Clinical trial NCT04211831
Conditions: Overactive Bladder With Urge Urinary Incontinence
Keywords: URO-902 hMaxi-K pVAX/hSlo Overactive Bladder Urge Urinary Incontinence
Sponsor: Urovant Sciences GmbH
Start date: 2019-12-16; Completion date: 2022-07-20
Enrollment: 80 participants; Allocation: RANDOMIZED; Intervention model: SEQUENTIAL

Brief summary:
This study will evaluate the efficacy, safety, and tolerability of a single dose of URO-902 24 milligrams (mg) and 48 mg (administered via intradetrusor injection), compared with placebo, in participants with overactive bladder (OAB) and urge urinary incontinence (UUI) up to 48 weeks post-dose.

Detailed description:

Arms: arm 1: Participants will receive either a single treatment of URO-902 24 milligrams (mg) or matching placebo. arm 2: Participants will receive either a single treatment of URO-902 48 mg or matching placebo.
Interventions: intervention 1: URO-902 intervention 2: Placebo
Intervention descriptions: intervention 1: intradetrusor injection intervention 2: intradetrusor injection

Protocol documents:
Prot_000.pdf: 
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CLINICAL PROTOCOL 
Protocol Title: An Exploratory Phase 2a Study Evaluating the Efficacy and Safety of URO-902 
in Subjects with Overactive Bladder and Urge Urinary Incontinence 
 
Protocol Number: 
URO-902-2001 
Version Number: 
3.0 (Amendment 2.0)  
 
Replaces Version 2.0, dated 21 February 2020 
Product Development Phase: 
Phase 2a 
Product: 
URO-902, formerly known as hMaxi-K, pVAX/hSlo 
Sponsor: 
 
Urovant Sciences GmbH 
Viaduktstrasse 8 
4051 Basel, Switzerland 
Telephone +41 (42) 2155999 
United States Contact: 
Senior Director, Clinical Development 
5281 California Avenue, Suite 100 
Irvine, CA 92617 USA  
Telephone:
Ext 
Email:
 
IND Number: 
013208 
EudraCT Number: 
Not applicable 
Emergency Contact Telephone 
Number: 
Refer to Study Contacts Page 
Contact for Serious Adverse Events 
(SAEs):  
Email: PHV_Urovant@iqvia.com 
Phone: 1-866-599-1341/Fax: 1-866-599-1342 
Approval Date: 
19 February 2021 
Sponsor Signatory: 
Chief Medical Officer 
 
CONFIDENTIAL 
This document contains proprietary and confidential information of Urovant Sciences GmbH Acceptance of this document 
constitutes agreement by the recipient that no previously unpublished information contained herein will be published or 
disclosed without the prior written approval of Urovant Sciences, GmbH with the exception that this document may be 
disclosed to study personnel under your supervision who need to know the contents for conducting the study and appropriate 
Institutional Review Boards (IRBs)/Ethics Committees (EC) under the condition that the personnel have agreed to keep this 
information confidential. The foregoing shall not apply to disclosure required by governmental regulations or laws, however, 
Urovant Sciences, GmbH shall be promptly notified of any such disclosure.  
 


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TABLE OF CONTENTS 
CLINICAL PROTOCOL .................................................................................................................1 
SPONSOR SIGNATURE PAGE ....................................................................................................2 
INVESTIGATOR STATEMENT ...................................................................................................3 
PROTOCOL AMENDMENT 2 - SUMMARY OF CHANGES ....................................................4 
TABLE OF CONTENTS .................................................................................................................6 
LIST OF TABLES .........................................................................................................................10 
1. 
PROTOCOL SUMMARY ..........................................................................................11 
1.1. 
Synopsis ......................................................................................................................11 
1.2. 
Schematic of Study Design: 2-Cohort, Dose-Escalation ............................................18 
1.3. 
Schedule of Assessments ............................................................................................19 
2. 
INTRODUCTION ......................................................................................................22 
2.1. 
Background .................................................................................................................22 
2.2. 
Study Rationale ...........................................................................................................25 
2.3. 
Benefit/Risk Assessment ............................................................................................25 
2.3.1. 
Potential Benefits ........................................................................................................25 
2.3.2. 
Potential Risks ............................................................................................................26 
3. 
OBJECTIVES AND ENDPOINTS ............................................................................27 
3.1. 
Objectives ...................................................................................................................27 
3.2. 
Endpoints ....................................................................................................................27 
3.2.1. 
Exploratory Efficacy Endpoints .................................................................................27 
3.2.2. 
Safety Endpoints .........................................................................................................28 
3.2.3. 
Other Endpoints ..........................................................................................................28 
4. 
STUDY DESIGN .......................................................................................................29 
4.1. 
Overall Design ............................................................................................................29 
4.1.1. 
Clinical Hypothesis .....................................................................................................30 
4.2. 
Scientific Rationale for Study Design ........................................................................30 
4.3. 
Justification of Dose ...................................................................................................31 
4.4. 
End of Study Definition ..............................................................................................32 
5. 
STUDY POPULATION .............................................................................................33 
5.1. 
Inclusion Criteria ........................................................................................................33 
5.2. 
Exclusion Criteria .......................................................................................................33 


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5.3. 
Lifestyle Considerations .............................................................................................35 
5.4. 
Screen Failures ............................................................................................................36 
6. 
STUDY TREATMENTS............................................................................................37 
6.1. 
Study Drugs Administered .........................................................................................37 
6.2. 
Day of Treatment Criteria ...........................................................................................38 
6.3. 
Treatment Administration ...........................................................................................39 
6.4. 
Preparation/Handling/Storage/Accountability ............................................................40 
6.5. 
Measures to Minimize Bias: Randomization and Blinding ........................................40 
6.6. 
Study Drug Compliance .............................................................................................42 
6.7. 
Dose Modification ......................................................................................................42 
6.8. 
Drug After the End of the Study .................................................................................42 
6.9. 
Prior and Concomitant Therapy ..................................................................................42 
6.9.1. 
Prohibited Drugs and Washout ...................................................................................42 
6.9.2. 
Permitted Treatments ..................................................................................................43 
7. 
DISCONTINUATION OF STUDY AND SUBJECT DISCONTINUATION 
OR WITHDRAWAL ..................................................................................................44 
7.1. 
Subject Discontinuation/Withdrawal from the Study .................................................44 
7.2. 
Lost to Follow-Up.......................................................................................................45 
7.3. 
Early Study Termination .............................................................................................45 
8. 
STUDY ASSESSMENTS AND PROCEDURES ......................................................46 
8.1. 
Screening Procedures ..................................................................................................46 
8.1.1. 
Informed Consent .......................................................................................................46 
8.1.2. 
Assignment of Subject Number ..................................................................................47 
8.1.3. 
Medical History ..........................................................................................................47 
8.1.4. 
Demographics .............................................................................................................47 
8.1.5. 
Prior and Concomitant Medications ...........................................................................47 
8.2. 
Efficacy Assessments .................................................................................................47 
8.2.1. 
Bladder Diaries ...........................................................................................................48 
8.2.2. 
Patient-Reported Outcomes ........................................................................................49 
8.2.3. 
Urodynamic Parameters ..............................................................................................49 
8.3. 
Pharmacokinetic Assessments ....................................................................................50 
8.4. 
Safety Assessments .....................................................................................................51 
8.4.1. 
Vital Signs ..................................................................................................................51 


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8.4.2. 
Physical Examinations ................................................................................................51 
8.4.3. 
Electrocardiograms .....................................................................................................51 
8.4.4. 
Post-Void Residual Volume .......................................................................................51 
8.4.5. 
Clean Intermittent Catheterization ..............................................................................52 
8.4.6. 
Clinical Safety Laboratory Assessments ....................................................................53 
8.4.7. 
Bladder and Kidney Ultrasound .................................................................................54 
8.4.8. 
Immunogenicity Blood Sample (Optional) ................................................................55 
8.5. 
Adverse Events and Serious Adverse Events .............................................................55 
8.5.1. 
Time Period and Frequency for Collecting Adverse Event and Serious 
Adverse Event Information .........................................................................................55 
8.5.2. 
Method of Detecting Adverse Events and Serious Adverse Events ...........................56 
8.5.3. 
Follow-up of Adverse Events and Serious Adverse Events .......................................56 
8.5.4. 
Regulatory Reporting Requirements for Serious Adverse Events .............................56 
8.5.5. 
Disease-Related Events and/or Disease-Related Outcomes Not Qualifying as 
Adverse Events or Serious Adverse Events ................................................................57 
8.5.6. 
Adverse Events of Special Interest .............................................................................57 
8.5.7. 
Medication Errors .......................................................................................................58 
8.6. 
Treatment of Overdose ...............................................................................................58 
9. 
STATISTICAL CONSIDERATIONS .......................................................................59 
9.1. 
Statistical Hypotheses .................................................................................................59 
9.2. 
Sample Size Determination ........................................................................................59 
9.3. 
Analyses Populations ..................................................................................................60 
9.4. 
Analysis Endpoints .....................................................................................................60 
9.4.1. 
Efficacy, Health Outcome, and Urodynamics Endpoints ...........................................60 
9.4.2. 
Safety Endpoints .........................................................................................................61 
9.4.3. 
Other Endpoints ..........................................................................................................61 
9.5. 
Statistical Analyses .....................................................................................................61 
9.5.1. 
Statistical Methods for Efficacy Analysis ..................................................................61 
9.5.2. 
Safety Analyses ..........................................................................................................62 
9.6. 
Multiplicity .................................................................................................................62 
9.7. 
Subgroup Analyses .....................................................................................................63 
9.8. 
Planned Analyses ........................................................................................................63 
9.8.1. 
Interim Analyses .........................................................................................................63 


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9.8.2. 
Final Analyses ............................................................................................................63 
9.8.3. 
Data and Safety Monitoring Board .............................................................................63 
10. 
SUPPORTING DOCUMENTATION AND OPERATIONAL 
CONSIDERATIONS ..................................................................................................65 
10.1. 
Appendix 1: Regulatory, Ethical, and Study Oversight Considerations ....................65 
10.1.1. 
Regulatory and Ethical Considerations ......................................................................65 
10.1.2. 
Financial Disclosure ...................................................................................................65 
10.1.3. 
Informed Consent Process ..........................................................................................66 
10.1.4. 
Data Protection ...........................................................................................................66 
10.1.5. 
Committees Structure .................................................................................................66 
10.1.6. 
Posting Clinical Study Data ........................................................................................66 
10.1.7. 
Data Quality Assurance ..............................................................................................67 
10.1.8. 
Source Documents ......................................................................................................68 
10.1.9. 
Study and Site Closure................................................................................................69 
10.1.10. 
Publication Policy .......................................................................................................69 
10.1.11. 
Compliance with Protocol ..........................................................................................70 
10.2. 
Appendix 2: Clinical Laboratory Tests .......................................................................70 
10.3. 
Appendix 3: Adverse Events: Definitions and Procedures for Recording, 
Evaluating, Follow-up, and Reporting ........................................................................71 
10.4. 
Appendix 4: Abbreviations .........................................................................................76 
10.5. 
Appendix 5: Study Tabular Summary ........................................................................78 
10.6. 
Appendix 6: Contraceptive Guidance and Collection of Pregnancy 
Information .................................................................................................................79 
10.7. 
Appendix 7: Prohibited Medications and Non-Drug Therapies .................................80 
10.8. 
Appendix 8: Study Schedule Supplement ..................................................................81 
10.9. 
Appendix 9: Patient-reported Outcomes Questionnaires Descriptions and 
Instructions .................................................................................................................82 
10.9.1. 
Incontinence-Specific Quality-of-Life Instrument (I-QOL) .......................................82 
10.9.2. 
Overactive Bladder Questionnaire (OAB-q) ..............................................................88 
10.9.3. 
Patient Global Impression of Change Scale (PGI-C) .................................................92 
10.10. 
Appendix 10: Protocol Amendment 1 – Summary of Changes .................................94 
11. 
REFERENCES ...........................................................................................................98 
 


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LIST OF TABLES 
Table 1: 
Summary of Study Drugs ...........................................................................................38 
Table 2: 
Clinical Laboratory Tests ...........................................................................................71 
 


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1. 
PROTOCOL SUMMARY 
1.1. 
Synopsis 
Protocol Title: An Exploratory Phase 2a Study Evaluating the Efficacy and Safety of URO-902 
in Subjects with Overactive Bladder and Urge Urinary Incontinence 
Protocol Number: URO-902-2001 
Brief Title: URO-902 in Female Subjects with Overactive Bladder and Urge Urinary 
Incontinence 
Study Rationale: There is a significant unmet need for treatment of overactive bladder (OAB) 
with agents with novel mechanisms of action and a well-tolerated safety profile, in particular, for 
patients who have been inadequately managed by pharmacologic therapy. URO-902 
(pVAX/hSlo) is a GMP manufactured double-stranded deoxyribonucleic acid (DNA) plasmid 
(pVAX vector) containing a cDNA insert encoding the pore-forming α subunit of the human 
smooth muscle Maxi-K channel, hSlo. The Maxi-K channel is a prominent and well-studied 
potassium (K) channel subtype involved in smooth muscle relaxation. Because heightened 
smooth muscle tone may be a causative factor of OAB with detrusor overactivity (DO), 
increased numbers of Maxi-K channels in bladder smooth muscle cells associated with effective 
URO-902 treatment may improve this condition. Based on the well tolerated favorable safety 
profile demonstrated in the completed studies in subjects with OAB and erectile dysfunction 
(ED) and the preliminary signals of efficacy in the treatment of OAB observed in Study ION-03, 
the current exploratory study is designed to evaluate the efficacy and safety of a single treatment 
of URO-902 administered via intradetrusor injection in female subjects with OAB and urge 
urinary incontinence (UUI). 
Objectives and Endpoints: 
The study objectives are as follows: 
x To evaluate the efficacy of a single dose of URO-902 24 mg and 48 mg (administered via 
intradetrusor injection), compared with placebo, in subjects with OAB and UUI up to 
48 weeks post-dose 
x To evaluate the safety and tolerability of a single dose of URO-902 24 mg and 48 mg 
(administered via intradetrusor injection), compared with placebo, in subjects with OAB 
and UUI up to 48 weeks post-dose 
This exploratory study has no formal statistical primary endpoint hypothesis. Study endpoints are 
as follows: 


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Exploratory Efficacy Endpoints 
x Change from baseline at Week 12 in average daily number of UUI episodes 
x Change from baseline at Week 12 in average daily number of micturitions 
x Change from baseline at Week 12 in average daily number of urinary incontinence (UI) 
episodes 
x Change from baseline at Week 12 in the average daily number of urgency episodes 
x Proportion of subjects achieving ≥ 50%, ≥ 75%, and 100% reduction from baseline at 
Week 12 in UUI episodes per day 
x Change from baseline at Week 12 in average volume voided per micturition 
x Health outcomes parameters:  
o Change from baseline at Week 12 in total summary score from the urinary 
Incontinence-Specific Quality-of-Life Instrument (I-QOL)  
o Change from baseline at Week 12 in OAB Questionnaire (OAB-q) scores  
o Overall change of bladder symptoms based on the Patient Global Impression of 
Change (PGI-C) scale score at Week 12 
x Urodynamic parameters (read by an independent central reviewer): 
o Cystometric volume at 1st sensation to void (FSV) 
o Maximum cystometric capacity (MCC) 
o Maximum detrusor pressure during the storage phase (corrected PdetMaxStorage ) 
o Presence/absence of the first involuntary detrusor contraction (IDC) and, if 
present: 
 Volume at first IDC (Vol@1stIDC)  
 Maximum detrusor pressure during the first IDC (corrected Pdet@1stIDC) 
Safety Endpoints 
Safety endpoints include adverse events, serious adverse events, physical examination, vital 
signs (pulse rate, blood pressure, respiration rate, and body temperature), hematology and 
clinical chemistry, post-void residual (PVR) urine volume, electrocardiograms (ECGs), 
concomitant medications and concurrent procedures.  


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Other Endpoints 
Other endpoints include hSlo cDNA concentrations (blood and urine)  
Overall Study Design:  
Structure: Multicenter, randomized, double-blind, placebo-controlled, single-treatment, 2-cohort, 
dose-escalation 
Study Treatment Groups: URO-902 (24 mg or 48 mg) administered as intradetrusor injections 
via cystoscopy. See Section 6.3 for details on treatment administration. A single treatment of 
URO-902 24 mg will be administered to subjects in Cohort 1. An independent Data and Safety 
Monitoring Board (DSMB) will make recommendations regarding dose escalation only after 
unblinded review of safety data from all subjects in Cohort 1 up to Week 6 (including subjects 
who prematurely exited the study prior to Week 6). Randomization into Cohort 2 will begin only 
after the DSMB has recommended it is safe to proceed. 
x Cohort 1: URO-902 24 mg  
x Cohort 2: URO-902 48 mg  
Controls: Matching placebo (phosphate buffered saline with 20% sucrose [PBS-20%]) in 
Cohort 1 and Cohort 2 (see Section 1.2). 
Dosage/Dose Regimen: For each subject in Cohort 1 or Cohort 2, a single treatment will be 
administered on Day 1 after fulfillment of the “day of treatment criteria” (see Section 6.2). 
Randomization/Stratification: An estimated total of 78 subjects will receive treatment across 
2 cohorts (Section 1.2), with approximately 39 subjects randomized into each cohort. In both 
cohorts, subjects will be randomized in a 2:1 ratio to receive either URO-902 (24 mg or 48 mg) 
or placebo. Each cohort will be randomized separately, and enrollment will be sequential, 
starting with Cohort 1 (URO-902 24 mg [n = 26] and placebo [n = 13]) and followed by Cohort 2 
(URO-902 48 mg [n = 26] and placebo [n = 13]). At the Randomization Visit (Visit 2), subjects 
in both Cohort 1 and Cohort 2 will be randomized centrally to receive either a single treatment of 
URO-902 or matching placebo. In Cohort 1a, randomization will be stratified by baseline UUI 
episodes per day (ie, ≤ 3 vs. > 3 UUI episodes per day) and the presence or absence of DO (as 
determined by the investigator). In Cohort 1b and Cohort 2, randomization will be stratified by 
baseline UUI episodes per day and previous use of onabotulinumtoxinA (naïve vs. prior use). 
Enrollment for subjects with previous onabotulinumtoxinA use will be capped at 50% of the total 
subject population per cohort.  
Visit Schedule: Study visits will be identical for Cohorts 1 and 2. A 5-week screening and 
randomization period (Days -35 to -1) will include screening subjects for eligibility (Visit 1) and 
randomization of eligible subjects (Visit 2). Subjects will be administered the study treatment via 
cystoscopy on Day 1 (Visit 3). All subjects will be evaluated at scheduled post-treatment visits at 


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Weeks 2, 6, 12, 18, and 24 (Visits 4 to 8, respectively), or until the subject exits the study. 
Afterwards, for subjects who complete the Week 24 Visit (Visit 8), 2 follow-up telephone (or 
other virtual technology) visits will be performed at Week 36 (Visit 9) and Week 48 (Visit 10). 
Additional OAB treatment: Starting at Week 24 (Visit 8), subjects can request and receive 
additional OAB treatment(s) at the clinical discretion of the investigator. Subjects who receive 
additional OAB treatment(s) at Week 24 or after will only be followed to assess adverse events 
at any future telephone (or other virtual technology) visits (Week 36 and/or Week 48). No 
efficacy assessments will be performed once a subject receives additional OAB treatment (see 
Section 6.9.2). 
Safety and efficacy assessments and study procedures are outlined in the study schema 
(Section 1.2) and the Schedule of Assessments (Section 1.3). 
Diagnosis and Criteria for Inclusion and Exclusion: 
Condition/Disease: OAB with UUI  
Listed below are key inclusion and exclusion criteria for the study. See Section 5 for the 
complete list of inclusion and exclusion criteria. 
Key Inclusion Criteria:  
x Subject is female, aged 40 to 79 years old, at the Screening Visit (Visit 1). 
x Subject has symptoms of OAB (frequency and urgency) with UUI for a period of at least 
6 months prior to the Screening Visit (Visit 1), determined by documented subject 
history. 
x Subject experiences an average of ≥ 1 episode of UUI per day (ie, a total of ≥ 3 UUI 
episodes over the 3-day subject Bladder Diary completed during the screening and 
randomization period). 
x Subject experiences urinary frequency, defined as an average of ≥ 8 micturitions (toilet 
voids) per day (ie, a total of ≥ 24 micturitions over the 3-day subject Bladder Diary 
completed during the screening and randomization period). 
x Subject has not been adequately managed with ≥ 1 oral or transdermal pharmacologic 
therapies for the treatment of their OAB symptoms (eg, anticholinergics, beta-3 agonist, 
etc), in the opinion of the investigator. Not adequately managed is defined as meeting one 
of the following: 
o an inadequate response after at least a 4-week period of pharmacologic therapy on 
a Food and Drug Administration (FDA)-approved dose(s) (ie, subject was still 
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o limiting side effects after at least a 2-week period of pharmacologic therapy on an 
FDA-approved dose(s) 
x Subject is of non-childbearing potential. See Section 10.6 for criteria to be satisfied. 
Key Exclusion Criteria:  
x Subject has symptoms of OAB due to any known neurological reason (eg, spinal cord 
injury, multiple sclerosis, cerebrovascular accident, Alzheimer’s disease, Parkinson’s 
disease, etc). 
x Subject has a predominance of stress incontinence in the opinion of the investigator, 
determined by subject history. 
x Subject currently uses or plans to use medications or therapies to treat symptoms of 
OAB, including nocturia. Subjects previously receiving these medications must have 
discontinued their use to achieve sufficient washout prior to beginning recording of 
symptoms into the Screening Bladder Diary as follows: 
o for desmopressin, at least one day prior 
o for anticholinergic therapy, at least 14 days prior 
o for intravesical anticholinergic therapy, at least 4 weeks prior 
o for β3 agonists, at least 14 days prior 
x Subjects who have previously been treated with: 
o onabotulinumtoxinA for urological indications within 12 months of starting the 
Screening Bladder Diary (subjects treated with onabotulinumtoxinA or other 
toxins for non-urological indications are eligible, regardless of when treated).  
o any other toxin for urological indications, regardless of when treated 
x Subject currently uses clean intermittent catheterization (CIC) or indwelling catheter to 
manage their urinary incontinence. 
x Subject has a PVR urine volume of > 100 mL at any time during Screening (before 
randomization). The PVR measurement can be repeated once on the same day; the 
subject is to be excluded if the repeated measure is above 100 mL. 
x Subject has had urinary retention or an elevated PVR urine volume that has been treated 
with an intervention (such as catheterization), within 6 months of the Screening Visit 
(Visit 1). Note: transient voiding difficulties as a result of surgical procedures that 
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x Subject has a history of 3 or more urinary tract infections (UTIs) within 6 months of the 
Screening Visit (Visit 1) or is taking prophylactic antibiotics to prevent chronic UTIs. 
Subjects with a current acute UTI during screening can be treated appropriately and are 
eligible.  
x Subject has current or previous uninvestigated hematuria. Subjects with investigated 
hematuria may enter the study if urological/renal pathology has been ruled out to the 
satisfaction of the investigator. 
Number of Subjects: Approximately 78 adult female subjects will be randomized into the 
2 cohorts, with approximately 39 subjects randomized into each cohort. 
Approximate Number of Sites: Up to 25 sites in North America  
Study Drug Groups and Study Duration: 
Refer to section on Overall Study Design above for treatment group information. 
Duration: Up to 53 weeks (5-week screening and randomization period, treatment administration 
on Day 1, and 48-week double-blind post-treatment/follow-up period) 
Statistical Methods: 
The following analysis populations will be evaluated: safety, safety (modified), and intent-to-
treat exposed (ITT-E). The safety population will consist of all subjects who received the study 
drug and will be used to assess treatment-emergent adverse events and other safety evaluations 
based on actual treatment received. A separate safety population, safety (modified), will be 
created to assess safety excluding subjects who received additional OAB medication at Week 24 
or after. ITT-E will be used for demographics, baseline characteristics, and efficacy analyses. 
The ITT-E population will consist of all subjects randomized and treated from Cohorts 1 and 2. 
Interim analyses may be conducted when ≥ 50% of subjects in Cohort 1 and/or when ≥ 50% of 
subjects in Cohort 2 have completed at least 12 weeks of follow-up post-randomization (or 
prematurely exited the study prior to Week 12) for future planning purposes. A planned interim 
analysis will be performed to evaluate the objectives of the protocol at Week 12, after all 
subjects in Cohorts 1 and 2 have completed the Week 12 Visit/Visit 6 (or prematurely exited the 
study prior to Week 12). The final analysis will be performed after all subjects have completed 
the study or exit the study prematurely. Details of the interim analyses and final analysis will be 
described in the Statistical Analysis Plan. 
The study has no formal statistical primary endpoint hypothesis. Descriptive statistics will be 
used to evaluate the efficacy and safety endpoints. For continuous efficacy endpoints, estimates 
of least squares means, standard error, and 95% confidence intervals (CI) will be presented for 
each treatment group. Nominal p-values from comparisons to placebo may be provided for 
descriptive purposes. 
The point estimate of the treatment difference and 95% confidence interval for the change from 
baseline at each visit for each continuous efficacy variable relative to placebo will be analyzed 
using a mixed-effect model for repeated measures (MMRM) method. The analysis model will 
include terms for baseline value as a covariate, in addition to the terms for treatment, visit, and 
treatment by visit interaction. For the urodynamic variables evaluated, only the independent 
central reviewer’s interpretation will be analyzed. 


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The proportion of subjects who achieve ≥ 50% reduction from baseline UUI episodes at 
Week 12 will be calculated for each treatment group. In addition, responder analyses will also be 
calculated for subjects who achieve ≥ 75% and 100% decrease in episodes of UUI at Week 12 
relative to baseline. The Cochran-Mantel-Haenszel (CMH) method will be utilized to compare 
the proportion of responders between the active and placebo groups by adjusting for the 
stratification factors. Data for all visits will also be presented. 
For safety variables, data from all subjects in the 2 cohorts who received study drug will be 
included. The incidence of adverse events will be summarized. The change from baseline in PVR 
urine volume will be analyzed. 
Sample Size Estimation: For this exploratory study, the sample size was empirically chosen. 
Please see Section 9.2 for details. 
Data and Safety Monitoring Board (DSMB): A DSMB will be retained to assess, on an 
ongoing basis, all safety aspects of this study including unblinded review of safety data after all 
subjects reach Week 6 (or prematurely exited the study prior to Week 6) in Cohort 1 
(URO-902 24 mg). Randomization into Cohort 2 will begin only after the DSMB has 
recommended it is safe to proceed to Cohort 2. This committee will be an external independent 
DSMB that monitors safety. The detailed activities including meeting plans will be described and 
documented in a separate DSMB Charter. A separate statistical analysis plan may also be 
prepared for the DSMB.


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1.2.
Schematic of Study Design: 2-Cohort, Dose-Escalation
Note: 
1)
Dose escalation to Cohort 2 is based on unblinded review of safety data by the Data and Safety Monitoring 
Board (DSMB) after all subjects reach Week 6 (or prematurely exited the study prior to Week 6) in 
Cohort 1. Randomization into Cohort 2 will begin only after the DSMB has recommended it is safe to 
proceed. 
2)
Starting at Week 24, subjects can request and receive additional overactive bladder (OAB) treatment(s) at 
the clinical discretion of the investigator. Subjects who receive additional OAB treatment(s) at Week 24 or 
after will only be followed to assess adverse events at any future telephone (or other virtual technology) 
visits (Week 36 and/or Week 48). No efficacy assessments will be performed once a subject receives
additional OAB treatment (see Section 6.9.2).


