diagnosis and Duration by Dose Level in Part 2 (Safety Analysis Set) 
 
Medical History 
Table 14.1.4.1 
Summary of Medical History by Dose Level in Part 1 (Safety Analysis Set) 
Table 14.1.4.2 
Summary of Medical History by Dose Level in Part 2 (Safety Analysis Set) 
 
Concomitant Medication 
Table 14.1.5.1.1 
Summary of Prior Medication by Dose Level in Part 1 (Safety Analysis Set) 
Table 14.1.5.1.2 
Summary of Prior Medication by Dose Level in Part 2 (Safety Analysis Set) 
Table 14.1.5.2.1 
Summary of Concomitant Medication by Dose Level in Part 1 (Safety Analysis Set) 
Table 14.1.5.2.2 
Summary of Concomitant Medication by Dose Level in Part 2 (Safety Analysis Set) 


 
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Efficacy Data 
 
Table 14.2.1.1 
Summary of ORR in Part 1 (Safety Analysis Set) 
Table 14.2.1.2 
Summary of ORR in Part 2 (Modified Intent-to-Treat Set) 
Table 14.2.1.3 
Summary of ORR in Part 2 (Per-Protocol Set) 
Table 14.2.1.4 
Summary of ORR in Part 2 (Safety Analysis Set) 
Table 14.2.2.1 
Summary of PFS Analysis in Part 1  (Safety Analysis Set) 
Table 14.2.2.2 
Summary of PFS Analysis in Part 2  (Modified Intent-to-Treat Set) 
Table 14.2.2.3 
Summary of PFS Analysis in Part 2  (Per-Protocol Set) 
Table 14.2.2.4 
Summary of PFS Analysis in Part 2  (Safety Analysis Set) 
Table 14.2.3.1 
Summary of OS Analysis in Part 1  (Safety Analysis Set) 
Table 14.2.3.2 
Summary of OS Analysis in Part 2  (Modified Intent-to-Treat Set) 
Table 14.2.3.3 
Summary of OS Analysis in Part 2  (Per-Protocol Set) 
Table 14.2.3.4 
Summary of OS Analysis in Part 2  (Safety Analysis Set) 
Table 14.2.4.1 
Summary of TTP Analysis in Part 1  (Safety Analysis Set) 
Table 14.2.4.2 
Summary of TTP Analysis in Part 2  (Modified Intent-to-Treat Set) 
Table 14.2.4.3 
Summary of TTP Analysis in Part 2  (Per-Protocol Set) 
Table 14.2.4.4 
Summary of TTP Analysis in Part 2  (Safety Analysis Set) 
 
 


 
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Safety Data 
 
Table 14.3.1.1.1 
Overview of Adverse Events by Dose Level in Part 1 (Safety Analysis Set) 
Table 14.3.1.1.2 
Overview of Adverse Events by Dose Level in Part 2 (Safety Analysis Set) 
Table 14.3.1.2.1 
Summary of Treatment-Emergent Adverse Event by Dose Level, System Organ Class 
and Preferred Term in Part 1 (Safety Analysis Set) 
Table 14.3.1.2.2 
Summary of Treatment-Emergent Adverse Event by Dose Level, System Organ Class 
and Preferred Term in Part 2 (Safety Analysis Set) 
Table 14.3.1.3.1 
Summary of Treatment Related Treatment-Emergent Adverse Event by Dose Level, 
System Organ Class and Preferred Term in Part 1 (Safety Analysis Set) 
Table 14.3.1.3.2 
Summary of Treatment Related Treatment-Emergent Adverse Event by Dose Level, 
System Organ Class and Preferred Term in Part 2 (Safety Analysis Set) 
Table 14.3.1.4.1 
Summary of Treatment-Emergent Adverse Event by Dose Level, System Organ Class, 
Preferred Term and Severity in Part 1 (Safety Analysis Set) 
Table 14.3.1.4.2 
Summary of Treatment-Emergent Adverse Event leading to study discontinuation by 
Dose Level, System Organ Class, Preferred Term and Severity in Part 2 (Safety 
Analysis Set) 
Table 14.3.1.5.1 
Summary of Treatment-Emergent Adverse Event by Dose Level, System Organ Class, 
Preferred Term and Causality in Part 1 (Safety Analysis Set) 
Table 14.3.1.5.2 
Summary of Treatment-Emergent Adverse Event by Dose Level, System Organ Class, 
Preferred Term and Causality in Part 2 (Safety Analysis Set) 
Table 14.3.1.6.1 
Summary of Treatment-Emergent Adverse Event leading to dose reductions by Dose 
Level, System Organ Class, Preferred Term in Part 1 (Safety Analysis Set) 
Table 14.3.1.6.2 
Summary of Treatment-Emergent Adverse Event leading to dose reductions by Dose 
Level, System Organ Class, Preferred Term in Part 2 (Safety Analysis Set) 
Table 14.3.1.7.1 
Summary of Treatment-Emergent Adverse Event leading to dose interruptions by Dose 
Level, System Organ Class, Preferred Term in Part 1 (Safety Analysis Set) 


