Phase 2, Open-Label Extension Study to Assess Long-Term Safety and Tolerability of Enobosarm (GTx 024) in Postmenopausal Women With Stress Urinary Incontinence
Phase: PHASE2
Conditions: Stress Urinary Incontinence
Sponsor: GTx

CLINICAL TRIAL PROTOCOL 
IND NUMBER: 126605 
Phase 2, Open-Label Extension Study to Assess Long-Term Safety and 
Tolerability of Enobosarm (GTx-024) in Postmenopausal Women With 
Stress Urinary Incontinence 
Protocol Number: G201004 
Sponsor: 
GTx, Inc. 
175 Toyota Center 
Memphis, TN 38103 
Sponsor Contact: 
Mayzie Johnston, PharmD 
Vice President, Clinical Development 
Telephone: +1 901-261-3858 
Project Manager: 
Shelley E. Evans 
Senior Project Manager, Medical Affairs Research 
Operations, PPD 
Shelley.Evans@ppdi.com 
Telephone: +1 910-558-5291 
Safety Hotline: 
1-866-966-8429 (US toll free) 
Version of Protocol:  
1.0 
Date of Protocol:  
21 May 2018 
 
CONFIDENTIAL  
All financial and nonfinancial support for this study will be provided by GTx. The concepts 
and information contained in this document or generated during the study are considered 
proprietary and may not be disclosed in whole or in part without the expressed, written 
consent of GTx. 
The study will be conducted according to the International Council for Harmonisation 
harmonised tripartite guideline E6(R2): Good Clinical Practice. 

GTx, Inc. 
GTx-024
Protocol G201004 Version 1.0 
21 May 2018
Protocol Approval -  Sponsor Signatory 
Study Title 
Phase 2, Open-Label Extension Study to Assess Long-Term Safety and
Tolerability of Enobosarm (GTx-024) in Postmenopausal Women With 
Stress Urinary Incontinence
Protocol Number 
G201004
Protocol Date 
21 May 2018
Protocol accepted and approved by:
Vice President, Clinical Development
Mayzie Johnston, PharmD 
GTx, Inc.
175 Toyota Plaza 
Memphis TN 38103
ignaturei
Date 
t

GTx, Inc.
Protocol 0201004 Version 1.0
Study Title
GTx-024 
21 May 2018
Protocol Approval -  Lead Statistician
Phase 2, Open-Label Extension Study to Assess Long-Term Safety and 
Tolerability of Enobosarm (GTx-024) in Postmenopausal Women With 
Stress Urinary Ineontinenee
Protocol Number 
G201004
Protocol Date
21 May 2018
Protocol accepted and approved by:
Lead Statistician
Elaine B. Hoffman, PhD 
Statistical Science Director 
Biostatistics 
PPD
3900 N Paramount Parkway 
Morrisville, NC 27560
PlaMe ilofbnui 
Stalistiail Science Director 
1 approve this docvnienl 
23 May 2018 15:37:39 -04:00
Signature
Date

GTx, Inc. 
GTx*024
Protocol G201004 Version 1.0 
21 May 2018
Protocol Approval -  Principal Investigator 
Study Title 
Phase 2, Open-Label Extension Study to Assess Long-Term Safety and
Tolerability of Enobosarm (GTx-024) in Postmenopausal Women With 
Stress Urinary Incontinence
Protocol Number 
G201004 
Protocol Date 
21 May 2018
Protocol accepted and approved by:
Principal Investigator
Kenneth M. Peters, MD 
*
Professor and Chair of Urology
Oakland University Wm. Beaumont School of Medicine
Beaumont Health -  Royal Oak, Ml 
3535 West 13 Mile Road, Suite 438 
Royal Oak, Ml 48073-6769
Tel. (248) 551-0387
Email: Kenneth.Peters@beaumont.edu
s\u\t
Signature 
Date

GTx, Inc. 
GTx-024 
Protocol G201004 Version 1.0 
21 May 2018 
 
Declaration of Investigator 
I have read and understood all sections of the protocol entitled “Phase 2, Open-Label 
Extension Study to Assess Long-Term Safety and Tolerability of Enobosarm (GTx-024) in 
Postmenopausal Women With Stress Urinary Incontinence” and the accompanying 
investigator’s brochure, version 16, dated 01 Aug 2017. 
I agree to supervise all aspects of the protocol and to conduct the clinical investigation in 
accordance with the Final Protocol Version 1.0, dated 21 May 2018, the International Council 
for Harmonisation harmonised tripartite guideline E6(R2): Good Clinical Practice and all 
applicable government regulations. I will not make changes to the protocol before consulting 
with GTx, Inc. or implement protocol changes without independent ethics committee 
approval except to eliminate an immediate risk to subjects. I agree to administer study 
treatment only to subjects under my personal supervision or the supervision of a 
subinvestigator. 
I will not supply the investigational drug to any person not authorized to receive it. 
Confidentiality will be protected. Subject identity will not be disclosed to third parties or 
appear in any study reports or publications. 
I will not disclose information regarding this clinical investigation or publish results of the 
investigation without authorization from GTx, Inc.  
 
 
 
Signature of Principal Investigator 
 
Date 
 
 
 
Printed Name of Principal Investigator 
 
 
 

GTx, Inc. 
GTx-024 
Protocol G201004 Version 1.0 
21 May 2018 
 
Statement of Compliance 
This study will be carried out in accordance with Good Clinical Practice (GCP) as required 
by the following: 
• United States (US) Code of Federal Regulations (CFR) applicable to clinical studies 
(45 CFR Part 46, 21 CFR Part 50, 21 CFR Part 56, and 21 CFR Part 312) 
• International Council for Harmonisation (ICH) E6; 62 Federal Register 25691 
(May 9, 1997) 
 

GTx, Inc. 
GTx-024 
Protocol G201004 Version 1.0 
21 May 2018 
 
Page 7 
Table of Contents 
Protocol Approval – Sponsor Signatory .................................................................................... 2 
Protocol Approval – Lead Statistician ....................................................................................... 3 
Protocol Approval – Principal Investigator................................................................................ 4 
Declaration of Investigator ........................................................................................................ 5 
Statement of Compliance ........................................................................................................... 6 
Table of Contents ....................................................................................................................... 7 
List of Tables ............................................................................................................................ 10 
Protocol Synopsis..................................................................................................................... 11 
List of Abbreviations................................................................................................................ 19 
1 
Introduction ...................................................................................................................... 20 
1.1 
Background and Rationale ...................................................................................... 20 
2 
Study Objectives............................................................................................................... 22 
2.1 
Primary Objective ................................................................................................... 22 
2.2 
Secondary Objectives.............................................................................................. 22 
3 
Investigational Plan .......................................................................................................... 23 
3.1 
Study Design ........................................................................................................... 23 
3.1.1 
Rationale of Study Design ....................................................................... 24 
3.1.1.1 
Risk Mitigation ........................................................................................ 24 
4 
Subject Selection and Withdrawal Criteria ...................................................................... 26 
4.1 
Selection of Study Population ................................................................................. 26 
4.1.1 
Inclusion Criteria ..................................................................................... 26 
4.1.2 
Exclusion Criteria .................................................................................... 27 
4.2 
Withdrawal of Subjects From the Study ................................................................. 28 
4.2.1 
Reasons for Withdrawal/Discontinuation ................................................ 28 
4.2.2 
Handling of Withdrawals ......................................................................... 29 
4.2.3 
Replacements ........................................................................................... 29 
5 
Study Treatments .............................................................................................................. 30 
5.1 
Method of Assigning Subjects to Treatment Groups .............................................. 30 
5.2 
Treatments Administered ........................................................................................ 30 
5.3 
Identity of Study Drug ............................................................................................ 30 
5.4 
Management of Clinical Supplies ........................................................................... 30 
5.4.1 
Study Drug Packaging and Storage ......................................................... 30 
5.4.2 
Study Drug Accountability ...................................................................... 31 

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5.5 
Overdose Management ........................................................................................... 31 
5.5.1 
Medication Errors .................................................................................... 31 
5.5.2 
Treatment of Medication Errors ............................................................... 31 
5.6 
Blinding................................................................................................................... 31 
5.6.1 
Breaking the Blind ................................................................................... 31 
5.7 
Treatment Compliance ............................................................................................ 32 
5.8 
Prior and Concomitant Medications ....................................................................... 32 
5.8.1 
Prohibited Concomitant Medications and Treatments ............................. 32 
6 
Study Assessments and Procedures .................................................................................. 34 
6.1 
Study Visits ............................................................................................................. 34 
6.1.1 
Baseline (Day 0) ...................................................................................... 34 
6.1.2 
Study Visits 1 to 3: Weeks 4 to 12, Clinic Visit ....................................... 35 
6.1.3 
Study Visit 4 Onwards: Week 24 Onwards, Clinic Visit ......................... 35 
6.1.4 
End-of-Study Visit ................................................................................... 36 
6.2 
Efficacy Assessments .............................................................................................. 36 
6.2.1 
Three-Day Voiding Diary ........................................................................ 36 
6.2.2 
Patient Global Impression of Severity ..................................................... 36 
6.2.3 
Patient Global Impression of Improvement ............................................. 37 
6.3 
Safety Assessments ................................................................................................. 37 
6.3.1 
Adverse Events ........................................................................................ 37 
6.3.1.1 
Definitions of Adverse Events ................................................................. 37 
6.3.1.2 
Eliciting and Documenting Adverse Events ............................................ 38 
6.3.1.3 
Reporting Adverse Events ....................................................................... 38 
6.3.1.4 
Assessment of Severity ............................................................................ 39 
6.3.1.5 
Assessment of Causality .......................................................................... 40 
6.3.1.6 
Follow-Up of Subjects Reporting Adverse Events .................................. 40 
6.3.2 
Halting Rules ........................................................................................... 40 
6.3.3 
Safety Assessments .................................................................................. 41 
6.3.3.1 
Vital Sign Measurements ......................................................................... 41 
6.3.3.2 
Height and Weight ................................................................................... 41 
6.3.3.3 
Prior and Concomitant Medications ........................................................ 41 
6.3.3.4 
Transvaginal Ultrasound .......................................................................... 42 
6.4 
Other Assessments .................................................................................................. 42 
6.5 
Pregnancy ................................................................................................................ 42 
6.6 
Laboratory Analyses ............................................................................................... 42 
7 
Statistical and Analytical Plan .......................................................................................... 44 
7.1 
Primary Safety Endpoint ......................................................................................... 44 
7.2 
Secondary Efficacy Endpoints ................................................................................ 44 