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1.3. 
Schedule of Assessments  
Study Period  
 
Screening 
Visit 
Visit 1 
Randomization 
Visit 
Visit 2 
Day 1 
(Treatment 
Administration 
Visit) 
Visit 3 
Week 2 
(Follow-
Up Clinic 
Visit) 
Visit 4 
Week 6 
(Follow-Up 
Clinic 
Visit) 
Visit 5 
Week 12 
(Follow-Up 
Clinic 
Visit) 
Visit 6 
Week 18 
(Follow-Up 
Phone 
Visitb) 
Visit 7 
Week 24 
(Exit or 
EW 
Clinic 
Visit) 
Visit 8 
Week 36 
(Follow-
Up Phone 
Visit)b 
Visit 9 
Week 48 
(Follow-
Up Phone 
Visit)b 
Visit 10 
Protocol-defined Visit 
Windows 
 
-35 to -1 Daysa 
± 3 Days 
± 7 Days 
± 7 Days 
± 7 Days 
± 7 Days 
± 7 Days 
± 7 Days 
COVID-19 Mitigation Visit 
Windows* 
 
 
+ 10 Days 
+ 4 Weeks 
+ 4 Weeks 
N/A 
+ 4 Weeks 
N/A 
N/A 
Consent/Authorization 
X 
 
 
 
 
 
 
 
 
 
Screening entry into IWRS 
X 
 
 
 
 
 
 
 
 
 
Inclusion/Exclusion Criteria 
X 
X 
 
 
 
 
 
 
 
 
Subject Randomization 
 
X 
 
 
 
 
 
 
 
 
Day of Treatment Criteria  
 
 
Xc 
 
 
 
 
 
 
 
Study Treatment 
Administration 
 
 
X 
 
 
 
 
 
 
 
Medical History and 
Demographics 
X 
 
 
 
 
 
 
 
 
 
Prior OAB Med Review 
X 
 
 
 
 
 
 
 
 
 
Physical Examination 
X 
 
 
 
 
 
 
X 
 
 
Vital Signs 
X 
X 
Xc 
X 
X 
X 
 
X 
 
 
ECG 
X 
 
 
X 
 
X 
 
X 
 
 
Urodynamics 
 
Xd 
 
 
 
X 
 
 
 
 
PVR Urine  
Xe 
 
 
X 
X 
X 
 
X 
 
 
Bladder and Kidney 
Ultrasound 
X 
 
 
 
 
 
 
X 
 
 
 


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Study Period  
 
Screening 
Visit 
Visit 1 
Randomization 
Visit 
Visit 2 
Day 1 
(Treatment 
Administration) 
Visit 3 
Week 2 
(Follow-
Up Clinic 
Visit) 
Visit 4 
Week 6 
(Follow-
Up Clinic 
Visit) 
Visit 5 
Week 12 
(Follow-Up 
Clinic 
Visit) 
Visit 6 
Week 18 
(Follow-Up 
Phone 
Visit)b 
Visit 7 
Week 24 
(Exit or 
EW 
Clinic 
Visit) 
Visit 8 
Week 36 
(Follow-
Up Phone 
Visit)b 
Visit 9 
Week 48 
(Follow-
Up Phone 
Visit)b 
Visit 10 
Protocol-defined Visit 
Windows 
 
-35 to -1 Daysa 
± 3 Days 
± 7 Days 
± 7 Days 
± 7 Days 
± 7 Days 
± 7 Days 
± 7 Days 
COVID-19 Mitigation Visit 
Windows* 
 
 
+ 10 Days 
+ 4 Weeks 
+ 4 Weeks 
N/A 
+ 4 Weeks 
N/A 
N/A 
Urine Dipstickf 
X 
X 
Xc 
X 
X 
X 
 
X 
 
 
Urine Cytology (local lab) 
X 
 
 
 
 
 
 
 
 
 
Hematology and Clinical 
Chemistry  
X 
 
 
 
X 
 
 
X 
 
 
Dispense Bladder Diary 
X 
 
X 
X 
X 
Xg 
 
Xh 
 
 
Review and data enter 
Bladder Diaryi 
 
X 
 
X 
X 
X 
X 
X 
Xh 
Xh 
Pharmacokinetic Assessment 
(urine and blood hSlo cDNA) 
 
 
Xc 
 
X 
 
 
Xj 
 
 
Prophylactic Antibiotics  
 
 
Xk 
 
 
 
 
 
 
 
I-QOL and OAB-q 
Questionnaires 
 
 
Xc 
 
X 
X 
 
 
 
 
PGI-C Questionnaire 
 
 
 
 
 
X 
 
 
 
 
Con Meds/Procedures 
Xl 
X 
X 
X 
X 
X 
X 
Xm 
Xm 
X 
AE/SAE Review 
X 
X 
X 
X 
X 
X 
X 
Xm 
Xm 
X 
Optional Immunogenicity 
Samplen 
 
 
 
 
 
 
 
 
 
 
Immunogenicity Consent 
Xo 
 
 
 
 
 
 
 
 
 
Sample Collection 
 
 
Xc 
 
X 
 
 
X 
 
 
AE = adverse event; Con Meds = concomitant medication(s); COVID-19 =coronavirus disease 2019; ECG = electrocardiogram; eCRF = electronic case report form; EW = early withdrawal; I-
QOL = Incontinence-Specific Quality of Life Instrument; IWRS = interactive web response system; lab = laboratory; Med = medication(s); N/A = not applicable; OAB = overactive bladder; 
OAB-q = Overactive Bladder Questionnaire; PGI-C = Patient Global Impression of Change; PVR = Post-void residual; SAE = serious adverse event 


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* To accommodate any COVID-19-related events that affect a subject’s scheduled visit, the COVID-19 Mitigation Visit Window may be used instead of the protocol-defined visit schedule 
window. However, if safe and feasible, study visits should adhere to the scheduled visit window for the study. Note that the COVID-19 Mitigation Visit Window is separate from the protocol-
defined visit window and the 2 visit windows may not be combined.  
a 
The actual number of days (duration) between the Screening Visit (Visit 1), Randomization Visit (Visit 2), and Day 1 (Visit 3) are flexible as long as the subject undergoes the required 
protocol-defined assessments at each respective visit and is fully evaluated for eligibility based on the clinical judgment of the investigator. The maximum Screening and Randomization 
period should not exceed 35 days prior to Day 1. 
b Visits at Weeks 18, 36, and 48 may be conducted by phone or other virtual technology. Follow-up visits at Week 36 and Week 48 are only conducted for subjects who have completed the 
Week 24 Visit (Visit 8) and are not applicable for early withdrawal (EW) subjects. 
c 
Performed prior to treatment. Additionally, for pharmacokinetic assessments only, these will also be performed approximately 2 hours ± 1 hour post-treatment on Day 1. 
d Urodynamic assessments at baseline should only be conducted after confirmation of subject eligibility during Visit 2 and must be performed prior to the actual randomization procedure. An 
historical urodynamic study, performed no more than 90 days prior to the first day of screening, may serve as the baseline urodynamic assessment if the criteria detailed in Section 8.2.3 are 
satisfied. At Week 12, all subjects will undergo a second urodynamic assessment. Note that the investigator does not need to wait for the results of the baseline urodynamic assessments to 
rule out bladder outlet obstruction if there are no other signs or symptoms of obstruction, in the investigator’s assessment, including elevated PVR volume (see Exclusion Criterion 14).  
e May be performed at any time during screening (before randomization), excluding diary data collection days. 
f 
A sample for urinalysis and urine culture/sensitivity will be sent to the laboratory only if the urine dipstick (performed at the site) results are suggestive of a urinary tract infection (ie, 
positive for the presence of leukocytes, nitrites, or blood cells). The urinalysis sample should be sent to the central laboratory, and the urine culture and sensitivity sample should be sent to 
the local laboratory. 
g Since the Week 18 visit (Visit 7) is a telephone (or other virtual technology) visit, sufficient diaries should be dispensed at Week 12 (Visit 6) to record data through Week 24.  
h At the Week 24 exit clinic Visit (Visit 8), a sufficient number of bladder diaries will be provided to subjects in advance for the remaining follow-up phone or other virtual technology visits; 
bladder diaries completed for the Week 36 (Visit 9) and Week 48 (Visit 10) follow-up visits will need to be collected by mail prior to the scheduled visit and reviewed by phone or other 
virtual technology with the subject. Diaries will not be collected if subjects receive additional OAB treatment(s) at Week 24 (Visit 8) or after. See Section 6.9.2 for further details. 
i 
Bladder diary must be completed by subjects for any 3 consecutive days in the 7 days prior to the protocol-defined visit timepoint. If a visit will be delayed per the COVID-19 mitigation 
window, the site should contact the subject to ensure that the diary completion occurs per the protocol-defined timepoints and not within the 7 days prior to the COVID-delayed visit. For 
screening only, the diary can be completed for 3 consecutive days at any time during the screening and randomization period. The total volume voided must be collected for one 24-hour 
period during the 3-day diary collection period. 
j 
If specimen(s) is still positive at Week 24, per the clinical discretion of the investigator, a subject may be instructed to return at Week 36 (Visit 9) and, if necessary, Week 48 (Visit 10) until 
 
the specimen(s) result is negative for hSlo cDNA. Pharmacokinetic assessment is to be performed at the EW Visit for subjects who withdraw early from the study. 
k All subjects must receive prophylactic antibiotics prior to treatment administration on Day 1 and for 1 additional day post-treatment at a minimum. 
l 
Prohibited medication washout of pharmacologic therapy for OAB must be complete prior to beginning recording of symptoms into the Screening Bladder Diary. 
m Starting at Week 24, subjects can request and receive additional OAB treatment(s) at the clinical discretion of the investigator. Subjects who receive additional OAB treatment(s) at 
Week 24 or after will only be followed to assess AEs at any future telephone (or other virtual technology) visits (Week 36 and/or Week 48). No efficacy assessments will be performed once 
a subject receives an additional OAB treatment. Any additional OAB concomitant medication used or procedure performed as well as any AEs reported must be recorded in the appropriate 
eCRF form. See Section 6.9.2 for further details. 
n If the subject consents and a baseline sample can be obtained prior to study treatment administration, then blood will be collected at each specified visit (applies to Cohort 2 only). Note that 
the sample will be stored, and antibody testing may be performed at a later date (up to 5 years from date of collection). 
o Consent may be obtained at any time before the sample is collected on Day 1. 
 
 


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2. 
INTRODUCTION 
Urovant Sciences GmbH (Urovant) is developing URO-902 (formerly known as hMaxi-K or 
pVAX/hSlo) for the treatment of overactive bladder (OAB) and urge urinary incontinence (UUI) 
in adults who have been inadequately managed by pharmacologic therapy. 
2.1. 
Background 
Overactive Bladder 
OAB is highly prevalent and affects approximately 16% of the population in the United States 
and Europe. Prevalence increases with age, affecting approximately a third of people 75 years 
and older [Stewart, 2003; Milsom, 2001]. The International Continence Society (ICS) defines 
OAB as urgency, with or without urge incontinence, usually associated with frequency and 
nocturia [Abrams, 2002]. Urgency is defined as a sudden compelling desire to void which is 
difficult to defer and, from the medical definition perspective, it is a necessary symptom for 
OAB. Urgency incontinence is the involuntary loss of urine accompanied by urgency (referred to 
as OAB Wet) and is present in approximately one-third of patients with OAB [Stewart, 2003; 
Milsom, 2001]. In the absence of incontinence, OAB is referred to as OAB Dry. OAB has been 
shown to negatively impact both social and medical well-being and represents a significant 
burden in terms of annual direct and indirect healthcare expenditures [Kelleher, 2002; 
Abrams, 2000; Reynolds, 2016]. 
Currently, the predominant class of drugs used to treat OAB is antimuscarinics. The clinical 
utility of antimuscarinics is limited by their modest efficacy and poor tolerability due to 
mechanism-based side effects including dry mouth and constipation. In addition to poor 
tolerability and modest efficacy, a recent body of literature suggests that chronic use of 
anticholinergics is associated with cognitive impairment and dementia [Risacher, 2016; 
Gray, 2015]. Mirabegron (Myrbetriq®; Astellas Pharma US, Inc), a β3-agonist and smooth 
muscle relaxer, is currently approved in multiple countries for the treatment of OAB 
[Nitti, 2013]. The reported adverse effects with mirabegron were similar to antimuscarinics, but 
with a lower incidence of dry mouth compared with tolterodine, an antimuscarinic. However, the 
effects of mirabegron on the cardiovascular system, pharmacokinetic interactions with other 
drugs, and increased incidence of new malignant events will require careful evaluation in the 
near future [Sacco, 2012]. In addition, BOTOX® (onabotulinumtoxinA), a neurotoxin which 
inhibits muscle contraction by blocking neuromuscular transmission through decreased 
acetylcholine release, has been approved for the treatment of neurogenic and idiopathic OAB 
(since 2011and 2012, respectively). BOTOX is administered via multiple intradetrusor injections 
into the bladder wall about every 6 months. BOTOX has been associated with urinary tract 
infections (UTIs), dysuria, and urinary retention. Other side effects are consistent with the 
mechanism of action of botulinum toxin and may include asthenia, generalized muscle weakness, 
diplopia, ptosis, dysphagia, dysphonia, dysarthria, urinary incontinence, and breathing 


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difficulties. These symptoms have been reported hours to weeks after injection 
[BOTOX Package Insert, 2018]. 
A major problem with currently available OAB treatments is that even when these drugs have 
documented efficacy, treatment is still limited by their side effects. There is a significant unmet 
need for the treatment of OAB with agents with novel mechanisms of action and a well-tolerated 
safety profile. 
URO-902 


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2.2. 
Study Rationale 
Overactive bladder is a highly prevalent disease shown to negatively impact both the social and 
medical well-being of patients. Many of the existing treatments are limited by their side effects. 
Thus, there is a significant unmet need for the treatment of OAB with agents with novel 
mechanisms of action and a well-tolerated safety profile, in particular, for patients who have 
been inadequately managed by pharmacologic therapy. URO-902 (pVAX/hSlo) is a GMP 
manufactured double-stranded DNA plasmid (pVAX vector) containing a cDNA insert encoding 
the pore-forming α subunit of the human smooth muscle Maxi-K channel, hSlo. The Maxi-K 
channel is a prominent and well-studied K channel subtype involved in smooth muscle 
relaxation. Because heightened smooth muscle tone may be a causative factor of OAB with DO, 
increased numbers of Maxi-K channels in the bladder smooth muscle cells associated with 
effective URO-902 treatment may improve this condition. Based on the favorable safety profile 
demonstrated in the completed clinical studies in subjects with OAB
 
 
 the current exploratory study is designed to 
evaluate the efficacy and safety of a single treatment of URO-902 administered via intradetrusor 
injection in subjects with OAB and UUI. 
2.3. 
Benefit/Risk Assessment 
2.3.1. 
Potential Benefits 
URO-902’s mechanism of action has the potential to demonstrate significant therapeutic benefit 
in the treatment of OAB and UUI. Nonclinical studies conducted with URO-902 have 
demonstrated no toxicity and no off-target effects in safety pharmacology studies. URO-902 has 
demonstrated a favorable safety profile in completed clinical studies
 
 
 
 
 
 


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2.3.2. 
Potential Risks 
Nonclinical studies evaluating URO-902 support the safety of the doses being evaluated in this 
study. Data from URO-902 nonclinical studies are summarized in the IB. See Section 4.3 for 
justification of dose selection. 
URO-902 at doses of up to 24 mg (which is the starting dose being evaluated in the current 
study) administered by direct injections into the bladder wall/detrusor muscle has been evaluated 
in a previous clinical study ION-03. No clinical safety signals (including no urinary retention), 
were identified in the studies. More detailed information about the known and reasonably 
expected adverse events of URO-902 administered via intradetrusor injections can be found in 
the IB. 
The risk to subjects in the current study will be minimized by compliance with the eligibility 
criteria, proper study design, close monitoring, and an independent Data and Safety Monitoring 
Board (DSMB). The study follows a dose-escalation design. The DSMB will make 
recommendations regarding dose escalation only after unblinded review of safety data from all 
subjects in Cohort 1 up to Week 6. 
URO-902 is an investigational drug and should only be used as specified in the clinical study 
protocol. Subjects should be excluded from treatment with URO-902 in accordance with 
inclusion/exclusion criteria of the protocol. 


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3. 
OBJECTIVES AND ENDPOINTS 
3.1. 
Objectives 
The objectives of this study are as follows: 
x To evaluate the efficacy of a single dose of URO-902 24 mg and 48 mg (administered via 
intradetrusor injection), compared with placebo, in subjects with OAB and UUI up to 
48 weeks post-dose 
x To evaluate the safety and tolerability of a single dose of URO-902 24 mg and 48 mg 
(administered via intradetrusor injection), compared with placebo, in subjects with OAB 
and UUI up to 48 weeks post-dose 
3.2. 
Endpoints 
This exploratory study has no formal statistical primary endpoint hypothesis. Study endpoints are 
as follows: 
3.2.1. 
Exploratory Efficacy Endpoints 
Exploratory efficacy endpoints include the following: 
x Change from baseline at Week 12 in average daily number of UUI episodes 
x Change from baseline at Week 12 in average daily number of micturitions 
x Change from baseline at Week 12 in average daily number of urinary incontinence (UI) 
episodes 
x Change from baseline at Week 12 in average daily number of urgency episodes 
x Proportion of subjects achieving ≥ 50%, ≥ 75%, and 100% reduction from baseline at 
Week 12 in UUI episodes per day 
x Change from baseline at Week 12 in average volume voided per micturition 
x Health outcomes parameters:  
o Change from baseline at Week 12 in total summary score from the urinary 
Incontinence-Specific Quality-of-Life Instrument (I-QOL)  
o Change from baseline at Week 12 in OAB Questionnaire (OAB-q) scores  
o Overall change of bladder symptoms based on the Patient Global Impression of 
Change (PGI-C) scale score at Week 12 


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x Urodynamic parameters (read by an independent central reviewer): 
o Cystometric volume at 1st sensation to void (FSV) 
o Maximum cystometric capacity (MCC) 
o Maximum detrusor pressure during the storage phase (corrected PdetMaxStorage) 
o Presence/absence of the first involuntary detrusor contraction (IDC) and, if 
present: 
 Volume at first IDC (Vol@1stIDC)  
 Maximum detrusor pressure during the first IDC (corrected Pdet@1stIDC) 
3.2.2. 
Safety Endpoints 
Safety endpoints include adverse events, serious adverse events, physical examination, vital 
signs (pulse rate, blood pressure, respiration rate, and body temperature), hematology and 
clinical chemistry, post-void residual (PVR) urine volume, electrocardiograms (ECGs), 
concomitant medications and concurrent procedures. 
3.2.3. 
Other Endpoints 
Other endpoints include hSlo cDNA concentrations (blood and urine). 


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4. 
STUDY DESIGN 
4.1. 
Overall Design 
This is a multicenter, randomized, double-blind, placebo-controlled, single-treatment, 2 cohort, 
dose-escalation study evaluating the efficacy and safety of URO-902 (24 mg or 48 mg) in the 
treatment of OAB and UUI in female subjects aged 40 to 79 years old. Subjects must complete 
all screening procedures and must meet all eligibility requirements to qualify for enrollment and 
randomization. 
Safety and efficacy assessments and study procedures are outlined in the study schema 
(Section 1.2) and the Schedule of Assessments (Section 1.3). 
The total duration of the study is up to 53 weeks including a 5-week screening and 
randomization period (Days -35 to -1); treatment on Day 1, and a 48-week double-blind 
post-treatment/follow-up period. Study visits will be identical for Cohorts 1 and 2. Subjects will 
be evaluated at the Screening Visit (Visit 1) for eligibility. Eligible subjects will be randomized 
to treatment within each cohort at the Randomization Visit (Visit 2); however, subjects will be 
administered study treatment via cystoscopy on Day 1 (Visit 3). All subjects will be evaluated at 
scheduled post-treatment visits at Weeks 2, 6, 12, 18 (phone/virtual), and 24 (Visits 4 to 8, 
respectively), or until the subject exits the study. Afterwards, for subjects who have completed 
the Week 24 Visit (Visit 8), 2 follow-up telephone visits (or other virtual technology visits) for 
assessment of safety will be performed at Week 36 (Visit 9) and Week 48 (Visit 10). 
An estimated total of 78 subjects will receive treatment across 2 cohorts (Section 1.2), with 
approximately 39 subjects randomized into each cohort. In both cohorts, subjects will be 
randomized in a 2:1 ratio to receive either URO-902 (24 mg or 48 mg) or placebo. Each cohort 
will be randomized separately, and enrollment will be sequential, starting with Cohort 1 
(URO-902 24 mg [n = 26] and placebo [n = 13]) and followed by Cohort 2 (URO-902 48 mg 
[n = 26] and placebo [n = 13]). Subjects in both Cohort 1 and Cohort 2 will be randomized 
centrally to receive either a single treatment of URO-902 or matching placebo.  
In Cohort 1a, randomization will be stratified by baseline UUI episodes per day (ie, ≤ 3 vs. 
> 3 UUI episodes per day) and presence or absence of DO (as determined by the investigator) at 
Visit 2. In Cohort 1b and Cohort 2, randomization will be stratified by baseline UUI episodes per 
day and previous use of onabotulinumtoxinA (naïve vs. prior use). OnabotulinumtoxinA 
treatment for urological indications must have occurred at least 12 months prior to starting the 
Screening Bladder Diary. Enrollment for subjects with previous onabotulinumtoxinA use will be 
capped at 50% of the total subject population per cohort. See details on stratification factors in 
Section 6.5. 
In Cohort 1, an unblinded review of safety data by the DSMB will be performed after all subjects 
reach Week 6 (or prematurely exited the study prior to Week 6). Randomization into Cohort 2 
will begin only after the DSMB has recommended it is safe to proceed. Details on tasks and 


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responsibilities and assessments of safety parameters will be provided in the DSMB Charter. The 
independent DSMB will review the safety data throughout the entire study. 
For each subject in Cohort 1 or Cohort 2, a single treatment will be administered on Day 1 
(Visit 3) after fulfillment of the “day of treatment criteria” (Section 6.2). Subjects will receive a 
single treatment of URO-902 or placebo administered by intradetrusor injections via cystoscopy. 
Details of the study treatment procedure are provided in Section 6.3. 
Subjects will be instructed to contact the study site to report any adverse events that occur within 
48 hours following administration of study treatment. A 3-day Bladder Diary will be used to 
collect information to assess exploratory efficacy endpoints related to the number of UUI, 
micturition, urgency, and UI episodes per day as well as one 24-hr volume voided of urine. 
Medications prohibited before and during the study and the associated washout periods as well as 
permitted medications are described in Sections 6.9.1 and 6.9.2, respectively. Starting at 
Week 24, subjects can request and receive additional OAB treatment(s) at the clinical discretion 
of the investigator. Subjects who receive additional OAB treatment(s) at Week 24 or after will 
only be followed to assess adverse events at any future telephone (or other virtual technology) 
visits (Week 36 and/or Week 48). No efficacy assessments will be performed once a subject 
receives additional OAB treatment. 
4.1.1. 
Clinical Hypothesis 
Due to the exploratory nature of this trial, there is no formal hypothesis. Statistical testing will be 
performed to estimate the treatment effect of URO-902 relative to placebo in female subjects 
with OAB with UUI. 
4.2. 
Scientific Rationale for Study Design 
Study URO-902-2001 is a randomized, double-blind, placebo-controlled, single-treatment, 
2 cohort, dose-escalation, exploratory efficacy and safety study. 
The dose-escalation design, including interim safety analyses and DSMB review between each 
cohort, helps minimize the risk to subjects. Randomization into Cohort 2 ( higher dose) will 
begin only after the DSMB has recommended it is safe to proceed. 
Randomization will be stratified by baseline UUI episodes per day, presence or absence of DO 
(as determined by the investigator), and previous use of onabotulinumtoxinA (naïve vs. prior 
use) to ensure balance across the treatment groups. See details on stratification factors in 
Section 6.5. 
This study is in the early stages of the development program for URO-902. There are no 
nonclinical studies that have investigated the genotoxic or reproductive effects of URO-902. 
Therefore, the study only includes female subjects of nonchildbearing potential. The study is 
randomized and double-blind to minimize investigator and subject bias and will provide a 


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placebo comparator for evaluating the safety and efficacy of the 2 doses of URO-902 being 
studied. In addition, relevant safety parameters including adverse events (AEs), SAEs, AEs of 
urinary retention and UTI as per protocol definition, physical examination, vital signs (pulse rate, 
blood pressure, respiration rate, and body temperature), urine dipstick reagent strip test, 
urinalysis/urine culture/sensitivity, as applicable, hematology and clinical chemistry, PVR urine 
volume, kidney and bladder ultrasound, concomitant medications and concurrent procedures will 
be closely monitored throughout the study. 
4.3. 
Justification of Dose 
An extensive series of in vitro and in vivo nonclinical studies evaluating the activity and safety of 
URO-902 have been conducted in OAB and ED animal models at doses up to 1 mg. The results 
of the nonclinical evaluations supported the initiation of URO-902 clinical studies. No toxicity 
was observed at any dose level in any of the preclinical studies at any dose in studies conducted 
to date, including multiple dosing in the ED rat model. Extensive data in the ED and OAB 
animal models has shown neither histopathological abnormality at any time point in any of 
40 organs evaluated or expression of the gene in any other organ other than the organ that 
underwent transfer more than 1 week after that transfer, as well as in ED studies conducted up to 
1 month after transfer. 
The bladder preclinical studies evaluated single doses of 0.01, 0.03, 0.1, 0.3, and 1 mg based on 
an obstructed bladder surface area of 12.56 cm2 and an average approximate bladder volume of 
4 mL. The formula for surface area is 4πr2. Hence, human bladder surface area (average bladder 
volume of 400 mL) is 263.5 cm2. Therefore, the approximate dose relationship of human to rat 
bladder is 20:1. In the completed OAB clinical studies, doses up to 25 mg by direct injection into 
the bladder wall/detrusor muscle were well-tolerated. Data from completed URO-902 nonclinical 
and clinical studies are summarized in the IB. 
In the
 
 
 
 No clinically meaningful safety signals were identified at either the 16 mg 
or 24 mg dose in 
 A starting dose of 24 mg will be initially tested in the planned 
Phase 2a clinical study URO-902-2001 to evaluate the safety and efficacy of URO-902 in 
subjects with OAB and UUI. Study URO-902-2001 has a dose-escalation design. Based on the 
unblinded review of observed safety data from all subjects in Cohort 1 (URO-902 24 mg) up to 
Week 6, the DSMB will make the recommendation to proceed with Cohort 2. Randomization 
into Cohort 2 (URO-902 48 mg) will begin only after the DSMB has recommended it is safe to 
proceed. 
The dose equivalent to the 24 mg human dose and the 48 mg human dose in the rat is no more 
than 0.480 mg and 0.960 mg, respectively. As described above, the obstructed bladder preclinical 
studies in the rat evaluated single doses of up to 1 mg based on surface area of the bladder. In the 
rat ED model, doses up to 1 mg were administered by intracavernous injection. Thus, the starting 


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dose concentration of URO-902 at 24 mg as well as the highest dose to be evaluated in the 
planned clinical study (48 mg) are well within the range of doses investigated in the preclinical 
studies. 
4.4. 
End of Study Definition 
The end of the study is defined as the date of the last visit or last scheduled procedure shown in 
the Schedule of Assessments (Section 1.3) for the last subject in the study. 
A subject is considered to have completed the study if she was treated, has not been discontinued 
for any reason, and attends the scheduled exit visit (Week 24; Visit 8) of the cohort she is 
enrolled in. 