 
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Table 14.3.1.7.2 
Summary of Treatment-Emergent Adverse Event leading to dose interruptions by Dose 
Level, System Organ Class, Preferred Term in Part 2 (Safety Analysis Set) 
Table 14.3.1.8.1 
Summary of Serious Adverse Event by Dose Level, System Organ Class, Preferred 
Term in Part 1 (Safety Analysis Set) 
Table 14.3.1.8.2 
Summary of Serious Adverse Event by Dose Level, System Organ Class, Preferred 
Term in Part 2 (Safety Analysis Set) 
Table 14.3.1.9.1 
Summary of Serious Adverse Event by Dose Level, System Organ Class, Preferred 
Term and Causality in Part 1 (Safety Analysis Set) 
Table 14.3.1.9.2 
Summary of Serious Adverse Event by Dose Level, System Organ Class, Preferred 
Term and Causality in Part 2 (Safety Analysis Set) 
Table 14.3.1.10.1 
Summary of Treatment-Emergent Adverse Even by Dose Level, System Organ Class, 
Preferred Term and patients experiencing toxicity Grade ≥3 according to CTCAE v4.03 
in Part 1 (Safety Analysis Set) 
Table 14.3.1.10.2 
Summary of Treatment-Emergent Adverse Even by Dose Level, System Organ Class, 
Preferred Term and patients experiencing toxicity Grade ≥3 according to CTCAE v4.03 
in Part 2 (Safety Analysis Set) 
Table 14.3.1.11 
Summary of DLTs by Dose Level in Part 1 (Safety Analysis Set) 
Table 14.3.2.1.1 
Hematology with N, mean, SD, median, minimum, maximum and change from baseline 
by Dose level in Part 1 (Safety Analysis Set) 
Table 14.3.2.1.2 
Hematology with N, mean, SD, median, minimum, maximum and change from baseline 
by Dose level in Part 2 (Safety Analysis Set) 
Table 14.3.2.2.1 
Biochemistry with N, mean, SD, median, minimum, maximum and change from 
baseline by Dose level in Part 2 (Safety Analysis Set) 
Table 14.3.2.2.2 
Biochemistry with N, mean, SD, median, minimum, maximum and change from 
baseline by Dose level in Part 2 (Safety Analysis Set) 
Table 14.3.2.3.1 
Coagulation with N, mean, SD, median, minimum, maximum and change from baseline 
by Dose level in Part 2 (Safety Analysis Set) 


 
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Table 14.3.2.3.2 
Coagulation with N, mean, SD, median, minimum, maximum and change from baseline 
by Dose level in Part 2 (Safety Analysis Set) 
Table 14.3.2.4.1 
Summary of Shift  in Hematology Results From Baseline to Post-Baseline by Dose 
level in Part 1 (Safety Analysis Set) 
Table 14.3.2.4.2 
Summary of Shift  in Hematology Results From Baseline to Post-Baseline by Dose 
level in Part 2 (Safety Analysis Set) 
Table 14.3.2.5.1 
Summary of Shift  in Biochemistry Results From Baseline to Post-Baseline by Dose 
level in Part 1 (Safety Analysis Set) 
Table 14.3.2.5.2 
Summary of Shift  in Biochemistry Results From Baseline to Post-Baseline by Dose 
level in Part 2 (Safety Analysis Set) 
Table 14.3.2.6.1 
Summary of Shift  in Coagulation Results From Baseline to Post-Baseline by Dose 
level in Part 1 (Safety Analysis Set) 
Table 14.3.2.6.2 
Summary of Shift  in Coagulation Results From Baseline to Post-Baseline by Dose 
level in Part 2 (Safety Analysis Set) 
Table 14.3.2.7.1 
Summary of Shift  in Urinalysis Results From Baseline to Post-Baseline by Dose level 
in Part 1 (Safety Analysis Set) 
Table 14.3.2.7.2 
Summary of Shift  in Urinalysis Results From Baseline to Post-Baseline by Dose level 
in Part 2 (Safety Analysis Set) 
Table 14.3.3.1.1 
Vital Signs with N, mean, SD, median, minimum, maximum and change from baseline 
by Dose level in Part 1 (Safety Analysis Set) 
Table 14.3.3.1.2 
Vital Signs with N, mean, SD, median, minimum, maximum and change from baseline 
by Dose level in Part 2 (Safety Analysis Set) 
Table 14.3.3.2.1 
Weight with N, mean, SD, median, minimum, maximum and change from baseline by 
Dose level in Part 1 (Safety Analysis Set) 
Table 14.3.3.2.2 
Weight Signs with N, mean, SD, median, minimum, maximum and change from 
baseline by Dose level in Part 2 (Safety Analysis Set) 
Table 14.3.3.3.1 
BMI with N, mean, SD, median, minimum, maximum and change from baseline by 