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Protocol G201004 Version 1.0 
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Page 9 
7.3 
Sample Size Calculation ......................................................................................... 44 
7.4 
Analysis Sets ........................................................................................................... 44 
7.5 
Description of Subgroups to be Analyzed .............................................................. 45 
7.6 
Statistical Analysis Methodology ........................................................................... 45 
7.6.1 
Analysis of Safety Endpoints ................................................................... 45 
7.6.2 
Analysis of Secondary Efficacy Endpoints ............................................. 46 
7.7 
Data Quality Assurance........................................................................................... 46 
7.7.1 
On-Site Audits.......................................................................................... 46 
7.7.2 
Data Management .................................................................................... 47 
8 
Ethics ................................................................................................................................ 48 
8.1 
Institutional Review Board ..................................................................................... 48 
8.2 
Ethical Conduct of the Study .................................................................................. 48 
8.3 
Subject Information and Consent............................................................................ 48 
9 
Investigator’s Obligations ................................................................................................ 50 
9.1 
Confidentiality ........................................................................................................ 50 
9.2 
Financial Disclosure and Obligations ..................................................................... 50 
9.3 
Investigator Documentation .................................................................................... 50 
9.4 
Study Conduct ......................................................................................................... 51 
9.5 
Adherence to Protocol............................................................................................. 51 
9.6 
Adverse Events and Study Report Requirements ................................................... 51 
9.7 
Investigator’s Final Report ..................................................................................... 51 
9.8 
Records Retention ................................................................................................... 52 
9.9 
Publications ............................................................................................................. 52 
10 Study Management ........................................................................................................... 53 
10.1 Monitoring .............................................................................................................. 53 
10.1.1 
Monitoring the Study ............................................................................... 53 
10.1.2 
Inspection of Records .............................................................................. 53 
10.2 Management of Protocol Amendments and Deviations.......................................... 53 
10.2.1 
Modification of the Protocol .................................................................... 53 
10.2.2 
Protocol Deviations.................................................................................. 53 
10.3 Study Termination ................................................................................................... 54 
10.4 Final Report ............................................................................................................ 54 
11 Reference List................................................................................................................... 56 
12 Appendices ....................................................................................................................... 57 
12.1 Appendix: Schedule of Events ................................................................................ 57 
12.2 Appendix: Patient Global Impression of Severity Scale......................................... 58 

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21 May 2018 
 
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12.3 Appendix: Patient Global Impression of Improvement Scale ................................ 59 
 
List of Tables 
Table 12–1 
Schedule of Events for Protocol G201004....................................................... 57 

GTx, Inc. 
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Protocol: G201004 Version 1.0 
21 May 2018 
 
Page 11 
PROTOCOL SYNOPSIS 
Protocol Number: 
G201004 
Name of Investigational 
Product:  
Enobosarm (GTx-024) 
Title of Study: 
Phase 2, Open-Label Extension Study to Assess 
Long-Term Safety and Tolerability of Enobosarm 
(GTx-024) in Postmenopausal Women With Stress Urinary 
Incontinence 
Sponsor: 
GTx, Inc. 
Phase of Development: 
Phase 2 
Trial Site(s): 
Approximately 65 (United States) 
Indication: 
Stress Urinary Incontinence 
Rationale: 
GTx-024 is an orally bioavailable and tissue-selective, 
nonsteroidal, selective androgen receptor modulator 
(SARM) that has demonstrated androgenic and anabolic 
activity and is currently being evaluated as a potential 
treatment for stress urinary incontinence (SUI) in 
postmenopausal women. 
Urinary incontinence and pelvic floor disorders are major 
health problems for women, especially as they age. 
Relaxation of pelvic floor muscles has been found to 
correlate with lower urinary tract symptoms, including 
SUI. Muscles of the pelvic floor and lower urinary tract are 
crucial for supporting the pelvic organs and micturition; 
however, damage to the muscles or lack of hormonal 
stimulation are thought to contribute to pelvic organ 
prolapse and urinary incontinence. 
Although anabolic steroids may increase muscle mass and 
strength, lack of oral bioavailability and known potential 
risks have limited their use. Nonsteroidal SARMs have 
potential to achieve benefits of anabolic steroid therapy 
(improved muscle mass, cholesterol/triglyceride levels, 
glucose metabolism, and bone density) with fewer adverse 
effects, such as hirsutism and acne, in women.  
Both nonclinical and clinical data suggest that SARMs 
may provide a new therapeutic option for pelvic floor and 
lower urinary tract disorders, as both testosterone and its 
more potent metabolite, dihydrotestosterone, have anabolic 
effects on muscle. 
GTx-024 has demonstrated androgenic and anabolic 
activity in male and female rat models. GTx-024 has 
consistently been observed to increase body weight, 

GTx, Inc. 
GTx-024 
Protocol: G201004 Version 1.0 
21 May 2018 
 
Page 12 
specifically muscle, in female rats. In a male rat model 
with castrate levels of serum testosterone, GTx-024 
induced hypertrophy of the levator ani muscle to 
approximately 120% of that of a noncastrated male. 
Another SARM (GSK2849466A), studied in an 
ovariectomized rat model that mimics SUI by disrupting 
urethral continence, was shown to increase urethral 
baseline pressure and the amplitude of urethral responses 
during sneezing by 64% and 74%, respectively, compared 
with the vehicle control. Furthermore, all of the rats (8/8) 
in the vehicle-treated group experienced fluid leakage 
during sneezing, whereas only 1 of the rats (1/8) in the 
SARM-treated group experienced such leakage upon 
similar challenge. Histologically, the SARM-treated 
animals had a reversal of the atrophy in urethral muscle 
observed in the control group. This preliminary in vivo 
study supports the clinical study of SARMs for the 
treatment of SUI.  
A proof-of-concept study is currently ongoing to 
investigate the effect of GTx-024 on symptoms of SUI in 
postmenopausal women. Available data from the 
18 subjects who received doses of GTx-024 3 mg once 
daily for 12 weeks showed a mean reduction in stress 
incontinence episodes of 81%, with all 18 subjects seeing a 
clinically significant reduction (>50% reduction in stress 
leaks) in the number of stress incontinence episodes by 
Week 12. 
A double-blind, placebo-controlled, parallel-design, 
randomized, multicenter, Phase 2 study (G201002) is 
currently ongoing to assess the clinical activity and safety 
of GTx-024 in postmenopausal women with SUI. The 
study consists of a screening period (4 weeks), treatment 
period (12 weeks), follow-up period (4 weeks), and 
durability period (16 weeks). 
 
The current 12-week, open-label extension study will 
enroll the following subjects from G201002:  
• From the first 225 subjects enrolled in G201002, 
only subjects randomly assigned to the placebo 
group and who have completed the 12-week 
treatment period and 16-week durability period of 
G201002 will be allowed to enroll in this study, 
once data from G201002 are unblinded. Placebo 
subjects who enrolled in the durability extension 
study (G201003) will be allowed to discontinue 
participation in G201003, once data from G201002 
have been unblinded. 

GTx, Inc. 
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Protocol: G201004 Version 1.0 
21 May 2018 
 
Page 13 
• From Subject 226 onwards, any subject who was 
randomly assigned to any treatment group and who 
has completed the 12-week treatment period and 
the 4-week follow-up period in Study G201002 will 
be allowed to enroll. 
This open-label extension study will provide additional 
long-term safety and tolerability data for GTx-024.  
Objectives: 
Primary Objective - Safety 
The primary objective in this study is to assess long-term 
safety and tolerability of GTx-024 (3 mg administered 
once daily) in postmenopausal women with SUI. 
Safety and tolerability will be assessed in relation to the 
following: 
• Incidence of adverse events (AEs) 
• Change from baseline in clinical laboratory 
evaluations 
• Change from baseline in sex hormone–binding 
globulin and testosterone levels 
• Change from baseline in vital sign measurements 
• Change from baseline in endometrial stripe 
thickness as assessed with transvaginal ultrasound 
(only in subjects with an intact uterus) 
Secondary Objectives - Efficacy 
The secondary objectives of this study are to describe the 
efficacy of GTx-024 (3 mg administered once daily) as a 
treatment for SUI in postmenopausal women in relation to: 
• Change from baseline in the mean number of stress 
incontinence episodes per day as measured with the 
3-day voiding diary  
• Change in subject-reported impression of SUI 
severity as measured with the Patient Global 
Impression of Severity scale (PGI-S) 
• Improvement relative to baseline in 
subject-reported impression of SUI improvement as 
measured with the Patient Global Impression of 
Improvement scale (PGI-I) 
• Change from baseline in the mean number of urge 
incontinence episodes per day as measured with the 
3-day voiding diary  
• Change from baseline in the mean number of total 
incontinence episodes (stress + urge) as measured 

GTx, Inc. 
GTx-024 
Protocol: G201004 Version 1.0 
21 May 2018 
 
Page 14 
with the 3-day voiding diary 
Subject Population: 
Inclusion Criteria 
Each subject must meet all of the following criteria to be 
enrolled in this study: 
1. Be an eligible subject from G201002, where an 
eligible subject is defined as: 
a. one of the first 225 subjects who were 
randomly assigned to the placebo group in 
G201002 and who have completed the 
required treatment and durability periods of 
that study, or;  
b. any subject from 226 onwards, who was 
randomly assigned to any treatment group 
and who completed the required treatment 
and follow-up periods of that study 
2. Be able to read, understand, and provide written, 
dated, informed consent prior to enrollment in the 
current study and be likely to comply with the 
study protocol and communicate with study 
personnel about AEs and other clinically important 
information 
3. Provide written consent to participate in the study 
within the following timeframes: 
a. for G201002 Subjects 1-225, within 30 days 
after unblinding of G201002 (subjects who 
consent to participate in G201003 will be 
allowed to discontinue from that study and 
consent to this study upon unblinding of 
G201002) 
b. for G201002 Subjects 226-493, within 30 
days of completing both the treatment and 
follow-up periods of G201002 
4. Agree to maintain a stable dose of any medication 
known to affect lower urinary tract function, 
including but not limited to anticholinergics, 
tricyclic antidepressants, beta-3 adrenergic 
agonists, or α-adrenergic blockers, throughout the 
duration of the study  
Exclusion Criteria 
A subject who meets any of the following criteria will be 
excluded from the study: 
1. Starts any new treatment (medication, pelvic floor 
physical therapy, or other treatment known to 
impact the pelvic floor) after completing G201002 

GTx, Inc. 
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Protocol: G201004 Version 1.0 
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Page 15 
that is known or suspected to affect lower urinary 
tract function, including vaginal rejuvenation 
2. Subject is currently taking systemic sex-hormone 
products (excludes intravaginal application of 
estradiol topical/tablet agents and hormones 
delivered via vaginal rings). The following washout 
periods are required if systemic hormonal products 
are discontinued prior to screening: 
a. minimum of 4 weeks for prior transdermal 
products or products with systemic absorption 
applied topically; 
b. minimum of 8 weeks for prior oral products; 
c. minimum of 8 weeks for prior intrauterine 
products, and;  
d. minimum of 3 months for prior hormonal 
implants or injectable drug therapy. 
3. Has a current cancer diagnosis (with the exception 
of nonmelanoma skin cancer) or any history of 
breast or endometrial cancer 
4. Has a known history or current episode of: 
a. New York Heart Association Stage ≥ 2 
hypertension (systolic blood pressure 
> 160 mmHg or diastolic blood pressure 
> 100 mmHg) at screening and/or baseline. 
Subjects with hypertension that has been treated 
and controlled with medication for ≥ 2 weeks 
prior to screening are eligible for participation 
b. Recent myocardial infarction or arterial or 
venous thromboembolic event (within 1 year) 
or a history of more than 1 myocardial 
infarction or arterial or venous thromboembolic 
event 
c. Cardiac-related syncopal event within the past 
year 
d. Cardio or cerebral vascular disease requiring 
surgical intervention (e.g., bypass surgery, 
angioplasty). For subjects with previous stent 
placement, please contact the medical monitor 
e. Congestive heart failure of Stage > 2 according 
to New York Heart Association criteria  
f. Angina pectoris 
5. Has a current or past history of any physical 
condition that, in the investigator’s opinion, might 
put the subject at risk, impact the absorption of the 
study drug, or interfere with interpretation of study 