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5. 
STUDY POPULATION 
The study is being conducted in female subjects with OAB and UUI. Specific inclusion and 
exclusion criteria are specified below. Prospective approval of protocol deviations to recruitment 
and enrollment criteria, also known as protocol waivers or exemptions, is not permitted. For 
screen failure procedures, see Section 5.4. 
5.1. 
Inclusion Criteria 
Subjects must meet all of the following inclusion criteria at the Screening Visit (Visit 1) and the 
Randomization Visit (Visit 2) to be eligible for participation in this study. 
1. 
Capable of giving written informed consent, which includes compliance with the 
requirements and restriction listed in the consent form. 
2. 
Subject is female, aged 40 to 79 years old, at the Screening Visit (Visit 1). 
3. 
Subject has symptoms of OAB (frequency and urgency) with UUI for a period of at 
least 6 months prior to Screening Visit (Visit 1), determined by documented subject 
history. 
4. 
Subject experiences an average of ≥ 1 episode of UUI per day (ie, a total of ≥ 3 UUI 
episodes over the 3-day subject Bladder Diary completed during the screening and 
randomization period). 
5. 
Subject experiences urinary frequency, defined as an average of ≥ 8 micturitions (toilet 
voids) per day (ie, a total of ≥ 24 micturitions over the 3-day subject Bladder Diary 
completed during the screening and randomization period). 
6. 
Subject has not been adequately managed with ≥ 1 oral or transdermal pharmacologic 
therapies for the treatment of their OAB symptoms (eg, anticholinergics, beta-3 agonist, 
etc), in the opinion of the investigator. Not adequately managed is defined as meeting 
one of the following: 
a 
an inadequate response after at least a 4-week period of pharmacologic therapy on 
a Food and Drug Administration (FDA)-approved dose(s) (ie, subject was still 
incontinent despite pharmacologic therapy), or 
b 
limiting side effects after at least a 2-week period of pharmacologic therapy on an 
FDA-approved dose(s) 
7. 
Subject is willing to use clean intermittent catheterization (CIC) to empty the bladder at 
any time after receiving study treatment if it is determined to be necessary by the 
investigator. 
8. 
Subject is of non-childbearing potential. See Section 10.6 for criteria to be satisfied. 
9. 
In the opinion of the investigator, subject is able to: complete study requirements, 
including using the toilet without assistance; collect urine volume voided per 
micturition measurements over a 24-hour period; complete bladder diaries and 
questionnaires; and attend all study visits. 
5.2. 
Exclusion Criteria 
Subjects will be excluded from participating in the study for any one of the following criteria 
assessed during the Screening Visit (Visit 1) and at the Randomization Visit (Visit 2): 


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1. 
Subject has symptoms of OAB due to any known neurological reason (eg, spinal cord 
injury, multiple sclerosis, cerebrovascular accident, Alzheimer’s disease, Parkinson’s 
disease, etc). 
2. 
Subject has a predominance of stress incontinence in the opinion of the investigator, 
determined by subject history. 
3. 
Subject currently uses or plans to use medications or therapies to treat symptoms of 
OAB, including nocturia. Subjects previously receiving these medications must have 
discontinued their use to achieve sufficient washout prior to beginning recording of 
symptoms into the Screening Bladder Diary as follows: 
a 
for desmopressin, at least one day prior 
b 
for anticholinergic therapy, at least 14 days prior 
c 
for intravesical anticholinergic therapy, at least 4 weeks prior 
d 
for β3 agonists, at least 14 days prior 
4. 
Subjects who have previously been treated with: 
a 
onabotulinumtoxinA for urological indications within 12 months of 
starting the Screening Bladder Diary (subjects treated with 
onabotulinumtoxinA or other toxins for non-urological indications are 
eligible, regardless of when treated) 
b 
any other toxin for urological indications, regardless of when treated 
5. 
Subject currently uses CIC or indwelling catheter to manage their urinary incontinence. 
6. 
Subject has been treated with any intravesical pharmacologic agent (eg, capsaicin, 
resiniferatoxin) within 12 months of beginning recording of symptoms into the 
Screening Bladder Diary. 
7. 
Subject has history or evidence of any pelvic or urological abnormalities, bladder 
surgery or disease, other than OAB, that may affect bladder function including but not 
limited to: 
a 
Bladder stones and/or bladder stone surgery at the time of screening or 
within 6 months prior to screening. 
b 
Surgery (including minimally invasive surgery) within 1 year of screening 
for: stress incontinence, uterine prolapse, rectocele, or cystocele. 
c 
Current or planned use of an implanted 
electrostimulation/neuromodulation device for treatment of urinary 
incontinence for the duration of the study. If a device is still implanted, it 
must be inactive 4 weeks prior to the Screening Visit (Visit 1) and for the 
duration of the study. 
d 
use of other non-implantable electrostimulatory devices for the duration of 
the study. 
8. 
Subject has a history of interstitial cystitis/painful bladder syndrome, in the opinion of 
the investigator. 
9. 
Subject has an active genital infection, other than genital warts, either concurrently or 
within 4 weeks prior to screening. 


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10. Subject has uterine prolapse of grade 3 or higher (ie, cervix descends outside of the 
introitus)  
11. Subject has a history or current diagnosis of bladder cancer or other urothelial 
malignancy, and/or has un-investigated suspicious urine cytology results. Suspicious 
urine cytology abnormalities require that urothelial malignancy is ruled out to the 
satisfaction of the investigator according to local site practice. 
12. Subject has evidence of bladder outlet obstruction, in the opinion of the investigator at 
screening or randomization. 
13. Subject has evidence of urethral outlet obstruction or urethral injury or stricture, in the 
opinion of the investigator at screening or randomization. 
14. Subject has a PVR urine volume of > 100 mL at any time during screening (before 
randomization). The PVR measurement can be repeated once on the same day; the 
subject is to be excluded if the repeated measure is above 100 mL. 
15. Subject has had urinary retention or an elevated PVR urine volume that has been 
treated with an intervention (such as catheterization), within 6 months of the Screening 
Visit (Visit 1). Note: transient voiding difficulties as a result of surgical procedures that 
resolved within 24 hours are not exclusionary. 
16. Subject has a 24-hour total volume of urine voided > 3000 mL, collected over 
24 consecutive hours during the 3-day Bladder Diary collection period prior to 
randomization. 
17. Subject has a history of 3 or more UTIs within 6 months of the Screening Visit (Visit 1) 
or is taking prophylactic antibiotics to prevent chronic UTIs. Subjects with a current 
acute UTI during screening can be treated appropriately and are eligible. 
18. Subject has a serum creatinine level > 2 times the upper limit of normal at the 
Screening Visit (Visit 1). 
19. Subject has current or previous uninvestigated hematuria. Subjects with investigated 
hematuria may enter the study if urological/renal pathology has been ruled out to the 
satisfaction of the investigator. 
20. Subject has a known allergy or sensitivity to URO-902, anesthetics, or antibiotics to be 
used during the study. 
21. Subject needs a walking aid on a permanent basis.  
22. Subject is currently participating in or has previously participated in another therapeutic 
study within 30 days of screening (or longer if local requirements specify). Subject has 
been previously treated with URO-902 in clinical trials (formerly known as hMaxi-K or 
pVAX/hSlo). 
23. Subject has a history or current evidence of any condition, therapy, laboratory 
abnormality or other circumstances that might, in the opinion of the investigator, 
confound the results of the study, interfere with the subject's ability to comply with the 
study procedure, or make participation in the study not in the subject's best interest. 
5.3. 
Lifestyle Considerations 
Not applicable for this study. 


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5.4. 
Screen Failures 
Screen failures are defined as subjects who consent to participate in the clinical study but are not 
subsequently randomized to treatment. A minimal set of screen failure information is required to 
ensure transparent reporting of screen failure subjects to meet the Consolidated Standards of 
Reporting Trials (CONSORT) publishing requirements and to respond to queries from regulatory 
authorities. Minimal information includes demography, screen failure details, eligibility criteria, 
and any SAE. 
Individuals who do not meet the criteria for participation in this study (screen failures) may be 
rescreened once upon consultation with, and approval by, the Sponsor or its designee. 
Rescreened subjects will be assigned a new subject number, and both subject numbers will be 
linked to the subject, and a new informed consent with the new subject number should be signed. 
Screening assessments specified in Section 1.3 should be repeated if subjects are rescreened. The 
following assessments would NOT need to be repeated if rescreening occurs within 2 to 8 weeks 
of the original screening date: Safety labs (if within 2 weeks); Urodynamics (if within 8 weeks). 
See Section 8.2.3 for information on valid historical urodynamic assessments. 
 
 


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6. 
STUDY TREATMENTS 
6.1. 
Study Drugs Administered 
All eligible subjects enrolled into the study will receive a single double-blind treatment of either 
URO-902 or placebo based on the cohort they are enrolled in. 
URO-902 (24 mg or 48 mg) or matching placebo will be administered as intradetrusor injections 
via cystoscopy. 
For Cohort 1, a single treatment of URO-902 24 mg or placebo will be administered. Based on 
the unblinded review of observed safety data from all subjects in Cohort 1 up to Week 6 
(including subjects who prematurely exited the study prior to Week 6), the DSMB will make the 
recommendation to proceed with Cohort 2. Randomization into Cohort 2 will begin only after 
the DSMB has recommended it is safe to proceed. 
x Cohort 1: URO-902 24 mg or matching placebo (phosphate buffered saline with 20% 
sucrose [PBS-20%]) 
x Cohort 2: URO-902 48 mg or matching placebo (PBS-20%) 
See Table 1 for a summary on study drugs. 


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Table 1: 
Summary of Study Drugs 
Study Drug Name 
URO-902 
Matched Placebo 
Identity of 
Formulation 
URO-902 Drug Product  
Phosphate Buffered Saline with 
20% Sucrose (PBS-20%) 
Dosage Formulation 
Dose 
24 mg or 48 mg 
Placebo 
Route of 
Administration  
Intradetrusor injection via cystoscopy 
Intradetrusor injection via 
cystoscopy 
Dosing Instructions 
Single treatment administered by the 
investigators or study site personnel 
qualified to perform cystoscopy. 
Single treatment administered by 
the investigators or study site 
personnel qualified to perform 
cystoscopy. 
Packaging and 
Labeling 
Manufacturer 
6.2. 
Day of Treatment Criteria 
For each subject in Cohort 1 or Cohort 2, a single treatment will be administered on Day 1 
(Visit 3) after fulfillment of the following “day of treatment criteria”: 
x Negative urine dipstick reagent strip test (for nitrates and leukocyte esterase) 
x If evaluated, negative urinalysis/urine culture/sensitivity results for a possible UTI have 
been reviewed 
x Subject is asymptomatic for a UTI, in the opinion of the investigator 
x No presence of bladder stones prior to or at cystoscopy 
x Investigator continues to deem that no condition or situation exists which, in the 
investigator’s opinion, puts the subject at significant risk from receiving URO-902 


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6.3. 
Treatment Administration 
x If a subject is taking any anticoagulants or anti-platelet drugs, consult with the subject’s 
primary care physician (or internist, cardiologist, etc), as deemed clinically necessary by 
the investigator, if the subject can discontinue these drugs for 2-3 days prior to the 
intradetrusor injections treatment and on the day of treatment. Subjects on an 
anticoagulant and/or anti-platelet therapy must be managed appropriately to decrease the 
risk of bleeding, per the clinical judgment of the investigator. 
x All subjects must receive prophylactic antibiotics at a minimum on Day 1 prior to 
treatment administration and for 1 additional day post-treatment. At the investigator’s 
discretion, subjects can receive prophylactic antibiotics up to 3 days prior to treatment per 
local site practice. 
x Prior to administration of study treatment, subjects will be instructed to void their bladder 
and then assume a supine position. 
x Use of anesthesia before treatment administration will be determined by the investigator. 
All study procedures are to be conducted using the appropriate antiseptic technique per 
local site practice for a cystoscopy. After disinfection of the urethral meatus, the 
following options are strongly recommended: 
o Lubricating gel, with or without local anesthetic, to facilitate insertion of the 
sterile, single-use transurethral catheter per local site practice 
o For all subjects, local anesthesia instillation in the bladder: 
 Instillation into the bladder of 1% to 4% lidocaine (or similar acting local 
anesthetic) prior to the procedure 
 Instillation solution should remain in the bladder for at least 15 minutes to 
achieve sufficient anesthesia; afterwards, the bladder will be drained of 
lidocaine, rinsed with saline, and drained again 
x A flexible or rigid cystoscope will be used for administration of study treatment. Per local 
site practice lubricating gel will be used to insert the cystoscope. The bladder will be 
instilled with a sufficient amount of saline to visualize the study injections. One syringe 
of appropriate size to hold a minimum of 12 mL of study drug and one syringe of 
appropriate size to hold a minimum of 1 mL PBS-20% flushing solution will be prepared 
and ready for treatment administration (refer to Section 6.5 as needed). The injection 
needle will be primed with approximately 0.5 mL of study drug. The 12 mL of study drug 
should be administered as 20 injections, each approximately 0.6 mL. Under direct 
visualization via cystoscopy, injections will be distributed evenly across the detrusor wall 
and spaced approximately 1 cm apart, avoiding the bladder dome and trigone.  


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x To administer study drug from the syringe, the needle should be inserted approximately 
2 mm into the detrusor for each injection. For the final injection site, a sufficient amount 
of PBS-20% (from the 1-mL syringe) will be pushed through the injection needle to 
ensure delivery of the remaining amount of study drug. 
x After injections are administered, the saline used for visualization must not be drained 
from the bladder to allow subjects to demonstrate the ability to void prior to leaving the 
clinic. Subjects must remain in the clinic until a spontaneous void has occurred, and to 
complete post-treatment assessments including observation as well as pharmacokinetic 
sample collection. 
x Subjects will be instructed to contact the study site to report any adverse events that occur 
within 48 hours following administration of study treatment. 
6.4. 
Preparation/Handling/Storage/Accountability  
Clinical supplies must be stored in a secure, limited-access location under the storage conditions 
specified on the label. Refer to the appropriate study manual for the storage and handling of 
URO-902 and placebo. In the event a subject does not fulfill the day of treatment criteria 
(Section 6.2), likely due to a suspected UTI that requires antibiotic treatment, study drug (URO-
902 or placebo) can remain in the refrigerator (2° to 8°C, in the original vial) as per instructions 
in the study manual to enable the subject to reschedule and return for the treatment visit (Day 1). 
Receipt and dispensing of study treatment must be recorded by an authorized person at the study 
site. 
Clinical supplies may not be used for any purpose other than that stated in the protocol. 
The investigator is responsible for keeping accurate records of the clinical supplies received from 
the Sponsor or designee, the amount dispensed and the amount remaining at the conclusion of 
the study. These records will be monitored throughout the study. 
For all sites, the local country Sponsor personnel or designee will provide appropriate 
documentation that must be completed for drug accountability and return. 
6.5. 
Measures to Minimize Bias: Randomization and Blinding 
To maintain blinding of the treatment groups, neither the investigator/physician subinvestigator 
(injector) nor site personnel who are involved in evaluating or injecting the subject should 
prepare the study drug. An additional individual (eg, unblinded pharmacist or nurse) with no 
subject evaluation or treatment involvement must be identified at each investigative site to 
prepare the study drug and the PBS-20% flushing solution. The responsibility of this individual 
will be to properly prepare the injection syringes with the accurate randomized study drug as per 
the volumes indicated by the Sponsor. They will draw the study drug into the syringes to be 
injected and dilute as needed as per instructions in the appropriate study manual. The study 
manual will be provided by the Sponsor/designee before the study start. 


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If an interim analysis(es) is/are performed (Section 9.8.1), the subject and the investigator or 
delegate(s) who are involved in the treatment or clinical evaluation of the subjects will remain 
blinded to the treatment assignments. Limited personnel from the Sponsor will be unblinded 
(double-blind, sponsor-open design). Sponsor personnel/designees who are directly involved 
with site interactions and communications (eg, study data manager and site monitors) will remain 
blinded to subject level treatment assignment. 
In both cohorts, subjects will be randomized in a 2:1 ratio to receive either URO-902 (24 mg or 
48 mg) or placebo. Each cohort will be randomized separately, and enrollment will be sequential, 
starting with Cohort 1 and followed by Cohort 2. At the Randomization Visit (Visit 2), subjects 
in both Cohort 1 and Cohort 2 will be randomized centrally to receive either a single treatment of 
URO-902 or matching placebo.  
In Cohort 1a, randomization will be stratified by baseline UUI episodes per day (≤ 3 vs. > 3 UUI 
episodes per day) and presence or absence of DO (as determined by the investigator) as per the 
original protocol. Subjects will be enrolled into Cohort 1b following implementation of 
Amendment 1 and updates to the interactive web response system (IWRS) system. In Cohort 1b 
and Cohort 2, randomization will be stratified by baseline UUI episodes per day and previous use 
of onabotulinumtoxinA (naïve vs. prior use).  
For Cohorts 1a, 1b, and 2, stratification factor for baseline UUI episodes is based on the average 
number of UUI episodes per day recorded over the 3-day diary period at baseline (ie, ≤ 3 vs. 
> 3 UU1 episodes per day).  
For Cohort 1a, the urodynamic assessment where an evaluation of the presence or absence of DO 
is performed to be used as a randomization factor, must be conducted at Visit 2 prior to 
performing the actual randomization procedure. For subjects with a valid historical urodynamic 
study (per protocol Section 8.2.3), the presence or absence of DO would already be known. 
For Cohorts 1b and 2, onabotulinumtoxinA treatment for urological indications must have 
occurred at least 12 months prior to starting the Screening Bladder Diary. Enrollment for 
subjects with previous onabotulinumtoxinA use will be capped at 50% of the total subject 
population per cohort. Subjects randomized to Cohort 1a will be assigned to the 
onabotulinumtoxinA naïve stratum for all analyses. 
Subjects will be centrally assigned to randomized study drug using an IWRS and the 
randomization schedule generated by the sponsor or designee. Before screening is initiated at 
each site, login information and directions for the IWRS will be provided. 
The IWRS will be programmed with blind-breaking instructions. In case of an emergency, the 
investigator or designee has the sole responsibility for determining if unblinding of a subject’s 
study drug assignment is warranted. Subject safety must always be the first consideration in 
making such a determination. If the investigator decides that unblinding is warranted, the 
investigator should make every effort to contact the Sponsor or designee prior to unblinding a 


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subject’s study drug assignment unless this could delay emergency treatment of the subject. If a 
subject’s study drug assignment is unblinded, the Sponsor or designee must be notified within 24 
hours after breaking the blind. The date and reason that the blind was broken must be recorded in 
the source documentation. 
6.6. 
Study Drug Compliance 
Study treatment will be administered by the investigator or physician subinvestigator qualified to 
perform intradetrusor injections. 
Study drug compliance will be closely monitored by counting the number of vials dispensed and 
returned. The study site personnel will make every effort to collect all unused study drug and 
empty vials. 
The study site will keep an accurate drug disposition record that specifies the amount of study 
drug administered to each subject and the date of administration. 
6.7. 
Dose Modification 
No dose modification of study drug is permitted. 
6.8. 
Drug After the End of the Study 
Not applicable. 
6.9. 
Prior and Concomitant Therapy 
The use of any concomitant medication, prescription or over-the-counter, is to be recorded on the 
subject’s electronic case report form (eCRF) at each visit along with the reason the medication is 
taken, the dates of administration, and the dose. 
6.9.1. 
Prohibited Drugs and Washout 
Section 10.7 provides a list of medications prohibited before the study and during the study (until 
Week 24) and the associated washout periods. If there is a clinical indication for any therapy that 
is specifically prohibited during the study, discontinuation from the study may be required. The 
investigator should discuss any questions regarding this with the Sponsor’s designated Medical 
Monitor. The final decision on any supportive therapy rests with the investigator and/or the 
subject’s primary physician. However, the decision to continue the subject in the study requires 
the mutual agreement of the investigator and the Sponsor’s designated Medical Monitor. Consult 
the Sponsor’s designated Medical Monitor if there is any uncertainty regarding subject use of a 
particular drug or drug class. 


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Starting at Week 24 and after, the OAB treatments listed in Section 10.7 are no longer prohibited 
and can be prescribed to a subject upon her request and in accordance with the investigator’s 
clinical judgment. See Section 6.9.2 for details. 
6.9.2. 
Permitted Treatments 
With the exception of the agents described in Section 10.7, any other concomitant medication 
deemed necessary for the welfare of the subject during the study may be given at the discretion 
of the investigator. If the permissibility of a specific medication/drug is in question, please 
contact the Sponsor’s designated Medical Monitor. Any medication or vaccine (including over-
the-counter or prescription medicines, vitamins, and/or herbal supplements) that the subject is 
receiving at the time of enrollment, start of screening, or receives during the study must be 
recorded in the eCRF. 
Furthermore, starting at Week 24 and after (at clinic visit, phone [or other virtual technology] 
visits or between visits), subjects can request and receive additional OAB treatment(s) such as 
those listed in Section 10.7 (see Section 6.9.1) at the clinical discretion of the investigator. The 
following criteria should be followed in order for subjects to receive additional OAB 
treatment(s): 
x Subject initiates a request for additional OAB treatment 
x Ensure subject’s last available OAB symptoms are recorded, as determined by the most 
recent 3-day patient Bladder Diary (if not already recorded) 
x Investigator deems the additional OAB treatment is appropriate and that no condition or 
situation exists which, in the investigator’s opinion, puts the subject at significant risk 
from treatment. 
Subjects who receive additional OAB treatment(s) at Week 24 or after will only be followed to 
assess adverse events at any future telephone (or other virtual technology) visits (Week 36 and/or 
Week 48). No bladder diaries will be collected, and no efficacy assessments will be performed 
once a subject receives additional OAB treatment. 
Additional OAB medications will not be provided by the Sponsor. 


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7. 
DISCONTINUATION OF STUDY AND SUBJECT 
DISCONTINUATION OR WITHDRAWAL 
A premature discontinuation will occur if a subject who signs the informed consent form (ICF) 
and is randomized ceases participation in the study, regardless of circumstances, before the 
completion of the protocol-defined study procedures. 
7.1. 
Subject Discontinuation/Withdrawal from the Study 
x A subject may choose to withdraw from the study at any time at her own request or may be 
withdrawn at any time at the discretion of the investigator or Sponsor for safety, behavioral, 
compliance, or administrative reasons. 
x If a subject withdraws consent for disclosure of future information, the Sponsor or designee 
may retain and continue to use any data collected before such a withdrawal of consent. 
x If a subject withdraws from the study, she may request destruction of any blood or urine 
samples taken and not tested, and the investigator must document this in the site study 
records and communicate this request to the Sponsor study team so that appropriate measures 
may be taken to ensure destruction of the materials. 
x Subjects who withdraw from the study will not be replaced (enrollment accounts for a 
predetermined dropout rate). 
The Week 24/Exit Clinic Visit (Visit 8) assessments as specified in the Schedule of Assessments 
(Section 1.3) will be completed when a subject withdraws or is withdrawn from the study, if 
possible. 
Reasons for discontinuation from the study include the following: 
x AE 
x Lack of efficacy 
x Noncompliance 
x Withdrawal of consent 
x Lost to follow-up 
x Physician decision 
x Protocol deviation 
x Pregnancy 
x Death 
x Administrative issues (eg, site closure) 
x Other 


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7.2. 
Lost to Follow-Up 
Should a subject fail to attend a required study visit, the site should attempt to contact the subject 
and re-schedule the missed visit as soon as possible. The site should also counsel the subject on 
the importance of maintaining the assigned visit schedule and ascertain whether or not the 
subject wishes to and/or should continue in the study based on previous non-compliance. In 
cases where the subject does not return for the rescheduled visit or cannot be reached to 
reschedule the missed visit, the site should make every effort to regain contact with the subject 
so that they can appropriately be withdrawn from the study with a primary reason of “Lost to 
Follow-up”, including at least three documented attempts to contact the subject (ie, phone, email, 
or certified letter). Efforts to establish the possible reason for discontinuation should be 
documented. 
7.3. 
Early Study Termination 
The Sponsor reserves the right to terminate the study for any reason. The study may be 
terminated early if the extent (incidence and/or severity) of emerging effects/clinical endpoints is 
such that the risk/benefit ratio to the study population as a whole is unacceptable. In addition, 
further recruitment in the study overall or at a particular study site may be stopped due to 
insufficient compliance with the protocol, Good Clinical Practice and/or other applicable 
regulatory requirements, procedure-related problems or the number of discontinuations for 
administrative reasons is too high. 


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8. 
STUDY ASSESSMENTS AND PROCEDURES 
Study procedures and their scheduled timepoints are summarized in the Schedule of Assessments 
(Section 1.3). Site personnel should note the following: 
x Protocol waivers or exemptions are not allowed; however, subjects who fail to meet 
eligibility criteria may be rescreened once, as appropriate (See Section 5.4). Any notable 
protocol deviations should be noted and raised to the Sponsor’s or designee’s attention. 
x Immediate safety concerns should be discussed with the Sponsor’s designated Medical 
Monitor immediately upon occurrence or awareness to determine if the subject should 
continue or be discontinued from the study. 
x Adherence to the study design requirements, including those specified in the Schedule of 
Assessments, is essential and required for study conduct. 
x Evaluations should be performed by the same evaluator throughout the study whenever 
possible. 
8.1. 
Screening Procedures 
Screening procedures can commence once the informed consent and data 
authorization/protection form have been obtained. Screening will be considered to have started at 
the time of the first screening activity or procedure and completed when all the required 
inclusion/exclusion criteria have been met and the subject is randomized. Screening procedures 
should be completed as soon as possible, with a maximum of a 35-day window. There is no 
minimum duration for screening procedures and randomization as long as the subject undergoes 
the required protocol-defined assessments at each respective visit and is fully evaluated for 
eligibility based on the clinical judgment of the investigator. 
All screening evaluations must be completed and reviewed to confirm that potential subjects 
meet all eligibility criteria. The investigator will maintain a screening log to record details of all 
subjects screened and to confirm eligibility or record reasons for screening failure, as applicable. 
Inclusion and exclusion criteria will be reviewed by the investigator or qualified designee at the 
Screening and Randomization Visits (Visits 1 and 2, respectively). 
For screen failure procedures, see Section 5.4. 
8.1.1. 
Informed Consent 
Documented consent must be obtained from each potential subject prior to participating in any 
study procedures. Refer to Section 10.1.3 for a detailed description of the informed consent 
process, including the voluntary nature of clinical study participation and the subject’s right to 
withdraw consent at any time. 


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8.1.2. 
Assignment of Subject Number 
All subjects who sign the ICF will be assigned a unique subject number by IWRS. This number 
will be used to identify the subject throughout the study. Subjects who are rescreened will be 
assigned a new subject number. If a new subject number is assigned, the subject will be linked to 
both subject numbers. 
8.1.3. 
Medical History 
A medical history will be obtained by the investigator or qualified designee. Medical history will 
include all chronic and ongoing conditions, regardless of year diagnosed, surgical history, and 
substance abuse history. All events occurring after the subject signs the ICF will be recorded as 
adverse events. 
8.1.4. 
Demographics 
Demographic data collection will include sex, age, race, and ethnicity. 
8.1.5. 
Prior and Concomitant Medications 
Any prior OAB medications (over the past 3 years), any other prior medications used up to 
6 months before the Screening Visit, as well as ongoing (concomitant) medications will be 
recorded beginning at the signing of the ICF and continuing until the Week 48 Safety Follow-up 
assessment. 
If additional OAB medication(s) is used or procedures are performed at Week 24 or after (see 
Section 6.9.2), these must be recorded in the appropriate eCRF form. 
8.2. 
Efficacy Assessments 
Efficacy assessments will be collected as outlined in the Schedule of Assessments (Section 1.3) 
in the form of a 3-day Bladder Diary. Information collected in the diaries will be used for 
assessment of the exploratory efficacy endpoints related to the number of UUI episodes, 
micturitions, urgency episodes, and UI episodes per day as well as one 24-hr period to record 
volume voided of urine. 
Subjects will complete questionnaires at the site during the study visits specified in the Schedule 
of Assessments (Section 1.3) before vital signs and blood draws to assess subject-perceived 
symptom relief and health-related quality of life. The patient-reported outcome (PRO) 
questionnaires are provided in Section 10.9. Urodynamic parameters will also be measured. 
Additional information on the Bladder Diary, questionnaires, and urodynamic parameters is 
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8.2.1. 
Bladder Diaries 
The Bladder Diary is used by subjects to record the frequency of daily OAB symptoms including 
all micturitions, urgency, UUI, UI, and main reason for incontinence, and volume voided per 
micturition (over one 24-hour period) by selecting the respective box for each symptom/event 
occurring during the course of a given day and night. 
Bladder diary must be completed by subjects for any 3 consecutive days in the 7 days prior to the 
visit (for screening only, it can be completed for 3 consecutive days at any time during the 
screening and randomization period); total volume voided must be collected for one 24-hour 
period during the 3-day diary collection period. Please refer to Section 10.7 for washout periods 
for prohibited medications. The Screening Bladder Diary should be started only after washout of 
prohibited medications is complete. 
A “Diary Day” is defined as the time between when the subject gets up for the day each morning 
(ie, the time the subject got up for the day yesterday to the time the subject got up for the day 
today; approximately a 24-hour period). 
To be eligible for the study, subjects must have a minimum of 3 consecutive Diary Days during 
the screening and randomization period (at any time during the period) and be capable in the 
investigator’s opinion of maintaining compliance with the diary requirements, including the 
measurement and recording of urine volume, as required throughout the course of the study. 
At all study visits, the site staff should inquire whether the subject had any difficulties with the 
diary and address any questions subjects may have. Instructions for proper completion of the 
diary should be re-reviewed. Subjects will be trained to enter data immediately following each 
event (in real time) and to input data from any “missed” events as soon as they are able. They 
will review and confirm that data from all events occurring within the preceding Diary Day 
(approximately 24 hours) have been entered at a consistent time each morning (eg, upon getting 
up for the day). 
Responses to the Bladder Diary will be reviewed by the site staff to assess whether subjects are 
capable of completing the diary and if subjects meet eligibility criteria. The daily averages for 
micturitions, urgency episodes, UUI episodes, and UI episodes will be calculated as average of 
the total by the number of events on Diary Days. Subjects who do not meet the OAB entry 
criteria will be excluded from the study. 
Note that diaries must be completed within 7 days prior to the protocol-defined timepoint 
regardless of any visit delay due to COVID-19. If a visit will be delayed per the COVID-19 
mitigation window, the site should contact the subject to ensure that the diary completion occurs 
per the protocol-defined timepoints and not within the 7 days prior to the COVID-delayed visit.  
More detailed instructions on the diary and instructions for use are provided in the appropriate 
study manual. 


