Clinical trial NCT04089735
Conditions: Ocular Inflammation and Pain After Cataract Surgery
Sponsor: Formosa Pharmaceuticals, Inc.
Start date: 2019-08-13; Completion date: 2020-04-07
Enrollment: 165 participants; Allocation: RANDOMIZED; Intervention model: PARALLEL

Brief summary:
This is a Phase 2a, 2-part study (designated Parts A and B) that will evaluate APP13007 dose strength and dosing frequency in a randomized double-masked fashion for comparison to the respective matching vehicle placebo. Part A will be conducted first to evaluate 0.05% APP13007 and matching vehicle placebo in an approximate 1:1 ratio in approximately 42 subjects who experience postoperative inflammation on the first day following routine, uncomplicated, cataract surgery and who meet all eligibility criteria. Based on the results of Part A, Part B of the study may be open for enrollment to evaluate 0.05% and/or 0.1% APP13007 at various dosing frequency in approximately 84 subjects, also in an approximate 1:1 ratio, active vs. placebo. In each Part, subjects will return periodically for study assessments during the treatment period and then for a follow-up visit approximately 1 week after stopping the study drug.

Detailed description:

Arms: arm 1: 1 drop 0.05% APP13007 twice daily for 21 days to the operated eye arm 2: 1 drop matching vehicle placebo for 0.05% APP13007 twice daily for 21 days to the operated eye arm 3: 1 drop 0.05% APP13007 twice daily for 3 days followed by 1 drop once daily for 11 days to the operated eye arm 4: 1 drop matching vehicle placebo for 0.05% APP13007 twice daily for 3 days followed by 1 drop once daily for 11 days to the operated eye arm 5: 1 drop 0.1% APP13007 twice daily for 3 days followed by 1 drop once daily for 11 days to the operated eye arm 6: 1 drop matching vehicle placebo for 0.1% APP13007 twice daily for 3 days followed by 1 drop once daily for 11 days to the operated eye
Interventions: intervention 1: APP13007, 0.05% intervention 2: APP13007, 0.1% intervention 3: APP13007 Placebo, 0.05% intervention 4: APP13007 Placebo, 0.1%
Intervention descriptions: intervention 1: APP13007 eye drop, 0.05% intervention 2: APP13007 eye drop, 0.1% intervention 3: APP13007 placebo eyedrop, 0.05% intervention 4: APP13007 placebo eyedrop, 0.1%

Protocol documents:
Prot_000.pdf: 























































































































































































































SAP_001.pdf: 
 
Document Number: CPN-201.SAP 
Version: 1 
Document Title:  Clinical Study CPN-201 Statistical 
Analysis Plan 
Page 1 of 35 
 
 
STATISTICAL ANALYSIS PLAN 
 
VERSION: 
1 
 
EFFECTIVE DATE: April 30th, 2020 
 
 
VERSION HISTORY 
SAP 
Number 
Version 
Number 
Effective 
Date 
Revision (Principal change from previous 
version) 
001 
1 
 
Not applicable, new SAP 
 
 
 
 
 
APPROVALS 
 
Prepared 
 
Date: 
30APR2020 
Name: 
 
 
 
Title: 
Head, Biostatistics and Data Management 
 
 
 
 
 
 
Approved 
 
Date: 
4May2020 
Name: 
 
 
 
Title: 
Chief Medical Officer 
 
 
 
 
 
 
Approved 
 
Date: 
4May2020 
Name: 
 
 
 
Title: 
Chief Executive Officer 
 
 
 
 
 
 
 
 


 
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Table of Contents 
 
 
INTRODUCTION ...................................................................................................... 5 
1.1. 
Study Design................................................................................................................. 5 
1.1.1. 
Study Schematics ...................................................................................................... 6 
1.1.2. 
Treatment Description for Data Analyses................................................................. 6 
1.1.3. 
Schedule of Events .................................................................................................... 8 
1.2. 
Objectives and Endpoints ........................................................................................... 10 
 
STATISTICAL HYPOTHESES ............................................................................. 11 
 
SAMPLE SIZE DETERMINATION ..................................................................... 11 
 
POPULATIONS FOR ANALYSES........................................................................ 11 
4.1. 
Safety Population ........................................................................................................ 11 
4.2. 
Intent-to-Treat (ITT) Population ................................................................................ 11 
4.3. 
Per-Protocol Population .............................................................................................. 12 
 
STATISTICAL ANALYSES ................................................................................... 12 
5.1. 
General Consideration ................................................................................................ 12 
5.1.1. 
General statistical analyses ..................................................................................... 12 
5.1.2. 
Tables ...................................................................................................................... 12 
5.1.3. 
Subject Listings ....................................................................................................... 13 
5.1.4. 
Reporting Conventions ........................................................................................... 13 
5.1.5. 
Visits, Days, PODs, EOT and EOS ........................................................................ 13 
5.1.6. 
Missing Data and Data Handling Rules .................................................................. 14 
5.1.7. 
Baseline Definitions ................................................................................................ 16 
5.1.8. 
Change from Baseline ............................................................................................. 16 
5.1.9. 
Multiple Assessments at One Time Point ............................................................... 17 
5.1.10. 
Final Analyses ..................................................................................................... 17 
5.1.11. 
Deviations from SAP .......................................................................................... 17 
5.2. 
Subject Disposition ..................................................................................................... 17 