GTx, Inc. 
GTx-024 
Protocol: G201004 Version 1.0 
21 May 2018 
 
Page 16 
results 
Study Design 
This is a multicenter, Phase 2, open-label extension study 
that will assess the long-term safety and tolerability of 
GTx-024.  
The current study will enroll the following subjects: 
• From the first 225 subjects enrolled in G201002, 
only subjects randomly assigned to the placebo 
group and who have completed the 12-week 
treatment period and 16-week durability period 
from G201002 will be allowed to enroll in this 
study, once data from G201002 are unblinded. 
Placebo subjects who enrolled in the durability 
extension study (G201003) will be allowed to 
discontinue participation in G201003, once data 
from G201002 have been unblinded 
• From Subject 226 onwards, any subject who was 
randomly assigned to any treatment group and who 
has completed the 12-week treatment period and 
the 4-week follow-up period in Study G201002 
All subjects in this study will receive GTx-024 3 mg orally, 
once daily. 
Baseline (Day 0) 
Subjects will be evaluated, and an additional written 
informed consent for the open-label extension study will 
be obtained. Procedures will be performed at baseline as 
specified in the schedule of events for the study.  
Long-term Open-label Extension Period 
Subjects who continue to meet the eligibility criteria will 
attend clinic visits at 4-week intervals for the first 
12 weeks of the open-label extension study (i.e., Weeks 4, 
8, and 12). Following the first 12 weeks, subjects will 
attend clinic visits every 12 weeks (i.e., 12 weeks from 
Visit 3 onwards). At each clinic visit, study procedures will 
be performed as specified in the schedule of events. 
There is currently no planned end to this study; instead, 
subjects will be on treatment and followed until marketing 
approval is granted to GTx-024 or until the withdrawal of 
the Investigational New Drug application. If marketing 
approval is granted to GTx-024, a plan for the transition of 
subjects from study drug to commercial drug product or 
other therapy will be implemented.  
Data from this open-label extension study will be used to 
assess the long-term safety and tolerability of GTx-024.  

GTx, Inc. 
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Protocol: G201004 Version 1.0 
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Page 17 
Estimated Study Duration: 
There is no scheduled end to the study, and duration will 
depend on the commercial availability of GTx-024. 
Safety Assessments: 
Safety assessments will include the following:  
• AEs 
• Clinical laboratory analyses 
• Levels of sex hormone–binding globulin and 
testosterone 
• Endometrial stripe thickness as assessed with 
transvaginal ultrasound (only for subjects with an 
intact uterus) 
Other standard safety assessments will include vital signs, 
height and weight measurements, concomitant 
medications, medical history, and brief physical 
examination findings. 
Efficacy Assessments: 
The following assessments will be conducted to evaluate 
efficacy: 
• 3-Day Voiding Diary 
The 3-day voiding diary is a study tool for subjects to 
report each episode of urinary void. The subject will 
be asked to record each episode of urinary leakage, 
the type (stress or urge) of leakage, and the severity 
of leakage. Subjects will also be asked to record each 
episode of voluntary urinary void and fluid intake. 
• PGI-S 
The PGI-S is a global rating of subject-reported 
impression of severity using a 4-point scale. Validity 
of the PGI-S has been established for application in 
SUI. 
• PGI-I 
The PGI-I is a global rating of subject-reported 
impression of improvement using a 7-point scale. 
Validity of the PGI-I has been established for 
application in SUI.  
Study Drug: 
Study drug will be provided as softgel capsules in bottle 
packaging. All subjects will receive GTx-024 3 mg once 
daily.  
Sample Size Calculation: 
No power calculations were carried out, as no formal 
statistical analysis will be performed. 
Analysis Sets: 
The safety analysis set (SAS) contains all subjects in the 
study who receive ≥ 1 dose of study drug. All safety 

GTx, Inc. 
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Protocol: G201004 Version 1.0 
21 May 2018 
 
Page 18 
analyses will use the SAS. 
The full analysis set (FAS) contains all randomized 
subjects who receive ≥ 1 dose of study drug and have ≥ 1 
postbaseline primary efficacy assessment (3-day voiding 
diary). All efficacy analyses will use the FAS. 
Methods of Analysis: 
A separate statistical analysis plan (SAP) will be finalized, 
providing detailed methods for all endpoints. In general, 
descriptive or summary statistics (n, mean, standard 
deviation, median, minimum and maximum values for 
continuous variables, and number [%] of subjects in each 
category for categorical variable) will be provided by visit. 
Source data for summary tables and statistical analyses 
will be presented as subject data listings. No imputation for 
missing values will be done. 
All statistical analyses will be performed using SAS 
(version 9.3 or higher). No formal statistical analysis will 
be performed as the open-label extension study is not 
powered for inferential statistics. Two-sided 95% 
confidence intervals will be provided where appropriate. 
Change from baseline will be between baseline (Day 0) 
and scheduled visits. The SAP will specify up to which 
visit summaries will be produced, as not all subjects will 
have been in the study for the same length of time. Further 
details on the analysis of the efficacy endpoints will be 
provided in the SAP. 
 
Safety Analyses 
All safety parameters (including AEs) will be summarized 
using the SAS.  
All AEs will be coded using the latest version of the 
Medical Dictionary for Regulatory Activities. All 
treatment-emergent AEs will be summarized and presented 
in the listings according to the number of subjects who 
report an event, the percentage of subjects with that event, 
the number of events, and the grade, duration, and 
relationship to treatment. Percentages will be based on the 
number of subjects who received treatment during the 
open-label extension study.  
Efficacy Analyses 
The primary analysis will be conducted on the primary 
endpoints at scheduled visits using the FAS.  
For continuous and ordinal endpoints, the treatment effect 
will be summarized descriptively by visit. 

GTx, Inc. 
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Protocol: G201004 Version 1.0 
21 May 2018 
 
Page 19 
List of Abbreviations 
Abbreviation 
Definition 
AE 
adverse event 
ALT 
alanine aminotransferase 
AST 
aspartate aminotransferase 
CBC 
complete blood count 
CFR 
Code of Federal Regulations 
CMP 
comprehensive metabolic panel 
EDC 
electronic data capture 
eCRF 
electronic case report form 
FAS 
full analysis set 
FDA 
US Food and Drug Administration 
GCP 
Good Clinical Practice 
HDL 
high-density lipoprotein 
ICF 
informed consent form 
ICH 
International Council for Harmonisation 
INR 
international normalized ratio 
IRB 
institutional review board 
MedDRA 
Medical Dictionary for Regulatory Activities 
NCI-CTCAE 
National Cancer Institute–Common Terminology Criteria for Adverse 
Events 
OTC 
over-the-counter 
PGI-I 
Patient Global Impression of Improvement scale 
PGI-S 
Patient Global Impression of Severity scale 
SAE 
serious adverse event 
SAP 
statistical analysis plan 
SARM 
selective androgen receptor modulator 
SAS 
safety analysis set 
SHBG 
sex hormone–binding globulin 
SUI 
stress urinary incontinence 
ULN 
upper limit of normal 
US 
United States 

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1 Introduction 
1.1 Background and Rationale 
Enobosarm (GTx-024) is an orally bioavailable and tissue-selective, nonsteroidal, selective 
androgen receptor modulator (SARM) that has demonstrated androgenic and anabolic activity 
and is currently being evaluated as a potential treatment for stress urinary incontinence (SUI) 
in postmenopausal women. 
Urinary incontinence and pelvic floor disorders are major health problems for women, 
especially as they age (Luber 2004). Relaxation of pelvic floor muscles has been found to 
correlate with lower urinary tract symptoms, including SUI. Muscles of the pelvic floor and 
lower urinary tract are crucial for supporting the pelvic organs and micturition; however, 
damage to the muscles and/or lack of hormonal stimulation are thought to contribute to pelvic 
organ prolapse and urinary incontinence.  
Although anabolic steroids may increase muscle mass and strength, lack of oral 
bioavailability and known potential risks have limited their use (Mohler et al 2009). 
Nonsteroidal SARMs have potential to achieve benefits of anabolic steroid therapy 
(improved muscle mass, cholesterol/triglyceride levels, glucose metabolism, and bone 
density) with fewer adverse effects, such as hirsutism and acne, in women.  
Both nonclinical and clinical data suggest that SARMs may provide a new therapeutic option 
for pelvic floor and lower urinary tract disorders, as both testosterone and its more potent 
metabolite, dihydrotestosterone, have anabolic effects on muscle.  
GTx-024 has demonstrated androgenic and anabolic activity in male and female rat models. 
GTx-024 has consistently been observed to increase body weight, specifically muscle, in 
female rats. In a male rat model with castrate levels of serum testosterone, GTx-024 induced 
hypertrophy of the levator ani muscle to approximately 120% of that of a noncastrated male. 
Another SARM (GSK2849466A), studied in an ovariectomized rat model that mimics SUI 
by disrupting urethral continence, was shown to increase urethral baseline pressure and the 
amplitude of urethral responses during sneezing by 64% and 74%, respectively, compared 
with the vehicle control (GTx, Inc. 2017; Herzog and Fultz 1990; Kadekawa et al. 2015). 
Furthermore, all of the rats (8/8) in the vehicle-treated group experienced fluid leakage during 
sneezing, whereas only 1 of the rats (1/8) in the SARM-treated group experienced such 
leakage upon similar challenge. Histologically, the SARM-treated animals had a reversal of 

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the atrophy in urethral muscle that was observed in the control group. This preliminary in 
vivo study supports the clinical study of SARMs for the treatment of SUI.  
A proof-of-concept study is currently ongoing to investigate the effect of GTx-024 on 
symptoms of SUI in postmenopausal women. Available data from the 18 subjects who 
received doses of GTx-024 3 mg once daily for 12 weeks showed a mean reduction in stress 
incontinence episodes of 81%, with all 18 subjects experiencing clinically significant 
reduction (>50% reduction in stress leaks) in the number of stress incontinence episodes by 
Week 12. 
A double-blind, placebo-controlled, parallel-design, randomized, multicenter, Phase 2 study 
(G201002) is currently ongoing to assess the clinical activity and safety of GTx-024 in 
postmenopausal women with SUI. The study consists of a screening period (4 weeks), 
treatment period (12 weeks), follow-up period (4 weeks), and durability period (16 weeks). 
The current 12-week, open-label extension study will enroll the following subjects from 
G201002:  
• From the first 225 subjects enrolled in G201002, only subjects randomly assigned to the 
placebo group and who have completed the 12-week treatment period and 16-week 
durability period from G201002 will be allowed to enroll in this study, once data from 
G201002 are unblinded. Placebo subjects who enrolled in the durability extension study 
(G201003) will be allowed to discontinue participation in G201003, once data from 
G201002 have been unblinded 
• From Subject 226 onwards, any subject who was randomly assigned to any treatment 
group and who has completed the 12-week treatment period and the 4-week follow-up 
period in Study G201002 will be allowed to enroll 
This open-label extension study will provide additional long-term safety and tolerability data 
for GTx-024. All subjects in this study will receive GTx-024 3 mg orally, once daily.  