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8.2.2. 
Patient-Reported Outcomes 
The subject’s perception of the level of impairment in functioning and well-being associated 
with the symptoms of OAB will be assessed using subject-completed questionnaires as specified 
below. Questionnaires should be administered prior to the subject undergoing any invasive 
procedure for any study visit, prior to vital signs measurement, and prior to study treatment at the 
Day 1 Visit. The versions of the questionnaires provided in the protocol are samples and will be 
replaced. Additional information on administering and completing these questionnaires is 
outlined in Section 10.9. These include the following questionnaires: 
x Urinary Incontinence-Specific Quality-of-Life Instrument (I-QOL) total summary score 
x OAB Questionnaire (OAB-q) score 
x Patient Global Impression of Change (PGI-C) scale score 
Examples of each of the PRO measures are provided in Section 10.9. 
8.2.3. 
Urodynamic Parameters 
Urodynamic assessments will only be performed at baseline after confirmation of subject 
eligibility during the Randomization Visit (Visit 2) prior to performing the actual randomization 
procedure. A historical urodynamic study, performed no more than 90 days prior to the first day 
of screening, may serve as the baseline urodynamic assessment if the criteria detailed below are 
satisfied.  
Historical urodynamic study may be substituted for the baseline urodynamic assessment, if the 
following 3 criteria are met: 
x Historical urodynamic study was obtained no more than 90 days prior to the first day of 
screening 
x Historical urodynamic results are available for evaluation by the central reader 
x Subject was not being treated with any OAB medication or had discontinued OAB 
treatment in accordance with Exclusion Criterion #3 
In case of subjects being rescreened when a historical urodynamic assessment was used for the 
original screening, it will NOT need to be repeated if rescreening still occurs within 90 days of 
the valid historical urodynamic assessment. If rescreening occurs more than 90 days since the 
historical urodynamic assessment, a new urodynamic test will need to be performed prior to 
randomization as the baseline assessment.  
At Week 12, all subjects will undergo a second urodynamic assessment.  


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Conduct of urodynamic testing will be standardized across all study sites and will be according 
to ICS standard guidelines [Homma 2002]. 
Urodynamic data will be reviewed by an independent central reviewer to determine final values 
for analysis purposes for the parameters listed below. The investigator, physician 
subinvestigator, or trained technician will perform urodynamic procedures on each subject, then 
interpret and record the results on the calculation worksheet. The worksheet and the tracings 
should be signed and dated by the investigator or physician subinvestigator. A test trace will be 
submitted by each site. A copy of the worksheet, along with the printed tracings will be 
submitted to the central reader. Only the central reader’s interpretation of the results will be 
recorded in the eCRF for final analysis. Study sites will not typically be informed of any 
differences between their urodynamic values and the urodynamic values determined by the 
central reviewer. However, study sites may receive feedback if persistent methodological issues 
arise per the central urodynamic reader. Note that the investigator does not need to wait for the 
results of the baseline urodynamic assessment to rule out bladder outlet obstruction if there are 
no other signs or symptoms of obstruction, in the investigator’s assessment, including elevated 
PVR volume (see Exclusion Criterion 13). 
The following urodynamic parameters are to be measured: 
x Cystometric volume at 1st sensation to void (FSV) 
x Maximum cystometric capacity (MCC) 
x Maximum detrusor pressure during the storage phase (corrected PdetMaxStorage) 
x Presence/absence of the first involuntary detrusor contraction (IDC) and, if present: 
o Volume at first IDC (Vol@1stIDC)  
o Maximum detrusor pressure during the first IDC (corrected Pdet@1stIDC) 
Additional related instructions will be provided in the appropriate study manual. 
8.3. 
Pharmacokinetic Assessments 
Urine and blood samples for hSlo cDNA assessment will be collected pre-treatment from 
subjects on Day 1 (Visit 3; treatment administration), 2 hours (± 1 hour) post-treatment on 
Day 1, Week 6 follow-up clinic visit (Visit 5), and Week 24 follow-up clinic/exit visit (Visit 8). 
If a specimen(s) is still positive at Week 24, per the clinical discretion of the investigator, a 
subject may be instructed to return at Week 36 (Visit 9) and, if necessary, Week 48 (Visit 10) 
until the specimen(s) result is negative for hSlo cDNA. Pharmacokinetic assessment is to be 
performed at the Early Withdrawal Visit for subjects who withdraw early from the study. 
Information on procedures for collection, handling, storage, and shipping the samples will be 
provided in a separate study manual. 


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8.4. 
Safety Assessments 
Planned timepoints for all safety assessments are provided in the Schedule of Assessments 
(Section 1.3). Immediate safety concerns should be discussed with the Sponsor’s designated 
Medical Monitor immediately upon occurrence or awareness to determine if the subject should 
continue or discontinue study drug. 
8.4.1. 
Vital Signs 
x Vital signs, including blood pressure (mmHg), heart rate (by pulse; beats per minute), 
respiration rate (breaths per minute), and body temperature (°C) will be assessed at the 
timepoints specified in the Schedule of Assessments (Section 1.3). 
x Blood pressure and pulse will be measured after the subject has been resting in a seated 
position for 5 minutes, after questionnaires have been administered, and before blood 
draws. 
x Blood pressure measurements will be taken on the same arm and by the same site staff 
throughout the study, if possible.  
x The same method for assessing temperature should be used at all visits for a particular 
subject. 
x Body weight will be measured with subjects in street clothing with jacket/coat and shoes 
removed, using the same scale throughout the study, if possible. 
x Standing height will be measured at screening only. 
8.4.2. 
Physical Examinations 
Brief physical examinations will include examination of the heart, lungs, abdomen, and visual 
pelvic examination. In addition, any organ system in which a previous abnormality was noted 
during screening or a subject has a complaint or AE will be examined. 
8.4.3. 
Electrocardiograms 
A 12-lead ECG will be performed in the supine position at the visits specified in the Schedule of 
Assessments (Section 1.3) using an ECG machine that automatically calculates the heart rate and 
measures PR, QRS, QT, and QTc intervals. ECGs will be read by trained personnel at the study 
site or appropriate delegates who are medically qualified. 
8.4.4. 
Post-Void Residual Volume 
The volume of urine that remains in the bladder after voiding (PVR) is an objective measurement 
that may serve as a proxy for impaired ability to void. The physician should assess subjects with 
an increase in PVR volume to determine whether an AE should be reported. (Refer to 
Section 8.5.6 for additional information on reporting AEs of urinary retention associated with 
increased PVR.)  


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The PVR assessment will be performed via ultrasound at the visits indicated in the Schedule of 
Assessments (Section 1.3). Note that since Visit 7 (Week 18) is a telephone (or other virtual 
technology) visit, subjects will not be seen in the clinic for 12 weeks between Visit 6 (Week 12) 
and Visit 8 (Week 24). Should the investigator identify a clinically relevant elevation in PVR 
volume at Visit 6, an unscheduled clinic visit should be performed at the investigator’s discretion 
to repeat the urine PVR assessments and urine dipstick. All efforts should be made to ensure the 
same device and operator are used for all PVR volume measurements for individual subjects.  
8.4.5. 
Clean Intermittent Catheterization 
The following guidance should be used for the initiation of CIC in this study. Sterile, single-use 
intermittent catheters should be used. Indwelling catheters should not be used in this study. 
Initiation of Clean Intermittent Catheterization 
As described above in the PVR section, CIC should be initiated per protocol when one of the 
following criteria is fulfilled: 
x PVR is ≥ 350 mL at any posttreatment visit, regardless of whether the subject reports 
associated symptoms 
x PVR is ≥ 200 mL and < 350 mL and the subject spontaneously reports associated 
symptoms (ie, voiding difficulties, sensation of bladder fullness) that in the opinion of the 
investigator requires CIC. 
The following will occur when initiating CIC (for elevated PVR as described above): 
1. 
CIC is implemented and the subject should be instructed to perform CIC using sterile, 
single-use catheters (which will be provided to the subject) 
2. 
An adverse event of urinary retention is recorded 
3. 
Central laboratory urine analysis and culture/sensitivity are performed as per routine 
requirements at each study visit 
4. 
The subject will be seen for a follow-up visit 1 week later where the PVR, associated 
symptoms, central laboratory urine analysis and culture/sensitivity will be reassessed. 
The investigator will determine whether the subject can then be followed per protocol 
scheduled study visits or whether additional follow-up visits should occur. 
Once CIC is initiated, the status of CIC use should be documented in the subject record at each 
visit (ie, use/nonuse). 
Cessation of Clean Intermittent Catheterization 
CIC should be discontinued when the following criteria are fulfilled: 
x the subject does not have any associated symptoms which in the opinion of the 
investigator require CIC AND 


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x the PVR is < 350 mL 
The date of discontinuation of CIC should be documented. If the investigator deems that CIC 
should not be discontinued even though the above criteria are fulfilled, then this should be 
documented, and the reason given. Upon discontinuing CIC, the subject will be seen for a 
follow-up visit 1 week later where the PVR, associated symptoms, central laboratory urine 
analysis and culture/sensitivity will be reassessed. The investigator will determine whether the 
subject can then be followed at regularly scheduled study visits or whether additional follow-up 
visits should occur based on PVR and/or associated symptoms. 
8.4.6. 
Clinical Safety Laboratory Assessments 
All protocol-defined laboratory assessments must be conducted in accordance with the 
laboratory manual and the Schedule of Assessments (Section 1.3). See Section 10.2 for the list of 
clinical laboratory tests to be performed and the Schedule of Assessments for the timing and 
frequency. Repeat or unscheduled samples may be taken for safety reasons or for technical issues 
with the samples. 
The clinical significance of test results will be evaluated as follows: 
x At screening, the investigator or physician subinvestigator will assess the clinical 
significance of any values outside the reference ranges provided by the laboratory, and 
subjects with abnormalities judged to be clinically significant will be excluded from the 
study. 
x The investigator or physician subinvestigator must review the laboratory report, document 
this review, and record any clinically relevant changes occurring during the study in the AE 
section of the eCRF. The laboratory reports must be filed with the source documents. 
Laboratory abnormalities associated with the underlying disease are not considered clinically 
significant unless judged by the investigator to have worsened or be more severe than 
expected for the subject’s condition. 
x All laboratory tests with values considered clinically significant during participation in the 
study should be repeated until the values return to normal or baseline or are no longer 
considered clinically significant by the investigator or Sponsor’s designated Medical 
Monitor. 
- If such values do not return to normal/baseline within a period of time judged reasonable 
by the investigator, the etiology should be identified and the Sponsor’s designated 
Medical Monitor notified. 
- If laboratory values from nonprotocol specified laboratory assessments performed at the 
institution’s local laboratory require a change in subject management or are considered 
clinically significant by the investigator (eg, SAE or AE or dose modification), then the 
results must be recorded in the eCRF. 
A urine dipstick will be performed at the study site at scheduled visits as noted in the Schedule of 
Assessments (Section 1.3) and when a subject presents with symptoms of a UTI at Unscheduled 


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Visits. If the urine dipstick is positive for leukocytes, nitrites, or blood cells, then a urinalysis and 
culture/sensitivity testing will be performed on the sample. The urinalysis should be performed at 
the central laboratory and the culture and sensitivity should be performed at the site’s designated 
local laboratory.  
If positive for nitrites and/or leukocyte esterase, indicating a possible infection, the subject 
should be treated with antibiotics per the clinical judgment of the investigator and in accordance 
with local site practice. Urine analysis, urine culture and sensitivity testing (performed from a 
sample collected on the same day as the dipstick) will be used to confirm the presence or absence 
of a UTI. If UTI is present, sensitivity testing will provide additional information to the 
investigator regarding pathogen susceptibility to the antibiotic selected for treatment based on the 
positive dipstick. 
x Screening urine dipstick reagent strip test results: If positive, the screening and 
randomization period can be extended to a total maximum of 35 days prior to randomization) 
in order to accommodate the additional time required for antibiotic treatment. Some of the 
screening procedures may be repeated, as deemed clinically necessary, at the discretion of 
the investigator (eg, repeat the Screening Bladder Diary if subject had a symptomatic UTI 
during initial diary collection). 
x Day 1 (treatment administration) urine dipstick reagent strip test results: Subjects with a 
dipstick positive for nitrites and/or leukocyte esterase on Day 1 (Visit 3) do not meet “day of 
treatment criteria” and therefore must not receive study treatment. The subject should be 
treated with antibiotics per the clinical judgment of the investigator and in accordance with 
local site practice and may then return for a rescheduled treatment Day 1 Visit within 10 days 
(but within a total maximum of 35 days of starting screening procedures). If the dipstick 
obtained at the rescheduled Day 1/treatment administration visit is negative for nitrites and 
leukocyte esterase and all “day of treatment criteria” are fulfilled, the subject can be treated. 
Urine cytology: Urine cytology is performed at the Screening Visit (Visit 1) only. Abnormal 
urine cytology suspicious for a urothelial malignancy should be investigated by the investigator 
according to local site practice, and only if such malignancy is ruled out should the subject be 
enrolled. Results of the investigation will be recorded in the source documents. 
8.4.7. 
Bladder and Kidney Ultrasound 
Abdominal ultrasound of the bladder and kidneys will be performed with the bladder at least half 
full. Subjects are excluded from this study if the screening ultrasound demonstrates the presence 
of bladder stones and/or intra-luminal filling defects of the bladder suggestive of malignancy. In 
the case of unclear new findings identified at the exit visit, an ultrasound which may be 
suggestive of stones (kidney, ureter, or bladder), other diagnostic measures must be performed in 
order to confirm the presence of stones, rule out bladder malignancy (eg, x-ray with or without 
contrast, urogram, computed tomography (CT) scan, magnetic resonance imaging (MRI) or 
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8.4.8. 
Immunogenicity Blood Sample (Optional) 
If the subject consents, blood samples for potential future immunogenicity testing will be 
collected at the timepoints specified in the Schedule of Assessments (Section 1.3) and stored for 
up to 5 years. If testing is not performed within 5 years of collection, the subject’s sample will be 
appropriately discarded. Sample collection is limited to subjects who are able to provide a blood 
sample prior to administration of study treatment on Day 1 (Visit 3) (Cohort 2 only).  
8.5. 
Adverse Events and Serious Adverse Events 
The definitions of an AE and SAE can be found in Section 10.3. 
AEs will be reported by the subject (or, when appropriate, by a caregiver, surrogate, or the 
subject’s legally authorized representative). 
The investigator and any qualified designees are responsible for detecting, documenting, and 
recording events that meet the definition of an AE or SAE and remain responsible for following 
up AEs that are serious, considered related to the study drug or study procedures, or that caused 
the subject to discontinue the study drug or the study (see Section 7). 
8.5.1. 
Time Period and Frequency for Collecting Adverse Event and Serious Adverse 
Event Information 
All AEs and SAEs will be collected from the signing of the ICF through 30 days after the end of 
the study at the timepoints specified in the Schedule of Assessments (Section 1.3), and as 
observed or reported spontaneously by study subjects. Medical occurrences that begin before the 
start of study drug, but after obtaining informed consent will be recorded in the AE section of the 
eCRF. 
All SAEs (including serious adverse events of special interest [AESIs]) will be recorded and 
reported on the eCRF within 24 hours of the study site personnel’s knowledge of the event. 
Marking the event as “serious” will automatically send required notifications for Sponsor or 
designee review. The investigator will also submit any updated SAE data within 24 hours of 
receipt of the information.  
Nonserious AESIs will be reported on the eCRF within 72 hours of the site’s knowledge of the 
information. 
Investigators are not obligated to actively seek AE or SAE information after conclusion of the 
study participation. However, if the investigator learns of any SAE, including a death, at any 
time after a subject has been discharged from the study, and he/she considers the event to be 
reasonably related to the study drug or study participation, the investigator must promptly notify 
the Sponsor or designee. 


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The method of recording, evaluating, and assessing causality of AEs and SAEs and the 
procedures for completing and transmitting SAE reports are provided in Section 10.3. 
8.5.2. 
Method of Detecting Adverse Events and Serious Adverse Events 
Care will be taken not to introduce bias when detecting AEs and SAEs. Open-ended and 
non-leading verbal questioning of the subject is the preferred method to inquire about AE 
occurrences. 
8.5.3. 
Follow-up of Adverse Events and Serious Adverse Events 
After the initial AE or SAE report, the investigator is required to proactively follow each subject 
at subsequent visits/contacts. All SAEs will be followed until resolution, stabilization, the event 
is otherwise explained, or the subject is lost to follow-up (as defined in Section 7.2). If a subject 
dies during participation in the study, the investigator will provide the Sponsor or designee with 
a copy of any available postmortem findings including an autopsy report with histopathology. 
The investigator is obligated to perform or arrange for the conduct of supplemental 
measurements and/or evaluations as medically indicated or as requested by the Sponsor or 
designee to elucidate the nature and/or causality of the AE or SAE as fully as possible. This may 
include additional laboratory tests or investigations, histopathological examinations, or 
consultation with other health care professionals. 
New or updated information will be recorded in the originally completed eCRF. As noted above, 
the investigator will submit any updated SAE data to the Sponsor or designee within 24 hours of 
receipt of the information. 
8.5.4. 
Regulatory Reporting Requirements for Serious Adverse Events 
x Prompt notification of an SAE by the investigator to the Sponsor or designee is essential so 
that legal obligations and ethical responsibilities towards the safety of subjects and the safety 
of a study drug under clinical investigation are met.  
x The Sponsor has a legal responsibility to notify both the local regulatory authority and other 
regulatory agencies about the safety of a study drug under clinical investigation. The Sponsor 
will comply with country-specific regulatory requirements relating to safety reporting to the 
regulatory authority, Institutional Review Boards (IRBs)/Independent Ethics Committees 
(IECs), and investigators.  
x Investigator safety reports must be prepared for suspected unexpected serious adverse 
reactions (SUSARs) according to local regulatory requirements and Sponsor policy and 
forwarded to investigators as necessary. 
x An investigator who receives an investigator safety report describing an SAE or other 
specific safety information (eg, summary or listing of SAEs) from the Sponsor or designee 
will review and then file it and will notify the IRB/IEC, if appropriate according to local 
requirements. 


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8.5.5. 
Disease-Related Events and/or Disease-Related Outcomes Not Qualifying as 
Adverse Events or Serious Adverse Events 
No disease-related events or outcomes are excluded from AE reporting. 
8.5.6. 
Adverse Events of Special Interest 
Selected nonserious and SAEs will be reported as AESIs. AESIs that also meet the definition of 
an SAE must be reported as described in Section 10.3. 
AESIs for this study include: 
x Adverse events consistent with urinary retention 
- An AE of urinary retention should only be recorded when a subject has a raised PVR that 
requires intervention with CIC or temporary placement of a urinary catheter according to the 
following criteria: 
a) subject has a PVR of ≥ 350 mL (regardless of symptoms), OR 
b) subject has a PVR ≥ 200 mL and < 350 mL and the subject reports associated symptoms, ie, 
sensation of bladder fullness or inability to void despite persistent effort, that in the investigator’s 
opinion require CIC. 
Note: An AE of residual urine volume should be recorded if, in the investigator’s opinion, the 
raised PVR is clinically significant but does not fulfill the above definition for urinary retention. 
x Adverse events suggestive of cystitis or UTI 
- An AE of UTI will be recorded if both the following criteria are fulfilled, regardless of subject 
symptoms: 
a) a positive urine culture result with a bacteriuria count of ≥ 105 CFU/mL  
(ie, 100 × 103 CFU/mL) 
b) leukocyturia of ≥ 5/hpf 
If a subject meets the criteria for the definition of a UTI, the investigator will record whether the 
UTI was “symptomatic” or “asymptomatic” on the AE eCRF. 
Note: If urinalysis/culture results are reported which, in the opinion of the investigator, are 
considered clinically significant but do not fulfill the above definition of a UTI, the findings 
should be recorded as AEs (eg, bacteriuria, leukocyturia). 


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8.5.7. 
Medication Errors 
Medication error refers to any unintended error in the dosing and/or administration of the study 
drug as per instructions in the protocol. Medication errors generally fall into 4 categories as 
follows: 
x Wrong study drug 
x Wrong dose (including dosing regimen, strength, form, concentration, amount) 
x Wrong route of administration 
x Wrong subject (ie, not administered to the intended subject) 
Medication errors include occurrences of overdose and underdose of the study drug. 
Overdose: Unintentional administration of a quantity of the study drug given per administration 
or per day that is above the maximum recommended dose according to the protocol. This also 
takes into account cumulative effects due to overdose (see Section 8.6 for treatment and 
reporting of overdose). For this study, any dose of URO-902 ≥ 20% higher than the dose that the 
subject has been randomized to is an overdose. There is no known antidote for an overdose.  
Underdose: No underdose is defined for this study. 
8.6. 
Treatment of Overdose 
In the event of an overdose (any dose of URO-902 ≥ 20% higher than the dose that the subject 
has been randomized to), the investigator or treating physician should: 
x Contact Sponsor’s designated Medical Monitor immediately 
x Closely monitor the subject for any AEs, SAEs, and laboratory abnormalities 
x Report all overdose events within 24 hours of awareness by the study site, in the eCRF 
according to Section 10.3, whether or not the overdose is associated with an AE 
x Document the quantity of the excess dose as well as the duration of the overdosing in the 
eCRF. 
Decisions regarding dose interruptions or modifications will be made by the investigator in 
consultation with the Sponsor’s designated Medical Monitor based on the clinical evaluation of 
the subject. 


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9. 
STATISTICAL CONSIDERATIONS 
This section contains a brief summary of the statistical analyses for this study; full details will be 
provided in the Statistical Analysis Plan (SAP). 
The randomized allocation schedule will be generated by the Sponsor or designee and 
implemented by the vendor of the study. Stratification will be performed across the study (not 
per site). The statistical analysis of the data obtained from this study will be the responsibility of 
the Sponsor or designee. At the end of the study, the official, final database will be frozen and 
unblinded after medical/scientific review has been performed, and data have been declared final 
and complete. The SAP will be approved prior to the final database lock. 
A subject is considered enrolled in the study at randomization. 
9.1. 
Statistical Hypotheses 
The study has no formal statistical primary endpoint hypothesis. The intent is to estimate the 
treatment effect of each dose group relative to placebo. For analysis of Cohort 1, subjects from 
Cohorts 1a and 1b will be combined. The primary analysis adjusted for randomization 
stratification will use the baseline UUI (≤ 3 vs. > 3 UUI episodes per day) and previous 
onabotulinumtoxinA use (naïve vs. prior use). Subjects in Cohort 1a will be assigned to the 
onabotulinumtoxinA naïve stratum. For the final analysis, the subjects receiving placebo from 
Cohort 1 and Cohort 2 will be pooled. 
9.2. 
Sample Size Determination 
For this exploratory study, the sample size was empirically chosen. With a total of 23 evaluable 
female subjects in each of the URO-902 treatment groups and 23 evaluable female subjects in 
the placebo group, this study would have approximately 55% power to detect a between-group 
difference of 1.8 episodes in the mean change from baseline to Week 12 in the number of UUI 
episodes per day between the URO-902 and placebo treatment groups (based on a 2-sample t-test 
with a 2-sided type I error rate of 0.05 and standard deviation [SD] of 2.86). Furthermore, the 
half-width of the confidence interval would be the following for different SD assumptions: 
SD 
N per Group 
Half-Width of Confidence 
Interval 
Lower  
Limit 
Upper  
Limit 
2.86 
23 
1.70 
0.10 
3.50 
3.00 
23 
1.78 
0.02 
3.58 
2.50 
23 
1.49 
0.31 
3.29 
In order to account for subject attrition (approximately 10%), the sample size was increased to 
26 subjects for each treatment group, amounting to a total of 78 randomized subjects required for 
exploratory efficacy analyses in both Cohort 1 (n = 39) and Cohort 2 (n = 39). 


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9.3. 
Analyses Populations 
The following analysis populations will be evaluated: safety, safety (modified), and intent-to-
treat exposed (ITT-E). 
ITT-E will be used for demographics, baseline characteristics, and efficacy analyses. The ITT-E 
population will consist of all subjects randomized and treated from Cohorts 1 and 2.  
The safety population will consist of all subjects from Cohorts 1 and 2 who were randomized and 
received study drug and will be used to analyze all safety endpoints based on actual treatment 
received. A separate safety population, safety (modified), will be created to assess safety 
excluding subjects who received additional OAB medication at Week 24 or after. 
Other populations, such as Intent-To-Treat and All Subjects Screened populations, will be 
defined in the SAP. 
9.4. 
Analysis Endpoints 
The exploratory study has no formal statistical primary analysis endpoint. 
9.4.1. 
Efficacy, Health Outcome, and Urodynamics Endpoints 
For the purposes of this study, the number of UUI episodes will be defined as the number of 
times a subject has marked “urge” as the main reason for the leakage as indicated on the Bladder 
Diary; regardless of whether more than one reason for leakage in addition to “urge” is checked. 
Average daily number of UUI episodes is calculated using the daily entries in the Bladder Diary, 
which is completed prior to each study visit. Average daily number of UUI episodes will be 
calculated as the total number of UUI episodes that occur on a Diary Day divided by the number 
of Diary Days in the Bladder Diary. 
A micturition is defined as “Urinated in Toilet” as indicated on the Bladder Diary. Average daily 
micturitions at each study visit will be calculated in the same manner as described above for UUI 
episodes. 
Urinary incontinence is defined as having any reason for leakage or “Accidental Urine Leakage” 
as indicated on the Bladder Diary. 
An urgency episode is defined as the “Need to Urinate Immediately” as indicated on the Bladder 
Diary. 
Exploratory Efficacy Endpoints are listed in Section 3.2.1. 