 
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5.3. 
Primary and Secondary Efficacy Endpoint(s) Analysis ............................................. 18 
5.3.1. 
Definition of Primary and Secondary Efficacy Endpoint(s) ................................... 18 
5.3.2. 
Main analytical approach ........................................................................................ 19 
5.3.3. 
Sensitivity analysis.................................................................................................. 21 
5.3.4. 
Supplementary analyses .......................................................................................... 21 
5.4. 
Secondary Efficacy Endpoint(s) Analysis .................................................................. 22 
5.5. 
Tertiary/Exploratory Endpoint(s) Analysis ................................................................ 22 
5.6. 
Safety Analyses .......................................................................................................... 22 
5.6.1. 
Extent of Exposure .................................................................................................. 23 
5.6.2. 
Adverse Events ....................................................................................................... 23 
5.6.3. 
Clinical chemistry and hematology parameters (Part A only) (Appendix B) .......... 24 
5.6.4. 
Serum Cortisol Concentration (Part A only) .......................................................... 24 
5.6.5. 
Intraocular Pressure ................................................................................................ 24 
5.6.6. 
Visual Acuity Worsening from Baseline ................................................................ 25 
5.6.7. 
Other Ocular Exams ................................................................................................ 25 
5.7. 
Other Analyses ........................................................................................................... 26 
5.8. 
Interim Analyses ......................................................................................................... 26 
 
SUPPORTING DOCUMENTATION .................................................................... 26 
6.1. 
Protocol deviation ....................................................................................................... 26 
6.2. 
Baseline characteristics and demographics ................................................................ 27 
6.3. 
Significant Medical/Surgical History ......................................................................... 27 
6.4. 
Prior/concomitant/follow-up medications (including dictionary) .............................. 27 
6.5. 
Changes to Protocol-Planned Analyses ...................................................................... 27 
6.6. 
Appendix A: ABBREVIATIONS .............................................................................. 28 
6.7. 
Appendix B:  Clinical Laboratory Parameters ........................................................... 29 
6.8. 
Appendix C:  Modifications for Part B according to Amendment 1 of the Protocol . 29 
 
REFERENCES ......................................................................................................... 30 
LIST OF TABLES ................................................................................................................ 31 


 
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FIGURES ............................................................................................................................... 34 
LISTINGS .............................................................................................................................. 34 
 
 
 
 


 
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INTRODUCTION 
The purpose of this statistical analysis plan (SAP) is to describe the data analyses and presentation 
to be performed following the completion of Study CPN-201 entitled  “A multicenter, randomized, 
double-masked, two-part, placebo-controlled, Phase 2A study to evaluate the safety, tolerability 
and preliminary efficacy of APP13007 for the treatment of inflammation and pain after cataract 
surgery”.  
Study endpoints and assessments, planned statistical methods, and derived variables are 
summarized in this plan. Planned tables, figures, and listings for data presentation are specified. 
All decisions regarding final analyses, as defined in this SAP document, are made prior to locking 
the study database. 
This is an exploratory Phase 2 study for the purpose of dosage regimen selection for Phase 3 
studies.  Further hypothesis-generating analyses may be performed outside those specified in this 
SAP. 
 
1.1. 
Study Design 
The study design is described by Figure A: Study Schematics in Section 1.1.1, Table B: Treatment 
Descriptions in Section 1.1.2, and Table C: Schedule of Events in Section 1.1.3.  
 
 


 
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1.1.1. Study Schematics 
Figure A 
 
 
 
1.1.2. Treatment Description for Data Analyses 
Three dosing regimens are evaluated, one in Part A and two in Part B. Each has its own 
matching vehicle placebo.  
Data display treatment descriptors with actual treatment dose will be used as shown in Table 
B below. 
 


 
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Table B:  Treatment Descriptions in Data Display 
Treatment ID 
Treatment Description 
Final Data Display 
Treatment Description 
Treatment 1: 
APP13007 0.05% one drop in the study 
eye BID for 21 days 
APP13007 0.05% BID 
Treatment 2: 
PLACEBO 0.05% one drop in the study 
eye BID for 21 days 
PLACEBO 0.05% BID 
Treatment 3: 
APP13007 0.05% one drop in the 
study eye BID for 3 days followed 
by QD for 11 days 
APP13007 0.05% BID/QD 
Treatment 4: 
PLACEBO 0.05% one drop in the study 
eye BID for 3 days followed by QD 
for 11 days 
PLACEBO 0.05% BID/QD 
Treatment 5: 
APP13007 0.1% one drop in the study 
eye BID for 3 days followed by QD 
for 11 days 
APP13007 0.1% BID/QD 
Treatment 6: 
PLACEBO 0.1% one drop in the 
study eye BID for 3 days followed 
by QD for 11 days 
PLACEBO 0.1% BID/QD 
 
 
 


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1.1.3. Schedule of Events 
Table C1: Schedule of Events for Part A 
 
 
 
 


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Table C2: Schedule of Events for Part B 
 
 
 
 


 
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1.2. 
Objectives and Endpoints 
Table A. 
Objectives and Endpoints 
OBJECTIVE 
ENDPOINT 
The primary safety objective of this 
study is to investigate the safety and 
tolerability of APP13007 versus 
corresponding matching vehicle 
placebo for the treatment of 
inflammation and pain through post-
operative day (POD) 22 in Part A and 
POD15 in Part B after cataract surgery. 
• Early Treatment Diabetic Retinopathy Study 
(ETDRS) Best corrected Visual Acuity by 
pinhole method 
• Slit-lamp biomicroscopy 
o Corneal edema 
o Ciliary flush 
o Bulbar conjunctival injection 
• Dilated indirect ophthalmoscopy 
o Vitreous 
o Retina  
o Macula 
o Choroid 
o Optic nerve 
• Intraocular pressure (IOP) 
• Adverse event (AE) monitoring 
• Clinical chemistry and hematology parameters 
(Part A only) (Appendix B) 
• Serum cortisol concentrations (Part A only) 
The primary efficacy objective of this 
study is to investigate the preliminary 
efficacy of APP13007 versus matching 
vehicle placebo for the treatment of 
inflammation and pain through POD15 
after cataract surgery. 
• Anterior chamber cell (ACC) count at POD15 
• Pain grade at POD15 
The secondary efficacy objective of 
this study is to compare the effects on 
markers of inflammation and pain and 
visual acuity between active 
APP13007 and matching vehicle 
placebo for each dose strength and 
frequency. 
• ACC count at PODs 4, 8 (and 22 for Part A). 
• Pain grade at PODs 4, 8 (and 22 for Part A) 
• Anterior chamber flare (ACF) grade at PODs 4, 
8, 15 (and 22 for Part A) 
• ACC grade at PODs 4, 8, 15 (and 22 for Part A). 
• Use of rescue medication and/or early 
withdrawal 