 
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Dose level in Part 1 (Safety Analysis Set) 
Table 14.3.3.3.2 
BMI Signs with N, mean, SD, median, minimum, maximum and change from baseline 
by Dose level in Part 2 (Safety Analysis Set) 
Table 14.3.3.4.1 
ECOG PS with N, mean, SD, median, minimum, maximum and change from baseline 
by Dose level in Part 1 (Safety Analysis Set) 
Table 14.3.3.4.2 
ECOG PS Signs with N, mean, SD, median, minimum, maximum and change from 
baseline by Dose level in Part 2 (Safety Analysis Set) 
Table 14.3.3.5.1 
ECOG PS Shift table by Dose level in Part 1 (Safety Analysis Set) 
Table 14.3.3.5.2 
ECOG PS Shift table by Dose level in Part 2 (Safety Analysis Set) 
Table 14.3.4.1.1 
Twelve-Lead Electrocardiograms with N, mean, SD, median, minimum, maximum and 
change from baseline by Dose level in Part 1 (Safety Analysis Set) 
Table 14.3.4.1.2 
Twelve-Lead Electrocardiograms with N, mean, SD, median, minimum, maximum and 
change from baseline by Dose level in Part 2 (Safety Analysis Set) 
Table 14.3.4.2.1 
Summary of Twelve-Lead Electrocardiograms by Dose level in Part 1 (Safety Analysis 
Set) 
Table 14.3.4.2.2 
Summary of Twelve-Lead Electrocardiograms by Dose level in Part 2 (Safety Analysis 
Set) 
Table 14.3.4.3.1 
Twelve-Lead Electrocardiograms Shift table by Dose level in Part 1 (Safety Analysis 
Set) 
Table 14.3.4.3.2 
Twelve-Lead Electrocardiograms Shift table by Dose level in Part 2 (Safety Analysis 
Set) 
Table 14.3.5.1.1 
Summary of Echocardiograms/ MUGA by Dose level in Part 1 (Safety Analysis Set) 
Table 14.3.5.1.2 
Summary of Echocardiograms/ MUGA by Dose level in Part 2 (Safety Analysis Set) 
Table 14.3.5.2.1 
Echocardiograms/ MUGA Shift table by Dose level in Part 1 (Safety Analysis Set) 
Table 14.3.5.2.2 
Echocardiograms/ MUGA Shift table by Dose level in Part 2 (Safety Analysis Set) 


 
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Table 14.3.6.1.1 
Summary of Physical Examination by Dose level in Part 1 (Safety Analysis Set) 
Table 14.3.6.1.2 
Summary of Physical Examination by Dose level in Part 2 (Safety Analysis Set) 
Table 14.3.6.2.1 
Physical Examination shift table by Dose level in Part 1 (Safety Analysis Set) 
Table 14.3.6.2.2 
Physical Examination shift table by Dose level in Part 2 (Safety Analysis Set) 
 
 
Exposure 
 
Table 14.4.1.1.1 
Summary of Study Drug Exposure by Dose Level in Part 1 (Safety Analysis Set) 
Table 14.4.1.1.2 
Summary of Study Drug Exposure by Dose Level in Part 2 (Safety Analysis Set) 
Table 14.4.1.2.1 
Summary of Study Drug Modifications by Dose Level in Part 1 (Safety Analysis Set) 
Table 14.4.1.2.2 
Summary of Study Drug Modifications by Dose Level in Part 2 (Safety Analysis Set) 
Table 14.4.1.3.1 
Summary of Study Drug Compliance by Dose Level in Part 1 (Safety Analysis Set) 
Table 14.4.1.3.2 
Summary of Study Drug Compliance by Dose Level in Part  (Safety Analysis Set) 
 
 
Pharmacokinetic Data 
Table 14.4.2.1.1 
Summary of Plasma Concentrations of dFdC versus Nominal Sampling Times by Dose 
Level in Part 1 (Pharmacokinetic Set) 
Table 14.4.2.1.2 
Summary of Plasma Concentrations of dFdC versus Nominal Sampling Times by Dose 
Level in Part 2 (Pharmacokinetic Set) 
Table 14.4.2.2.1 
Summary of Plasma Concentrations of dFdU versus Nominal Sampling Times by Dose 
Level in Part 1 (Pharmacokinetic Set) 
Table 14.4.2.2.2 
Summary of Plasma Concentrations of dFdU versus Nominal Sampling Times by Dose 
Level in Part 2 (Pharmacokinetic Set) 
Table 14.4.2.3.1 
Summary of Pharmacokinetic Parameters of dFdC by Dose Level in Part 1 
(Pharmacokinetic Set) 


 
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Table 14.4.2.3.2 
Summary of Pharmacokinetic Parameters of dFdC by Dose Level in Part 2 
(Pharmacokinetic Set) 
Table 14.4.2.4.1 
Summary of Pharmacokinetic Parameters of dFdU by Dose Level in Part 1 
(Pharmacokinetic Set) 
Table 14.4.2.4.2 
Summary of Pharmacokinetic Parameters of dFdU by Dose Level in Part 2 
(Pharmacokinetic Set) 
Table 14.4.2.5.1 
Summary of Metabolic Ratio by Dose Level in Part 1 (Pharmacokinetic Set) 
Table 14.4.2.5.2 
Summary of Metabolic Ratio by Dose Level in Part 2 (Pharmacokinetic Set) 
Table 14.4.2.6.1 
Food-Effect Evaluation for dFdC in the Higher Dose-Expansion Cohort in Part 1 
(Pharmacokinetic Set) 
Table 14.4.2.6.2 
Food-Effect Evaluation for dFdU in the Higher Dose-Expansion Cohort in Part 2 
(Pharmacokinetic Set) 
Table 14.4.2.7.1 
Dose Proportionality for dFdC by Dose Level in Part 1 (Pharmacokinetic Set) 
Table 14.4.2.7.2 
Dose Proportionality for dFdC by Dose Level in Part 2 (Pharmacokinetic Set) 
Table 14.4.2.8.1 
Dose Proportionality for dFdU by Dose Level in Part 1 (Pharmacokinetic Set) 
Table 14.4.2.8.2 
Dose Proportionality for dFdU by Dose Level in Part 2 (Pharmacokinetic Set) 
 