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9.4.2. 
Safety Endpoints 
Safety will be assessed via clinical review of all relevant safety parameters including AEs, 
clinical laboratory assessments, vital sign assessments, PVR volume, physical examination, 
ECGs, and concomitant medications and procedures. 
9.4.3. 
Other Endpoints 
Blood and urine hSlo cDNA concentrations. 
9.5. 
Statistical Analyses 
9.5.1. 
Statistical Methods for Efficacy Analysis 
Descriptive statistics will be used to evaluate the efficacy endpoints. For continuous efficacy 
endpoints, estimates of least squares means, standard error, and 95% confidence intervals (CI) 
will be presented for each treatment group and the difference between each dose group relative 
to placebo. Nominal p-values from comparisons from each dose group relative to placebo may 
be provided for descriptive purposes. 
Baseline will be defined as the diary assessments collected prior to randomization for all diary 
related efficacy endpoints and results of the questionnaires collected at the Day 1 Visit (Visit 3) 
for all health outcome endpoints. 
For the analysis of continuous change from baseline endpoints (eg, change from baseline in 
average daily number of UUI episodes, change from baseline in average daily number of 
micturitions, change from baseline in average daily number of UI episodes, change from baseline 
in average daily number of urgency episodes, change from baseline in average volume voided 
per micturition, change from baseline in average I-QOL total summary score, change from 
baseline in OAB-q score, and change from baseline in PGI-C score), a mixed-effect model for 
repeated measures (MMRM) with restricted maximum likelihood estimation will be used. 
Analysis will be stratified by the randomization factors assuming there are greater than 5 subjects 
per stratum. This model corrects for dropout and accounts for the fact that measurements taken 
on the same subject over time tend to be correlated, by using all available information on 
subjects within the same covariate set to derive an estimate of the treatment effect for a drop-out 
free population. 
The analysis model for each efficacy endpoint will include terms for treatment, visit, visit by 
treatment interaction term, and baseline score as covariates. If warranted, non-parametric 
methods will be used. For interim analyses, analysis of covariance (ANCOVA) may be 
performed instead of MMRM due to limited sample size. If there is a sufficient sample size, 
randomization factors will be considered for use in the model. Data for all visits will also be 
presented. 


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The proportion of subjects who has ≥ 50% reduction from baseline in UUI episodes at Week 12 
will be calculated for each treatment group. In addition, responder analyses will also be 
calculated for subjects who achieve ≥ 75% and 100% decrease in episodes of UUI at Week 12 
relative to baseline. The Cochran-Mantel-Haenszel (CMH) method will be utilized to compare 
the proportion of responders between the active and placebo groups by adjusting for the 
stratification factors. Data for all visits will also be presented. Further details of handling of 
missing data will be described in the SAP. 
For the urodynamic variables evaluated, only the independent central reviewer’s interpretation 
will be analyzed. 
9.5.2. 
Safety Analyses 
Safety analysis will be conducted using the safety population and summarized by treatment 
group. The treatment-emergent period will be defined as the period from initial exposure of the 
study drug through 48 weeks after treatment administration or the date of the initiation of another 
investigational agent or surgical intervention, whichever occurs first. Safety will be assessed 
through summaries of adverse events and clinical laboratory evaluations. 
AEs will be coded using the latest version of Medical Dictionary for Regulatory Activities 
(MedDRA; version 21.0 or higher). All reported AEs (whether treatment emergent or not) will 
be included in by-patient AE listings. The severity of all adverse events will be evaluated by the 
investigator. The incidence of the adverse events will be presented by system organ class and 
preferred term, relationship to study treatment, and severity. Treatment emergent AESIs and 
treatment related AESIs will also be summarized by system organ class (SOC) and preferred 
term (PT). A list of all AESIs can be found in Section 8.5.6. 
Laboratory data will consist of chemistry, hematology, and urinalysis data. Only data collected 
by the laboratory will be included in the analysis. 
Descriptive statistics of observed values and change from baseline for vital signs and post void 
residual volume will be listed and summarized. 
Supplemental safety analyses will be performed to separate subjects that did or did not receive 
additional OAB treatment at or after Week 24. Tables that are to be repeated with the alternative 
safety population will be described further in the SAP. 
9.6. 
Multiplicity 
All efficacy endpoints will be considered descriptive and no multiplicity adjustments will be 
performed for these endpoints. 


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9.7. 
Subgroup Analyses 
Subgroup analyses of endpoints may be conducted. Additional details of any planned subgroup 
analyses will be provided in the SAP. 
9.8. 
Planned Analyses 
9.8.1. 
Interim Analyses 
Two or more interim analyses may be conducted; when ≥ 50% of subjects in Cohort 1, and/or 
when ≥ 50% of subjects in Cohort 2 have completed at least 12 weeks of follow-up 
post-randomization (or prematurely exited the study prior to Week 12). Any interim analysis 
performed is for future planning purposes. 
The planned interim analysis will be conducted to evaluate the objectives of the protocol at 
Week 12. These analyses will be performed after the completion of the following steps: 
x All subjects in Cohorts 1 and 2 have completed Week 12 (or prematurely exited the study 
prior to Week 12) 
x All required database cleaning activities have been completed and final database release 
and freeze has been declared by data management 
x All criteria for unblinding the randomization code have been met. 
For any interim analysis, the subject and the investigator or delegate(s) who are involved in the 
treatment or clinical evaluation of the subjects will remain blinded to the treatment assignments. 
Limited personnel from the Sponsor will be unblinded (double-blind, sponsor-open design). 
Sponsor personnel who are directly involved with site interactions and communications (eg, 
study data manager and site monitors) will remain blinded to subject level treatment assignment. 
9.8.2. 
Final Analyses 
The planned final end-of-study analysis will be performed once the final clinical database lock 
(after all subjects complete the study or exit the study prematurely) has been achieved and 
treatment codes have been unblinded to the statistics and programming team. The clinical study 
report will be based on final analysis results. 
9.8.3. 
Data and Safety Monitoring Board 
A DSMB will be retained to assess, on an ongoing basis, all safety aspects of this study including 
unblinded review of safety data after all subjects reach Week 6 (or prematurely exited the study 
prior to Week 6) in Cohort 1 (URO-902 24 mg). Randomization into Cohort 2 will begin only 
after the DSMB has recommended it is safe to proceed. This committee will be an external 
independent DSMB that monitors safety. The detailed activities including meeting plans will be 


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described and documented in the DSMB Charter. A separate statistical analysis plan may be 
prepared for the DSMB. 


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10. 
SUPPORTING DOCUMENTATION AND OPERATIONAL 
CONSIDERATIONS 
10.1. 
Appendix 1: Regulatory, Ethical, and Study Oversight 
Considerations 
10.1.1. 
Regulatory and Ethical Considerations 
x This study will be conducted in accordance with the protocol and with the following: 
- Consensus ethical principles derived from international guidelines including the 
Declaration of Helsinki and Council for International Organizations of Medical Sciences 
(CIOMS) International Ethical Guidelines 
- Applicable International Conference on Harmonisation of Technical Requirements for 
Registration of Pharmaceuticals for Human Use (ICH)/International Organization for 
Standardization Good Clinical Practice (GCP) guidelines 
- Applicable laws and regulations 
x The protocol, protocol amendments, ICF, Investigator’s Brochure, and other relevant 
documents (eg, subject-oriented materials) must be submitted to an IRB/IEC by the 
investigator and reviewed and approved by the IRB/IEC before the study is initiated. 
x Any amendments to the protocol will require IRB/IEC approval before implementation of 
changes made to the study design, except for changes necessary to eliminate an immediate 
hazard to study subjects.  
x The investigator will be responsible for the following: 
- Providing written summaries of the status of the study to the IRB/IEC annually or more 
frequently in accordance with the requirements, policies, and procedures established by 
the IRB/IEC 
- Notifying the IRB/IEC of SAEs or other significant safety findings as required by 
IRB/IEC procedures 
- Providing oversight of the overall conduct of the study at the site and adherence to 
requirements of applicable local regulations, for example 21 Code of Federal Regulations 
(CFR), ICH guidelines, the IRB/IEC, and European regulation 536/2014 for clinical 
studies (if applicable) 
10.1.2. 
Financial Disclosure 
Investigators and subinvestigators will provide the Sponsor or designee with sufficient, accurate 
financial information as requested to allow the Sponsor or designee to submit complete and 
accurate financial certification or disclosure statements to the appropriate regulatory authorities. 
Investigators are responsible for providing information on financial interests during the course of 
the study and for 1 year after completion of the study. 


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10.1.3. 
Informed Consent Process 
x The investigator or his/her representative will explain the nature of the study to the subject or 
her legally authorized representative and answer all questions regarding the study.  
x Subjects must be informed that their participation is voluntary. Subjects or their legally 
authorized representatives will be required to sign a statement of informed consent that meets 
the requirements of 21 CFR 50, local regulations, ICH guidelines, Health Insurance 
Portability and Accountability Act requirements, where applicable, and the IRB/IEC or study 
center. 
x The medical record must include a statement that written informed consent was obtained 
before the subject was enrolled in the study and the date the written consent was obtained. 
The authorized person obtaining the informed consent must also sign the ICF. 
x Subjects must be re-consented to the most current version of the ICF(s) during their 
participation in the study. 
x A copy of the ICF(s) must be provided to the subject or the subject’s legally authorized 
representative. 
x Subjects who are rescreened are required to sign a new ICF. 
10.1.4. 
Data Protection 
x Subjects will be assigned a unique identifier. Any subject records or datasets that are 
transferred to the Sponsor or designee will contain the identifier only; subject names or any 
information which would make the subject identifiable will not be transferred. If a subject is 
rescreened and assigned a new subject number, both subject numbers for that individual will 
be linked. 
x The subject must be informed that her personal study-related data will be used by the 
Sponsor or designee in accordance with local data protection law. The level of disclosure 
must also be explained to the subject. 
x The subject must be informed that her medical records may be examined by Clinical Quality 
Assurance auditors or other authorized personnel appointed by the Sponsor or designee, by 
appropriate IRB/IEC members, and by inspectors from regulatory authorities. 
10.1.5. 
Committees Structure 
Not applicable. 
10.1.6. 
Posting Clinical Study Data 
Clinical study information will be posted on external registries and websites (eg, US National 
Institutes of Health’s website www.ClinicalTrials.gov and European Clinical Trial Register) as 
per applicable regulatory requirements. 


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10.1.7. 
Data Quality Assurance 
x All subject data relating to the study will be recorded on the eCRFs unless transmitted to the 
Sponsor or designee electronically (eg, laboratory data). The investigator is responsible for 
verifying that data entries are accurate and correct by physically or electronically signing the 
eCRF. 
x The investigator must maintain accurate documentation (source data) that supports the 
information entered in the eCRF. 
x The investigator must permit study-related monitoring, audits, IRB/IEC review, and 
regulatory agency inspections and provide direct access to source data documents. 
x The Sponsor is responsible for the data management of this study including quality checking 
of the data. Management of clinical data will be performed in accordance with applicable 
Sponsor-approved standards and data cleaning procedures to ensure the integrity of the data. 
x Study monitors will perform ongoing source data verification to confirm that data entered 
into the eCRF by authorized site personnel are accurate, complete, and verifiable from source 
documents; that the safety and rights of subjects are being protected; and that the study is 
being conducted in accordance with the currently approved protocol and any other study 
agreements, ICH GCP, and all applicable regulatory requirements. 
10.1.7.1. Study Site 
All clinical study documentation must be retained by the investigator until at least 2 years after 
the last approval of a marketing application in an ICH region (ie, United States, Europe, or 
Japan) and until there are no pending or contemplated marketing applications in an ICH region; 
or, if no application is filed or if the application is not approved for such indication, until 2 years 
after the investigation is discontinued and regulatory authorities have been notified. Investigators 
may be required to retain documents longer if required by applicable regulatory requirements, by 
local regulations, or by an agreement with the Sponsor. The investigator must notify the Sponsor 
before destroying any clinical study records. 
The study site and the record retainer should take measures in such a way that these records are 
not lost or abandoned during the designated period of preservation and that they are presented 
upon request. 
10.1.7.2. Institutional Review Board, Independent Ethics Committee, and Research 
Ethics Board (IRB/IEC/REB) 
The protocol, protocol amendments, informed consent form, Investigator’s Brochure, and any 
other relevant materials, including accompanying material to be provided to the subject (eg, 
advertisements, subject information sheets, or descriptions of the study used to obtain informed 
consent) will be submitted by the investigator to an IRB/REB/IEC. Initial approval from the 
IRB/REB/IEC must be obtained before starting the study and should be documented in a letter to 
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x Protocol number 
x Protocol version 
x Protocol date 
x Documents reviewed  
x Date on which the committee met and granted the approval 
Any amendments to the protocol will require IRB/REB/IEC approval before implementation of 
changes made to the study design, except for changes necessary to eliminate an immediate 
hazard to study subjects. 
The investigator will be responsible for the following (and must retain applicable notices and 
records as above under Study Site): 
x Providing written summaries of the status of the study to the IRB/REB/IEC’s annually or 
more frequently in accordance with the requirements, policies, and procedures 
established by the IRB/REB/IEC 
x Notifying the IRB/REB/IEC of serious adverse events or other significant safety findings 
as required by procedures established by the IRB/REB/IEC 
x Notifying the IRB/REB/IEC when the study has been completed or when his/her 
individual study site has been closed 
10.1.8. 
Source Documents 
x Source documents provide evidence for the existence of the subject and substantiate the 
integrity of the data collected. Source documents are filed at the investigator’s site. 
x Data entered in the eCRF that are transcribed from source documents must be consistent with 
the source documents or the discrepancies must be explained. The investigator may need to 
request previous medical records or transfer records, depending on the study. Current 
medical records must be available during the site monitor’s visit. 
x The required source documents are: 
− Subject identification (name, date of birth, sex) 
− Documentation that the subject meets eligibility criteria, (eg, history, physical 
examination, and confirmation of diagnosis to support inclusion and exclusion 
criteria) 
− Participation in the study (including study number) 
− Study discussed and date of informed consent 
− Dates of all visits 


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− Documentation that protocol-defined procedures were performed 
− Results of efficacy parameters, as required by the protocol 
− Start and end date (including dose regimen) of study drug (drug dispensing and 
return should be documented as well) 
− Record of all AEs and other safety parameters (start and end date, and causality 
and intensity as assigned by the investigator) 
− Concomitant medication (including start and end date) 
− Date of study completion and reason for early discontinuation, if applicable. 
10.1.9. 
Study and Site Closure 
The Sponsor designee reserves the right to close the study site or terminate the study at any time 
for any reason at the sole discretion of the Sponsor. Study sites will be closed upon study 
completion. A study site is considered closed when all required documents and study supplies 
have been collected, and a study-site closure visit has been performed. 
The investigator may initiate study-site closure at any time, provided there is reasonable cause 
and sufficient notice is given in advance of the intended termination. 
Reasons for the early closure of a study site by the Sponsor or investigator may include but are 
not limited to: 
x Failure of the investigator to comply with the protocol, the requirements of the IRB/IEC/REB 
or local health authorities, the Sponsor’s procedures, or GCP guidelines 
x Inadequate recruitment of subjects by the investigator 
x Discontinuation of further study drug development 
As stated above in Section 10.1.7, the investigator is responsible for notifying the IRB/IEC/REB 
upon closure of the site or completion of the study. 
10.1.10. 
Publication Policy 
x The Sponsor has proprietary interest in this study. Authorship and manuscript composition 
will reflect joint cooperation between multiple investigators and sites and Sponsor or 
designee personnel. Authorship will be established prior to the writing of the manuscript. As 
this study involves multiple centers, no individual publications will be allowed prior to 
completion of the final report of the multicenter study except as agreed with the Sponsor. 
x The Sponsor will comply with the requirements for publication of study results. In 
accordance with standard editorial and ethical practice, the Sponsor will generally support 
publication of multicenter studies only in their entirety and not as individual site data. 


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10.1.11. 
Compliance with Protocol 
The investigator is responsible for compliance with the protocol at the investigational site. A 
representative of the Sponsor will make frequent contact with the investigator and his/her 
research staff and will conduct regular monitoring visits at the site to review subject and study 
drug accountability records for compliance with the protocol. Protocol deviations will be 
discussed with the investigator upon identification, and the investigator will document the 
deviation in the subject records and/or any applicable eCRF(s). Significant protocol deviations 
will be reported to the IRB/IEC/REB according to the IRB/IEC/REB’s reporting requirements. 
10.2. 
Appendix 2: Clinical Laboratory Tests 
The clinical laboratory tests to be performed are detailed in Table 2. Subjects do not need to fast 
prior to laboratory testing. Refer to the Laboratory Manual for information on where testing 
should be performed (site, local laboratory, central laboratory). 
A urine dipstick will be performed at the study site at scheduled visits as noted in the Schedule of 
Assessments (Section 1.3) and when a subject presents with symptoms of a UTI at Unscheduled 
Visits. If the urine dipstick is positive for leukocytes, nitrites, or blood cells, then a urinalysis and 
culture/sensitivity testing will be performed on the sample. The urinalysis should be performed at 
the central laboratory and the culture and sensitivity should be performed at the site’s designated 
local laboratory.  














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Term 
Description 
ICF 
informed consent form 
ICH 
International Conference on Harmonisation of Technical Requirements for 
Registration of Pharmaceuticals for Human Use 
ICS 
International Continence Society 
IDC 
involuntary detrusor contraction 
IEC 
independent ethics committee 
I-QOL 
Incontinence-Specific Quality-of-Life Instrument 
IRB 
institutional review board 
ITT-E 
intent-to-treat exposed 
IWRS 
interactive web response system  
K 
potassium 
MCC 
maximum cystometric capacity 
MedDRA 
Medical Dictionary for Regulatory Activities 
MMRM 
mixed-effect model for repeated measures  
OAB 
overactive bladder 
OAB-q 
Overactive Bladder Questionnaire 
PBS 
phosphate buffered saline 
Pdet@1stIDC 
maximum detrusor pressure during the first involuntary detrusor 
contraction 
PdetMaxStorage 
maximum detrusor pressure during the storage phase  
PGI-C 
Patient Global Impression of Change 
PK 
pharmacokinetic 
PRO 
patient-reported outcome 
PT 
preferred term 
PUO 
partial urethral outlet 
PVR 
post-void residual 
REB 
research ethics board 
SAE 
serious adverse event 
SAP 
statistical analysis plan 
SD 
standard deviation 
SOA 
Schedule of Assessments 
SOC 
system organ class  
TEAE 
treatment-emergent adverse event 
UI 
urinary incontinence 


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Term 
Description 
URO-902 
formerly known as hMaxi-K or pVAX/hSlo. GMP manufactured double-
stranded DNA plasmid (pVAX) containing a cDNA insert encoding the 
pore-forming α subunit of the human smooth muscle Maxi-K channel, hSlo 
Urovant 
Urovant Sciences GmbH 
US 
United States 
UTI 
urinary tract infection 
UUI 
urge urinary incontinence 
Vol@1stIDC 
volume at first involuntary detrusor contraction 
10.5. 
Appendix 5: Study Tabular Summary 
 
Parameter 
Group 
Parameter 
Value 
Trial 
information 
Trial Title 
An Exploratory Phase 2a Study 
Evaluating the Efficacy and Safety of 
URO-902 in Subjects with Overactive 
Bladder and Urge Urinary 
Incontinence 
 
Clinical Study Sponsor 
Urovant Sciences GmbH 
 
Trial Phase Classification 
Phase 2a 
 
Trial Indication 
Overactive Bladder (OAB) and Urge 
Urinary Incontinence (UUI) 
 
Trial Indication Type 
Treatment 
 
Trial Type 
Multicenter, randomized, double-blind, 
placebo-controlled, single-treatment, 
2-cohort, dose-escalation 
 
Trial Length 
Up to 53 weeks  
 
Planned Country of Investigational Sites 
North America 
 
Planned Number of Subjects 
78  
 
FDA-Regulated Device Study 
No 
 
FDA-Regulated Drug Study 
Yes  
 
Pediatric Study 
No 
Subject 
information 
Diagnosis Group 
OAB with UUI 
Healthy Subject Indicator 
No 
 
Planned Minimum Age of Subjects 
40 
 
Planned Maximum Age of Subjects 
79 
 
Sex of Subjects 
Female 
 
Stable Disease Minimum Duration 
At least 6 months prior to screening 


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Parameter 
Group 
Parameter 
Value 
Treatments 
Investigational Therapy or Treatment 
URO-902 
 
Drug Type 
URO-902 consists of the gene for the α 
pore of the Maxi-K channel, hSlo, 
inserted into a plasmid vector, pVAX, 
called pVAX/hSlo 
 
Pharmacological Class of Invest. Therapy 
Gene therapy 
 
Dose per Administration 
24 or 48 
 
Dose Units 
mg 
 
Dosing Frequency 
Single administration 
 
Route of Administration 
Intradetrusor injections via cystoscopy 
 
Current Therapy or Treatment 
Current therapy or treatment for 
symptoms of OAB not allowed 
 
Added on to Existing Treatments 
Not applicable 
 
Control Type 
Placebo 
 
Comparative Treatment Name 
Not applicable 
Trial design 
Study Type 
Randomized, double-blind, placebo-
controlled, single-treatment 
 
Drug Model 
2-cohort, sequential, dose-escalation 
 
Planned Number of Arms 
Placebo and 2 dose groups of 
URO-902 
 
Trial is Randomized 
Yes 
 
Randomization Quotient 
2:1 
 
Trial Blinding Schema 
Double-blind 
 
Stratification Factor 
Cohort 1a: Baseline UUI episodes per 
day (ie, ≤ 3 vs. > 3 UUI episodes per 
day) and presence or absence of DO 
(as determined by the investigator) at 
randomization. Cohort 1b and 
Cohort 2: randomization will be 
stratified by baseline UUI episodes per 
day and previous use of 
onabotulinumtoxinA (naïve vs. prior 
use). OnabotulinumtoxinA use for 
urological indications must have 
occurred at least 12 months prior to the 
beginning of recording symptoms into 
the Screening Bladder Diary 
 
Adaptive Design 
No 
10.6. 
Appendix 6: Contraceptive Guidance and Collection of Pregnancy 
Information  
The current study will enroll eligible female subjects aged 40 to 79 years old at screening who 
are non-childbearing and satisfy at least one of the following: 
x is postmenopausal with documentation of cessation of menses for ≥ 12 consecutive months at 
the start of screening, without an alternative medical cause.  


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 If the investigator believes that additional confirmation of postmenopausal status is 
necessary, a follicle-stimulating hormone (FSH) level in the postmenopausal range 
(≥ 30 mU/mL at Screening) may be used to confirm a postmenopausal state; systemic 
HRT must be temporarily discontinued (duration determined by the investigator) prior to 
testing FSH levels. Women whose postmenopausal status is in doubt must be deemed 
ineligible for study participation. 
x is permanently sterile, following bilateral tubal ligation (performed ≥ 6 months prior to the 
start of screening), hysterectomy, bilateral salpingectomy, or bilateral oophorectomy. 
Pregnancy tests will not be conducted during this study. 
10.7. 
Appendix 7: Prohibited Medications and Non-Drug Therapies 
The following medications/procedures are prohibited prior to the study and during the study 
(until Week 24), as outlined in Section 6.9.1.  


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Prohibited Medications Class 
Examples  
Washout Period/Comments  
Anticholinergicsa 
Darifenacin, fesoterodine, 
hyoscyamine, oxybutynin, 
propantheline, solifenacin, 
tolterodine, and trospium 
Subject must discontinue use at least 
14 days prior to beginning recording of 
symptoms into the Screening Bladder 
Diary and remain off this therapy during 
the study (until Week 24). Inhaled 
anticholinergics used on an as needed 
basis are permitted  
Anticholinergic intravesical therapy 
Oxybutynin, trospium  
Subject must discontinue use at least 
4 weeks prior to beginning recording of 
symptoms into the Screening Bladder 
Diary and remain off this therapy during 
the study (until Week 24) 
Antidiuretic hormones 
Desmopressin 
Subject must discontinue use at least 1 day 
prior to beginning recording of symptoms 
into the Screening Bladder Diary and 
remain off this therapy during the study 
(until Week 24) 
Beta-3 adrenergic agonists 
Mirabegron 
Subject must discontinue use at least 
14 days prior to beginning recording of 
symptoms into the Screening Bladder 
Diary and remain off this therapy during 
the study (until Week 24) 
Botulinum toxins 
Intradetrusor injection or 
intravesical administration of 
botulinum toxin 
Subject must discontinue 
onabotulinumtoxinA use at least 
12 months prior to starting the Screening 
Bladder Diary and remain off this therapy 
or any other toxin used for urological 
indications during the study (until 
Week 24) 
Intravesical pharmacologic agent  
Capsaicin, resiniferatoxin 
Subject must discontinue use within 
12 months of randomization and remain 
off this therapy during the study (until 
Week 24) 
Implanted electrostimulation/ 
neuromodulation device  
Use of other non-implantable 
electrostimulatory devices 
InterStimTM, NUROTM 
Current or planned use prohibited for the 
duration of the study (until Week 24) 
a 
Refers to prescription medications used on a daily basis to treat chronic conditions.  
10.8. 
Appendix 8: Study Schedule Supplement 
Please see Section 1.3 for the Schedule of Assessments. 


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10.9. 
Appendix 9: Patient-reported Outcomes Questionnaires Descriptions 
and Instructions 
Information on each of the scales and questionnaires for patient-reported outcomes are provided 
below. A sample for each assessment is also provided. 
10.9.1. 
Incontinence-Specific Quality-of-Life Instrument (I-QOL) 
The I-QOL is a validated, disease-specific quality of life questionnaire designed to measure the 
impact of urinary incontinence on patients’ lives [Patrick 1999]. It contains 22 items and is 
designed for self-administration, requiring approximately 5 minutes to complete. The response 
options and values for each item are: “Extremely” (value = 1), “Quite a bit” (value = 2), 
“Moderately” (value = 3), “A little” (value = 4), and “Not at all” (value = 5). A total summary 
score is calculated based on the 22 items; additionally, three domain scores (Avoidance and 
Limiting Behavior, Psychosocial Impact, and Social Embarrassment) can be calculated based on 
responses to items corresponding to each respective domain. The total summary and domain 
scores range from 0 to 100, with higher scores indicating better quality of life. 


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11. 
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function: Report from the standardisation sub-committee of the international continence society. 
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Abrams P, Kelleher CJ, Kerr LA, Rodgers RG. Overactive bladder significantly affects quality 
of life. Am J Manag Care. 2000;11:S580-S590. 
BOTOX® (onabotulinumtoxinA) Package Insert, 2018. 
Coyne K, Revicki D, Hunt T, Corey R, Stewart W, Bentkover J, et al. Psychometric validation of 
an overactive bladder symptom and health-related quality of life questionnaire: the OAB-q. Qual 
Life Res. 2002 Sep;11(6):563-574. 
Gray SL, Anderson ML, Dublin S, Hanlon JT, Hubbard R, Walker R, Yu O, Crane PK, Larson 
EB. Cumulative use of strong anticholinergics and incident dementia: a prospective cohort study. 
JAMA Intern Med. 2015 Mar;175(3):401-407. 
Homma Y, Batista J, Bauer S, Griffiths D, Hilton P, Kramer G, et al. Urodynamics. In: Abrams 
P, Cardozo L, Khoury S, Wein A, editors. Incontinence. Plymouth, UK: Plymbridge Distributors 
Ltd; 2002. p. 317-372. 
Kelleher CJ, Kreder KJ, Pleil AM, Burges SM, Reese PR. Long-term health- related quality of 
life of patients receiving extended-release tolterodine for overactive bladder. Am J Manag Care 
2002;8:S616-S630. 
Milsom I, Abrams P, Cardozo L, Roberts RG, Thüroff J, Wein AJ. How widespread are the 
symptoms of an overactive bladder and how are they managed? A population-based prevalence 
study. BJU International 2001;87:760-766. 
Nitti VW, Auerbach S, Martin N, Calhoun A, Lee M, Herschorn S. Results of a Randomized 
Phase III Trial of Mirabegron in Patients with Overactive Bladder. J Urol 2013;189: 1388-1395. 
Patrick DL, Martin ML, Bushnell DM. The I-QOL: a quality-of-life instrument specific to 
persons with urinary incontinence: user’s manual and scoring diskette for United States version. 
Seattle, Washington: Health Research Associates, Inc., 1999. 58p. 
Reynolds WS, Fowke J, Dmochowsi R. The Burden of Overactive Bladder on US Public Health. 
Curr Bladder Dysfunct Rep. 2016 Mar;11(1):8-13. 
Risacher SL, McDonald BC, Tallman EF, West JD, Farlow MR, et al; Alzheimer’s Disease 
Neuroimaging Initiative. Association Between Anticholinergic Medication Use and Cognition, 
Brain Metabolism, and Brain Atrophy in Cognitively Normal Older Adults. JAMA Neurol. 2016 
Jun 1;73(6):721-732. 