 
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OBJECTIVE 
ENDPOINT 
• Proportion of subjects with ACC count = 0 at 
PODs 4, 8, 15 (and 22 for Part A) without 
receiving rescue medication; 
• Proportion of subjects who are pain-free at 
PODs 4, 8, 15 (and 22 for Part A) without 
receiving rescue medication; 
• Proportion of subjects with ACF grade = 0 at 
PODs 4, 8, 15 (and 22 for Part A) without 
receiving rescue medication 
• Visual acuity (ETDRS Best corrected Visual 
Acuity by pinhole method) at PODs 4, 8, 15 (and 
22 for Part A) without receiving rescue 
medication 
 
Statistical Hypotheses 
No formal hypotheses are being tested. 
All reported p-values will be considered exploratory and hypothesis-generating. 
 
Sample Size Determination 
This is a Phase 2a trial to explore dosing regimens, and no formal sample size calculations 
were used to determine the number of subjects to be enrolled into the trial. The sample size of 
21 subjects in each study drug group was selected based on feasibility. Assuming a dropout 
rate of 5%, approximately 21 subjects will be enrolled into each treatment arm so that 
approximately 20 subjects per treatment arm would be expected to complete study drug dosing 
through the POD15 visit. 
 
Populations for Analyses 
The analysis populations are defined as safety population, intent-to-treat population, and per-protocol 
population.  
4.1. 
Safety Population 
Safety population consists of all subjects who received at least one dose of study drug. 
4.2. 
Intent-to-Treat (ITT) Population 
ITT population consists of all subjects who are randomized to a study drug.   


 
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4.3. 
Per-Protocol Population  
Per-Protocol population consists of all randomized subjects who complete the study drug 
dosing through POD14 and do not have any major protocol deviations as defined in Section 
6.1 that could affect the efficacy endpoints.  
 
Statistical Analyses 
5.1. 
General Consideration 
Data analyses will be performed by the Sponsor designee, PharStat, Inc. Raleigh, NC. 
Tables and listings will be prepared in accordance with the current International Conference 
on Harmonization (ICH) Guidelines (ICH E3 Structure and Content of Clinical Study Reports 
1996).   
Version 9.3 or higher of the SAS system will be used to analyze the data and to generate tables, 
figures, and listings. All SAS programs prepared to analyze the data will be properly annotated.  
The format of this Statistical Analysis Plan follows the TransCelerate BioPharma’s template, 
version Nov. 2018. 
5.1.1. General Statistical Analyses 
All analyses will be done by each study part separately.  
All analysis variables will be summarized descriptively by each active dosing regimen and its 
matching vehicle placebo group.  
Continuous measures will be summarized as mean and standard deviation. In most cases, 95% 
confidence interval, median, and range will also be provided. Inferential statistics for 
continuous primary and secondary efficacy endpoints will include Student t-test and/or Mann-
Whitney test.  
Categorical measures will be summarized by counts. Due to the small sample size, the 
percentage of subjects will be provided only when it is necessary. The total number for the 
relevant population and the number of subjects who are still on study drug will be listed with 
the counts of categorical measures. “On study drug” will be defined in Section 5.1.6.2. 
5.1.2. Tables 
All tables are organized by study part and by treatment, i.e., two treatments on each table: the 
active dosing regimen and its matching vehicle placebo. All statistical comparisons are 
between the active dosing regimen vs. its matching vehicle placebo in each study part 
separately.  


 
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5.1.3. Subject Listings 
All supporting data to tables and figures will be sorted and listed by study part, treatment, 
investigator, subject, and visit.  
5.1.4. Reporting Conventions 
The information and explanatory notes in the “footer” or bottom of each table and listing will 
include the following information: 
 
Date of output generation 
 
SAS® program name, including the path where the program is stored 
 
Any other output specific details that require further elaboration 
 
In general, tables will be formatted with a column displaying findings for all 
subjects by treatment group in each study part.   
 
For summary tables, the following reporting convention in Table D will be used 
unless otherwise stated:  
Table D Decimal Places 
Categorical measures 
Counts (n) 
None 
Percentages (%) 
1 decimal place 
Continuous measures 
n 
None 
Mean 
i+1 decimal places 
Median 
i+1 decimal places 
SD 
i+1 decimal places 
Min 
i decimal places 
Max 
i decimal places 
Derived variables 
p-values 
if <0.001: presented as 
<0.001 
if > 0.001: presented to 3 
decimal places 
i refers to the number of decimal places reported in the CRF or other appropriate source data 
for the original data. 
5.1.5. Visits, Days, PODs, EOT and EOS 
5.1.5.1. Definition of Visits 
Based on Tables C1 and C2, Schedule of Events, visits and their nominal days relative to the 
surgery are further described in Table E below.  


 
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Table E: Visits 
Visit 
Number 
Visit Name  
Notes 
10 
Screening  
Days -28 to -1. Baseline for most safety 
endpoints 
20 
Surgery  
Day 0 
30 
POD1 
Randomization and start of study drug. 
Baseline for efficacy endpoints 
40 
POD4 
3-5 days post-surgery 
50 
POD8 
7-9 days post-surgery 
60 
POD15 (Part A only) 
14 -16 days post-surgery for Part A  
70 
End of Treatment (EOT) 
POD22 (Part A) 
POD15 (Part B) 
1-2 days post last dose of study drug. 
Part A: 22-23 days post-surgery  
Part B: 15-16 days post-surgery 
80 
End of Study (EOS) 
POD28 (Part A) 
POD22 (Part B) 
6-8 days post the last dose of study drug.  
Part A: 27-29 days post-surgery.  
Part B: 20-22 days post-surgery 
90 
UNSCHEDULED 
 