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2 Study Objectives 
2.1 Primary Objective 
The primary objective in this study is to assess long-term safety and tolerability of GTx-024 
(3 mg administered once daily) in postmenopausal women with SUI. 
Safety and tolerability will be assessed in relation to the following: 
• Incidence of adverse events (AEs) 
• Change from baseline in clinical laboratory evaluations 
• Change from baseline in sex hormone–binding globulin (SHBG) and testosterone levels 
• Change from baseline in vital sign measurements 
• Change from baseline in endometrial stripe thickness as assessed with transvaginal 
ultrasound (only in subjects with an intact uterus) 
2.2 Secondary Objectives 
The secondary objectives of this study are to describe the efficacy of GTx-024 (3 mg 
administered once daily) as a treatment for SUI in postmenopausal women in relation to: 
• Change from baseline in the mean number of stress incontinence episodes per day as 
measured with the 3-day voiding diary  
• Change in subject-reported impression of SUI severity as measured with the Patient 
Global Impression of Severity scale (PGI-S) 
• Improvement relative to baseline in subject-reported impression of SUI improvement as 
measured with the Patient Global Impression of Improvement scale (PGI-I) 
• Change from baseline in the mean number of urge incontinence episodes per day as 
measured with the 3-day voiding diary  
• Change from baseline in the mean number of total incontinence episodes (stress + urge) 
as measured with the 3-day voiding diary 

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3 Investigational Plan 
3.1 Study Design 
This is a multicenter, Phase 2, open-label extension study that will assess the long-term safety 
and tolerability of GTx-024.  
The current study will enroll the following subjects from G201002: 
• From the first 225 subjects enrolled in G201002, only subjects randomly assigned to the 
placebo group and who have completed the 12-week treatment period and 16-week 
durability period will be allowed to enroll in this study, once data from G201002 are 
unblinded. Placebo subjects who enrolled in the durability extension study (G201003) 
will be allowed to discontinue participation in G201003, once data from G201002 have 
been unblinded 
• From Subject 226 onwards, any subject who was randomly assigned to any treatment 
group and who has completed the 12-week treatment period and the 4-week follow-up 
period in Study G201002 
All subjects in this study will receive GTx-024 3 mg orally, once daily. 
The study consists of the following visits:  
Baseline (Day 0) 
Subjects will be evaluated, and an additional written informed consent for the open-label 
extension study will be obtained. Procedures will be performed at baseline as specified in the 
schedule of events (Table 12–1) for the study.  
Long-term Open-label Extension Period 
Subjects who continue to meet the eligibility criteria will attend clinic visits at 4-week 
intervals for the first 12 weeks of the open-label extension study (i.e., Weeks, 4, 8, and 12). 
Following the first 12 weeks, subjects will attend clinic visits every 12 weeks (i.e., 12 weeks 
from Visit 3 onwards). At each clinic visit, study procedures will be performed as specified in 
the schedule of events (Table 12–1). 
There is currently no planned end to this study; instead, subjects will be on treatment and 
followed until marketing approval is granted to GTx-024 or until the withdrawal of the 
Investigational New Drug application. If marketing approval is granted to GTx-024, a plan 

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for the transition of subjects from study drug to commercial drug product or other therapy 
will be implemented.  
Data from this open-label extension study will be used to assess the long-term safety and 
tolerability of GTx-024. 
3.1.1 Rationale of Study Design  
In prior clinical studies, GTx-024 has been generally well tolerated, including single doses up 
to 100 mg and multiple doses up to 30 mg once daily for up to 14 days. In longer studies, 
GTx-024 has also been generally well tolerated at 1, 3, and 9 mg daily doses for up to 
833 days (GTx, Inc. 2017). The open-label extension study will provide additional long-term 
safety and tolerability data for GTx-024. 
3.1.1.1 Risk Mitigation 
This study has been designed to mitigate known risks associated with the use of GTx-024. 
Clinical study G200501 observed the effects of administering doses of 0.1, 0.3, 1, and 3 mg 
of GTx-024 (or placebo) for 86 days to healthy subjects: postmenopausal women and elderly 
men. Dose-dependent increases in serum alanine aminotransferase (ALT) and aspartate 
aminotransferase (AST) were observed. A higher incidence of transient, asymptomatic 
increases in ALT levels reported as AEs occurred in the group receiving GTx-024 3 mg 
(20.8%) compared with the placebo group (0%). One subject who received 3 mg/day of 
GTx-024 was discontinued due to an ALT level >3 times the upper limit of normal (ULN). 
The ALT levels returned to normal with continued exposure to GTx-024 in most cases and, 
furthermore, in instances when dosing was not continued, levels returned to normal. No 
significant increases in levels of total bilirubin, gamma glutamyl transferase, alkaline 
phosphatase, or lactate dehydrogenase have been observed in subjects with elevated ALT 
levels. Consistent with the effects of other orally administered anabolic agents, GTx-024 
causes a dose-dependent reduction in high-density lipoprotein (HDL), the clinical 
significance of which is unknown at this time. The proposed mechanism for reduction in 
HDL is due to stimulation of reverse cholesterol transport and increased HDL catabolism by 
hepatic lipase. Reductions in HDL levels are temporary and typically return to baseline levels 
12 months after treatment initiation.  
In an ongoing, Phase 2 study of GTx-024 in estrogen receptor–positive/androgen 
receptor-positive breast cancer that is examining daily doses of GTx-024 at 9 and 18 mg, 
several cases of clinically significant hypercalcemia have been observed that were assessed 
by the investigators as possibly related to GTx-024. Subjects with hypercalcemia had 

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estrogen receptor–positive breast cancer with bone metastases. Hypercalcemia has not been 
observed in previous studies of healthy individuals or at doses <9 mg. 
Subjects will be monitored at each visit for health changes since the previous visit and 
adverse effects of treatment. Since elevations in ALT levels have been observed in prior 
studies, serum ALT and other liver function analytes will be monitored at each visit during 
treatment (Table 12–1). Stopping rules have been included for severe elevations of liver 
function tests or other signs of hepatotoxicity (Section 6.3.2). Subjects will also be monitored 
for changes in lipid profile and serum calcium. 
The effect of GTx-024 on a fetus has not been evaluated; therefore, the subjects in the current 
investigation will be postmenopausal.  

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4 Subject Selection and Withdrawal Criteria 
4.1 Selection of Study Population 
Up to 500 subjects will be enrolled at around 65 sites in the United States (US). Subjects will 
be enrolled in the current study only if they meet all of the inclusion criteria and none of the 
exclusion criteria. 
Deviations from the inclusion and exclusion criteria are not allowed because they can 
potentially jeopardize the scientific integrity of the study, regulatory acceptability, or subject 
safety. Therefore, adherence to the criteria as specified in the protocol is essential. 
4.1.1 Inclusion Criteria 
Each subject must meet all of the following criteria to be enrolled in this study: 
1. Be an eligible subject from G201002, where an eligible subject is defined as: 
a. one of the first 225 subjects who were randomly assigned to the placebo 
group in G201002 and who have completed the required treatment and 
durability periods of that study, or;  
b. any subject from 226 onwards, who was randomly assigned to any 
treatment group and who completed the required treatment and follow-up 
periods of that study 
2. Be able to read, understand, and provide written, dated informed consent prior to 
enrollment in the current study and be likely to comply with the study protocol and 
communicate with study personnel about AEs and other clinically important 
information 
3. Provide written consent to participate in the study within the following timeframes: 
a. for G201002 Subjects 1-225, within 30 days after the unblinding of 
G201002 (subjects who consent to participate in G201003 will be allowed 
to discontinue from that study and consent to this study upon unblinding of 
G201002) 
b. for G201002 Subjects 226-493, within 30 days of completing both the 
treatment and follow-up period of G201002 

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4. Agree to maintain a stable dose of any medication known to affect lower urinary 
tract function, including but not limited to anticholinergics, tricyclic 
antidepressants, beta-3 adrenergic agonists, or α-adrenergic blockers, throughout 
the duration of the study  
4.1.2 Exclusion Criteria 
A subject who meets any of the following criteria will be excluded from the study: 
1. Starts any new treatment (medication, pelvic floor physical therapy, or other 
treatment known to impact the pelvic floor) after completing G201002 that is 
known or suspected to affect lower urinary tract function, including vaginal 
rejuvenation 
2. Subject is currently taking systemic sex-hormone products (excludes intravaginal 
application of estradiol topical/tablet agents and hormones delivered via vaginal 
rings). The following washout periods are required if systemic hormonal products 
are discontinued prior to screening: 
a. minimum of 4 weeks for prior transdermal products or products with systemic 
absorption applied topically; 
b. minimum of 8 weeks for prior oral products; 
c. minimum of 8 weeks for prior intrauterine products, and;  
d. minimum of 3 months for prior hormonal implants or injectable drug therapy. 
3. Has a current cancer diagnosis (with the exception of nonmelanoma skin cancer) or 
any history of breast or endometrial cancer 
4. Has a known history or current episode of: 
a. New York Heart Association Stage ≥ 2 hypertension (systolic blood pressure 
> 160 mmHg or diastolic blood pressure > 100 mmHg) at screening and/or 
baseline. Subjects with hypertension that has been treated and controlled with 
medication for ≥ 2 weeks prior to screening are eligible for participation 
b. Recent myocardial infarction or arterial or venous thromboembolic event 
(within 1 year) or a history of more than 1 myocardial infarction or arterial or 
venous thromboembolic event 
c. Cardiac-related syncopal event within the past year 

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d. Cardio or cerebral vascular disease requiring surgical intervention (e.g., 
bypass surgery, angioplasty). For subjects with previous stent placement, 
please contact the medical monitor 
e. Congestive heart failure of Stage > 2 according to New York Heart 
Association criteria  
f. Angina pectoris 
5. Has a current or past history of any physical condition that, in the investigator’s 
opinion, might put the subject at risk, impact absorption of the study drug, or 
interfere with interpretation of study results  
4.2 Withdrawal of Subjects from the Study 
The duration of the study is not fixed and is defined for each subject as the date signed, 
written informed consent is provided through the last follow-up visit depending on the 
commercial availability of GTx-024. 
4.2.1 Reasons for Withdrawal/Discontinuation 
Subjects are free to withdraw from the study at any time for any reason.  
In addition, subjects may be withdrawn from the study by the principal investigator in 
consultation with GTx, Inc. for any of the following reasons: 
• AEs that require treatment with a prohibited medication or procedure 
• Development of any condition that may pose an additional risk to the subject or if the 
principal investigator decides it is in the best interest of the subject to withdraw from the 
study 
• Sponsor’s decision 
• Subject is unable to follow investigators’ instructions and/or to comply with the study 
procedures 
• Protocol deviation 
• Disease progression 
The clinical study report will include reasons for all subject withdrawals from treatment as 
well as details relevant to violations of study prohibitions and concomitant therapy. 