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19 February 2021 
Sacco E, Bientinesi R. Mirabegron: a review of recent data and its prospects in the management 
of overactive bladder. Ther Adv Urol. 2012;4(6): 315–324. 
Stewart WF, Van Rooyen JB, Cundiff GW, Abrams P, Herzog AR, Corey R, et al. Prevalence 
and burden of overactive bladder in the United States. World J Urol. 2003;20:327-336. 

SAP_001.pdf: 
 
 
 
STATISTICAL ANALYSIS PLAN 
 
Protocol Number: 
URO-902-2001 
Protocol Title: 
An Exploratory Phase 2a Study Evaluating the Efficacy and Safety 
of URO-902 in Subjects with Overactive Bladder and Urge Urinary 
Incontinence 
Protocol Version and 
Date: 
Version 3.0 (Amendment 2); 19-Feb-2021 
Investigational 
Product: 
URO-902, formerly known as hMaxi-K, pVAX/hSlo 
Indication 
Overactive Bladder and Urge Urinary Incontinence 
Development Phase: 
Phase 2a 
US IND Number: 
013208 
EudraCT Number: 
Not Applicable 
Sponsor: 
Urovant Sciences, GmbH  
Grosspeteranlage 29 Suites 2006-2010  
4052 Basel, SwitzerlandTelephone +41 (42) 2155999 
SAP Version 
Effective Date 
 
Version 2.0 
21-JUL-2022 
SAP Author 
 
 
 


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TABLE OF CONTENTS 
LIST OF ABBREVIATIONS ......................................................................................... 8 
SAP VERSION HISTORY........................................................................................... 10 
1. 
INTRODUCTION ..................................................................................... 11 
1.1. 
Study Objectives and Endpoints ................................................................ 11 
1.1.1. 
Efficacy Objectives .................................................................................... 11 
1.1.2. 
Safety Objectives ....................................................................................... 12 
1.1.3. 
Other Objectives ........................................................................................ 12 
1.2. 
Study Design .............................................................................................. 12 
1.2.1. 
Overall Study Design ................................................................................. 12 
1.2.2. 
Randomization and Blinding ..................................................................... 14 
1.2.3. 
Statistical Hypotheses ................................................................................ 15 
1.2.4. 
Sample Size Justification ........................................................................... 15 
2. 
PLANNED ANALYSES ........................................................................... 15 
2.1. 
Interim Analysis ......................................................................................... 15 
2.2. 
Final Analysis ............................................................................................ 16 
2.3. 
Data and Safety Monitoring Board ............................................................ 16 
3. 
ANALYSIS POPULATION ..................................................................... 17 
3.1. 
Analysis Population ................................................................................... 17 
3.1.1. 
Screened Population .................................................................................. 17 
3.1.2. 
Randomized Population ............................................................................. 17 
3.1.3. 
Safety Population ....................................................................................... 17 
3.1.4. 
Intent-to-treat Exposed (ITT-E) ................................................................. 17 
3.2. 
Violations and Deviation ........................................................................... 17 
3.2.1. 
Protocol Deviations ................................................................................... 17 
4. 
GENERAL STATISTICAL CONSIDERATIONS ................................... 18 
4.1. 
General Principles for Data Analysis ........................................................ 18 
4.1.1. 
Multicenter Study ...................................................................................... 18 
4.1.2. 
Testing Strategy and Multiplicity Adjustments ......................................... 18 
4.1.3. 
Examination of Subgroups ........................................................................ 18 
4.2. 
General Data Handling Conventions ......................................................... 19 


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4.2.1. 
Study Treatment Description ..................................................................... 19 
4.2.2. 
Reporting Conventions .............................................................................. 19 
4.2.3. 
Premature Withdrawal and Missing Data .................................................. 20 
4.2.4. 
Analysis Visiting Windows ....................................................................... 21 
4.3. 
Data Definitions and Deviations ................................................................ 23 
4.3.1. 
Efficacy Data Collection Schedule ............................................................ 23 
4.3.2. 
Bladder Diary Data .................................................................................... 24 
4.3.2.1. 
Average Daily Number of UUI Episodes .................................................. 24 
4.3.2.2. 
Average Daily Number of Micturitions ..................................................... 24 
4.3.2.3. 
Average Daily Number of UI Episodes ..................................................... 24 
4.3.2.4. 
Average Daily Number of Urgency Episodes ........................................... 25 
4.3.2.5. 
Proportion of Subjects Achieving ≥ 50%, ≥ 75%, and 100% Reduction 
from Baseline at Week 12 in UUI Episodes per Day ................................ 25 
4.3.2.6. 
Average Volume Voided per Micturition .................................................. 25 
4.3.2.7. 
Average Daily Number of Nighttime Voids .............................................. 25 
4.3.2.8. 
Proportion of Subjects Achieving ≥ 50% and 100% Reduction from 
baseline in Number of Average Daily UI Episodes ................................... 25 
4.3.3. 
Questionnaires Data and Scoring ............................................................... 26 
4.3.3.1. 
Total Summary Score from I-QOL ............................................................ 26 
4.3.3.2. 
OAB-q Scores ............................................................................................ 27 
4.3.3.3. 
PGI-C Scale Score ..................................................................................... 28 
4.3.4. 
Study Day and Duration ............................................................................ 28 
4.3.5. 
Baseline and Change from Baseline .......................................................... 28 
5. 
DEMOGRAPHIC, OTHER BASELINE CHARACTERISTICS AND 
MEDICATION .......................................................................................... 29 
5.1. 
Subject Disposition and Withdrawals ........................................................ 29 
5.2. 
Demographic and Baseline Characteristics ............................................... 30 
5.3. 
Medical History and Concomitant Disease ............................................... 30 
5.4. 
Prior and Concomitant Medication ............................................................ 30 
5.4.1. 
Additional OAB Treatment ....................................................................... 31 
6. 
EFFICACY ANALYSIS ........................................................................... 31 
6.1. 
Continuous Efficacy Endpoints ................................................................. 31 


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6.2. 
Categorical Endpoints ................................................................................ 33 
6.3. 
Urodynamic Endpoints .............................................................................. 34 
6.4. 
hSlo cDNA concentrations ........................................................................ 34 
7. 
SAFETY ANALYSIS ............................................................................... 34 
7.1. 
Extent of Exposure and Compliance ......................................................... 34 
7.2. 
Adverse Events .......................................................................................... 34 
7.3. 
Laboratory Evaluations .............................................................................. 36 
7.4. 
Vital Signs ................................................................................................. 37 
7.5. 
ECG ........................................................................................................... 37 
7.6. 
Physical Examination ................................................................................ 37 
7.7. 
PVR............................................................................................................ 38 
7.8. 
Clean Intermittent Catheterization (CIC) .................................................. 38 
7.9. 
Bladder and Kidney Ultrasound ................................................................ 38 
8. 
COVID-19 CONSIDERATIONS .............................................................. 38 
9. 
CHANGE FROM ANALYSIS PLANNED IN PROTOCOL................... 39 
10. 
REFERENCES .......................................................................................... 39 
11. 
APPENDIX................................................................................................ 41 
11.1. 
Table of Contents for Data Display Specifications ................................... 41 
11.1.1. 
Output Tables ............................................................................................. 41 
11.1.2. 
Output Figures ........................................................................................... 48 
11.1.3. 
Output Listings .......................................................................................... 49 
 
LIST OF TABLES 
Table 1: 
Sample Size Determination Parameters ..................................................... 15 
Table 2: 
Diary Data Analysis Visit Window Slotting ............................................. 22 
Table 3: 
Analysis Visit Window Slotting ................................................................ 23 
Table 4: 
Efficacy Assessments ................................................................................ 23 
Table 5: 
I-QOL Scoring ........................................................................................... 26 
Table 6: 
OAB-q Scoring .......................................................................................... 27 
 
LIST OF FIGURES 


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Figure 1: 
Schematic of Study Design: 2-Cohort, Dose-Escalation ........................... 14 
 
 
 


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LIST OF ABBREVIATIONS  
Abbreviation 
Term 
AE 
adverse event 
AESI 
adverse event of special interest 
ANCOVA 
analysis of covariance 
ATC 
Anatomical Therapeutical Chemical 
BMI 
body mass index 
CFB 
change from baseline 
CI 
confidence interval 
CIC 
clean intermittent catheterization 
CMH 
Cochran-Mantel-Haenszel 
CSR 
clinical study report 
DBP 
diastolic blood pressure 
DNA 
double-stranded deoxyribonucleic acid 
DO 
detrusor overactivity 
DSMB 
data safety monitoring board 
ECG 
electrocardiogram 
eCRF 
electronic case report form 
ED 
erectile dysfunction 
EDC 
electronic data capture 
EOS 
end of study 
FSV 
Cystometric volume at 1st sensation to void 
HRQL 
health-related quality of life 
IDC 
involuntary detrusor contraction 
I-QOL 
Incontinence Quality-of-Life Instrument 
ITT-E 
intent-to-treat exposed 
IxRS 
interactive voice or web response system 
LSMEANS 
least squares means 
MCC 
maximum cystometric capacity 
MedDRA 
Medical Dictionary for Regulatory Activities 
MID 
minimally important difference 
MMRM 
mixed model for repeated measures 
OAB 
overactive bladder 
PdetMaxStorage 
maximum detrusor pressure during the storage phase 
Pdet@1stIDC 
maximum detrusor pressure during the first IDC 
PD 
protocol deviation 
PGI-C 
Patient Global Impression of Change 
PT 
preferred term 
PVR 
post-void residual 
REML 
restricted (or residual) maximum likelihood 
SAP 
statistical analysis plan 
SBP 
systolic blood pressure 


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Abbreviation 
Term 
SD 
standard deviation 
SE 
standard error 
SOC 
system organ class 
TEAE 
treatment emergent adverse event 
TL 
topline 
TLF 
table, listing, and figure 
UI 
urinary incontinence 
UUI 
urge urinary incontinence 
Vol@1stIDC 
volume at first IDC 
WHO 
World Health Organization  


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SAP VERSION HISTORY 
Version 
Date 
Description of Changes 
1.0 
01-NOV-2021 
Original Document 
2.0 
21-JUL-2022 
Added second interim analysis, revise the MIC definition 
as well as the average Urine volume per MIC calculation 




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o Change from baseline at Week 12 in OAB 
Questionnaire (OAB-q) scores 
o Overall change of bladder symptoms based 
on the Patient Global Impression of 
Change (PGI-C) scale score at Week 12 
x 
Urodynamic parameters (read by an independent 
central reviewer): 
o Cystometric volume at 1st sensation to 
void (FSV) 
o Maximum cystometric capacity (MCC) 
o Maximum detrusor pressure during the 
storage phase (Pdetmax) 
o Presence/absence of the first involuntary 
detrusor contraction (IDC) and, if present: 
 
Volume at first IDC (VPmaxIDC) 
 
Maximum detrusor pressure 
during the first IDC (PmaxIDC) 
x 
Change from baseline at Week 12 in average daily 
number of nighttime voids 
1.1.2. 
Safety Objectives  
Safety Objectives 
 Endpoints 
To evaluate the safety and tolerability 
of a single dose of URO-902 24 mg 
and 48 mg (administered via 
intradetrusor injection), compared 
with placebo, in subjects with OAB 
and UUI up to 48 weeks post-dose 
Safety endpoints include adverse events, serious adverse 
events, physical examination, vital signs (pulse rate, blood 
pressure, respiration rate, and body temperature), 
hematology and clinical chemistry, post-void residual 
(PVR) urine volume, electrocardiograms (ECGs), 
concomitant medications and concurrent procedures. 
 
1.1.3. 
Other Objectives  
Other Efficacy Objectives 
 Endpoints 
Other endpoints include hSlo cDNA 
concentrations (blood and urine). 
x 
Proportion of subjects with detectable hSlo cDNA 
concentrations 
x 
Summary of mean hSlo cDNA concentrations 
1.2. 
Study Design 
1.2.1. 
Overall Study Design 
This is a multicenter, randomized, double-blind, placebo-controlled, single-treatment, 2-cohort, 
dose-escalation study. 
URO-902 24 mg will be administered as intradetrusor injections to subjects in Cohort 1. An 
independent Data and Safety Monitoring Board (DSMB) will make the recommendation 


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regarding dose escalation only after unblinded review of safety data once all subjects in Cohort 
1 reach Week 6 (or prematurely discontinue). Randomization at the higher dose (URO-902 48 
mg) will begin only after the DSMB has recommended it is safe to proceed to Cohort 2 
x Cohort 1: URO-902 24 mg and matching placebo 
x Cohort 2: URO-902 48 mg and matching placebo 
An estimated total of 78 subjects will be enrolled into 2 cohorts with approximately 39 subjects 
randomized into each cohort. In both cohorts, subjects will be randomized in a 2:1 ratio to 
receive either URO-902 (24 mg or 48 mg) or placebo. Each cohort will be randomized 
separately, and enrollment will be sequential, starting with Cohort 1 (URO-902 24 mg [n = 26] 
and placebo [n = 13]) and followed by Cohort 2 (URO-902 48 mg [n = 26] and placebo [n = 
13]).  
Prior to protocol amendment 1, subjects in Cohort 1 were randomized based on two 
stratification factors: baseline UUI episodes per day (≤3 vs. >3 UUI episodes per day), and 
presence and absence of detrusor overactivity (DO). Post-protocol amendment 1, subjects were 
randomized based on a new randomization scheme and presence or absence of DO was 
replaced with prior onabotulinumtoxinA use. All subjects randomized prior to and post 
amendment 1 will have the baseline UUI episodes per day as a stratification factor. The 
subjects randomized under the original randomization scheme will be considered Cohort 1a 
and subjects randomized under the new randomization scheme will be considered Cohort 1b. 
For reporting purposes, Cohort 1a and Cohort 1b will be combined and subjects enrolled in 
Cohort 1a will be considered onabotulinumtoxinA naïve.  
Study visits will be identical for Cohorts 1 and 2. Subjects will be evaluated during a 5-week 
screening and randomization period for eligibility (Days -35 to -1). Eligible subjects will be 
randomized to treatment at the Randomization Visit (Visit 2) within each cohort; however, 
subjects will be administered the study treatment via cystoscopy on Day 1 (Visit 3). All 
subjects will be evaluated at scheduled post-treatment clinic visits at Weeks 2, 6, 12, 18, and 
24 (Visits 4 to 8, respectively), or until the subject exits the study. Week 18 may be conducted 
by phone or other virtual technology. Afterwards, 2 follow-up visits by phone or other virtual 
technology will be performed at Week 36 (Visit 9) and Week 48 (Visit 10) for subjects who 
have completed the Week 24 Visit. 
Starting at Week 24 (Visit 8), subjects can request and receive additional OAB treatment(s) at 
the clinical discretion of the investigator. Subjects who receive additional OAB treatment(s) at 
Week 24 or after will only be followed to assess adverse events at any future telephone (or 
other virtual technology) visits (Week 36 and/or Week 48). No bladder diaries will be 
collected, and no efficacy assessments will be performed once a subject receives an additional 
OAB treatment. 
Refer to Section 1.3 of the protocol for a detailed time and events schedule. Study schema is 
shown below: 


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Figure 1: 
Schematic of Study Design: 2-Cohort, Dose-Escalation 
 
 
1.2.2. 
Randomization and Blinding 
Randomization will occur centrally using an interactive voice or web response system (IxRS) 
using central, stratified block randomization. Randomization will be done separately for Cohort 
1 and Cohort 2 and will be stratified based on the following stratification factors: 
x Baseline UUI episodes per day (≤ 3 vs. > 3), and  
x Prior onabotulinumtoxinA use (naïve vs. prior use) 
As mentioned in the previous section, subjects enrolled under the randomization scheme prior 
to protocol amendment 1 (Cohort 1a) will be considered onabotulinumtoxinA naïve when 
combined with Cohort 1b for analyses and summaries. 
To maintain blinding of the treatment groups, neither the investigator/physician subinvestigator 
(injector) nor site personnel who are involved in evaluating or injecting the subject should 
prepare the study drug. An additional individual (eg, unblinded pharmacist or nurse) with no 
subject evaluation or treatment involvement must be identified at each investigative site to 
prepare the study drug and the PBS-20% flushing solution. The responsibility of this individual 
will be to properly prepare the injection syringes with the accurate randomized study drug as 
per the volumes indicated by the Sponsor. They will draw the study drug into the syringes to be 
injected and dilute as needed as per instructions in the study manual.  
For the interim analysis(es), the subject and the investigator or delegate(s) who are involved in 
the treatment or clinical evaluation of the subjects will remain blinded to the treatment 
assignments. Limited personnel from the Sponsor will be unblinded (double-blind, sponsor-


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open design). Sponsor personnel/designees who are directly involved with site interactions and 
communications (eg, study operations lead and site monitors) will remain blinded to subject 
level treatment assignment. 
1.2.3. 
Statistical Hypotheses  
The study has no formal statistical primary endpoint hypothesis. The intent is to estimate the 
treatment effect of each dose group relative to placebo. For the final analysis, the subjects 
receiving placebo from Cohort 1 and Cohort 2 will be pooled. 
1.2.4. 
Sample Size Justification 
For this exploratory study, the sample size was empirically chosen. With a total of 23 evaluable 
female subjects in each of the URO-902 treatment groups and 23 evaluable female subjects in 
the placebo group, this study would have approximately 55% power to detect a between-group 
difference of 1.8 episodes in the mean change from baseline to Week 12 in the number of UUI 
episodes per day between the URO-902 and placebo treatment groups (based on a 2-sample t-
test with a 2-sided type I error rate of 0.05 and standard deviation [SD] of 2.86). Furthermore, 
the half-width of the confidence interval would be the following for different SD assumptions: 
Table 1: 
Sample Size Determination Parameters 
SD 
N per Group 
Half-Width of 
Confidence Interval 
Lower Limit 
Upper Limit 
2.86 
23 
1.70 
0.10 
3.50 
3.00 
23 
1.78 
0.02 
3.58 
2.55 
23 
1.49 
0.31 
3.29 
In order to account for subject attrition (approximately 10%), the sample size was increased to 
26 subjects for each treatment group, amounting to a total of 78 randomized subjects required 
for exploratory efficacy analyses in both Cohort 1 (n = 39) and Cohort 2 (n = 39). 
2. 
PLANNED ANALYSES  
2.1. 
Interim Analysis  
Two or more interim analyses may be conducted; when ≥ 50% of subjects in Cohort 1, and/or 
when ≥ 50% of subjects in Cohort 2 have completed at least 12 weeks of follow-up post-
treatment administration (or prematurely exited the study prior to Week 12). Any interim 
analysis performed is for future planning purposes and no adjustment to the type I error will be 
made. 
The planned interim analysis will be conducted to evaluate the objectives of the protocol at 
Week 12. These analyses will be performed after the completion of the following steps: 


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x All subjects in Cohorts 1 and 2 have completed Week 12 (or prematurely exited the 
study prior to Week 12). 
x All required database cleaning activities have been completed and final database 
release and freeze has been declared by data management for all subjects to be 
included in the interim analysis. 
x All criteria for unblinding the randomization code have been met.  
Similar to the planned Week 12 interim analysis, a second planned interim analysis will be 
conducted after all subjects complete Week 24 (or prematurely exited the study prior to Week 
24). This analysis will evaluate the objectives of the protocol at Week 24.  
2.2. 
Final Analysis  
The 
will perform the production and quality control of all tables, 
figures and listings on behalf of Urovant Sciences, Inc. 
Statistical programming will start after data have been collected and are available in the 
database.  Blinded dry-runs using dummy treatment code will be performed prior to database 
lock and unblinding to ensure programming displays and algorithms are developed as planned.  
The planned final end-of-study analysis will be performed once the final clinical database lock 
(after all subjects complete the study or exit the study prematurely) has been achieved and 
treatment codes have been unblinded to the statistics and programming team. The clinical 
study report will be based on final analysis results. 
2.3. 
Data and Safety Monitoring Board 
A DSMB will be retained to assess, on an ongoing basis, all safety aspects of this study 
including unblinded review of safety data after all subjects reach Week 6 (or prematurely 
discontinue) in Cohort 1 (URO-902 24 mg). Randomization at the higher dose (URO-902 48 
mg) will begin only after the DSMB has recommended it is safe to proceed to Cohort 2. This 
committee will be an external independent DSMB that monitors safety. The detailed activities 
including meeting plans will be described and documented in the DSMB Charter. A separate 
statistical analysis plan may be prepared for the DSMB. 


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3. 
ANALYSIS POPULATION  
3.1. 
Analysis Population 
3.1.1. 
Screened Population 
The screened population consists of all subjects who are screened for the study. This 
population is used primarily for subject accounting purposes and will generally not be used for 
summary or analysis. 
3.1.2. 
Randomized Population 
The randomized population consists of all subjects who are randomized to receive any double-
blind study drug regardless of whether they received the study drug.  
3.1.3. 
Safety Population   
The safety population consists of all subjects from Cohorts 1 and 2 who were randomized and 
received study drug and will be used to analyze all safety endpoints based on actual treatment 
received.  
3.1.4. 
Intent-to-treat Exposed (ITT-E) 
The ITT-E population consists of all subjects who are randomized and treated from Cohorts 1 
and 2. ITT-E will be used for demographics, baseline characteristics, and efficacy analyses. 
Subjects will be included in the treatment group to which they are randomized. 
3.2. 
Violations and Deviation  
Subjects who do not meet eligibility criteria but are still randomized will be analyzed 
according to the analysis population described in Section 3.1. 
3.2.1. 
 Protocol Deviations  
The final list of major protocol deviations will be finalized and documented prior to database 
lock except for the deviation category of wrong treatment which will be confirmed upon study 
unblinding. Only major protocol deviations will be summarized and listed in the Clinical Study 
Report (CSR). Major deviations will be those which are considered to potentially impact upon 
the interpretation of the primary efficacy endpoint in the study or may potentially impact the 
interpretation of safety. Major protocol deviation categories may include, but are not limited to 
the following: 
x Randomized subjects who do not meet the inclusion criteria 
x Randomized subjects who meet any of the exclusion criteria 
x Subjects who received the wrong treatment  


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x Concomitant use of prohibited medications not approved by the medical monitor 
x Randomized subjects who met withdrawal criteria during the study but were not 
withdrawn
Due to the limited sample size of the study, no sensitivity analysis will be performed on a per-
protocol population. 
4. 
GENERAL STATISTICAL CONSIDERATIONS 
4.1. 
General Principles for Data Analysis  
The general formats and layouts of tables, listings, and figures (TLFs) are provided in a 
separate “programming consideration” document. Actual formats and layouts may be altered 
slightly to accommodate actual data or statistics. Minor format changes will not require 
updates to the SAP. 
The TLF numbering and general content follow the ICH E3 guidelines. 
4.1.1. 
Multicenter Study  
In this study the stratified permuted block randomization is not done within centers.  The 
analyses will be conducted by pooling data from all study centers and will not include study 
center as a covariate in the statistical modeling. 
4.1.2. 
Testing Strategy and Multiplicity Adjustments  
No formal multiplicity adjustment will be performed. Nominal p-values will be provided for all 
exploratory efficacy analysis as a measure of the strength of association between the endpoint 
and the treatment effect rather than formal tests of hypotheses.  
4.1.3. 
Examination of Subgroups  
To determine whether the treatment effect is consistent across various subgroups, the efficacy 
endpoints related to UUI, micturitions, UI, and urgency episodes will be summarized 
descriptively for each of the following subgroups: 
x Baseline UUI episodes per day (≤3 vs >3)  
x Presence or absence of DO  
x Previous use of onabotulinumtoxinA (naïve vs. prior use) 
Baseline UUI episodes per day will be based on the actual value collected at baseline, presence 
or absence of DO will be based on the central reader’s assessment on the urodynamics form, 
and prior onabotulinumtoxinA use will be based on the randomization stratification from the 
new randomization scheme after the amendment 1. The subjects enrolled in Cohort 1a will be 
considered onabotulinumtoxinA naïve. 


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4.2. 
General Data Handling Conventions 
4.2.1. 
Study Treatment Description 
Randomized treatment groups will be displayed as shown in the following table: 
Data Displays for Reporting 
Description 
Order in TLF 
Placebo 
1 
URO-902 24 mg 
2 
URO-902 48 mg 
3 
Overall (if applicable) 
4 
For the reporting done within each cohort, the treatment group applicable to the cohort will be 
used in the output (i.e. URO-902 24 mg for Cohort 1 and URO-902 48 mg for Cohort 2). 
4.2.2. 
Reporting Conventions 
General rules 
In general, all collected safety data, any derived efficacy parameters from the bladder diary, 
questionnaire data, and urodynamic data will be presented in subject data listings. Listings will 
be ordered by treatment, subject number, and assessment week or event date. The treatment 
group presented in listings will be based on the planned assignment for efficacy endpoints and 
actual assignment for safety endpoints. 
Summary tables will be provided for all randomized subjects. All demographic and baseline 
data will be presented by treatment arm and overall, unless otherwise specified. Bladder diary, 
questionnaire, urodynamic data, and safety data will be presented by treatment arm only. In 
general, continuous variables will be summarized to include the population sample size (N), 
number of subjects with available data (n), arithmetic mean, standard deviation (SD), median, 
minimum, Q1, Q3 and maximum values. Categorical variables will be summarized by the 
population size (N), number of subjects with available data (n), number of subjects in each 
category, and the percentage of subjects in each category. Unless otherwise noted, the 
denominator to determine the percentage of subjects in each category will be based on the 
number of subjects with available data (n) in the analysis set of interest. Selective ordinal data 
may be summarized using both descriptive statistics as well as counts and percentages of 
subjects in each category, as appropriate.  


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20 
The data analyses will be conducted using the SAS® System (SAS Institute Inc., Cary, NC) 
version 9.3 or above. The eClinical Solutions’ standard operating procedures will be followed 
for the validation of all SAS programs and outputs. 
Formats 
Numeric data will be reported at the precision collected on the electronic case report form 
(eCRF).  The reported precision from non eCRF sources may be adjusted to a clinically 
interpretable number or decimal places. 
Unscheduled Visits 
Unscheduled visits will be assigned to an analysis visit using the all-inclusive windows defined 
in Section 4.2.4. If multiple assessments are done in the same visit, the selection rule in 
Section 4.2.4 will apply. All assessments at unscheduled visits will be included for deriving 
last post-baseline or maximum value post-baseline and in the data listings. 
4.2.3. 
Premature Withdrawal and Missing Data  
If any missing data are present in diary data for any reasons, no imputation will be applied to 
derive responder. No explicit missing data imputation will be performed for change from 
baseline since a mixed model for repeated measures (MMRM) will be applied to change from 
baseline analysis. For responder analyses, subjects with missing assessments will be considered 
non-responders. Missing items from the questionnaires will be handled according to the 
respective measure instructions as described in Section 4.3.3. 
In general, missing safety data will not be imputed and only observed values will be analyzed. 
If the relationship of an AE record (“Relationship to Study Treatment” on AE eCRF) is 
missing this AE will be considered “Probably Related” to the study treatment. If the AE 
severity is missing every effort should be made to acquire the information from the 
investigator. “Severe” will be assigned to a missing intensity for reporting purposes. 
The general imputation rules of partial missing date for both AE and concomitant medication 
are detailed below: 
Dates with missing the day of the year will adhere to the following conventions:  
x The missing day of onset date will be set to: 
 First day of the month that the event occurred, if the onset YYYY-MM is after 
the YYYY-MM of the study treatment 
 The day of the study treatment, if the onset YYYY-MM is the same as YYYY- 
MM of the study treatment  


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 The date of informed consent, if the onset YYYY-MM is before the YYYY-
MM of the study treatment. 
x The missing day of end date will be set to: 
 The last day of the month of the occurrence. If the subject died in the same 
month, then set the imputed date as the death date. 
Dates with missing both the day and month of the year will adhere to the following 
conventions: 
x Missing day and month of onset date will be set to: 
 January 1 of the year of the onset, if the onset YYYY is after the YYYY of the 
study treatment 
 The date of the study treatment, if the onset YYYY is the same as YYYY of the 
study treatment 
 The date of informed consent, if the onset YYYY is before YYYY of the study 
treatment 
x The missing date of end date will be set to: 
 December 31 of the year of occurrence. If the subject died in the same year, 
then set the imputed date as the death date. 
4.2.4. 
Analysis Visiting Windows 
No analysis visit window will be defined for urodynamic data, and questionnaire data. Nominal 
clinical visits will be used for summary and analysis. Any unscheduled data will be listed only.  
Diary data used for analyses will be windowed as follows:  
1. For baseline, the 3-day diary completed closest to study drug administration will be 
used regardless of when the diary is completed within the screening and randomization 
period. 
2. For post-baseline the analysis visit windowing will be based on the scheduled visit 
dates relative to the study drug administration date according to the schedule of 
assessments in the protocol instead of the actual visit dates. This is set up to take into 
account the visit delays due to COVID-19 should it happen. Table below provides 
details on the target study day and corresponding visit windowing rules for the post-
baseline visits. 