 
Tumor Markers 
 
Table 14.5.1 
Summary of Tumor Marker by Dose Level in Part 1 (Safety Analysis Set) 
Table 14.5.2 
Summary of Tumor Marker by Dose Level in Part 2 (Safety Analysis Set) 
 
 
Protocol 
Deviations 
 
Table 14.6.1 
 Summary of Protocol Deviation by Dose Level in Part 1 (Safety Analysis Set) 


 
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Table 14.6.2 
Summary of Protocol Deviation by Dose Level in Part 2 (Safety Analysis Set) 
9. 
FIGURES 
Figure 14.2.1.1 
Kaplan-Meier Plot for PFS in Part 1  (Safety Analysis Se) 
Figure 14.2.1.2 
Kaplan-Meier Plot for PFS in Part 2  (Modified Intent-to-Treat Set) 
Figure 14.2.1.3 
Kaplan-Meier Plot for PFS in Part 2  (Per-Protocol Set) 
Figure 14.2.1.4 
Kaplan-Meier Plot for PFS in Part 2  (Safety Analysis Set) 
Figure 14.2.2.1 
Kaplan-Meier Plot for OS in Part 1  (Safety Analysis Set) 
Figure 14.2.2.2 
Kaplan-Meier Plot for OS in Part 2  (Modified Intent-to-Treat Set) 
Figure 14.2.2.3 
Kaplan-Meier Plot for OS in Part 2  (Per-Protocol Set) 
Figure 14.2.2.4 
Kaplan-Meier Plot for OS in Part 2  (Safety Analysis Set) 
Figure 14.2.3.1 
Kaplan-Meier Plot for TTP in Part 1  (Safety Analysis Set) 
Figure 14.2.3.2 
Kaplan-Meier Plot for TTP in Part 2  (Modified Intent-to-Treat Set) 
Figure 14.2.3.3 
Kaplan-Meier Plot for TTP in Part 2  (Per-Protocol Set) 
Figure 14.2.3.4 
Kaplan-Meier Plot for TTP in Part 2  (Safety Analysis Set) 
Figure 14.4.1.1 
Subject Profiles for dFdC Plasma Concentration Time Data (Linear Scale) 
(Pharmacokinetic Set) 
Figure 14.4.1.2 
Subject Profiles for dFdU Plasma Concentration Time Data (Linear Scale) 
(Pharmacokinetic Set) 
Figure 14.4.1.3 
Subject Profiles for dFdC Plasma Concentration Time Data (Semi-logarithmic Scale) 
(Pharmacokinetic Set) 
Figure 14.4.1.4 
Subject Profiles for dFdU Plasma Concentration Time Data (Semi-logarithmic Scale) 
(Pharmacokinetic Set) 
 Figure 14.4.2.1 
Mean (± Standard Deviation) Profiles for dFdC Plasma Concentration Time Data in 


 
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Part 1 (Linear Scale) (Pharmacokinetic Set) 
Figure 14.4.2.2 
Mean (± Standard Deviation) Profiles for dFdC Plasma Concentration Time Data in 
Part 2 (Linear Scale) (Pharmacokinetic Set) 
 Figure 14.4.2.3 
Mean (± Standard Deviation) Profiles for dFdC Plasma Concentration Time Data in 
Part 1 (Semi-logarithmic Scale) (Pharmacokinetic Set) 
Figure 14.4.2.4 
Mean (± Standard Deviation) Profiles for dFdC Plasma Concentration Time Data in 
Part 2 (Semi-logarithmic Scale) (Pharmacokinetic Set) 
Figure 14.4.3.1 
Mean (± Standard Deviation) Profiles for dFdU Plasma Concentration Time Data in 
Part 1 (Linear Scale) (Pharmacokinetic Set) 
Figure 14.4.3.2 
Mean (± Standard Deviation) Profiles for dFdU Plasma Concentration Time Data in 
Part 2 (Linear Scale) (Pharmacokinetic Set) 
Figure 14.4.3.3 
Mean (± Standard Deviation) Profiles for dFdU Plasma Concentration Time Data in 
Part 1 (Semi-logarithmic Scale) (Pharmacokinetic Set) 
Figure 14.4.3.4 
Mean (± Standard Deviation) Profiles for dFdU Plasma Concentration Time Data in 
Part 2 (Semi-logarithmic Scale) (Pharmacokinetic Set) 
Figure 14.4.4.1 
Assessment of dFdC Dose Proportionality  in Part 1 (Pharmacokinetic Set)  
Figure 14.4.4.2 
Assessment of dFdC Dose Proportionality  in Part 2 (Pharmacokinetic Set)  
Figure 14.4.4.3 
Assessment of dFdU Dose Proportionality in Part 1 (Pharmacokinetic Set) 
Figure 14.4.4.4 
Assessment of dFdU Dose Proportionality in Part 2 (Pharmacokinetic Set) 
10. 
LISTINGS TO BE INCLUDED IN SECTION 16 OF THE CLINICAL 
STUDY REPORT 
Subject Disposition 
Listing 16.2.1.1.1 
Disposition of the Patients in Part 1 (Enrolled Set) 
Listing 16.2.1.1.2 
Disposition of the Patients in Part 2 (Enrolled Set) 