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4.2.2 Handling of Withdrawals  
Subjects are free to withdraw from the study or study treatment at any time upon request. 
Subject participation in the study may be stopped at any time at the discretion of the 
investigator or at the request of the Sponsor. 
Subjects who discontinue study treatment or active participation in the study will no longer 
receive GTx-024 and will be considered to have withdrawn from the study. When a subject 
withdraws from the study, the reason(s) for withdrawal shall be recorded by the investigator 
on the relevant page of the electronic case report form (eCRF). Subjects who discontinue 
from study drug will be monitored for AEs for 30 days after they stop treatment with study 
drug. Every effort will be made to obtain all end-of-treatment measures as outlined in Table 
12–1, in the event that a subject withdraws, or is withdrawn, from the study before 
transitioning to commercial product or other therapy. Subjects who are not returning for final 
assessments will be contacted (2 documented telephone calls followed by 1 registered letter) 
by the site in an attempt to improve their compliance. 
It is vital to obtain follow-up data on any subject withdrawn because of an AE or serious AE 
(SAE). In every case, efforts must be made to undertake protocol-specified, safety follow-up 
procedures. All data collected from all subjects, including early withdrawals and early 
discontinuations of treatment, will be used in the reporting and analysis of the study. 
4.2.3 Replacements 
Subjects who discontinue prematurely from the study will not be replaced.  

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5 Study Treatments 
Refer to Section 3.1 for a full description of the study design. All subjects will receive 
GTx-024 3 mg administered orally, once daily. Study drug will be supplied as opaque, white 
to off-white, size 5, oval softgel capsules. 
5.1 Method of Assigning Subjects to Treatment Groups 
Subjects who meet all eligibility criteria will continue the current open-label extension study 
with randomization code as assigned by an interactive web response system. 
5.2 Treatments Administered 
Study drug (Section 5) will be taken orally with water at approximately the same time each 
day, with or without food. Subjects will be required to take one 3 mg Softgel capsule per day. 
In the event of a missed dose, subjects will be instructed to skip the missed dose and continue 
dosing as per label instructions from the next day. 
5.3 Identity of Study Drug 
The GTx-024 study drug is an opaque, white to off-white, size 5, oval softgel capsule that 
contains the active ingredient GTx-024 (3 mg) dissolved in the inactive excipient 
polyethylene glycol 400. All study drug capsules will be imprinted with “GTx” in black ink 
on the outer shell of the capsule.  
5.4 Management of Clinical Supplies 
GTx, Inc. will provide adequate supplies of study drug through a central distribution center to 
clinical sites. 
5.4.1 Study Drug Packaging and Storage 
Study drug will be packaged in high-density polyethylene bottles with induction seal and 
child-resistant closure. Each bottle will contain sufficient study drug for 35 days of dosing 
according to the study protocol. Three bottles (a total of 105 dosing days) will be packaged 
together in one subject kit. For the first 3 visits, sites will collect the study drug previously 
dispensed, perform accountability, and re-dispense the same kit. 
Each subject kit, and the bottles contained in the kit, will be labeled with a unique medication 
identification number. A clinical label with the medication identification number will be 
affixed to the outside of each bottle and subject kit. The label will also specify storage and 
dosing information. 

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At the study site, study drug must be stored in a secure area (e.g., a locked cabinet), protected 
from moisture, and kept at a controlled room temperature of 15oC to 25oC (59oF-77oF), with 
excursions permitted to 30oC (86oF). 
5.4.2 Study Drug Accountability 
The investigator will maintain accurate records of receipt of all study drug, including dates of 
receipt. In addition, accurate records will be kept regarding when and how much study drug 
is dispensed to and used by each subject in the study. Reasons for departure from the 
expected dispensing regimen must be recorded. At the completion of the study, to satisfy 
regulatory requirements regarding drug accountability, all study drug will be reconciled and 
retained or destroyed according to applicable regulations and Sponsor requirements. 
5.5 Overdose Management 
In Phase 1 studies, subjects have safely received single doses of up to GTx-024 100 mg. The 
likelihood of a need for other than symptomatic treatment of overdose is considered 
extremely low. In the event of an overdose reported to the study site, the medical monitor 
should be contacted immediately. 
5.5.1 Medication Errors 
Dispensing study drug to subjects for self-administration in an outpatient study increases the 
risk of medication errors. All errors in medication dispensing or administration must be 
carefully documented. These errors may include (but are not limited to) providing the wrong 
dose, medication loss, or administration at the wrong time of day. Adherence to the 
medication protocol will be emphasized at every visit.  
5.5.2 Treatment of Medication Errors 
The treatment of medication errors should be discussed with the medical monitor on a 
case-by-case basis.  
5.6 Blinding 
This is an open-label study, and all subjects will receive study drug (GTx-024 3 mg). 
5.6.1 Breaking the Blind 
Not applicable, as this is an open-label study. 

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5.7 Treatment Compliance 
Individual subject compliance in taking study drug will be monitored by counting the unused 
medication that is returned by the subject at visits. Compliance will be documented. If 
compliance is < 80% or > 120%, the investigator or designee is to counsel the subject and 
ensure that steps are taken to improve compliance. Subjects who are < 80% or > 120% 
compliant with the dose regimen for any 2 consecutive visit periods during the study may be 
withdrawn from the study. 
5.8 Prior and Concomitant Medications 
The investigator or designee must record the use of prior medications (all medication taken 
within 30 days prior to baseline, Day 0) and current treatment (including both drug and 
nondrug therapies and all prescribed, over-the-counter [OTC], and alternative medicines) in 
the eCRFs. The minimum requirement is that the drug name and dates of administration are 
to be recorded. This also includes drugs used on a chronic or as-needed basis. Subjects must 
be instructed not to start any new medication, either prescribed or OTC, without consulting 
the investigator, unless the new medication is required for emergency use. Subjects must be 
instructed to notify the investigator immediately if medications are required for emergency 
use. 
Any changes in concomitant medications also will be recorded in the subject’s eCRF. 
Any concomitant medication deemed necessary for the welfare of the subject during the 
study may be given at the discretion of the investigator. However, it is the responsibility of 
the investigator to ensure that details regarding the medication are recorded in full in the 
eCRF. 
5.8.1 Prohibited Concomitant Medications and Treatments 
Prohibited medications and treatments during the study duration include the following: 
• Systemic hormonal products including, but not limited to, estrogens; progestins; 
testosterone; methyltestosterone; oxandrolone (Oxandrin®); oxymetholone; danazol; 
fluoxymesterone (Halotestin®); other androgenic compounds, including herbals such as 
fenugreek, Korean ginseng, goat weed, forskolin, and ashwagandha; and Testro-X®. 
Intravaginal application of estradiol topical/tablet agents and hormones delivered via 
vaginal ring are allowed 

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• Any new treatment (medication or otherwise), including vaginal rejuvenation, that is 
known or suspected to affect lower urinary tract function throughout the treatment and 
follow-up periods 
• Treatment with any other investigational agent  
• New pelvic floor exercises (including Kegel) throughout the follow-up period  

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6 Study Assessments and Procedures 
Before performing any study procedures, all potential subjects will sign an additional 
informed consent form (ICF) for this open-label extension study. Subjects will have the 
opportunity to have any questions answered before they sign the ICF. The investigator must 
address all questions raised by the subject. The investigator will also sign the ICF.  
6.1 Study Visits  
The schedule of events for this study is presented in Table 12–1.  
6.1.1 Baseline (Day 0) 
The study will be explained in detail, and adequate time will be allowed for answering 
questions from a potential subject before she signs the institutional review board (IRB)–
approved informed consent document. Informed consent will be obtained from those 
potential subjects who meet initial inclusion/exclusion criteria and express willingness to 
participate before study site personnel conduct any study assessments of such subjects.  
Once informed consent is obtained, the subjects will be assigned a subject number and a 
detailed interview will be conducted with the subject about her concomitant medications, and 
recent medical history will be evaluated. Height and weight will be measured, and vital signs 
will be assessed as presented in Table 12–1.  
The subject’s eligibility criteria will be reviewed. During the visit, the subject will complete 
the PGI-S. Blood will be drawn for a comprehensive metabolic panel (CMP), complete blood 
count (CBC), lipid panel, hormone panel, and SHBG tests. A baseline transvaginal ultrasound 
examination will also be performed in subjects with an intact uterus. 
The subject will be provided with a 3-day voiding diary and instructed to complete the 3-day 
voiding diary within the week preceding her next study visit. 
Study drug will be dispensed with administration instructions. The medication identification 
number assigned to the subject will be noted in the eCRF for study drug accountability. The 
subject will take the first dose of study drug during the baseline visit. The subject will be 
reminded to bring all unused study drug (in its original packaging) to her next clinic visit. 
Any subject who prematurely discontinues the study or study drug will be asked to return to 
the study site and complete end-of-study assessments. 

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6.1.2 Study Visits 1 to 3: Weeks 4 to 12, Clinic Visit 
The subject will return to the study site for a clinic visit as per the schedule of events 
described in Table 12–1. Concomitant medications and AEs will be reviewed. Weight and 
vital signs will be measured. 
The 3-day voiding diary provided to the subject at her previous visit will be collected and 
completion verified. The subject will be provided with a new 3-day voiding diary and 
instructed to complete it within the week preceding her next study visit. The subject will also 
complete the PGI-S and PGI-I during each site visit. Blood will be drawn for a CMP, CBC, 
lipid panel, hormone panel, and SHBG tests.  
Study drug will be dispensed with administration instructions. The medication identification 
number assigned to the subject will be noted in the eCRF for study drug accountability. The 
subject will be reminded to bring all unused study drug (in its original packaging) to her next 
clinic visit. 
A transvaginal ultrasound examination will be performed in subjects with an intact uterus 
only at Visit 3. 
6.1.3 Study Visit 4 Onwards: Week 24 Onwards, Clinic Visit 
During the long-term safety period (after Visit 3), the subject will return to the study site for 
clinic visits. Concomitant medications and AEs will be reviewed. Weight and vital signs will 
be measured. Blood will be drawn for a CMP, CBC, and lipid panel. Completion of a 3-day 
voiding diary will be verified.  
The hormone panel and SHBG tests will be assessed every 24 weeks only (i.e., every other 
clinic visit).  
A transvaginal ultrasound examination will be performed every 24 weeks (i.e., every other 
clinic visit) in subjects with an intact uterus. Study drug will be dispensed, with 
administration instructions, at each clinic visit. The medication identification number 
assigned to the subject will be noted in the eCRF for study drug accountability. The subject 
will be reminded to bring all unused study drug (in its original packaging) to her next clinic 
visit. 
The subject will be provided with a new 3-day voiding diary and instructed to complete it 
within the week preceding her next study visit.  