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Table 2: 
Diary Data Analysis Visit Window Slotting 
Analysis Visit 
Target Study Day 
Range 
Visit Window 
Week 2 
15 
15 ± 7 days 
[8, 22] 
Week 6 
43 
43 ± 14 days 
[29, 57] 
Week 12 
85 
85 ± 14 days 
[71, 99] 
Week 18 
127 
127 ± 14 days 
[113, 141] 
Week 24 
169 
169 ± 14 days 
[155, 183] 
Week 36 
253 
253 ± 21 days 
[232, 274] 
Week 48 
337 
337 ± 21 days 
[316, 358] 
 
For safety endpoints such as vital signs, laboratory tests, PVR, and ECGs, data records will be 
slotted to one of the protocol specified visits using the analysis visit window rules below:  
After all the records have been assigned to an analysis visit, if there are more than one records 
in the same analysis visit, the one closest to the target day will be used (for records in screening 
and randomization visits, the later one will be selected). If equal distance, the earlier one will 
be used. If there are more than one results in the same day, the time portion of the collection 
date and time, the “repeat” status, or the inherent sequence in the electronic data capture (EDC) 
system will be used for differentiation and the later one will be used.  


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Table 3: 
Analysis Visit Window Slotting 
Analysis 
Window Label 
Nominal Visit 
Nominal Day 
Visit Window 
Screening 
Screening Visit 
Not Applicable 
Not Applicable 
Randomization 
Randomization Visit 
Not Applicable 
Not Applicable 
Day 1 
Day 1 
1 
Not Applicable 
Week 2 
Week 2 
15 
VS, PVR: [2, 29] 
ECG: [2, 50] 
Week 6 
Week 6 
43 
VS, PVR: [30, 64] 
Lab: [1, 106] 
Week 12 
Week 12 
85 
VS, PVR: [65, 106] 
ECG: [51, 127] 
Week 18 
Week 18 
127 
VS, PVR: [107, 148] 
Week 24 
Week 24 
169 
VS, PVR: 149+ 
ECG: 128+ 
Lab: 107+ 
4.3. 
Data Definitions and Deviations 
4.3.1. 
Efficacy Data Collection Schedule 
Efficacy assessments will be collected as outlined in Table 4 below. Refer to protocol section 
1.3 (Schedule of Assessments) for the details on the day of visit and timing of measurement. 
Table 4: 
Efficacy Assessments 
Assessment 
Form  
Measurement and Timing 
Bladder Diary 
Bladder Diary – Day 1 
Bladder Diary – Day 2 
Bladder Diary – Day 3 
eCRF form 
 
For any 3 consecutive days within the 
Screening and Randomization period (Visits 1 
and 2) and any 3 consecutive days in the 7 
days prior to the clinical visit (Weeks 2, 6, 12, 
18, 24, 36, and 48 (Visits 4-10, respectively)), 
subjects will record daily urination data 
including  
“Need to urinate immediately” 
“Urinated in toilet” 
“Accidental urine leakage” 
“Main reason for leakage”  
For one 24-hour period, subject will also 
record the urine volume for one of the diary 
days 


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Assessment 
Form  
Measurement and Timing 
Health Outcome 
parameters 
x Incontinence Quality of Life 
Instrument (I-QOL) eCRF 
x Overactive Bladder Questionnaire 
(OAB-q) eCRF 
x  Patient Global Impression of 
Change Scale (PGI-C) eCRF 
x I-QOL: Day 1, Weeks 6, 12 
x OAB-q: Day 1, Weeks 6, 12 
x PGI-C: Week 12 
  
Urodynamics 
Urodynamics – Site 
Urodynamics – Central Reader 
Prior to Randomization at Visit 2 and Week 
12 
4.3.2. 
Bladder Diary Data 
Bladder Diary data are collected following the schedule of assessments in Section 1.3 of the 
protocol. Per protocol diaries will not be collected if subjects receive additional OAB 
treatment(s) at Week 24 or after. For summary and analysis purpose, any bladder diaries 
collected after the first additional OAB treatment will not be included in defining the 
subsequent endpoints based on the bladder diaries. All bladder diaries collected will be 
included in the listings. 
4.3.2.1. 
Average Daily Number of UUI Episodes 
Average daily number of UUI episodes is calculated as the total number of UUI episodes 
recorded on the bladder diary days that fall within the visit window divided by the number of 
days the Bladder Diary is filled out. A UUI episode is defined as when a subject checked 
“Urge” to the question “select main reason for leakage” on the bladder diary eCRF. Subjects 
may have more than one bladder diary filled out on the same day and all the UUI episodes will 
be summed together. The denominator used for calculating the average number will only count 
the days in which there is a completed bladder diary day within the visit window. 
4.3.2.2. 
Average Daily Number of Micturitions 
Average daily number of micturitions is defined as the total number of times a subject checked 
“Urinated in toilet” or recorded non-zero urine volume in a completed bladder diary day within 
the visit window divided by the number of days the Bladder Diary is filled out. Subjects may 
have more than one bladder diary filled out on the same day and all the micturitions will be 
summed together. The denominator used for calculating the average number will only count 
the days in which there is a completed bladder diary day within the visit window. 
4.3.2.3. 
Average Daily Number of UI Episodes 
Average daily number of UI episodes is calculated as the total number of UI episodes recorded 
on a completed diary day within the visit window divided by the number of days the Bladder 
Diary is filled out. A UI episode is defined as when a subject answered “Yes” to the question 
“Accidental urine leakage” or indicates any reason for leakage on the bladder diary eCRF. 


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Subjects may have more than one bladder diary filled out on the same day and all the UI 
episodes will be summed together. The denominator used for calculating the average number 
will only count the days in which there is at least one completed bladder diary day within the 
visit window. 
4.3.2.4. 
Average Daily Number of Urgency Episodes 
Average daily number of urgency episodes is calculated as the total number of urgency 
episodes recorded on a completed diary day within the visit window divided by the number of 
days the Bladder Diary is filled out. An urgency episode is defined as when a subject answered 
“Yes” to the question “Need to urinate immediately” on the bladder diary eCRF. Subjects may 
have more than one bladder diary filled out on the same day and all the urgency episodes will 
be summed together. The denominator used for calculating the average number will only count 
the days in which there is at least one completed bladder diary day within the visit window. 
4.3.2.5. 
Proportion of Subjects Achieving ≥ 50%, ≥ 75%, and 100% Reduction from 
Baseline at Week 12 in UUI Episodes per Day 
Proportion of subjects achieving x% reduction from baseline is defined as: - (Average daily 
number of UUI episodes at Week n – UUI at Baseline) / UUI at Baseline * 100%. This is 
defined when both baseline and post-baseline average daily number of UUI episodes are non-
missing. For responder analyses, missing reduction from baseline will be considered non-
responders for the visit in question. 
4.3.2.6. 
Average Volume Voided per Micturition 
A subject is expected to fill out the volume voided for one 24-hour period during the 3-day 
diary collection window. The average volume voided per micturition is calculated as the 
arithmetic mean of all voids for which a subject has recorded a positive volume, even if 
volume voided is filled out for more than one 24-hour period. 
4.3.2.7. 
Average Daily Number of Nighttime Voids 
Average daily number of nighttime voids is defined as the total number of times a subject 
checked “Urinated in toilet” or recorded non-zero urine volume in a completed bladder diary 
day within the visit window between the hours of 11:00 PM and 6:00 AM the following day, 
divided by the number of days the Bladder Diary is filled out. Subjects may have more than 
one bladder diary filled out on the same day and all the nighttime voids will be summed 
together. The denominator used for calculating the average number will only count the days in 
which there is at least one completed bladder diary day within the visit window. 
4.3.2.8. 
Proportion of Subjects Achieving ≥ 50% and 100% Reduction from baseline in 
Number of Average Daily UI Episodes 
Proportion of subjects achieving x% reduction from baseline is defined as: - (Average daily 
number of UI episodes at Week n – UI at Baseline) / UI at Baseline * 100%. This is defined 
when both baseline and post-baseline average daily number of UI episodes are non-missing. 


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For responder analyses, missing reduction from baseline will be considered non-responders for 
the visit in question. 
4.3.3. 
Questionnaires Data and Scoring 
4.3.3.1. 
Total Summary Score from I-QOL 
I-QOL is a validated, disease-specific quality of life questionnaire designed to measure the 
impact of urinary incontinence on patients’ lives. It contains 22 items. The response options 
and values for each item are:  
x Extremely (value = 1) 
x Quite a bit (value = 2) 
x Moderately (value = 3) 
x A little (value = 4) 
x Not at all (value = 5) 
A total summary score is calculated based on the 22 items; additionally, three domain scores 
(Avoidance and Limiting Behavior, Psychosocial Impact, and Social Embarrassment) can be 
calculated based on responses to items corresponding to each respective domain. The score 
will only be derived when at least 50% of the items are completed: i.e. ≥ 11 items are 
completed at one visit [1]. The three domains will be considered missing if the total score is 
missing. The three domains include the following items: 
Table 5: 
I-QOL Scoring 
Domain 
Items 
Lowest Possible 
Scores 
Possible 
Range 
Total Summary Score 
1 to 22 
22 
88 
Avoidance and Limiting 
Behavior 
1, 2, 3, 4, 10, 11, 13, 20 
8 
32 
Psychosocial Impact 
5, 6, 7, 9, 15, 16, 17, 21, 22 
9 
36 
Social Embarrassment 
8, 12, 14, 18, 19 
5 
20 
The total score and each domain score are based on the transformation of the raw score to a 0 
to 100 scale using the formula below: 
ൌݏݑ݉݋݂ݐ݄݁݅ݐ݁݉ݏെ݈݋ݓ݁ݏݐ݌݋ݏݏܾ݈݅݁ݏܿ݋ݎ݁
݌݋ݏݏܾ݈݅݁ݎܽݓݏܿ݋ݎ݁ݎܽ݊݃݁
ൈͳͲͲ 
The higher score corresponds to a better quality of life. 


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Subjects who had at least a 10-point increase of I-QOL total summary score from baseline to 
post-baseline visits are considered responders who achieved the minimally important 
difference (MID). Subjects who had an increase less than 10 points at any post-baseline or 
change from baseline is missing are considered non-responders.  
4.3.3.2. 
OAB-q Scores 
The OAB-q was developed to assess symptom bother and the impact of OAB on health-related 
quality of life (HRQL). It contains 33 items and the response options and values for each item 
are:  
x Not at all / None of the time (value = 1) 
x A little bit / A little of the time (value = 2) 
x Somewhat / Some of the time (value = 3) 
x Quite a bit / A good bit of the time (value = 4) 
x A great deal / Most of the time (value = 5) 
x A very great deal / All the time (value = 6).  
The instrument yields a HRQL total score and four subscales as listed in the table below: 
Table 6: 
OAB-q Scoring 
Scale 
Items 
Lowest, Highest 
Possible Scores 
Possible Range 
Total HRQL 
9 to 33 
25, 150 
125 
Symptom Bother 
1 to 8 
8, 48 
40 
Coping 
9, 11, 16, 21, 22, 26, 32, 33 
8, 48 
40 
Concern 
12, 13, 14, 19, 23, 25, 29 
7, 42 
35 
Sleep 
10, 15, 24, 17, 30 
5, 30 
25 
Social Interaction 
18, 20, 27, 28, 31 
5, 30 
25 
For the Symptom Bother Score, the transformed score is: 
ൌݏݑ݉݋݂ݐ݄݁݅ݐ݁݉ݏെ݈݋ݓ݁ݏݐ݌݋ݏݏܾ݈݅݁ݏܿ݋ݎ݁
݌݋ݏݏܾ݈݅݁ݎܽݓݏܿ݋ݎ݁ݎܽ݊݃݁
ൈͳͲͲ 
The higher score corresponds to a larger bother. 
For Coping, Concern, Sleep, Social Interaction, and Total HRQL scores, the score is: 


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ൌ݄݄݅݃݁ݏݐ݌݋ݏݏܾ݈݅݁ݏܿ݋ݎ݁െݏݑ݉݋݂ݐ݄݁݅ݐ݁݉ݏ
݌݋ݏݏܾ݈݅݁ݎܽݓݏܿ݋ݎ݁ݎܽ݊݃݁
ൈͳͲͲ 
The higher score corresponds to a higher quality of life. 
If < 50% of the scale items are missing, the scale will be retained with the mean scale of the 
items present used to impute a score for the missing items. If ≥ 50% of the items are missing, 
no scale score will be calculated, the subscale score should be considered missing [2].  
4.3.3.3. 
PGI-C Scale Score 
The PGI scale in this study is an OAB-specific quality of life questionnaire. The tool was 
designed to assess the impact (and improvement) of urinary incontinence on activities of daily 
living, wellbeing, and function. The eCRF collects PGI severity, control, frequency, leakage, as 
well as change. All PGI scales are ordinal assessments going from lower impact of disease to 
highest impact of disease. PGI-Severity ranges from 1 to 4, PGI-Control, PGI-Frequency, PGI-
Leakage range from 1 to 5. PGI-Change ranges from 1 to 7. These questions will be displayed 
as categorical variables with count and percent for each category at Week 12. Also, PGI-
Change will be analyzed as a dichotomous responder variable. Subjects who answer “Much 
better” or “Moderately better” will be considered responders, while all other answers and 
missing values will be considered non-responders. 
4.3.4. 
Study Day and Duration  
Study day is relative to the start date of the double-blind treatment. This is used to describe the 
relative time of an event or assessment that happened during the study. The first day of the 
study is defined as the day a subject receives either URO-902 (24 or 48 mg) or placebo. There 
is no study Day 0 defined.  
x For an event or assessment that occurred on or after the study treatment:  Study day 
= Date of event or examination – date of study treatment + 1 
x For an event or assessment that occurred prior to the study treatment:  Study day = 
Date of event or examination – date of study treatment 
A duration between any two dates (such as AE duration) expressed in days will be calculated 
using the following conventions: Duration = later date – earlier date + 1. 
4.3.5. 
Baseline and Change from Baseline 
In general, the last recorded value prior to the study treatment will serve as the baseline 
measurement for the safety endpoints such as lab, vital signs, and ECGs. For numeric result, 
the mean of the multiple values will be used as baseline if multiple measurements are collected 
on the same baseline day without the time or “repeat” status to differentiate the records. For 
categorical results, the record with the later sequence number or collection time will be used if 
multiple measurements are collected on the same baseline day. For the bladder diary data, the 3 


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diary days collected during the screening and randomization period and closest to the treatment 
administration is considered baseline. All 3 diary days are required for the change from 
baseline derivation.  For the questionnaires, the baseline is the data collected on the Day 1 Visit 
prior to treatment. 
Change from baseline will be calculated as the post-baseline value minus the baseline value. 
Percentage change from baseline will be calculated as the change from baseline divided by the   
baseline value, multiplied by 100. If either the baseline or post-baseline value is missing, then 
change from baseline and percentage change from baseline will be set to missing. 
5. 
DEMOGRAPHIC, OTHER BASELINE CHARACTERISTICS 
AND MEDICATION 
5.1. 
Subject Disposition and Withdrawals 
Subject disposition will be summarized for Screened population and by treatment group for the 
Randomized population separately. The summary for the screened population will include 
number of subjects who passed screening and who were screen failures and corresponding 
screen failure reasons.  
For the disposition summary of the randomized population, the following categories will be 
presented; randomized, completed Week 24, completed Week 48 follow-up, withdrawn from 
study (and reason), received study treatment, did not receive any treatment, additional OAB 
treatment prescribed. 
Reasons for exclusion from analysis population will also be summarized based on the 
populations defined in Section 3.1.  
The frequency and percentage of subjects with at least one major Protocol Deviation (PD), 
major PD by classification and reasons/category for PD will be summarized by treatment arm 
for the Randomized population.  
Eligibility criteria, screen failures (including date and primary reason for failure), and informed 
consent (protocol version, informed consent version date and date signed) will be listed for all 
subjects screened. 
A summary of randomized subjects by investigator name and site identification will be 
provided. 
Randomization details will also be listed, including the date of randomization, randomization 
number and randomization strata. 


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5.2. 
Demographic and Baseline Characteristics 
All demographic and baseline characteristic data will be summarized by treatment group using 
descriptive statistics for all subjects in the ITT-E population.  
The summary table will include age, age category (<65, ≥65 years), ethnicity, race, baseline 
UUI episodes per day (<=3, >3) (as randomized and actual baseline), presence or absence of 
DO (site and central assessments), previous use of onabotulinumtoxinA (naïve vs. prior use) 
(as randomized and actual baseline), weight, height and body mass index (BMI).  
The actual baseline for UUI episodes per day is derived based on the logic described in 
Section 4.3.2.1.  The actual previous use of onabotulinumtoxinA status is based on searching 
for the preferred term of “BOTULINUM TOXIN TYPE A” in subject’s medications taken 
before randomization.  
Age (years), height (cm), weight (kg), and BMI (kg/m2) captured at Screening will be 
summarized as a continuous variable. 
All demographic data will be listed. 
5.3. 
Medical History and Concomitant Disease 
Descriptions of medical history findings will be coded using Medical Dictionary for 
Regulatory Activities (MedDRA) version 22.1 or higher. 
A disease or illness reported as medical history without a start date will be included in medical 
history without a date assigned. Medical history will be sorted by descending overall 
frequency, by system organ class (SOC) and preferred term (PT) in the summary table. 
Medical history data listings will be sorted by treatment, subject number, start date, SOC and 
PT. 
5.4. 
Prior and Concomitant Medication  
Prior and concomitant medications will be coded using the World Health Organization (WHO) 
Drug B3-Sept Format, 2019 version. These medications will be further classified as follows.  
x 
Prior medication is defined as those medications that started prior to the study treatment.  
x 
Concomitant medication is defined as those taken on or after the study treatment. If 
medications started prior to the study treatment and were ongoing after the study 
treatment, those medication will be considered as concomitant medication as well.  
The number and percentage of subjects taking prior and concomitant medications will be 
summarized overall by Anatomical Therapeutical Chemical (ATC) Levels 2 and 4 for all 
subjects in the safety population. Prior and concomitant medications will be listed for all 
subjects in the safety population.  


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5.4.1. 
Additional OAB Treatment 
Starting at Week 24 and after (at clinic visit, phone visits or between visits), subjects can 
request and be prescribed additional OAB treatment(s). A treatment is considered additional 
OAB treatment if on the “Concomitant Medications/Procedures” eCRF the answer to “Is this 
an additional OAB medication” is “Yes”, regardless of when the additional OAB treatment 
was received.   
The additional OAB medication will be summarized by ATC level 2, 4, and Preferred Term 
while additional OAB procedures will be summarized by preferred term. Separate listings for 
additional OAB medications and procedures will be provided. 
6. 
EFFICACY ANALYSIS 
In general, the ITT-E will be used for all analyses of efficacy endpoints unless stated 
otherwise. 
6.1. 
Continuous Efficacy Endpoints 
For continuous efficacy endpoints, the by-visit means and SD, as well as change from baseline 
estimated with least squares means, standard error, and 95% confidence intervals (CI) will be 
presented for each treatment group and the difference between each dose group relative to 
placebo. Nominal p-values from comparisons from each dose group relative to placebo will be 
provided for descriptive purposes. The change from baseline will be analyzed by a mixed 
model for repeated measures (MMRM) with restricted maximum likelihood estimation 
incorporating all applicable post-baseline visits. For average daily number of urge urinary 
incontinence (UUI) episodes, the MMRM model will include terms for treatment, visit, visit by 
treatment interaction term, baseline value, and previous use of onabotulinumtoxinA as 
randomized (naïve vs. prior use). For the other endpoints, one additional term of baseline UUI 
episodes per day category as randomized (≤ 3 vs. > 3) will be included in the MMRM model. 
If any of the stratification factors has a stratum with number of subjects less than 5, the factor 
in question will not be included in the MMRM model. If >15% of the subjects were incorrectly 
stratified based on the actual baseline UUI episodes per day and previous use of 
onabotulinumtoxinA, a sensitivity analysis using the same MMRM model but with actual 
values of the two stratification factors as covariates will be carried out. 
The Kenward-Roger adjustment will be used with restricted (or residual) maximum likelihood 
(REML) to make statistical inference.  An unstructured covariance matrix will be used to 
model the correlation among repeated measurements. If the unstructured covariance model 
fails to converge with the default Newton-Raphson algorithm, the Fisher scoring algorithm will 
be used to provide initial values of the covariance parameters.  In the rare event that none of 
the above methods yield convergence, the following structures will be investigated:  
heterogeneous Toeplitz, Toeplitz, heterogeneous First-Order Autoregressive [AR (1)], 


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heterogeneous compound symmetry (HCS), and compound symmetry (CS).  The covariance 
structure converging to the best fit, as determined by Akaike’s information criterion (AIC), will 
be used. 
Results will be presented in terms of least square means (LSMEANS), treatment differences in 
LSMEANS, nominal p-value and 95% confidence intervals for the treatment difference for all 
timepoints. Type III p-values for all factors in the MMRM model will be listed on the table.  
An example of the SAS code for the base procedure is given below: 
proc mixed data = datain method = reml; 
      class subject treatment visit UUI_cat onabA_use; 
      model CFB = treatment visit treatment*visit baseline UUI_cat onabA_use;   
                  /ddfm=KR solution chisq; 
      repeated visit / subject=subject type=UN r rcorr; 
      lsmeans treatment*visit / pdiff=all cl alpha=0.05; 
run; 
The normality assumptions for the primary analysis model will be assessed by inspection of the 
residuals from the model and normal probability plots. If assumptions of normality are grossly 
violated, additional supportive analysis of the data will be performed in order to assess the 
robustness of the conclusions drawn from the primary analysis.  
Sensitivity analyses using analysis of covariance (ANCOVA) will be carried out for change 
from baseline at Week 12 in average daily number of UUI episodes and micturitions. The 
model will use the change from baseline data at Week 12 and include treatment arms and 
baseline value as covariates. 
The following efficacy endpoints will be summarized and analyzed in this fashion (Data for all 
visits will also be presented): 
x Change from baseline at Week 12 in average daily number of UUI episodes 
x Change from baseline at Week 12 in average daily number of micturitions 
x Change from baseline at Week 12 in average daily number of UI episodes 
x Change from baseline at Week 12 in average daily number of urgency episodes 
x Change from baseline at Week 12 in average volume voided per micturition 


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x Change from baseline at Week 12 in total summary score from I-QOL 
x Change from baseline at Week 12 in OAB-q scores 
x Change from baseline at Week 12 in average daily number of nighttime voids 
For all domains and subscales in I-QOL and OAB-q the mean value and change from baseline 
will be summarized by visit. 
6.2. 
Categorical Endpoints 
The following categorical endpoints are included in this study (Data for all visits will also be 
presented) 
x Proportion of subjects who has ≥ 50% reduction from baseline in UUI episodes at 
Week 12  
x Proportion of subjects who has ≥ 75% reduction from baseline in UUI episodes at 
Week 12 
x Proportion of subjects who has 100% reduction from baseline in UUI episodes at 
Week 12 
x Proportion of subjects who has ≥ 50% reduction from baseline in average daily 
number of UI episodes at Week 12  
x Proportion of subjects who has ≥ 75% reduction from baseline in average daily 
number of UI episodes at Week 12 
x Proportion of subjects who has 100% reduction from baseline in average daily 
number of UI episodes at Week 12 
x Proportion of responders (subjects who achieve MID for I-QOL total score) at 
Week 6 and 12 
x Proportion of responders based on the PGI-C scale score at Week 12 
The Cochran-Mantel-Haenszel (CMH) common risk difference estimate stratified by the 
updated stratification factors (baseline UUI episodes per day (≤ 3 vs. > 3), and prior 
onabotulinumtoxinA use (naїve vs. prior use)) used during randomization with weights 
proposed by Greenland and Robins [3] will be used to compare the proportion of responders 
between the active and placebo groups. If there is a stratum for a treatment group that has 0 
subjects in it, the 0 count will be replaced by 0.5 in order to prevent dividing by 0 as suggested 
in Greenland and Robins.  The estimated common risk difference, and associated p-value and 
2-sided 95% confidence interval will be tabulated. 