Some subjects may discontinue their study drug before the scheduled EOT.  Situations like 
this will be discussed in the following sections.  
5.1.5.2 Visit windows  
It is expected that all visits should occur according to the protocol schedule described in Table 
C1 and Table C2, the Schedule of Events; visits outside of this schedule will be addressed as 
protocol deviations. A new visit variable, AVISIT, will be defined based on the visit date 
relative to the date of surgery and a windowing strategy described by Table F. AVISIT will 
only be used for efficacy variables.  
Table F: Windowing Strategy: 
5.1.6. Missing Data and Data Handling Rules 
Incomplete dates, missing visits, missing assessments, and situations for subjects who 
prematurely discontinued study drug are discussed in the following.  
Surgery 
Day 1 post-
surgery 
2-6 days 
post-
surgery 
7-11 
days 
post-
surgery 
12-18 days 
post-
surgery 
19-25 days 
post-
surgery 
26+ days 
post-
surgery 
Day 0 
POD1 
POD4 
POD8 
POD15 
POD22 
POD28 


 
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5.1.6.1. Incomplete dates for birthday, adverse events, and medications 
The day of birth date will not be collected in order to protect subjects’ privacy. To calculate 
the age from birth date, the day will be assigned as 1st of the birth month.  
For analysis of AEs and medications, a complete date should be established in order to 
determine whether AEs or medications as occurring during study drug or not. For the 
purpose of handling partially reported start and stop dates for AEs or medications, the 
following algorithm will be applied: 
Missing start 
day, but month 
and year 
present 
If study drug had been taken in the same month and year as the 
occurrence of the AE/medication, then the start day of the 
event/medication will be assigned to the day of first dose of study 
drug.   
Otherwise the start day will be set to the first day of the month. 
Missing start 
day and month, 
but year 
present 
If study drug had been taken in the same year as the occurrence of the 
AE/medication, then the start date of the event/medication will be 
assigned to the date of first dose of study drug. 
Otherwise the start day and month will be set to 01 January. 
Missing end 
day and month, 
but year 
present 
The end day and month will be set to the date of end of study. 
However, if trial termination year is greater than the year of the 
event/medication, then the day and month will be set to 31December. 
In subject data listings, start and stop date of AEs or medication will 
be displayed as reported. 
5.1.6.2. Missing data, rescue, last observation carried forward (LOCF) 
For efficacy analyses of POD1 to POD22 (Part A) and POD1 to POD15 (Part B), LOCF 
method will be used to impute all assessment values after the subjects that are rescued or 
early withdrawn and/or have stopped being on study drug to calculate mean, standard 
deviation, 95% confidence interval, median, Q1, Q3, minimum, and maximum for anterior 
chamber cell (ACC) count, ACC grade, ACF grade, pain grade, and Best Corrected Visual 
Acuity.  
LOCF 
The last on study drug assessment is carried forward to impute 
a later missing value. 
On study drug 
Subjects are considered as being on study drug when they start 
their first dose until two days after their last dose of the study 
drug.  


 
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Therefore, if a subject has an observed POD1 value for an efficacy endpoint, this subject should 
have observed or imputed values for the same endpoint through POD1 – POD22 for Part A or 
POD1-POD15 for Part B. Consequently, to avoid confusion, “POD22” will be used in place 
of “EOT” for Part A and “POD15” for Part B. 
LOCF will not be used for “complete” subjects even if they have stopped study drug earlier 
than POD21 (Part A) and POD14 (Part B). 
LOCF will not be used for safety analyses. For safety endpoints, the total number of subjects 
in the safety population and the summary statistics of all observed data will be calculated for 
each active dosing regimen and its matching vehicle placebo group for each visit.  
Since the eye exams at Screening and EOS visits are evaluated as safety endpoints, the last 
non-missing observation from the study-eye at post-surgery PODs will not be carried forward 
to impute missing values at EOS visit for the Screening vs. EOS analyses. 
To be clear, in any cases, EOS measurements will not be modified and Screening 
measurements will not be carried forward.  
5.1.7. Baseline Definitions 
Baseline is the last observed (without LOCF or any other imputation) assessment prior to the 
time when the first dose of study drug is administered unless otherwise specified.   
Screening will be considered as the baseline for the Screening vs EOS comparison. EOS data 
will be summarized with Screening data in the same table.  
For all other PODs, i.e., POD 1, 4, 8, 15, and 22 (for Part A), POD1, 4, 8, and 15 (for Part B), 
ocular examinations are carried out for the study eye only. These data will be summarized in 
the same table for each active dosing regimen and its matching vehicle placebo. The Pre-dose 
assessment at POD1 will be the baseline.  
5.1.8. Change from Baseline 
Change from baseline 
(CFB) is calculated as: 
CFB value = ‘visit’ value – baseline value 
CFB will be indicated as missing, if either the baseline or post-
dose value is missing.  


 
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5.1.9. Multiple Assessments at One Time Point 
Where multiple assessments are recorded for a particular POD, e.g., ACC counts at the same 
visit or when two visits fall in the same POD window (Table F), the mean of the assessments 
will be calculated and used in any derivation of summary statistics.  All observed data will be 
listed. 
Note, for any efficacy endpoint: 
If EOS falls in the same POD window (Table F) with another visit, the assessment obtained 
from that other visit, while the subject is still on study drug, will be used for the efficacy 
analyses.  
If there are no other assessments in the same POD window (Table F) while the subject is still 
on study drug, e.g., within 2 days of the last dose of study drug as defined in 5.1.6.2, the EOS 
assessment will be used for efficacy analyses.  
For any safety categorial endpoint, instead of taking the mean of all assessments within that 
POD window, the assessment obtained at the latest visit within that POD window will be used 
for that POD.  
EOS assessments will not be modified in any case as mentioned in 5.1.6.2. 
5.1.10. Final Analyses 
The final analysis will be performed when the last randomized subject has completed the final 
study visit, the data are monitored and cleaned, and the database has been locked and 
authorized for analysis.  A data review meeting will precede database lock. 
5.1.11. Deviations from SAP 
Any deviations from the SAP will be documented and a justification given in the clinical 
study report. 
5.2. 
Subject Disposition 
Total number of subjects screened, proportion of screen failures, proportion of randomization 
failures will be presented in Table 14.1.1.  
The total number of subjects for each active dosing regimen and its matching vehicle placebo 
in each study population, namely, safety, ITT, and per protocol, will also be presented in Table 
14.1.1.  