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Any subject who prematurely discontinues study or study drug will be asked to return to the 
study site and complete the end-of-study assessments. 
6.1.4 End-of-Study Visit 
Approximately 30 days after the last dose of GTx-024, the subject will return to the study site 
for an end-of-study clinic visit. Unused study drug will be collected. Concomitant 
medications and AEs will be reviewed. Weight and vital signs will be measured. Blood will 
be drawn for a CMP, CBC, lipid panel, hormone panel, and SHBG test. Completion of a 3-
day voiding diary will be verified.  
The subject will complete the PGI-S and PGI-I questionnaires. 
The subject will be instructed on the transition to commercial drug or other therapy by the 
physician. 
6.2 Efficacy Assessments 
Efficacy assessments will include a 3-day voiding diary, PGI-S, and PGI-I.  
6.2.1 Three-Day Voiding Diary 
Subjects will record all urinary voids, specifying each episode of urinary leakage, including 
type (stress or urge). Fluid intake will also be recorded as well as any episode of vaginal 
discharge or spotting.  
In each 3-day voiding diary, subjects are to record voiding data for 3 consecutive days in a 
paper diary. To be considered valid, each 3-day voiding diary must be recorded within 7 days 
on either side of a planned visit.  
Three-day voiding diaries are reliable measures of the mean number of urinary incontinence 
episodes per day, incontinence type, mean number of voids per 24 hours, and mean number 
of voids during sleeping hours.  
6.2.2 Patient Global Impression of Severity 
The PGI-S is a global rating of subject-reported impression of SUI severity using a 4-point 
scale (Appendix 12.2). Validity of the PGI-S has been established for application in SUI. 

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6.2.3 Patient Global Impression of Improvement  
The PGI-I is a global rating of subject-reported impression of SUI improvement using a 
7-point scale (Appendix 12.3). Validity of the PGI-I has been established for application in 
SUI. 
6.3 Safety Assessments 
6.3.1 Adverse Events 
6.3.1.1 Definitions of Adverse Events 
The investigator is responsible for reporting all AEs that are observed or reported during the 
study regardless of their possible relationship to study drug or their clinical significance. 
An AE is defined as any untoward medical occurrence in a subject enrolled into this study 
regardless of its causal relationship to study drug. Subjects will be instructed to contact the 
investigator at any time after randomization if any symptoms develop. 
A treatment-emergent AE is defined as any event not present before exposure to study drug or 
any event already present that worsens in either intensity or frequency after exposure to study 
drug and within the 30 days following discontinuation of drug treatment. 
An SAE is defined as any event that results in one of the following conditions: 
• Death 
• Life-threatening event (defined as a subject at immediate risk of death at the time of the 
event) 
• An event that requires inpatient hospitalization or prolongation of existing hospitalization  
• An event that results in persistent or significant disability/incapacity  
• An event that results in a congenital anomaly/birth defect  
• Any other important medical event that may not result in death, be life threatening, or 
require hospitalization may be considered an SAE when, based upon appropriate medical 
judgment, the event may jeopardize the subject or may require medical or surgical 
intervention to prevent one of the outcomes listed in this definition. Examples of such 
medical events include allergic bronchospasm that requires intensive treatment in an 
emergency room or at home, blood dyscrasias or convulsions that do not result in 
inpatient hospitalization, or the development of drug dependency or drug abuse 

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6.3.1.2 Eliciting and Documenting Adverse Events 
Adverse events will be assessed beginning at enrollment (date of signed informed consent) 
through the end of the study.  
Serious AEs that occur more than 30 days after the last dose of study drug need not be 
reported unless the investigator considers them to be related to study drug. 
At every study visit, subjects will be asked a standard nonleading question to elicit any 
medically related changes in their well-being. They will also be asked if they have been 
hospitalized, had any accidents, used any new medications, or changed concomitant 
medication regimens (both prescription and OTC medications). 
In addition to subject observations, AEs identified from any study data (e.g., laboratory 
values, physical examination findings) or identified from review of other documents (e.g., 
subject diaries) that are relevant to subject safety will be documented on the AE page in the 
eCRF. 
6.3.1.3 Reporting Adverse Events 
All AEs reported or observed during the study will be recorded on the AE page in the eCRF. 
Information to be collected includes drug treatment, dose, event term, time of onset, 
investigator-specified assessment of severity and relationship to study drug, time of 
resolution of the event, seriousness, any required treatment or evaluations, and outcome. 
Adverse events resulting from concurrent illnesses, reactions to concurrent illnesses, 
reactions to concurrent medications, or progression of disease states must also be reported. 
All AEs will be followed to adequate resolution. The Medical Dictionary for Regulatory 
Activities (MedDRA) will be used to code all AEs.  
Any medical condition that is present at the time that the subject is screened should not be 
reported as an AE. However, if a pre-existing condition changes in intensity or frequency at 
any time during the study, it should be recorded as an AE. 
Any AE that meets SAE criteria (Section 6.3.1.1) must be entered into the electronic data 
capture (EDC) system immediately (i.e., within 1 business day) after site personnel first learn 
of the event. Once the qualifying SAE data are entered, pharmacovigilance will be notified by 
an email alert, which will contain high-level safety information. Additional safety 
information will be obtained from the EDC system via applicable eCRF pages. If the EDC 
system is not available, the site should send a completed paper SAE report form to 
pharmacovigilance by fax or email: 

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• Safety fax line: 1-866-966-2970 (US toll-free) 
• Safety email: sae@cmedresearch.com 
When the EDC system is again available, the site must enter all applicable information into 
the EDC system. All supporting source information concerning the SAE (e.g., hospital 
records) should be provided by fax or email.  
If there is a question concerning an SAE, the site needs guidance regarding reporting of an 
SAE, the site is returning a call from a safety specialist, or the site urgently needs to report an 
SAE or make pharmacovigilance aware of an SAE, the Safety Hotline should be used:  
• Safety Hotline: 1-866-966-8429 (US toll-free) 
If a site makes an initial report of an SAE by the safety hotline, the site must subsequently 
enter all applicable information into the EDC system immediately thereafter.  
6.3.1.4 Assessment of Severity 
All AEs will be assessed by the investigator according to National Cancer Institute–Common 
Terminology Criteria for Adverse Events (NCI-CTCAE) version 4.0. For any AE that is not 
specifically covered in NCI-CTCAE version 4.0, the criteria that should be used are as 
follows:  
Grade 
Description 
0 
No AE or within normal limits 
1 
Mild; asymptomatic or mild symptoms; clinical or diagnostic observations 
only; intervention not indicated  
2 
Moderate; minimal, local, or noninvasive intervention indicated; limiting 
age-appropriate instrumental activities of daily living  
3 
Severe or medically significant but not immediately life-threatening; 
hospitalization or prolongation of hospitalization indicated; disabling; limiting 
self-care activities of daily living  
4 
Life-threatening consequences; urgent intervention indicated 
 
Changes in the severity of an AE should be documented to allow an assessment of the 
duration of the event at each level of intensity to be performed. Adverse events characterized 
as intermittent do not require documentation of onset and duration of each episode. 

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6.3.1.5 Assessment of Causality 
The investigator’s assessment of an AE’s relationship to study drug is part of the 
documentation process, but it is not a factor in determining what is or is not reported in the 
study. If there is any doubt as to whether a clinical observation is an AE, the event should be 
reported. 
The relationship or association of the test article in causing or contributing to the AE will be 
characterized using the following classification and criteria: 
Unrelated: 
This relationship suggests that there is no association between the study drug 
and the reported event. 
 
Possible: 
This relationship suggests that treatment with the study drug caused or 
contributed to the AE, i.e., the event follows a reasonable temporal sequence 
from the time of drug administration or follows a known response pattern to 
the study drug but could also have been produced by other factors. 
 
Probable: 
This relationship suggests that a reasonable temporal sequence of the event 
with drug administration exists and, based upon the known pharmacological 
action of the drug, known or previously reported adverse reactions to the drug 
or class of drugs, or judgment based on the investigator’s clinical experience, 
the association of the event with the study drug seems likely. The event 
disappears or decreases on cessation or reduction of the dose of study drug. 
 
Definite: 
This relationship suggests that a definite causal relationship exists between 
drug administration and the AE, and other conditions (concurrent illness, 
progression/expression of disease state, or concurrent medication reaction) do 
not appear to explain the event. The event reappears or worsens if the study 
drug is re-administered.  
6.3.1.6 Follow-Up of Subjects Reporting Adverse Events 
All AEs must be reported in detail on the appropriate page in the eCRF and be monitored to 
satisfactory resolution, until the investigator deems the event to be chronic or not clinically 
significant, or until the subject is considered to be stable. 
6.3.2 Halting Rules 
If, in the opinion of the investigator, the participation in the study is or is becoming 
detrimental to the well-being of a particular subject, this issue should be discussed with the 
medical monitor for this study and the subject’s participation in the study may be 
discontinued. 

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Discontinuation of treatment should be considered if:  
• ALT or AST > 8 × ULN 
• ALT or AST > 5 × ULN for more than 2 weeks 
• ALT or AST > 3 × ULN and total bilirubin > 2 × ULN or international normalized ratio 
(INR) > 1.5 
• ALT or AST > 3 × ULN with the appearance of fatigue, nausea, vomiting, right upper 
quadrant pain or tenderness, fever, rash, and/or eosinophilia (differential count > 5%) 
In the event that ALT or AST is > 3 × ULN, the subject will return for an unscheduled clinic 
visit, and blood will be drawn for a coagulation panel to determine the INR. The INR will be 
determined at a local laboratory.  
All subjects discontinued from the study should be followed until abnormal values return to 
normal, and all discontinuations should be discussed with the medical monitor prior to 
discontinuation.  
6.3.3 Safety Assessments 
The safety assessments will include AEs (Section 6.3.1), vital sign measurements, height and 
weight, concomitant medications, medical history, physical examination findings, 
transvaginal ultrasound, and clinical laboratory analyses (Section 6.6). Refer to Table 12–1 
for a detailed schedule of the timing of study procedures. 
6.3.3.1 Vital Sign Measurements 
Vital sign measurements include oral temperature, seated blood pressure, pulse rate, and 
respiration rate. Vital sign measurements will be collected as indicated in Table 12–1.  
6.3.3.2 Height and Weight 
Height will be measured at baseline only and weight will be measured as indicated in Table 
12–1. 
6.3.3.3 Prior and Concomitant Medications 
At the baseline visit, the investigator or designee must record the use of prescription and OTC 
medication taken within the prior 30 days. Concomitant medications (including prescription 
and OTC medications) will be assessed and recorded throughout the study (Table 12–1).  