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Other PGI questions collected on the PGI form, i.e. PGI-severity, PGI-control, PGI-frequency, 
and PGI-leakage will be summarized with count and frequency of each response at Week 12. 
6.3. 
Urodynamic Endpoints 
For urodynamic assessments only the independent central reviewer’s interpretation will be 
summarized. The frequency and percentage of central reader’s assessment compared to the 
investigator’s reading and descriptive summary of the results of each parameter will be 
provided for baseline and Week 12. The change from baseline at Week 12 will also be 
provided.  
6.4. 
hSlo cDNA concentrations 
The presence and absence of hSlo cDNA concentrations will be summarized at each protocol 
specified visit. If present, numeric results of the concentration will also be summarized at each 
visit.  
7. 
SAFETY ANALYSIS 
The safety population will be used for all safety analyses. Safety will be based on adverse 
events reporting, clinical laboratory data, ECGs, PVR, physical examinations, and vital signs. 
No inferential statistical testing is planned on the safety data, all data will be descriptively 
summarized by treatment and visit.  
7.1. 
Extent of Exposure and Compliance 
Since this is a treatment with single injection at the start of the study, no summary of exposure 
or compliance will be provided. A listing of study injection will be provided for all subjects in 
the safety population. 
7.2. 
Adverse Events 
Adverse events (AEs) will be coded using MedDRA version 22.1. or later. 
All reported AEs (whether treatment emergent or not) will be included in by-subject AE 
listings. Sorting will be by treatment, subject, date of event, SOC, PT and then verbatim 
description. 
An AE will be considered a treatment emergent adverse event (TEAE) if it begins or worsens 
in severity after initial exposure of the study treatment through 48 weeks after treatment 
administration or the date of the initiation of another OAB medication, investigational agent or 
surgical intervention, whichever occurs first. The determination of “48 weeks” will be based on 
the study day of the AE start date instead of the visit date of the Week 48 visit. The OAB 
medication will be determined based on the checkbox “Is this an additional OAB medication” 


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on the concomitant medication CRF. After data are collected and reviewed by the clinical 
team, a blinded list of investigational agents and surgical interventions will be provided to the 
programming team prior to database lock to determine which AEs should no longer be 
considered treatment emergent due to possible confounding effects. Partial AE start dates will 
be imputed as detailed in Section 4.2.3.  
Summary tables will be based on TEAEs. The incidence of TEAEs will be presented using 
counts and percentages of subjects with TEAEs and tabulated by SOC and PT. SOC will be 
sorted in descending frequency and PT within SOC will be sorted by descending frequency 
based on the incidence of all subjects treated with URO-902 included in the summary. If a 
subject has multiple occurrences (start and stop) of an event associated with a specific SOC or 
PT within a SOC, a subject will only be counted once in the incidence count for the SOC or PT 
within SOC respectively. 
An overall summary table of AEs by treatment group will be presented detailing the number 
and percentage of subjects, and number of events for the following categories: 
x At least one TEAE; 
x At least one Treatment-Related TEAE; 
x At least one Grade ≥ 3 TEAE (Mild = Grade 1, Moderate = Grade 2, Severe = 
Grade 3, Life-Threatening = Grade 4, Death = Grade 5) 
x At least one Grade ≥ 3 Treatment-Related TEAE; 
x At least one Serious TEAE; 
x At least one Serious Treatment-Related TEAE; 
x At least one TEAE leading to Discontinuation from Study; 
x At least one TEAE of Special Interest; 
x At least one Treatment-Related TEAE of Special Interest 
Except for all TEAEs which will be summarized by SOC and PT, the incidence of all other 
TEAEs by PT will be presented for the following in descending frequency based on the 
incidence of all subjects treated with URO-902. 
x All TEAEs by SOC and PT;  
x All TEAEs 
x Treatment-related TEAEs (i.e., Possibly Related or Probably Related); 
x All TEAEs by PT, and maximum severity (where the maximum intensity per 
subject will be counted at each level of summarization);  
x TEAEs with Grade ≥ 3; 


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x Treatment-related TEAEs with Grade ≥ 3; 
x Serious TEAEs; 
x Treatment-related serious TEAEs; 
x Fatal TEAE; 
x TEAEs leading to discontinuation from study; 
x TEAE of Special Interest; 
x Treatment-related TEAE of Special Interest; 
x Non-fatal serious TEAE; 
An AE is considered an AE of Special Interest if on the AE eCRF the answer to the item “Is 
this an AE of Special Interest?” is “Yes”. For specific criteria used to identify AEs of Special 
Interest refer to section 8.5.6 of the protocol. 
Overall AE listing will include the treatment arm, start and stop dates/times of the AE, 
treatment dates/times, and treatment emergent status. 
A Treatment related AE is defined as an AE for which the investigator classifies the AE as 
being “Possibly Related” or “Probably Related” to study treatment on Adverse Event eCRF. 
Missing relationship and severity (intensity) will be imputed per Section 4.2.3. 
Additional summary table will be provided for AEs that occurred on or after the study dose 
through 48 weeks after the study dose administration and after additional OAB medication was 
taken, or initiation of investigational agent, or surgical intervention. 
The following additional listings will be provided: 
x Listing of deaths 
x Listing of SAEs 
x Listing of TEAEs of Special Interest 
x Listing of TEAEs leading to study discontinuation 
7.3. 
Laboratory Evaluations 
All continuous laboratory parameters in hematology and clinical chemistry will be summarized 
descriptively by absolute value at each visit by treatment group, together with the 
corresponding changes from baseline. All parameters will be summarized in SI units. 
Hemoglobin and Hematocrit will also be summarized in Conventional units. 
The number and percentage of subjects with laboratory measurements outside of the central 
laboratory normal range will also be summarized by treatment group and visit. Shift tables 
from baseline to maximum post-baseline value, to minimum post-baseline value, last post-


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baseline value, and at each post-baseline visit will be provided for hematology and chemistry 
parameters to display low, normal, high, and missing values by treatment group in a 3-by-3 
contingency table. Denominators for percentages will be the number of subjects with non-
missing data at the specific assessment and baseline. 
A sample for the urinalysis and urine culture will be sent to the central laboratory only if the 
urine dipstick performed at the site tests positive for the presence of leukocytes, nitrites, or 
blood cells. Central urinalysis data will only be presented in the listing. 
Any data outside the central laboratory normal reference ranges will be explicitly noted on the 
listings that are produced.  
7.4. 
Vital Signs 
Vital sign data including blood pressure, pulse rate, respiration rate, and body temperature will 
be collected at all study visits except for the telephone follow-up visits. Height will be 
measured at Screening only.  
For all parameters, absolute values and change from baseline will be presented for scheduled 
visits using descriptive statistics for continuous variables. In addition, the maximum post-
baseline, change from baseline to the maximum post-baseline, and change from baseline to the 
last post-baseline value will be summarized for each of the vital signs. 
A by-subject listing, sorted by treatment and subject identifier, will be presented including all 
vital sign results (scheduled or unscheduled). 
7.5. 
ECG 
12-Lead ECG data will be collected at the screening visit and at Week 2, 12, and 24 visits. For 
all parameters, absolute values and change from baseline will be presented for scheduled visits 
using descriptive statistics for continuous variables. The evaluation result will be summarized 
at each visit as well.  
A by-subject listing, sorted by treatment and subject identifier, will be presented including all 
ECG results (scheduled or unscheduled). 
7.6. 
Physical Examination 
Physical examination data will be collected at Screening and Week 24. Shift tables (normal, 
abnormal, not done) of baseline versus the last observation post-baseline [normal, abnormal 
(same as baseline), abnormal (new or aggravated), not done] may be generated, presenting the 
assessment for each component of the physical examination separately. Listing of abnormal 
results will be produced.  


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7.7. 
PVR 
The volume of urine that remains in the bladder after voiding is an objective measurement that 
may serve as a proxy for impaired ability to void. Absolute values and change from baseline 
will be presented for scheduled visits using descriptive statistics for PVR measurements. In 
addition, the maximum post-baseline, change from baseline to the maximum post-baseline, and 
change from baseline to the last post-baseline value will be summarized. The maximum and 
the last post-baseline value will not use the visit windows rules and any non-missing post-
baseline values will be used for the derivation. A categorical summary will be based on the 
following levels: 
x < 100 mL 
x ≥ 100 and < 200 mL 
x ≥ 200 and < 350 mL 
x ≥ 350 mL 
All PVR measurements will be listed. 
7.8. 
Clean Intermittent Catheterization (CIC) 
For subjects with CIC initiated, time from study drug injection to the start of CIC (in days) and 
length of CIC use (in days) will be summarized by treatment arms. If CIC is used more than 
once, the length will be summed together for summary purpose. No imputation will be done for 
CIC start and stop dates. All CIC information will be provided in the listing. 
7.9. 
Bladder and Kidney Ultrasound 
Bladder and Kidney ultrasound results will not be summarized and will be provided in the 
listing. 
8. 
COVID-19 CONSIDERATIONS 
This study was conducted during the COVID-19 global pandemic.  During the time period of 
the pandemic, it is anticipated that changes in study visit schedules, missed visits, or patient 
discontinuations may lead to missing information (e.g., for protocol-specified procedures). In 
accordance with the FDA guidance of conduct of clinical trials of medical produces during 
COVID-19 public health emergency, it is important to capture specific information in the case 
report form that explains the basis of the missing data, including the relationship to COVID-19 
for missing protocol-specified information (e.g., from missed study visits or study 
discontinuations due to COVID-19).  


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The proportion of subjects with COVID-19 impact on visits will be summarized by treatment 
group overall and by visit.  The reasons (e.g. subject acquired COVID-19, Subject unable to 
travel due to COVID-19, investigative site closure due to COVID-19, etc) will also be 
summarized. A listing of subjects who had a COVID-19 information collected will be 
generated which will document the date of contact, update to AE or concomitant medication 
since the last visit, and whether the bladder diaries were completed. 
9. 
CHANGE FROM ANALYSIS PLANNED IN PROTOCOL 
Protocol section 9.3 defined analyses population. In the SAP Section 3.1, safety (modified) 
population was removed. 
Protocol section 3.2.1 defined exploratory efficacy endpoints. In the SAP Section 1.1.1 and 
Section 4.3.2 , ‘Nighttime voids’ was added as an exploratory efficacy endpoint. 
In the SAP Section 7.2 the definition of treatment-emergent adverse event was updated to 
include the additional OAB medication as the end of the treatment-emergent period in addition 
to the initiation of the investigational agent and/or surgical intervention.  
In Protocol section 9.5.1 PGI-C change from baseline was analyzed using MMRM method. In 
the SAP section 4.3.3 and 6.2 the analysis method for PGI-C was changed to use the responder 
analysis with CMH method.    
SAP added additional endpoint of proportion of subjects achieving ≥ 50%, ≥ 75% and 100% 
reduction in average daily UI episodes.  
Since no analysis plan is definitive especially for this exploratory study, we may perform 
additional data driven sensitivity analysis. 
10. 
REFERENCES 
[1] B. Schurch, P. Denys, C. M. Kozma, P. R. Reese, T. Slaton, and R. Barron, “Reliability 
and Validity of the Incontinence Quality of Life Questionnaire in Patients With 
Neurogenic Urinary Incontinence,” Arch. Phys. Med. Rehabil., vol. 88, no. 5, pp. 646–652, 
May 2007, doi: 10.1016/j.apmr.2007.02.009. 
[2] K. S. Coyne, C. L. Thompson, J.-S. Lai, and C. C. Sexton, “An overactive bladder 
symptom and health-related quality of life short-form: Validation of the OAB-q SF: 


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Statistical Analysis Plan – URO-902-2001 
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40 
Validation of the OAB-q SF,” Neurourol. Urodyn., vol. 34, no. 3, pp. 255–263, Mar. 2015, 
doi: 10.1002/nau.22559. 
[3] S. Greenland and J. M. Robins, “Estimation of a Common Effect Parameter from Sparse 
Follow-Up Data,” Biometrics, vol. 41, no. 1, p. 55, Mar. 1985, doi: 10.2307/2530643. 
 


 
 
11. 
APPENDIX 
11.1. 
Table of Contents for Data Display Specifications 
11.1.1. 
Output Tables 
Table 8: 
List of Output Tables  
Title 
Population 
 
Programming Note 
Deliverable 
Study Population  
14.1.1.1 
Subject Enrollment 
Screened 
 
EOS 
14.1.1.2 
Subject Disposition  
Randomized 
 
TL, EOS 
14.1.1.3 
Subject Randomization by Investigator 
Randomized 
 
EOS 
14.1.2.1 
Major Protocol Deviations  
Randomized 
 
EOS 
14.1.2.2 
Reasons for Exclusion from Analysis  
Randomized 
 
EOS 
14.1.3.1 
Demographics and Baseline Characteristics  
ITT-E 
 
TL, EOS 
14.1.4 
Medical History  
ITT-E 
 
EOS 
14.1.5.1 
Prior Medication  
ITT-E 
ATC Level 2 and 4 
EOS 
14.1.5.2 
Concomitant Medication  
ITT-E 
ATC Level 2 and 4 
EOS 
14.1.5.3 
Additional OAB Treatment Received Prior to Week 24 
(Medication)  
ITT-E 
ATC Levels 2, 4 and PT 
TL, EOS 
14.1.5.4 
Additional OAB Treatment Received at or After Week 24 
(Medication)  
ITT-E 
ATC Levels 2, 4 and PT 
TL, EOS 
14.1.5.5 
Additional OAB Treatment Received Prior to Week 24 
(Procedure) 
ITT-E 
ATC Levels 2, 4 and PT 
EOS 


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Title 
Population 
 
Programming Note 
Deliverable 
14.1.5.6 
Additional OAB Treatment Received at or After Week 24 
(Procedure) 
ITT-E 
ATC Levels 2, 4 and PT 
EOS 
14.1.6 
COVID-19 Impact 
ITT-E 
 
EOS 
Efficacy Endpoints  
 
 
EOS 
14.2.1.1 
Change from Baseline in Average Daily Number of Urge 
Urinary Incontinence Episodes (MMRM) 
ITT-E 
Week 12 first, then other 
visits sequentially  
TL, EOS 
14.2.1.2 
Summary of Average Daily Number of Urge Urinary 
Incontinence Episodes 
ITT-E 
By-visit Descriptive 
Summary 
TL, EOS 
14.2.1.3 
Summary of Average Daily Number of Urge Urinary 
Incontinence Episodes by Baseline UUI Episodes per Day  
ITT-E 
Subgroup columns within 
each treatment arm 
EOS 
14.2.1.4 
Summary of Average Daily Number of Urge Urinary 
Incontinence Episodes by Presence or absence of DO  
ITT-E 
Subgroup columns within 
each treatment arm 
EOS 
14.2.1.5 
Summary of Average Daily Number of Urge Urinary 
Incontinence Episodes by Previous Use of onabotulinumtoxinA 
ITT-E 
Subgroup columns within 
each treatment arm 
EOS 
14.2.1.6 
Change from Baseline in Average Daily Number of Urge 
Urinary Incontinence Episodes at Week 12 
ITT-E 
 
EOS 
14.2.1.7 
Change from Baseline in Average Daily Number of Urge 
Urinary Incontinence Episodes (MMRM) - Sensitivity Analysis 
ITT-ES 
Week 12 first, then other 
visits sequentially 
EOS 
14.2.2.1 
Change from Baseline in Average Daily Number of 
Micturitions (MMRM) 
ITT-E 
Week 12 first, then other 
visits sequentially 
TL, EOS 
14.2.2.2 
Summary of Average Daily Number of Micturitions 
ITT-E 
By-visit Descriptive 
Summary 
TL, EOS 
14.2.2.3 
Summary of Average Daily Number of Micturitions by 
Baseline UUI Episodes per Day  
ITT-E 
Subgroup columns within 
each treatment arm 
EOS 
14.2.2.4 
Summary of Average Daily Number of Micturitions by 
Presence or absence of DO  
ITT-E 
Subgroup columns within 
each treatment arm 
EOS 


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Title 
Population 
 
Programming Note 
Deliverable 
14.2.2.5 
Summary of Average Daily Number of Micturitions by 
Previous Use of onabotulinumtoxinA 
ITT-E 
Subgroup columns within 
each treatment arm 
EOS 
14.2.2.6 
Change from Baseline in Average Daily Number of 
Micturitions at Week 12 
ITT-E 
 
EOS 
14.2.2.7 
Change from Baseline in Average Daily Number of 
Micturitions (MMRM) - Sensitivity Analysis 
ITT-ES 
Week 12 first, then other 
visits sequentially 
EOS 
14.2.3.1 
Change from Baseline in Average Daily Number of Urinary 
Incontinence Episodes (MMRM) 
ITT-E 
Week 12 first, then other 
visits sequentially 
 TL, EOS 
14.2.3.2 
Summary of Average Daily Number of Urinary Incontinence 
Episodes 
ITT-E 
By-visit Descriptive 
Summary 
TL, EOS 
14.2.3.3 
Summary of Average Daily Number of Urinary Incontinence 
Episodes by Baseline UUI Episodes per Day  
ITT-E 
Subgroup columns within 
each treatment arm 
EOS 
14.2.3.4 
Summary of Average Daily Number of Urinary Incontinence 
Episodes by Presence or absence of DO  
ITT-E 
Subgroup columns within 
each treatment arm 
EOS 
14.2.3.5 
Summary of Average Daily Number of Urinary Incontinence 
Episodes by Previous Use of onabotulinumtoxinA 
ITT-E 
Subgroup columns within 
each treatment arm 
EOS 
14.2.4.1 
Change from Baseline in Average Daily Number of Urgency 
Episodes (MMRM) 
ITT-E 
Week 12 first, then other 
visits sequentially 
TL, EOS 
14.2.4.2 
Summary of Average Daily Number of Urgency Episodes 
ITT-E 
By-visit Descriptive 
Summary 
TL, EOS 
14.2.4.3 
Summary of Average Daily Number of Urgency Episodes by 
Baseline UUI Episodes per Day  
ITT-E 
Subgroup columns within 
each treatment arm 
EOS 
14.2.4.4 
Summary of Average Daily Number of Urgency Episodes by 
Presence or absence of DO  
ITT-E 
Subgroup columns within 
each treatment arm 
EOS 
14.2.4.5 
Summary of Average Daily Number of Urgency Episodes by 
Previous Use of onabotulinumtoxinA 
ITT-E 
Subgroup columns within 
each treatment arm 
EOS 
14.2.5.1 
Change from Baseline in Average Volume Voided per 
Micturition (MMRM) 
ITT-E 
Week 12 first, then other 
visits sequentially 
TL, EOS 


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Title 
Population 
 
Programming Note 
Deliverable 
14.2.5.2 
Summary of Average Volume Voided per Micturition 
ITT-E 
By-visit Descriptive 
Summary 
TL, EOS 
14.2.6.1 
Proportion of Subjects Achieving >= 50% Reduction in 
Number of Average Daily Urge Urinary Incontinence Episodes 
from Baseline 
ITT-E 
Week 12 first, then other 
visits sequentially 
EOS 
14.2.6.2 
Proportion of Subjects Achieving >= 75% Reduction in Urge 
Urinary Incontinence Episodes from Baseline 
ITT-E 
Week 12 first, then other 
visits sequentially 
EOS 
14.2.6.3 
Proportion of Subjects achieving 100% Reduction in Urge 
Urinary Incontinence Episodes from Baseline 
ITT-E 
Week 12 first, then other 
visits sequentially 
TL, EOS 
14.2.7.1 
Change from Baseline I-QOL Domains (MMRM) 
ITT-E 
Week 12 first, then Week 6 
TL, EOS 
14.2.7.2 
Summary of I-QOL Scores 
ITT-E 
By-visit Descriptive 
Summary 
TL, EOS 
14.2.7.3 
Proportion of Subjects Achieving the Minimally Important 
Difference in I-QOL Total Summary Score 
ITT-E 
Week 12 first, then Week 6 
EOS 
14.2.8.1 
Change from Baseline in OAB-q Scores (MMRM) 
ITT-E 
Week 12 first, then Week 6 
TL, EOS 
14.2.8.2 
Summary of OAB-q Scores 
ITT-E 
By-visit Descriptive 
Summary 
TL, EOS 
14.2.9.1 
Change from Baseline on Patient Global Impression Change 
Score at Week 12 
ITT-E 
Week 12 only 
TL, EOS 
14.2.9.2 
Summary of Patient Global Impression 
ITT-E 
By-visit Descriptive 
summary and change from 
baseline of numeric score 
EOS 
14.2.10 
Summary of Urodynamic Parameters 
ITT-E 
Baseline and Week 12 only 
EOS 
14.2.11 
Summary of hSlo cDNA Concentrations 
ITT-E 
 
TL, EOS 
14.2.12.1 
Change from Baseline in Average Daily Number of Nighttime 
Voids (MMRM) 
ITT-E 
Week 12 first, then other 
visits sequentially 
 EOS 


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Title 
Population 
 
Programming Note 
Deliverable 
14.2.12.2 
Summary of Average Daily Number of Nighttime Voids 
ITT-E 
By-visit Descriptive 
Summary 
EOS 
14.2.13.1 
Proportion of Subjects Achieving >= 50% Reduction in 
Number of Average Daily Urinary Incontinence Episodes from 
Baseline 
ITT-E 
Week 12 first, then other 
visits sequentially 
EOS 
14.2.13.2 
Proportion of Subjects Achieving >= 75% Reduction in 
Number of Average Daily Urinary Incontinence Episodes from 
Baseline 
ITT-E 
Week 12 first, then other 
visits sequentially 
EOS 
14.2.13.3 
Proportion of Subjects Achieving 100% Reduction in Number 
of Average Daily Urinary Incontinence Episodes from Baseline 
ITT-E 
Week 12 first, then other 
visits sequentially 
EOS 
Safety Endpoints 
Adverse Events 
14.3.1.1 
Overall Treatment-Emergent Adverse Events 
Safety 
 
TL, EOS 
14.3.1.2.1 
Treatment-Emergent Adverse Events by System Organ Class 
and Preferred Term 
Safety 
Sort by SOC and PT in 
descending frequency of 
URO-902 arms combined. 
TL, EOS 
14.3.1.2.2 
Treatment-Emergent Adverse Events Occurring between 
Weeks 1 and 24 by System Organ Class and Preferred Term 
Safety 
Sort by SOC and PT in 
descending frequency of 
URO-902 arms combined. 
EOS 
14.3.1.2.3 
Treatment-Emergent Adverse Events Occurring between 
Weeks 24 and 48 by System Organ Class and Preferred Term 
Safety 
Sort by SOC and PT in 
descending frequency of 
URO-902 arms combined. 
EOS 
14.3.1.3.1 
Treatment-Emergent Adverse Events by Preferred Term 
Safety 
Sort by PT in descending 
frequency of URO-902 arms 
combined. Same for all 
subsequent tables 
TL, EOS 
14.3.1.3.2 
Treatment-Related Treatment-Emergent Adverse Events by 
Preferred Term 
Safety 
 
EOS 


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Title 
Population 
 
Programming Note 
Deliverable 
14.3.1.4 
Treatment-Emergent Adverse Events by Preferred Term and 
Maximum Intensity 
Safety 
 
EOS 
14.3.1.5 
Treatment-Emergent Adverse Events with Grade ≥ 3 by 
Preferred Term 
Safety 
 
EOS 
14.3.1.6 
Treatment-Related Treatment-Emergent Adverse Events with 
Grade ≥ 3 by Preferred Term  
Safety 
 
EOS 
14.3.1.7 
Serious Treatment-Emergent Adverse Events by Preferred 
Term 
Safety 
 
TL, EOS 
14.3.1.8 
Treatment-Related Serious Treatment-Emergent Adverse 
Events by Preferred Term 
Safety 
 
EOS 
14.3.1.9 
Fatal Treatment-Emergent Adverse Events by Preferred Term 
Safety 
 
EOS 
14.3.1.10 
Treatment-Emergent Adverse Events Leading to Study 
Discontinuation by Preferred Term  
Safety 
 
TL, EOS 
14.3.1.11 
Treatment-Emergent Adverse Events of Special Interest by 
Preferred Term 
Safety 
 
EOS 
14.3.1.12 
Treatment-Related Treatment-Emergent Adverse Events of 
Special Interest by Preferred Term 
Safety 
 
EOS 
14.3.1.13 
Non-Fatal Serious Treatment-Emergent Adverse Events by 
Preferred Term 
Safety 
 
EOS 
14.3.1.14 
Adverse Events Occurred After Study Dose Through Week 48 
and After Initiation of Another OAB Medication, 
Investigational Agent, or Surgical Intervention 
Safety 
Same Layout as the Overall 
AE Summary Table 14.3.1.1 
EOS 
14.3.2.6 
List of Deaths 
Safety 
 
EOS 
14.3.2.7 
Listing of Serious Adverse Events 
Safety 
 
EOS 
14.3.2.8 
Listing of Treatment-Emergent Adverse Events Leading to 
Study Discontinuation 
Safety 
 
EOS 


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Title 
Population 
 
Programming Note 
Deliverable 
14.3.2.9 
Listing of Treatment-Emergent Adverse Events of Special 
Interest 
Safety 
 
EOS 
Laboratory data 
14.3.3.1 
Hematology Laboratory Parameters 
Safety 
Include observed and CFB 
EOS 
14.3.3.2 
Abnormal Classification of Hematology Laboratory Parameters 
Safety 
 
EOS 
14.3.3.3 
Shift Table of L/N/H Classification for Hematology Laboratory 
Parameters from Baseline  
Safety 
Include max/min post-dose, 
by visit, last-post dose  
EOS 
14.3.3.4 
Clinical Chemistry Laboratory Parameters 
Safety 
Include observed and CFB 
EOS 
14.3.3.5 
Abnormal Classification of Clinical Chemistry Laboratory 
Parameters 
Safety 
 
EOS 
14.3.3.6 
Shift Table of L/N/H Classification for Clinical Chemistry 
Laboratory Parameters from Baseline  
Safety 
Include max/min post-dose, 
by visit, last-post dose 
EOS 
Other Safety 
14.3.4 
Vital Sign Parameters 
Safety 
Include all observed and CFB 
EOS 
14.3.5 
Electrocardiograms Parameters 
Safety 
Include all observed and CFB 
EOS 
14.3.6 
Physical Examination Shift from Baseline 
Safety 
 
EOS 
14.3.7 
Post-void Residual Urine Volume 
Safety 
Include all observed and CFB 
TL, EOS 
14.3.8 
Clean Intermittent Catheterization 
Safety 
 
EOS 
 


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11.1.2. 
Output Figures 
Table 9: 
List of Output Figures  
Title 
Population 
 
Programming Note 
Deliverable 
Efficacy Endpoints  
 
 
 
14.2.1.1 
Line Plots of LSMEAN (SE) of Change from Baseline in 
Average Daily Number of UUI Episodes from MMRM 
ITT-E 
Include all individual weeks 
TL, EOS 
14.2.1.2 
Line Plots of LSMEAN (SE) of Change from Baseline in 
Average Daily Number of Micturitions from MMRM 
ITT-E 
Include all individual weeks.  
TL, EOS 
14.2.1.3 
Line Plots of LSMEAN (SE) of Change from Baseline in 
Average Daily Number of UI Episodes from MMRM 
ITT-E 
Include all individual weeks.  
EOS 
14.2.1.4 
Line Plots of LSMEAN (SE) of Change from Baseline in 
Average Daily Number of Urgency Episodes from MMRM 
ITT-E 
Include all individual weeks.  
EOS 
14.2.1.5 
Line Plots of LSMEAN (SE) of Change from Baseline in 
Average Volume Voided per Micturition from MMRM 
ITT-E 
Include all individual weeks. 
EOS 
14.2.1.6 
Line Plots of LSMEAN (SE) of Change from Baseline in 
Average Daily Number of Nighttime Voids from MMRM 
ITT-E 
Include all individual weeks. 
EOS 
14.2.1.7 
Line Plots of LSMEAN (SE) of Change from Baseline in Total 
Summary Score from I-QOL 
ITT-E 
 
EOS 
14.2.1.8 
Kaplan Meier Plot of Time to Study Discontinuation 
Safety 
 
EOS 
 
 


Urovant Sciences 
Statistical Analysis Plan – URO-902-2001 
21-JUL-2022 
49 
11.1.3. 
Output Listings 
Table 8: 
List of Output Listings  
Title 
Population 
        Programming Note 
Deliverable 
Disposition and Demographics 
 
 
 
16.2.1.1 
Listing of Subject Disposition for Screen Failures 
Screened 
 
EOS 
16.2.1.2 
Listing of Subject Disposition 
Randomized 
 
EOS 
16.2.1.3 
Listing of Subjects who did not Satisfy Inclusion/Exclusion 
Criteria  
Screened 
 
EOS 
16.2.1.4 
Listing of Subject Randomization Details 
Randomized 
 
EOS 
16.2.1.5 
Listing of Major Protocol Deviations 
Randomized 
 
EOS 
16.2.1.6 
Listing of Subject Demographic and Baseline Characteristics 
ITT-E 
 
EOS 
16.2.1.7 
Listing of Medical History  
ITT-E 
 
EOS 
16.2.1.8 
Listing of Prior and Concomitant Medications 
ITT-E 
 
EOS 
16.2.1.9 
Listing of Additional OAB Medications 
ITT-E 
 
EOS 
16.2.1.10 
Listing of Additional OAB Procedures 
ITT-E 
 
EOS 
16.2.1.11 
COVID-19 Impact 
ITT-E 
 
EOS 
16.2.1.12 
Listing of Analysis Populations 
Randomized 
 
EOS 
Efficacy 
 
 
EOS 
16.2.2.1 
Listing of Bladder Diary  
ITT-E 
 
EOS 
16.2.2.2 
Listing of I-QOL Scores 
ITT-E 
 
EOS 
16.2.2.3 
Listing of OAB-q Scores 
ITT-E 
 
EOS 
16.2.2.4 
Listing of Patient Global Impression Scale Scores 
ITT-E 
 
EOS 
16.2.2.5 
Listing of Urodynamic Parameters 
ITT-E 
 
EOS 
16.2.2.6 
Listing of hSlo cDNA Concentrations 
ITT-E 
 
EOS 
Safety  
 
 
 


Urovant Sciences 
Statistical Analysis Plan – URO-902-2001 
21-JUL-2022 
50 
Title 
Population 
        Programming Note 
Deliverable 
16.2.3.1 
Listing of Treatment Exposure  
Safety 
 
EOS 
16.2.3.2 
Listing of Adverse Events 
Safety 
 
EOS 
16.2.3.3 
Listing of Hematology Laboratory Results 
Safety 
 
EOS 
16.2.3.4 
Listing of Clinical Chemistry Laboratory Results 
Safety 
 
EOS 
16.2.3.5 
Listing of Urinalysis Laboratory Results 
Safety 
 
EOS 
16.2.3.6 
Listing of Vital Sign Results 
Safety 
 
EOS 
16.2.3.7 
Listing of ECG Results 
Safety 
 
EOS 
16.2.3.8 
Listing of Physical Examination 
Safety 
 
EOS 
16.2.3.9 
Listing of Post-void Residual Urine Volume 
Safety 
 
EOS 
16.2.3.10 
Listing of Clean Intermittent Catheterization 
Safety 
 
EOS 
16.2.3.11 
Listing of Bladder and Kidney Ultrasound 
Safety 
 
EOS