 
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Furthermore, the number and proportion of subjects who discontinued study drug prior to 
POD21 for Part A and POD14 for Part B will be summarized for each treatment according to 
reason of discontinuation in Table 14.1.1.   
All subjects’ disposition status information, reasons of screen/randomization failure and 
discontinuation of study drug prior to POD21 will be presented in Listing 16.2.1.  
5.3. 
Primary and Secondary Efficacy Endpoint(s) Analysis 
5.3.1. Definition of Primary and Secondary Efficacy Endpoint(s) 
The primary efficacy endpoints are: 
• Anterior chamber cell (ACC) count at POD15 (mean of two counts) 
• Pain grade at POD15 
Pain grade is defined as follows:  
0 
None: Absence of pain. 
1 
Minimal: Presence of minimal throbbing or aching pain (expected 
following cataract surgery) 
2 
Mild: Presence of mild throbbing or aching pain, easily tolerated. 
3 
Moderate: Presence of moderate throbbing or aching pain leading to the 
desire to use an analgesic. 
4 
Moderate: Presence of moderate throbbing or aching pain leading to the 
desire to use an analgesic. 
The secondary efficacy endpoints are:  
• Anterior chamber cell (ACC) count at PODs 4, 8 (and 22 for Part A), mean of two counts 
at each POD. 
• ACC grade at PODs 4, 8, 15 (and 22 for Part A), mean of two grades at each POD 
ACC grade is defined as the following:  
0 
1 
2 
3 
4 
0 cell 
1-5 cells 
6-15 cells 
16-30 cells 
> 30 
• Pain grade at PODs 4, 8 (and 22 for Part A) 
• ACF grade at PODs 4, 8, 15 (and 22 for Part A) 
ACF grade is defined as follows: 
ACF Grade 
ACF Grade Used 
for Analyses 
Description 


 
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0 
0 
None 
1+ 
1 
Faint 
2+ 
2 
Moderate (iris and lens details clear) 
3+ 
3 
Marked (iris and lens details hazy) 
4+ 
4 
Intense (fibrin or plastic aqueous) 
• Use of rescue medication and/or early withdrawal 
• Proportion of subjects with ACC count = 0 (first measurement only) at PODs 4, 8, 15 (and 
22 for Part A) without receiving rescue medication; 
• Proportion of subjects who are pain-free at PODs 4, 8, 15 (and 22 for Part A) without 
receiving rescue medication; 
• Proportion of subjects with ACF grade = 0 at PODs 4, 8, 15 (and 22 for Part A) without 
receiving rescue medication 
• Visual acuity (ETDRS Best corrected Visual Acuity by pinhole method) at PODs 4, 8, 15 
(and 22 for Part A) without receiving rescue medication 
See Section 7.2 of the protocol for more details about the definitions of the endpoints.   
5.3.2. Main analytical approach  
The primary and secondary efficacy endpoints analyses will follow the General Consideration 
described in Section 5.1. 
Efficacy analysis will be performed for the ITT population.  
The primary efficacy endpoints and the secondary endpoints will be summarized in the same 
tables, figures, and listings.  
Rescue and Early Withdrawal: 
Rescue criteria: After randomization 
 anterior chamber cells count >30 cells,  
 an increase in anterior chamber cell count by > 15 cells from POD1/pre-dose baseline, or 
 ≥2 grade of increase in ACF from POD1/pre-dose baseline. 
For visits POD4 through EOS, the total number of subjects randomized, number of subjects 
who are still on study drug, and the number of subjects rescued for each reason and for subjects 
withdrawn early will be summarized in Table 14.2.1 for each active dosing regimen and its 
matching vehicle placebo.  
The denominator of each proportion is the number of subjects being randomized.  