 
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Listing 16.2.1.2.1 
Failed Inclusion and Exclusion Criteria in Part 1 (Enrolled Set) 
Listing 16.2.1.2.2 
Failed Inclusion and Exclusion Criteria in Part 2 (Enrolled Set) 
Listing 16.2.1.3.1 
Discontinued patients in Part 1 (Safety Analysis Set) 
Listing 16.2.1.3.2 
Discontinued patients in Part 2 (Safety Analysis Set) 
Listing 16.2.2.1 
Protocol Deviations in Part 1 (Safety Analysis Set) 
Listing 16.2.2.2 
Protocol Deviations in Part 2 (Safety Analysis Set) 
Baseline and Demographic Data 
Listing 16.2.4.1.1 
Subject Demographics and Baseline Characteristics in Part 1 (Safety Analysis Set ) 
Listing 16.2.4.1.2 
Subject Demographics and Baseline Characteristics in Part 2 (Safety Analysis Set ) 
Listing 16.2.4.2.1 
Medical History in Part 1 (Safety Analysis Set ) 
Listing 16.2.4.2.2 
Medical History in Part 2 (Safety Analysis Set ) 
Listing 16.2.4.3.1 
Primary Diagnosis and Duration in Part 1 (Safety Analysis Set ) 
Listing 16.2.4.3.2 
Primary Diagnosis and Duration in Part 2 (Safety Analysis Set ) 
Listing 16.2.4.4.1 
Substance use - drugs in Part 1 (Safety Analysis Set ) 
Listing 16.2.4.4.2 
Substance use - drugs in Part 2 (Safety Analysis Set ) 
 
 
Concomitant Medication 
Listing 16.2.4.5.1 
Prior Medication in Part 1 (Safety Analysis Set ) 
Listing 16.2.4.5.2 
Prior Medication in Part 2 (Safety Analysis Set ) 
Listing 16.2.4.6.1 
Concomitant Medication in Part 1 (Safety Analysis Set) 
Listing 16.2.4.6.2 
Concomitant Medication in Part 2 (Safety Analysis Set) 
 
 


 
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Exposure 
Listing 16.2.5.1.1 
Study Drug Administration in Part 1 (Safety Analysis Set) 
Listing 16.2.5.1.2 
Study Drug Administration in Part 2 (Safety Analysis Set) 
Listing 16.2.5.2.1 
Listing of Derived Study Drug Administration in Part 1 (Safety Analysis Set) 
Listing 16.2.5.2.2 
Listing of Derived Study Drug Administration in Part 2 (Safety Analysis Set) 
 
 
Pharmacokinetic Data 
Listing 16.2.5.3.1 
Individual Blood Sampling Times in Part 1 (Pharmacokinetic Set) 
Listing 16.2.5.3.2 
Individual Blood Sampling Times in Part 2 (Pharmacokinetic Set) 
Listing 16.2.5.4.1 
Individual Plasma Concentrations of dFdC in Part 1 (Pharmacokinetic Set) 
Listing 16.2.5.4.2 
Individual Plasma Concentrations of dFdC in Part 2 (Pharmacokinetic Set) 
Listing 16.2.5.5.1 
Individual Plasma Concentrations of dFdU in Part 1 (Pharmacokinetic Set) 
Listing 16.2.5.5.2 
Individual Plasma Concentrations of dFdU in Part 2 (Pharmacokinetic Set) 
Listing 16.2.5.6.1 
Pharmacokinetic Parameters of  dFdC  (Pharmacokinetic Set) 
Listing 16.2.5.6.2 
Pharmacokinetic Parameters of  dFdU  (Pharmacokinetic Set) 
 
 
Efficacy Data 
 
Listing 16.2.6.1.1 
Tumor Assessment – Target Lesions and Clinical Overall Visit Response in Part 1 
(Safety Analysis Set) 
Listing 16.2.6.1.2 
Tumor Assessment – Target Lesions and Clinical Overall Visit Response in Part 2 
(Safety Analysis Set) 
Listing 16.2.6.2.1 
Tumor Assessment – Non-Target Lesions in Part 1(Safety Analysis Set) 
Listing 16.2.6.2.2 
Tumor Assessment – Non-Target Lesions in Part 2(Safety Analysis Set) 


 
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Listing 16.2.6.3.1 
Tumor Assessment – New Lesions in Part 1(Safety Analysis Set) 
Listing 16.2.6.3.2 
Tumor Assessment – New Lesions (Safety Analysis Set) 
Listing 16.2.6.4.1 
Best Overall Response and ORR in Part 1(Safety Analysis Set) 
Listing 16.2.6.4.2 
Best Overall Response and ORR in Part 2(Safety Analysis Set) 
Listing 16.2.6.5.1 
PFS, OS and TTP in Part 1 (Safety Analysis Set) 
Listing 16.2.6.5.2 
PFS, OS and TTP in Part 2 (Safety Analysis Set) 
 
 
Safety Variables 
Listing 16.2.7.1.1 
All Adverse Events in Part 1 (Safety Analysis Set) 
Listing 16.2.7.1.2 
All Adverse Events in Part 2 (Safety Analysis Set) 
Listing 16.2.7.2.1 
All Serious Adverse Events in Part 1 (Safety Analysis Set) 
Listing 16.2.7.2.2 
All Serious Adverse Events in Part 2 (Safety Analysis Set) 
Listing 16.2.7.3.1 
Treatment-Emergent Adverse Event  leading to study discontinuation in Part 1 (Safety 
Analysis Set) 
Listing 16.2.7.3.2 
Treatment-Emergent Adverse Event  leading to study discontinuation in Part 2 (Safety 
Analysis Set) 
Listing 16.2.7.4.1 
Treatment-Emergent Adverse Event  leading to treatment discontinuation in Part 1 
(Safety Analysis Set) 
Listing 16.2.7.4.2 
Treatment-Emergent Adverse Event  leading to treatment discontinuation in Part 2 
(Safety Analysis Set) 
Listing 16.2.7.5.1 
Treatment-Emergent 
Adverse 
Event 
 
leading 
to 
dose 
modification 
(reduction/interruption/delay) in Part 1 (Safety Analysis Set) 
Listing 16.2.7.5.2 
Treatment-Emergent 
Adverse 
Event 
 
leading 
to 
dose 
modification 
(reduction/interruption/delay) in Part 2 (Safety Analysis Set) 