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6.3.3.4 Transvaginal Ultrasound 
Only for subjects with an intact uterus, transvaginal ultrasound will be performed at the 
baseline visit, Week 12 and every 24 weeks only (i.e., every other clinic visit) to observe 
changes in the endometrial stripe thickness (mm). Transvaginal ultrasound examinations will 
be evaluated locally.  
6.4 Other Assessments 
6.5 Pregnancy 
This study will enroll only postmenopausal women who are not taking hormone products. In 
the unlikely event of a pregnancy, any pregnancy must be reported to pharmacovigilance 
within 2 weeks of learning of its occurrence, using the EDC system (Section 6.3.1.3). The 
pregnancy must be followed up to determine the outcome (including spontaneous 
miscarriage, elective termination, normal birth, or congenital abnormality) and status of 
mother and child, even if the subject was discontinued from the study. Pregnancy 
complications and elective terminations for medical reasons must be reported as an AE or 
SAE. Spontaneous miscarriages must be reported as an SAE. 
Any SAE occurring in association with a pregnancy, brought to the investigator’s attention 
after the subject has completed the study, and considered by the investigator as possibly 
related to the study treatment must be promptly reported to pharmacovigilance.  
6.6 Laboratory Analyses 
Any abnormal laboratory test results or other safety assessments, including those that worsen 
from baseline, felt to be clinically significant in the medical and scientific judgment of the 
investigator are to be recorded as AEs or SAEs. 
However, any clinically significant safety assessments that are associated with the underlying 
disease, unless judged by the investigator to be more severe than expected for the subject’s 
condition, are not to be reported as AEs or SAEs. 
Laboratory analyses include the following: 
• Complete blood count, including red blood cells, white blood cells, mean corpuscular 
volume, hemoglobin, hematocrit, platelets, and differential (basophils, eosinophils, 
lymphocytes, monocytes, and neutrophils) 

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• Comprehensive metabolic panel (Chem 16), including calcium, carbon dioxide, glucose, 
potassium, sodium, total protein, and kidney and liver function tests (serum ALT, AST, 
γ-glutamyl transpeptidase, and total and direct bilirubin) will be performed (the 
investigator may repeat these checks if clinically indicated) 
• Fasting lipid panel, including total cholesterol, low-density lipoprotein, HDL, and 
triglycerides 
• Hormone panel, including testosterone, free testosterone, and estradiol 
• SHBG 

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7 Statistical and Analytical Plan  
7.1 Primary Safety Endpoint 
The primary objective in this study is to assess the safety and tolerability of GTx-024 3 mg 
administered once daily in relation to the following: 
• Incidence of AEs 
• Change from baseline in clinical laboratory evaluations 
• Change from baseline in SHBG and testosterone levels 
• Change from baseline in vital sign measurements 
• Change from baseline in endometrial stripe thickness as assessed with transvaginal 
ultrasound (only in subjects with an intact uterus) 
7.2 Secondary Efficacy Endpoints 
The secondary objective in this study is to assess the efficacy of GTx-024 3 mg administered 
once daily at each scheduled visit in relation to the following:  
• Change from baseline in the mean number of stress incontinence episodes per day as 
measured with the 3-day voiding diary  
• Change from baseline in subject-reported impression of SUI severity as measured with 
the PGI-S 
• Improvement relative to baseline in subject-reported impression of SUI improvement as 
measured with the PGI-I 
• Change from baseline in the mean number of urge incontinence episodes per day as 
measured with the 3-day voiding diary  
• Change from baseline in the mean number of total incontinence episodes (stress + urge) 
as measured with the 3-day voiding diary 
These analyses will be described in further detail in the statistical analysis plan (SAP).  
7.3 Sample Size Calculation 
No power calculations were carried out, as no formal statistical analysis will be performed. 
7.4 Analysis Sets 
Data analysis will be performed on a safety analysis set (SAS) and a full analysis set (FAS).  

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The SAS set contains all subjects in the study who receive ≥ 1 dose of study drug. All safety 
analyses will use the SAS.  
The FAS is used for efficacy analysis. The FAS consists of all randomized subjects who 
receive ≥ 1 dose of study drug and have ≥ 1 postbaseline primary efficacy assessment (3-day 
voiding diary). Further details will be provided in the SAP.  
7.5 Description of Subgroups to be Analyzed 
No subgroup analyses are planned. 
7.6 Statistical Analysis Methodology 
A separate SAP will be finalized, providing detailed methods for all endpoints. In general, 
descriptive or summary statistics (n, mean, standard deviation, median, minimum and 
maximum values for continuous variables, and number [%] of subjects in each category for 
categorical variable) will be provided by visit. Source data for summary tables and statistical 
analyses will be presented as subject data listings. 
All statistical analyses will be performed using SAS (version 9.3 or higher). No formal 
statistical analysis will be performed, as the open-label extension study is not powered for 
inferential statistics. Two-sided 95% confidence intervals will be provided where appropriate. 
No imputation will be done for missing data. The SAP will specify up to which visit 
summaries will be produced, as not all subjects will have been in the study for the same 
length of time. Further details on the analysis of efficacy endpoints will be provided in the 
SAP. 
The study period is defined as baseline (Day 0) through end of study. Change from baseline 
will be between baseline (Day 0) and scheduled visits.  
7.6.1 Analysis of Safety Endpoints 
All safety parameters (including AEs, vital signs, laboratory evaluations, SHBG, testosterone, 
and endometrial stripe thickness) will be summarized using the SAS set. No formal 
hypothesis testing will be performed.  
All AEs will be coded using the latest version of MedDRA. All treatment-emergent AEs will 
be summarized and presented in the listings by the number of subjects who report an event, 
the percentage of subjects with that event, the number of events, and the grade, duration, and 
relationship to treatment. Percentages will be based on the number of subjects who received 
each treatment during the study. 

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Clinical laboratory safety tests will be performed, and normal ranges will be provided for 
each laboratory test. Values outside the normal range will be assessed for clinical significance 
by the investigator. Shift tables (change from baseline value to on-treatment values) will be 
presented for each laboratory measurement and assessment time. Each parameter outside the 
normal range will be designated as high (H) or low (L) in the individual data listings.  
7.6.2 Analysis of Secondary Efficacy Endpoints 
For continuous primary endpoints, treatment effects will be summarized descriptively 
according to visit.  
For ordinal primary endpoints, treatment effects will be summarized descriptively according 
to visit.  
Further details on the analysis of efficacy endpoints will be provided in the SAP. Further 
details on the analysis of secondary efficacy endpoints will be provided in the SAP. 
7.7 Data Quality Assurance 
Standard operating procedures are available for all activities relevant to the quality of this 
study. Designated personnel will be responsible for implementing and maintaining quality 
assurance and quality control systems to ensure that the study is conducted and that data are 
generated, documented, and reported in compliance with the study protocol, Good Clinical 
Practice (GCP), and Good Laboratory Practice requirements as well as applicable regulatory 
requirements and local laws, rules, and regulations relating to the conduct of the clinical trial. 
An authorized quality assurance auditor will audit the study data and procedures at periodic 
intervals as indicated. Domestic or foreign regulatory authorities, the IRB, and a 
Sponsor-authorized auditor may request access to all study documentation for an on-site 
inspection or audit. The investigator must notify GTx, Inc. of any regulatory authority 
inspections and forward copies of the inspection report to GTx, Inc. 
Electronic data systems will be in accordance with applicable aspects of 21 Code of Federal 
Regulations (CFR) Part 11, International Council for Harmonisation (ICH) Guidelines, GCP, 
local laws and legislation, and the Health Insurance Portability and Accountability Act. 
7.7.1 On-Site Audits 
At any time, quality assurance representatives of the Sponsor and/or regulatory bodies may 
visit the unit to carry out an audit of the study in compliance with regulatory guidelines and 
company policy. Such audits will require access to study records, documentation, and 

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regulatory files. At all times, subject privacy will be of utmost importance and respected. 
Typically, sufficient notice will be given to the investigator to prepare for the visit. 
7.7.2 Data Management 
As part of the responsibilities assumed by participating in the study, the investigator agrees to 
maintain adequate case histories for the subjects treated as part of the research under this 
protocol. The investigator agrees to maintain accurate eCRFs and source documentation as 
part of the case histories. Source documents may include, but are not restricted to, such 
documents as laboratory reports and 3-day voiding diaries.  
Investigative site personnel will enter subject data into InForm® (the eCRF program). The 
analysis data sets will be a combination of these data and data from other sources (e.g., 
laboratory data). 
Clinical data management will be performed in accordance with applicable GTx standards 
and data cleaning procedures to ensure the integrity of the data, e.g., removing errors and 
inconsistencies in the data. Adverse events and concomitant medication terms will be coded 
using the MedDRA, an internal validated medication dictionary. 
After database lock, each study site will receive a CD-ROM containing all of their 
site-specific eCRF data as entered into Oracle Clinical Remote Data Capture for the study, 
including full discrepancy and audit history. Additionally, a CD-ROM copy of all of the study 
site’s data from the study will be created and sent to the Sponsor for storage. PPD will 
maintain a duplicate CD-ROM copy for their records. In all cases, subject initials will not be 
collected or transmitted to the Sponsor. 

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8 Ethics 
8.1 Institutional Review Board 
Federal regulations and the ICH guidelines require that approval be obtained from an IRB 
before participation of human subjects in research studies. Before study onset, the protocol, 
informed consent, advertisements to be used for the recruitment of study subjects, and any 
other written information regarding this study to be provided to the subject must be approved 
by the IRB. Documentation of all IRB approvals and of the IRB compliance with ICH 
harmonised tripartite guideline E6(R2): GCP will be maintained by the site and will be 
available for review by the Sponsor or its designee. 
All IRB approvals should be signed by the IRB chairman or designee and must identify the 
IRB name and address, the clinical protocol by title or protocol number or both, and the date 
approval or a favorable opinion was granted. 
The investigator is responsible for providing written summaries of the progress and status of 
the study at intervals not exceeding 1 year or otherwise specified by the IRB. The investigator 
must promptly supply the Sponsor or its designee, the IRB, and, where applicable, the 
institution, with written reports on any changes significantly affecting the conduct of the 
study or increasing the risk to subjects. 
8.2 Ethical Conduct of the Study 
The study will be performed in accordance with the ethical principles that have their origin in 
the Declaration of Helsinki, ICH GCP, and all applicable regulations. 
8.3 Subject Information and Consent 
A written informed consent in compliance with US Title 21 CFR Part 50 shall be obtained 
from each subject before entering the study or performing any unusual or nonroutine 
procedure that involves risk to the subject. An informed consent template may be provided by 
the Sponsor to investigative sites. If any institution-specific modifications to study-related 
procedures are proposed or made by the site, the consent should be reviewed by the Sponsor 
or its designee or both before IRB submission. Once reviewed, the consent will be submitted 
by the investigator to his or her IRB for review and approval before the start of the study. If 
the ICF is revised during the course of the study, all active participating subjects must sign 
the revised form. 
Before recruitment and enrollment, each prospective subject will be given a full explanation 
of the study and be allowed to read the approved ICF. Once the investigator is assured that 

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the subject understands the implications of participating in the study, the subject will be asked 
to give consent to participate in the study by signing the ICF. 
The investigator shall retain the signed original ICF(s) and give a copy of the signed original 
form to the subject.  