 
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Subjects’ rescue and early withdrawn information together with reasons will be presented in 
Listing 16.2.6.1.  
Inflammation and Best Corrected Visual Acuity (BCVA) endpoints:  
Both eyes at Screening and End of Study (EOS): 
Although the tables for these data are numbered 14.2.xxx and listed with other efficacy 
endpoint tables, the analyses of parameters for eye exams at Screening and EOS visits, 
especially, those for the non-study-eye, are primarily considered as safety analyses and, 
therefore, they will be carried out for the safety population.   
ACC count, ACC grade, pain grade, ACF grade, and ETDRS acuity (logMAR score) will be 
summarized descriptively for Screening and EOS visits for each active dosing regimen and its 
matching vehicle placebo. The summary statistics include total number of subjects in the safety 
population, mean, standard deviation, 95% confidence interval, median, Q1, Q3, minimum, 
and maximum of observed data. See Tables 14.2.2.1.1-14.2.2.1.4, and 14.2.5.1. 
The distribution of ACC, pain, and ACF grades for each treatment group will be described by 
the frequency within each grade for Screening and EOS visits. See the top part of Tables 
14.2.3.1 – 14.2.3.3.  
Last non-missing observation from the other PODs will not be carried forward to impute 
missing values at EOS visit as discussed in Section 5.1.6.2. 
Study eye only:  
ACC count, ACC grade, pain grade, ACF grade, and ETDRS acuity (logMAR score) will be 
summarized descriptively in Tables 14.2.2.2.1-14.2.2.2.4, and 14.2.5.2 for POD1-22 (Part A) 
and POD1-15 (Part B) by each active dosing regimen and its matching vehicle placebo. LOCF 
method will be used to impute all assessments after the subject stopped being on study drug to 
calculate mean, standard deviation, 95% confidence interval, median, Q1, Q3, minimum, and 
maximum. On study drug is defined in Section 5.1.6.2.  
The observed data (without LOCF or any other imputation) when the subjects are still on study 
drug of above listed endpoints will also be summarized in Tables 14.2.2.3.1 – 14.2.2.3.4, and 
14.2.5.3. The treatment difference will also be reflected by the number of subjects that are still 
on study drug. PODs defined by Table F will be used for calculating the treatment group 
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The same statistics will be calculated for CFB of the same endpoints, with POD1 pre-dose 
measure as the baseline, in Tables 14.2.2.4.1-14.2.2.4.4, and 14.2.5.4. LOCF will be used to 
impute missing values and all assessments after subjects stopped being on study drug. 
Statistical comparison between each active dosing regimen and its matching vehicle placebo 
will be carried out for each visit during POD4 - POD22 (Part A) and POD4 – POD15 (Part B) 
using t-test and Mann-Whitney non-parametric tests noting that p-values will only be 
considered descriptive and hypothesis-generating.  
These endpoints will also be visually presented in Figures 14.2.2.1, 14.2.2.2, 14.2.5.1, and 
14.2.5.2. 
The distribution of ACC, pain, and ACF over different grades for each treatment will be 
described by the frequency and proportion of subjects within each grade for each active dosing 
regimen and its matching vehicle placebo for each visit during POD4 – POD22 (Part A) and 
POD4 –POD15 (Part B) in the lower part of Tables 14.2.3.1 – 14.2.3.3 without LOCF. The 
denominator of the proportion is the number of subjects randomized. The treatment difference 
will also be reflected by the number of subjects that are still on study drug.  
The proportion of subjects with ACC count = 0, pain-free, or ACF grade = 0 without receiving 
rescue medication will be calculated for each active dosing regimen and its matching vehicle 
placebo for each visit during POD1 – POD22 (Part A) and POD1 – POD15 (Part B) in Table 
14.2.4 and Figure 14.2.4. The denominator will be the total number of subjects randomized in 
each treatment group. LOCF will be used to impute missing values and all assessments after 
subjects stopped being on study drug.  
All subjects’ inflammation and pain information will be presented in Listing 16.2.6.2 and best 
correct visual acuity information will be presented in Listing 16.2.6.3.  
5.3.3. Sensitivity analysis 
A subset of efficacy analyses will be examined for the per-protocol population as supportive 
analysis.   
Since only first ACC count will be used to define the proportion of subject with ACC count = 
0, analyses of using second ACC count and/or a combination of the two counts will be 
explored.  
5.3.4. Supplementary Analyses 
Exploratory hypothesis-generating analyses may be performed as necessary.  


 
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5.3.4.1. Proportions of “Resolved”, “Unresolved”, and “Early discontinuation of study drug” 
To understand the persistence of grade=0 for ACC (the first of two counts), ACF, and pain, 
proportions of “resolved”, “unresolved”, and “early discontinuation of study drug” will be 
calculated for each active dosing regimen and its matching vehicle placebo group at each POD 
in Table 14.2.4 and Figure 14.2.4 
 A subject is defined as “Early discontinuation of study drug” at a POD when the subject is not 
on study drug at that POD. See Section 5.1.6.2 for the definition of on study drug.  
A subject’s ACC (ACF, or pain) is defined as “Resolved” at a POD when the first of the two 
ACC counts (grade for ACF and pain) at that POD and all PODs beyond is equal to zero while 
the subject is still on study drug for all these PODs. Otherwise, the subject is “Unresolved”. 
For example, if a subject’s first of two ACC counts equal to zero at POD8 but the first of two 
ACC counts >0 at POD15, this subject would be “Unresolved” at both POD8 and POD15.  
If a subject’s first of two ACC counts >0 at POD22 (Part A) or at POD15 (Part B) this subject 
cannot be “Resolved” at any POD.  
Apparently, a subject having an “Early discontinuation of study drug” can never be “Resolved” 
at any PODs. More precisely, this subject will be an “Early discontinuation of study drug” for 
all PODs beyond.  
5.4. 
Secondary Efficacy Endpoint(s) Analysis 
Secondary efficacy endpoints will be analyzed with the primary efficacy endpoints together. 
See Section 5.3 for details.  
5.5. 
Tertiary/Exploratory Endpoint(s) Analysis 
None are planned.  
5.6. 
Safety Analyses 
Safety data will be analyzed for all subjects in the Safety population.  
The safety endpoints are:  
• Adverse event (AE)  
• Serum cortisol concentrations (Part A) 
• Clinical chemistry and hematology parameters (Part A) (Appendix B) 
• Intraocular pressure (IOP) 
• Early Treatment Diabetic Retinopathy Study (ETDRS)  


 
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• Best corrected Visual Acuity by pinhole method 
• Slit-lamp biomicroscopy 
o Corneal edema 
o Ciliary flush 
o Bulbar conjunctival injection 
• Dilated indirect ophthalmoscopy 
o Vitreous 
o Retina  
o Macula 
o Choroid 
o Optic nerve 
See Section 7.2 of the protocol for detail about these endpoints.  The analyses of these safety 
endpoints are described in the following sub-sections.  
For all safety endpoints, the total number of subjects in the safety population and the 
summary statistics of all observed data will be calculated for each active dosing regimen and 
its matching vehicle placebo group and each visit as defined in the CRF. No imputation will 
be used.  
5.6.1.  Extent of Exposure  
The summary statistics of number of eye drops that were instilled correctly in the study eye 
during the entire treatment course will be calculated for each active dosing regimen and its 
matching vehicle placebo group and will be presented in Table 14.1.4. Individual dosing diary 
will be presented in Listing 16.2.5.1  
5.6.2. Adverse Events 
Adverse events (AE) will be summarized by total number of any AE, number of subjects 
reporting the AE, ocular AEs (affecting the study eye and/or the non-study eye and/or both 
eyes, when appropriate), systemic AEs, SAEs, discontinuing study drug due to an AE, by 
relationship to study drug only, to ocular surgery only, or to both drug and ocular surgery, and 
by severity. Adverse events will be coded using Medical Dictionary for Regulatory Activities 
(MedDRA version 22.0 – March 2019) and categorized by system organ class and preferred 
terms.  
See Tables 14.3.1.1 – 14.3.1.3 for details.  
All adverse events will be listed by both reported term and MedDRA term in Listing 16.2.7.1 
and 16.2.7.2.  