 
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Listing 16.2.7.6.1 
Treatment-Emergent Adverse Event leading to death in Part 1 (Safety Analysis Set) 
Listing 16.2.7.6.2 
Treatment-Emergent Adverse Event leading to death in Part 2 (Safety Analysis Set) 
Listing 16.2.7.7.1 
Treatment-Emergent Adverse Event with Grade≥ 3 in Part 1(Safety Analysis Set) 
Listing 16.2.7.7.2 
Treatment-Emergent Adverse Event with Grade≥ 3 in Part 2(Safety Analysis Set) 
Listing 16.2.7.8.1 
Patients experiencing toxicity Grade ≥3 according to CTCAE v4.03 in Part 1 (Safety 
Analysis Set) 
Listing 16.2.7.8.2 
Patients experiencing toxicity Grade ≥3 according to CTCAE v4.03 in Part 2 (Safety 
Analysis Set) 
Listing 16.2.7.8.2 
DLTs in Part 2 (Safety Analysis Set) 
Listing 16.2.8.1.1 
Biochemistry Laboratory Values in Part 1 (Safety Analysis Set) 
Listing 16.2.8.1.2 
Biochemistry Laboratory Values  in Part 2(Safety Analysis Set) 
Listing 16.2.8.2.1 
Hematology Laboratory Values in Part 1 (Safety Analysis Set) 
Listing 16.2.8.2.2 
Hematology Laboratory Values in Part 2 (Safety Analysis Set) 
Listing 16.2.8.3.1 
Coagulation Laboratory Values in Part 1 (Safety Analysis Set) 
Listing 16.2.8.3.2 
Coagulation Laboratory Values in Part 2 (Safety Analysis Set) 
Listing 16.2.8.4.1 
Urinalysis Laboratory Values in Part 1 (Safety Analysis Set) 
Listing 16.2.8.4.2 
Urinalysis Laboratory Values in Part 2 (Safety Analysis Set) 
Listing 16.2.9.1.1 
Vital Signs in Part 1 (Safety Analysis Set) 
Listing 16.2.9.1.2 
Vital Signs in Part 2 (Safety Analysis Set) 
Listing 16.2.9.2.1 
Body Height, Weight and BMI in Part 1 (Safety Analysis Set) 
Listing 16.2.9.2.2 
Body Height, Weight and BMI in Part 2 (Safety Analysis Set) 
Listing 16.2.9.3.1 
ECOG PS in Part 1 (Safety Analysis Set) 
Listing 16.2.9.3.2 
ECOG PS in Part 2 (Safety Analysis Set) 


 
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Listing 16.2.10.1 
Twelve-Lead Electrocardiogram in Part 1 (Safety Analysis Set) 
Listing 16.2.10.2 
Twelve-Lead Electrocardiogram in Part 2 (Safety Analysis Set) 
Listing 16.2.11.1 
Echocardiogram /MUGA in Part 1 (Safety Analysis Set) 
Listing 16.2.11.2 
Echocardiogram /MUGA in Part 2 (Safety Analysis Set) 
Listing 16.2.12.1.1 
Physical Examination in Part 1 (Safety Analysis Set) 
Listing 16.2.12.1.2 
Physical Examination in Part 2 (Safety Analysis Set) 
Listing 16.2.12.2.1 
Pregnancy Test in Part 1 (Safety Analysis Set) 
Listing 16.2.12.2.2 
Pregnancy Test in Part 2 (Safety Analysis Set) 
 
 


 
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11. 
DOCUMENTATION OF STATISTICAL METHODS 


 
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TP-EP.BS-WW-001-05  
Effective date:  29 Jul 15 
Related to:  SOP-EP.BS-WW-002 
 
                                                                         Page 60 of 63 
12. 
EVENT SCHEDULES 
Table 5 
Part 1 Event Schedule (Dose Escalation) 
 
 
SV 
Cycle 1a 
Cycle 2 
Cycle 3 
onwardsb 
EOTc 
30-
day 
FU 
FU 
for 
OS 
 
D -28 
to 0 
D1 
D2 
D3 
D8 
D15 
D16 
D17 
D1 
(±1d) 
D8 
(±1d) 
D15 
(±1d) 
D1 
(±1d) 
(±3d) 
(±3d) 
(±3d) 
Informed consent 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Inclusion/Exclusion criteria 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
Medical history 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Demographics 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Prior medication 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Concomitant medication 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
Height 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Weight 
X 
X 
 
 
X 
X 
 
 
X 
X 
X 
X 
X 
X 
 
Physical examination 
X 
X 
 
 
X 
X 
 
 
X 
X 
X 
X 
X 
X 
 
Vital signs 
X 
X 
 
 
X 
X 
 
 
X 
X 
X 
X 
X 
X 
 
ECG 
X 
X 
 
 
X 
X 
 
 
X 
 
 
X 
X 
X 
 
Echocardiogram or MUGA 
X 
 
 
 