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9 Investigator’s Obligations 
The following administrative items are meant to guide the investigator in the conduct of the 
study but may be subject to change based on industry and government standard operating 
procedures, working practice documents, or guidelines. Changes will be reported to the IRB 
but will not result in protocol amendments. 
9.1 Confidentiality 
All laboratory specimens, evaluation forms, reports, and other records will be identified in a 
manner designed to maintain subject confidentiality. All records will be kept in a secure 
storage area with limited access. Clinical information will not be released without the written 
permission of the subject, except as necessary for monitoring and auditing by the Sponsor, its 
designee, the US Food and Drug Administration (FDA), or the IRB. 
The investigator and all employees and coworkers involved with this study may not disclose 
or use for any purpose other than performance of the study any data, record, or other 
unpublished, confidential information disclosed to those individuals for the purpose of the 
study. Prior written agreement from the Sponsor or its designee must be obtained for the 
disclosure of any said confidential information to other parties. 
9.2 Financial Disclosure and Obligations 
Investigators are required to provide financial disclosure information to allow the Sponsor to 
submit the complete and accurate certification or disclosure statements required under 
21 CFR 54. In addition, the investigator must provide to the Sponsor a commitment to 
promptly update this information if any relevant changes occur during the course of the 
investigation and for 1 year following the completion of the study. 
Neither the Sponsor nor PPD is financially responsible for further testing or treatment of any 
medical condition that may be detected during the screening process. In addition, in the 
absence of specific arrangements, neither the Sponsor nor PPD is financially responsible for 
further treatment of the subject’s disease. 
9.3 Investigator Documentation 
Prior to beginning the study, the investigator will be asked to comply with ICH E6(R2) 8.2 
and Title 21 of the CFR by providing the following essential documents, including but not 
limited to: 
• IRB approval 

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• Original investigator-signed investigator agreement page of the protocol 
• Form FDA 1572, fully executed, and all updates on a new fully executed Form FDA 1572 
• Curriculum vitae for the investigator and each subinvestigator listed on Form FDA 1572 
• Financial disclosure information to allow the Sponsor to submit complete and accurate 
certification or disclosure statements required under 21 CFR 54. In addition, the 
investigators must provide to the Sponsor a commitment to promptly update this 
information if any relevant changes occur during the course of the investigation and for 
1 year after the completion of the study 
• IRB-approved informed consent, samples of site advertisements for recruitment for this 
study, and any other written information regarding this study that is to be provided to the 
subject 
• Laboratory certifications and normal ranges for any local laboratories used by the site, in 
accordance with 42 CFR 493 
9.4 Study Conduct 
The investigator agrees that the study will be conducted according to the principles of ICH 
E6(R2). The investigator will conduct all aspects of this study in accordance with all national, 
state, and local laws or regulations. Study information from this protocol will be posted on 
publicly available clinical trial registers before the enrollment of subjects begins. 
9.5 Adherence to Protocol 
The investigator agrees to conduct the study as outlined in this protocol in accordance with 
ICH E6(R2) and all applicable guidelines and regulations. 
9.6 Adverse Events and Study Report Requirements 
By participating in this study, the investigator agrees to submit reports of SAEs according to 
the timeline and method outlined in the protocol. In addition, the investigator agrees to 
submit annual reports to the study-site IRB as appropriate. 
9.7 Investigator’s Final Report 
Upon completion of the study, the investigator, where applicable, should inform the 
institution; the investigator/institution should provide the IRB with a summary of the study’s 
outcome and the Sponsor and regulatory authority(ies) with any reports required. 

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9.8 Records Retention 
Essential documents should be retained until at least 2 years after the last approval of a 
marketing application in an ICH region and until there are no pending or contemplated 
marketing applications in an ICH region or at least 2 years have elapsed since the formal 
discontinuation of clinical development of the investigational product. It is the responsibility 
of the Sponsor to inform the investigator/institution as to when these events stated in 21 CFR 
312.62(c) have occurred. These documents should be retained for a longer period, however, if 
required by the applicable regulatory requirements or by an agreement with the Sponsor. 
9.9 Publications 
After completion of the study, the data may be considered for reporting at a scientific meeting 
or for publication in a scientific journal. In these cases, the Sponsor will be responsible for 
these activities and will work with the investigators to determine how the manuscript is 
written and edited, the number and order of authors, the publication to which it will be 
submitted, and other related issues. The Sponsor has final approval authority over all such 
issues. 
Data are the property of the Sponsor and cannot be published without prior authorization 
from the Sponsor, but data and publication thereof will not be unduly withheld. 

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10 Study Management 
10.1   Monitoring 
10.1.1   Monitoring the Study 
The clinical monitor, as a representative of the Sponsor, has the obligation to follow the study 
closely. In doing so, the monitor will visit the investigator and study site at periodic intervals, 
in addition to maintaining necessary telephone and letter contact. The monitor will maintain 
current personal knowledge of the study through observation, review of study records and 
source documentation, and discussion of the conduct of the study with the investigator and 
personnel. 
All aspects of the study will be carefully monitored, by the Sponsor or its designee, for 
compliance with applicable government regulation with respect to current GCP and current 
standard operating procedures. 
10.1.2   Inspection of Records 
Investigators and institutions involved in the study will permit study-related monitoring, 
audits, IRB review, and regulatory inspections by providing direct access to all study records. 
In the event of an audit, the investigator agrees to allow the Sponsor, representatives of the 
Sponsor, FDA, or other regulatory agency access to all study records. 
The investigator should promptly notify the Sponsor and PPD of any audits scheduled by any 
regulatory authorities and promptly forward copies of any audit reports received to the 
Sponsor. 
10.2   Management of Protocol Amendments and Deviations 
10.2.1   Modification of the Protocol 
Any changes in this research activity, except those necessary to remove an apparent, 
immediate hazard to the subject, must be reviewed and approved by the Sponsor or its 
designee. Amendments to the protocol must be submitted in writing to the investigator’s IRB 
for approval before subjects can be enrolled into an amended protocol. 
10.2.2   Protocol Deviations 
The investigator or designee must document and explain in the subject’s source 
documentation any deviation from the approved protocol. The investigator may implement a 
deviation from, or a change of, the protocol to eliminate an immediate hazard to study 

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subjects without prior IRB approval. As soon as possible after such an occurrence, the 
implemented deviation or change, the reasons for it, and any proposed protocol amendments 
should be submitted to the IRB for review and approval, to the Sponsor for agreement, and to 
the regulatory authorities, if required. 
A deviation from the protocol is an unintended or unanticipated departure from the 
procedures or processes approved by the Sponsor and the IRB and agreed to by the 
investigator. A significant deviation occurs when there is nonadherence to the protocol by the 
subject or investigator that results in a significant, additional risk to the subject. Significant 
deviations can include nonadherence to inclusion or exclusion criteria, enrollment of the 
subject without prior Sponsor approval, or nonadherence to FDA regulations or ICH GCP 
guidelines, and will lead to the subject being withdrawn from the study (Section 4.2). 
Protocol deviations will be documented by the clinical monitor throughout the course of 
monitoring visits. Principal investigators will be notified in writing by the monitor of 
deviations. The IRB should be notified of all protocol deviations in a timely manner. 
10.3   Study Termination 
Although GTx has every intention of completing the study, GTx reserves the right to 
discontinue the study at any time for clinical or administrative reasons. 
The end of the study is defined as the date on which the last subject completes the last visit 
(includes follow-up visit). 
10.4   Final Report 
Whether the study is completed or prematurely terminated, the Sponsor will ensure that the 
clinical study reports are prepared and provided to the regulatory agency(ies) as required by 
the applicable regulatory requirement(s). The Sponsor will also ensure that the clinical study 
reports in marketing applications meet the standards of the ICH harmonised tripartite 
guideline E3: Structure and content of clinical study reports. 
Where required by applicable regulatory requirements, an investigator signatory will be 
identified for the approval of the clinical study report. The investigator will be provided 
reasonable access to statistical tables, figures, and relevant reports and will have the 
opportunity to review the complete study results. 
Upon completion of the clinical study report, the Sponsor will provide the investigator with 
the full summary of the study results. The investigator is encouraged to share the summary 

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21 May 2018 
 
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results with the study subjects, as appropriate. The study results will be posted on publicly 
available clinical study registers. 

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11  Reference List 
GTx, Inc. GTx-024. Investigator’s brochure, 16th ed. Memphis (TN); 2017. 103 p. 
Herzog AR, Fultz NH. Prevalence and incidence of urinary incontinence in 
community-dwelling populations. J Am Geriatr Soc. 1990;38(3):273-81. 
Kadekawa K, Kawamorita N, Turnbull P, et al. Therapeutic effects of a selective androgen 
receptor modulator, GSK2849466A on stress urinary incontinence in ovariectomized rats 
[abstract PD27-11]. J Urol. 2015;193 Suppl 4S;e575-6. 
Luber KM. The definition, prevalence, and risk factors for stress urinary incontinence. 
Rev Urol. 2004;6 Suppl 3:S3-9. 
Mohler ML, Bohl CE, Jones A, et al. Nonsteroidal selective androgen receptor modulators 
(SARMs): dissociating the anabolic and androgenic activities of the androgen receptor for 
therapeutic benefit. J Med Chem. 2009;52(12):3597-617. 

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12  Appendices 
12.1   Appendix: Schedule of Events  
Table 12–1 
Schedule of Events for Protocol G201004 
Procedure 
Baseline  
Day 0 
V1 
V2 
V3 
Long-term 
Safety 
End of Study 
Day or Week 
Week 0 
Week 4 
Week 8 
Week 12 
Every 
12 weeks 
following 
V3 
30 days after 
last dose of 
GTx-024 
Visit Window (days) 
– 
±7 
±7 
±7 
±7 
±7 
Informed consent 
X 
 
 
 
 
 
Eligibility criteria review 
X 
 
 
 
 
 
Medical history 
X 
 
 
 
 
 
Concomitant medications 
X 
X 
X 
X 
X 
X 
Heighta and weight 
X 
X 
X 
X 
X 
X 
Vital signs 
X 
X 
X 
X 
X 
X 
3-Day voiding diary 
X 
X 
X 
X 
X 
X 
PGI-S 
X 
X 
X 
X 
X 
X 
PGI-I 
 
X 
X 
X 
X 
X 
Adverse event reviews 
X 
X 
X 
X 
X 
X 
CMP (Chem 16 panel) 
X 
X 
X 
X 
X 
X 
CBC 
X 
X 
X 
X 
X 
X 
Lipid panel 
X 
X 
X 
X 
X 
X 
Hormone panelb 
X 
X 
X 
X 
X 
X 
SHBGb 
X 
X 
X 
X 
X 
X 
Transvaginal ultrasoundc 
X 
 
 
X 
X 
 
Dispense study drug 
X 
X 
X 
X 
X 
 
Collect unused study drug 
 
X 
X 
X 
X 
X 
Abbreviations: CBC, complete blood count; CMP, comprehensive metabolic panel; PGI-I, Patient Global 
Impression of Improvement scale; PGI-S, Patient Global Impression of Severity scale; SHBG, sex 
hormone–binding globulin; V, visit. 
a  
Height is measured at baseline only. 
b  
During the long-term safety period, the hormone panel and SHBG will be assessed every 24 weeks only 
(i.e., every other clinic visit). 
c  
During the long-term safety period, the transvaginal ultrasound will be assessed every 24 weeks only (i.e., 
every other clinic visit) 

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12.2   Appendix: Patient Global Impression of Severity Scale 
 
 
 
 

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12.3   Appendix: Patient Global Impression of Improvement Scale