 
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All serious and significant adverse events will be listed in Table 14.3.2 and narrated in Table 
14.3.3.  
5.6.3. Clinical Chemistry and Hematology Parameters (Part A only) (Appendix B)  
All laboratory data will be summarized by mean, SD, 95% CI, median, Q1, Q3, minimum, and 
maximum for POD1 Pre-Dose, EOT, and EOS for the active and its matching vehicle placebo 
groups in Part A in Table 14.3.5.1. The frequencies of shift from Low, Normal, or High, 
defined by Covance Laboratory Services, to other categories will also be summarized for both 
treatments in Table 14.3.5.2. All values will be listed for all subjects in the safety population 
by treatment for POD1 Pre-Dose, EOT and EOS in Listing 16.2.8.1 and 16.2.8.2. All values 
that are out of normal range will be listed in Table 14.3.4 and Listing 16.2.8.3.  
5.6.4. Serum Cortisol Concentration (Part A only)  
Serum cortisol concentration will be summarized in the same way as other laboratory tests 
for POD1 pre-randomization and EOT for the active and its matching vehicle placebo groups 
in Part A in Table 14.3.5.3 and 14.3.5.4 and visually presented in Figures 14.3.5.1.  
All serum cortisol values and their flags (H or L) defined by Covance Laboratory Services 
will be listed for all subjects by treatment for Screening and EOT visits in Listing 16.2.8.4. 
All individual subject values will be visually presented in Figure 14.3.5.2.  
5.6.5. Intraocular Pressure  
For both eyes at Screening and EOS, the analyses of IOP will follow the methods discussed 
in Section 5.3.2 for Both eyes at Screening and End of Study (EOS). The results will be 
presented in the top part of Table 14.3.6.1 and Table 14.3.6.2. 
For study eye at POD1-POD22 (Part A) and POD1-POD15 (Part B), the analyses will follow 
the methods discussed in Section 5.3.2 for “observed data” under Study eye only. The results 
will be presented in Table 14.3.6.1 and its CFB (Baseline = Screening for EOS; Baseline = 
POD1/pre-dose for other PODs) for the active and its matching vehicle placebo groups at 
each visit in Table 14.3.6.2 and visually presented in Figure 14.3.6.1.  
The distribution of IOP over the ranges of <10, 10-15, 16-20, 21-25, 25-30, and >30(mmHg) 
for each treatment will be described by the frequencies in each range in Table 14.3.6.3. The 
distribution of IOP increase over ranges of ≤0, 1-5, 6-10, 11-15, >15 (mmHg) for the active 
and its matching vehicle placebo groups will be described by the frequency in each range at 
each visit in Table 14.3.6.4.  


 
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The number of subjects with an IOP ≥ 24 mmHg AND an increase ≥ 10 mmHg from pre-dose 
baseline, and the number of subjects with an IOP > 30 mmHg at any time following initiation 
of study drug will be summarized in the adverse event tables, Table 14.3.1.1- 14.3.1.3.  
All subjects’ intraocular pressure information will be presented in Listing 16.2.9.2 and visually 
presented in Figure 14.3.6.2.  
5.6.6. Visual Acuity Worsening from Baseline  
The number of subjects with a visual acuity worsening of ≥ 2 lines (equivalent to ≥ 0.2 
logMAR CFB), together with ≤ 0 (≤ 0 logMAR CFB) and in between (0< logMAR CFB < 
0.2) at EOS from Screening and at each visit during POD4-POD22 (Part A) and POD4-
POD15 (Part B) from POD1/pre-dose visit will be summarized in Table 14.3.6.5. 
As mentioned earlier, all subjects’ best correct visual acuity information will be presented in 
Listing 16.2.6.3.  
5.6.7. Other Ocular Exams 
Ocular symptoms, comfort grading assessment, ophthalmoscopy findings, and slit lamp 
biomicroscopy results will be categorically summarized. 
Slit Lamp Biomicroscopy:  
The distribution of Bulbar Conjunctival Injection, Sclera – Ciliary Flush, and Corneal Edema 
over the following categories: Absent, Mild, Moderate, and Severe for each treatment at each 
visit will be described by the frequencies in each category for each active dosing regimen and 
its matching vehicle placebo in Tables 14.3.6.6 -14.3.6.8.  
All subjects’ slit lamp biomicroscopy information will be presented in Listing 16.2.9.3. 
Inflammation Endpoints: 
As mentioned in Section 5.3.2, although the Tables 14.2.2.1, 14.2.3.1, 14.2.3.2, 14.2.3.3, and 
14.2.5.1 are listed with other efficacy endpoint tables, the analyses of the eye exam 
parameters at the Screening and EOS visits, especially those for the non-study-eye, are 
primarily considered as safety analyses. 
Dilated Indirect Ophthalmoscopy:  


 
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The number of subjects with abnormal vitreous retina, macula, choroid, and optic nerve for 
each active dosing regimen and its matching vehicle placebo will be summarized for 
Screening and EOS visits in Table 14.3.6.9 – 14.3.6.13. 
Cup-to-Disc Ratio will be summarized by mean, SD, 95% CI, median, Q1, Q3, minimum, 
and maximum for each active dosing regimen and its matching vehicle placebo at Screening 
and EOS visits in Table 14.3.6.14.  
Lens pathology findings for non-study eye will be summarized in Table 14.3.6.15 for subjects 
with phakic lens by the total number of subjects with abnormal findings and by abnormal 
cataract category: Nuclear, Cortical, and Posterior Subcapsular and by cataract Grade 1, 2, and 
3 for each active dosing regimen and its matching vehicle placebo at Screening and EOS visits.   
All dilated indirect ophthalmoscopy ocular exam data will be presented in Listing 16.2.9.4.   
5.7. 
Other Analyses 
None are planned.  
5.8. 
Interim Analyses 
No formal interim analyses are planned for this study.   
Masked and, if warranted, unmasked data reviews to determine the progression from Part A to 
Part B are described in Section 3.3 of the protocol.  
 