 
 
 
 
 
 
 
Xd 
 
 
 
ECOG PS 
X 
X 
 
 
X 
X 
 
 
X 
X 
X 
X 
X 
X 
 
Pregnancy teste 
X 
X 
 
 
 
 
 
 
X 
 
 
X 
X 
X 
 
Hematology, serum chemistry, 
coagulation parametersf  
X 
Xg 
  
 
X 
X 
 
 
X 
X 
X 
X 
X 
X 
 
Urinalysis 
X 
Xg 
 
 
X 
X 
 
 
X 
 
 
X 
X 
X 
 
Tumor markers 
X 
Xg 
 
 
 
 
 
 
X 
 
 
X 
X 
 
 
AE/SAEf 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
Study drug administrationh 
 
X 
 
X 
X 
X 
 
X 
X 
X 
X 
X 
 
 
 
Dispense study drug  
 
X 
 
 
 
 
 
 
X 
 
 
X 
 
 
 
Provide/review diary 
 
X 
 
 
 
 
 
 
X 
 
 
X 
X 
 
 
PK samplingi 
 
X 
X 
X 
X 
X 
X 
X 
 
 
 
 
 
 
 
Tumor assessment 
X 
 
 
 
 
 
 
 
 
 
 
Xj 
 
 
 


 
Statistical Analysis Plan 
InnoPharmax Inc. 
 
Final 1.0 
Protocol No.: Inno-GO-03 
 
21/Jan/2019 
TP-EP.BS-WW-001-05  
Effective date:  29 Jul 15 
Related to:  SOP-EP.BS-WW-002 
 
                                                                         Page 61 of 63 
(RECIST v1.1) 
Contact of patient by site 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Xk 
Biomarker sample 
Xl 
 
 
 
 
 
 
 
 
 
 
X 
X 
 
 
Abbreviations: AE=adverse event; D/d=day; ECG=electrocardiogram; ECOG= Eastern Cooperative Oncology Group; EOT=end of treatment; 
FU=follow up; MUGA=multigated acquisition; OS=overall survival; PK=pharmacokinetic; PS=performance status; RECIST=Response Evaluation 
Criteria in Solid Tumors; SAE=serious adverse event; SV=Screening Visit; v=version. 
a. Only the days on which the patient will visit the study site are displayed in this table. 
b. Patients will receive D07001-softgel until withdrawal due to a DLT, disease progression according to RECIST v1.1, withdrawn consent, or when 
another treatment discontinuation criterion is met. 
c. Patients who are discontinued from study drug for a reason other than disease progression, death, or withdrawal of consent are to continue tumor 
assessments on the same schedule. 
d. Cycles 3 and 5 only. 
e. In women of childbearing potential only.  A serum pregnancy test will be done at Screening and on Cycle 1 Day 1.  A urine pregnancy test will be 
done on Day 1 of each subsequent cycle, at the EOT visit, and at the safety follow-up visit. 
f. Abnormal liver function test values and AEs will be followed as indicated until resolution or return to baseline. 
g. To be repeated only if screening was more than 7 calendar days prior. 
h. Patients will receive study drug in the fasted state on Days 1, 3, 5, 8, 10, 12, 15, 17, and 19 of each cycle.  During Cycle 1, patients may take study 
drug at home on Days 5, 10, 12, and 19.  During Cycle 2, patients may take study drug at home on Days 3, 5, 10, 12, 17, and 19.  From Cycle 3 
onwards, patients may take study drug at home on all dosing days except for Day 1 of each cycle.  Dosing on 2 consecutive days will not be allowed; 
there will be at least 1 day between 2 doses. 
i. 
For PK sampling, there may be a time window of within 30 minutes before the study drug administration for the pre-dose time point, ±5 minutes for 
time points up to 4 hours of study drug administration, ±15 minutes for time points after 4 hours of study drug administration, and ±60 minutes for 
the time points on return visits. 
j. 
Tumor assessment will be performed within 0-7 calendar days before the start of every odd-numbered cycle, starting from 0-7 calendar days before 
the start of Cycle 3. 
k. Every 6 weeks. 
l. 
Samples for pharmacogenomic, pharmacoproteomic and cellular analysis will be collected if the patient provides additional consent.   
 
 
 
 
 
 


 
Statistical Analysis Plan 
InnoPharmax Inc. 
 
Final 1.0 
Protocol No.: Inno-GO-03 
 
21/Jan/2019 
TP-EP.BS-WW-001-05  
Effective date:  29 Jul 15 
Related to:  SOP-EP.BS-WW-002 
 
                                                                         Page 62 of 63 
Table 6 
Part 2 Event Schedule (Dose Expansion) 
 
 
SV 
Cycle 1a 
Cycle 2 
Cycle 3 
onwardsb 
EOTc 
30-
day 
FU 
FU 
for 
OS 
 
D -28 to 
0 
D1 
D2 
D3 
D8 
D 
15 
D 
16 
D 
17 
D1 
(±1d) 
D2d 
(±1d) 
D3d 
(±1d) 
D8 
(±1d) 
D15 
(±1d) 
D1  
(±1d) 
(±3d) 
(±3d) 
(±3d) 
Informed consent 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Inclusion/Exclusion criteria 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Medical history 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Demographics 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Prior medication 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Concomitant medication 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Xd 
Xd 
X 
X 
X 
X 
X 
 
Height 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Weight 
X 
X 
 
 
X 
X 
 
 
X 
 
 
X 
X 
X 
X 
X 
 
Physical examination 
X 
X 
 
 
X 
X 
 
 
X 
 
 
X 
X 
X 
X 
X 
 
Vital signs 
X 
X 
 
 
X 
X 
 
 
X 
 
 
X 
X 
X 
X 
X 
 
ECG 
X 
X 
 
 
X 
X 
 
 
X 
 
 
 
 
X 
X 
X 
 
Echocardiogram or MUGA 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Xe 
 
 
 
ECOG PS 
X 
X 
 
 
X 
X 
 
 
X 
 
 
X 
X 
X 
X 
X 
 
Pregnancy testf 
X 
X 
 
 
 
 
 
 
X 
 
 
 
 
X 
X 
X 
 
Hematology, serum 
chemistry, coagulation 
parametersg  
X 
Xh 
  
 
X 
X 
 
 
X 
 
 
X 
X 
X 
X 
X 
 
Urinalysis 
X 
Xh 
 
 
X 
X 
 
 
X 
 
 
 
 
X 
X 
X 
 
Tumor markers 
X 
Xh 
 
 
 
 
 
 
X 
 
 
 
 
X 
X 
 
 
AE/SAEg 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Xd 
Xd 
X 
X 
X 
X 
X 
 
Study drug administrationi 
 
X 
 
X 
X 
X 
 
X 
X 
 
Xd 
X 
X 
X 
 
 
 
Dispense study drug 
 
X 
 
 
 
 
 
 
X 
 
 
 
 
X 
 
 
 
Provide/review diary 
 
X 
 
 
 
 
 
 
X 
 
 
 
 
X 
X 
 
 
PK samplingj 
 
X 
X 
X 
X 
X 
X 
X 
Xd 
Xd 
Xd 
 
 
 
 
 
 
Tumor assessment 
(RECIST v1.1) 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Xk 
 
 
 
Contact of patient by site 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Xl 


 
Statistical Analysis Plan 
InnoPharmax Inc. 
 
Final 1.0 
Protocol No.: Inno-GO-03 
 
21/Jan/2019 
TP-EP.BS-WW-001-05  
Effective date:  29 Jul 15 
Related to:  SOP-EP.BS-WW-002 
 
                                                                         Page 63 of 63 
Biomarker sample 
Xm 
 
 
 
 
 
 
 
 
 
 
 
 
X 
X 
 
 
 
Abbreviations: AE=adverse event; D/d=day; ECG=electrocardiogram; ECOG= Eastern Cooperative Oncology Group; EOT=end of treatment; 
FU=follow up; MUGA=multigated acquisition; OS=overall survival; PK=pharmacokinetic; PS=performance status; RECIST=Response Evaluation 
Criteria in Solid Tumors; SAE=serious adverse event; SV=Screening Visit; v=version. 
a. Only the days on which the patient will visit the study site are displayed in this table. 
b. Patients will receive D07001-softgel until withdrawal due to disease progression according to RECIST v1.1, withdrawn consent, or when another 
treatment discontinuation criterion is met. 
c. Patients who are discontinued from study drug for a reason other than disease progression, death, or withdrawal of consent are to continue tumor 
assessments on the same schedule. 
d. Patients in food-effect cohort only. Patients who are not in the food-effect cohort will not attend the study site on Cycle 2 Days 2 and 3, and so may 
take the study drug at home on Cycle 2 Day 3. 
e. Cycles 3 and 5 only. 
f. In women of childbearing potential only.  A serum pregnancy test will be done at Screening and on Cycle 1 Day 1.  A urine pregnancy test will be 
done on Day 1 of each subsequent cycle, at the EOT visit, and at the safety follow-up visit. 
g. Abnormal liver function test values and AEs will be followed as indicated until resolution or return to baseline. 
h. To be repeated only if screening was more than 7 calendar days prior. 
i. 
Patients will receive study drug in the fasted state on Days 1, 3, 5, 8, 10, 12, 15, 17, and 19 of each cycle, besides who was randomized to the high-fat 
fed in Cycle 1 Day 15 or Cycle 2 Day 1.  During Cycle 1, patients may take study drug at home on Days 5, 10, 12, and 19.  During Cycle 2, patients 
in the lower dose-expansion cohort may take study drug at home on Days 3, 5, 10, 12, 17, and 19; patients in the higher dose-expansion cohort (food-
effect cohort) may take study drug at home on Days 5, 10, 12, 17, and 19.  From Cycle 3 onwards, patients may take study drug at home on all dosing 
days except for Day 1 of each cycle.  Dosing on 2 consecutive days will not be allowed; there will be at least 1 day between 2 doses. 
j. 
For PK sampling, there may be a time window of within 30 minutes before the study drug administration for the pre-dose time point, ±5 minutes for 
time points up to 4 hours of study drug administration, ±15 minutes for time points after 4 hours of study drug administration, and ±60 minutes for 
the time points on return visits. 
k. Tumor assessment will be performed within 0-7 calendar days before the start of every odd-numbered cycle, starting from 0-7 calendar days before 
the start of Cycle 3. 
l. 
Every 6 weeks. 
m. Samples for pharmacogenomic, pharmacoproteomic and cellular analysis will be collected if the patient provides additional consent.