Supporting Documentation 
6.1. 
Inclusion/Exclusion criteria 
 Inclusion and Exclusion criteria and comments will be presented in Listing 16.2.2.1 -16.2.2.3. 
6.2. 
Protocol deviation  
Major Protocol Deviations are defined as those deviations that have the potential to  
(i) 
affect subject rights, safety and/or wellbeing, and/or  
(ii) 
affect study objectives and/or endpoints assessments.   
Only major protocol deviations are documented in the clinical study report (CSR). 
All details of protocol deviation cases will be presented in Listing 16.2.2.4.  


 
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There will be a separate class of major protocol deviations due to the COVID-19 pandemic in 
the clinical study report. 
Subjects excluded from Per Protocol population will be presented in Listing 16.2.3. 
6.3. 
Baseline characteristics and demographics 
The Safety population will be the primary population for the summary of demographics. 
Demographics, including mean and standard deviation of age, count and proportion of 
gender, race, ethnicity, iris color, study eye (OD or OS) will be summarized for each active 
dosing regimen and its matching vehicle placebo in Table 14.1.2. 
All subjects’ demographic characteristics will be presented in Listing 16.2.4.1.  
6.4. 
Significant Medical/Surgical History 
Significant medical, surgical, ocular medical, and ocular surgical history will be coded using 
Medical Dictionary for regulatory Activities (MedDRA version 22.0 – March 2019) and will 
be summarized by system organ class (SOC) and by preferred term (PT) by each active 
dosing regimen and its matching vehicle placebo for the safety population in Table 14.1.3. 
All medical/surgical history will be presented in Listing 16.2.4.2.  
6.5. 
Prior/concomitant/follow-up medications (including dictionary) 
Prior/concomitant/follow-up medications, including rescue medications, will be coded using 
World Health Organization (WHO) Drug Dictionary WHODRUG B3 GLOBAL (MARCH 
2019) and will be summarized by medication class and medication name presented for each 
active dosing regimen and its matching vehicle placebo group for the safety population in 
Table 14.1.5.  
All prior/concomitant/follow-up medications will be presented in Listing 16.2.9.1.  
6.6. 
Changes to Protocol-Planned Analyses 
• Added analyses for ACC grade at PODs 4, 8, 15 (and 22 for Part A).  
 
 


 
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6.7. 
Appendix A: ABBREVIATIONS 
Abbreviation 
Definition 
ACC 
Anterior Chamber Cell 
ACF 
Anterior Chamber Flare 
AE 
Adverse Event 
ALP  
Alkaline Phosphatase 
ALT  
Alanine Amino Transferase 
AST  
Aspartate Amino Transferase 
BCVA 
Best Corrected Visual Acuity  
BID 
Twice Daily 
BSL 
Baseline 
BUN 
Blood Urea Nitrogen 
CFB 
Change from Baseline 
CI 
Confidence Interval 
ConMed 
Concomitant Medications 
CSR 
Clinical Study Report 
EOT 
End of Treatment 
EOS 
End of Screening 
IOP 
Intraocular Pressure 
ITT 
Intent-to-Treat 
MCH 
Mean Corpuscular Hemoglobin 
MCHC 
Mean Corpuscular Hemoglobin Concentration 
MCV 
Mean Corpuscular Volume 
MedDRA 
Medical Dictionary for Regulatory Activities 
OD 
Right Eye 
OS 
Left Eye 
OU 
Both Eyes 
PP 
Per Protocol 
QD 
Once Daily 
RBC 
Red Blood Cell 
SAP 
Statistical Analysis Plan 
SD 
Standard Deviation 
WHO 
World Health Organization 
 
 
 


 
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6.8. 
Appendix B:  Clinical Laboratory Parameters 
Part A ONLY:  Clinical chemistry and hematology parameters.  Venous blood samples are 
drawn at POD1 (pre-dose of study drug), POD22 (or EOT visit) and at the EOS visit.  
If a subject is rescued prior to POD22, then the venous blood samples are drawn on the day 
that the subject discontinues the study drug (EOT), or as soon as possible after that. All 
rescued subjects have another blood draw at EOS visit.  
If a subject withdraws from study early (early termination), then the venous blood samples 
are drawn on the day that the subject discontinues the study drug (EOT). They do not have 
another blood test for EOS visit.  
Hematology 
Platelet Count           
RBC Indices: 
RBC Count 
MCV 
Absolute WBC Count (absolute) and automated differential 
WBC count 
MCH 
Hemoglobin 
MCHC 
Clinical Chemistry 
Glucose 
Chloride 
Sodium 
ALT (alanine 
amino transferase) 
Calcium 
BUN (blood urea 
nitrogen) 
Potassium 
AST (aspartate 
amino transferase) 
Albumin 
Creatinine 
CO2 
Bilirubin 
Total 
protein 
ALP (alkaline 
phosphatase) 
 
 
Serum cortisol concentration - Blood drawn at POD1/pre-Randomization and at POD22/EOT 
in Part A. 
6.9. 
Appendix C:  Modifications for Part B according to Amendment 1 of the Protocol 
Based on the Amendment 1 of the protocol, the treatment duration of the study in Part B is 
shortened by one week. Accordingly, EOT visit (VISITNUM = 70) is planned at POD15 and 
EOS visit (VISITNUM=80) is planned at POD22.  
 
 


 
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REFERENCES 
ICH E3 
 
SAP template:  
https://transceleratebiopharmainc.com/assets/clinical-content-reuse-assets/ 
 


 
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Tables 


 
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Figures 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Listings 
 


 
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