GTx-024 as a Treatment for Stress Urinary Incontinence in Women

• Sponsor: GTx (Industry)
• Overall Status: Completed
• CT.gov ID: NCT02658448
• Collaborator: (none)
• Enrollment: 19
• Locations: 3
• Arm: 1
• Actual Duration (Months): 29
• Patients Per Site: 6.3
• Patients Per Site Per Month: 0.2

Study Details

Study Description

Brief Summary

The purpose of this study is to determine whether GTx-024 is safe and effective in the treatment of stress urinary incontinence in postmenopausal women.

Condition or Disease	Intervention/Treatment	Phase
Stress Urinary Incontinence	Drug: GTx-024	Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 19 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: GTx-024 as a Treatment for Stress Urinary Incontinence in Women: A Proof of Concept Study
• Actual Study Start Date: Jan 1, 2016
• Actual Primary Completion Date: Jun 1, 2018
• Actual Study Completion Date: Jun 1, 2018

Arms and Interventions

Arm	Intervention/Treatment
Experimental: GTx-024 3 mg GTx-024 softgel capsules will be administered once daily to a total dose of 3 mg for up to 12 weeks.	Drug: GTx-024; GTx-024 softgel capsules will be administered once-daily to a total dose of 3 mg; Other Names: enobosarm

Outcome Measures

Primary Outcome Measures

1. The Mean Percent Change in Number of Stress Incontinence Episodes/Day as Assessed by Patient Completion of the 3 Day Voiding Diary [12 weeks]

   To describe the effect of 12 weeks of treatment of GTx-024 on the number of stress incontinence episodes/day as assessed by patient completion of the 3 day voiding diary

Secondary Outcome Measures

1. Stress Urinary Incontinence as Assessed by 24 Hour Pad Weight Test. Mean Percent Change in Pad Weight [12 weeks]

   To describe the effect of 12 weeks of treatment of GTx-024 on SUI as assessed by 24 hour pad weight test. Mean percent reduction in pad weight

Other Outcome Measures

1. Number of Participants With Adverse Events [12 weeks]

   To describe the safety profile of GTx-024 3 mg PO daily in subjects with stress urinary incontinence.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 80 Years
• Sexes Eligible for Study: Female
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Give voluntary, written and signed, informed consent

• Female

• Age18 to 80 years old be clinically confirmed as postmenopausal. Subjects must have undergone the onset of spontaneous, medically induced or surgical menopause prior to the start of this study. Spontaneous menopause is defined as 12 months of spontaneous amenorrhea or 6 months of spontaneous amenorrhea with serum FSH levels > 40 mIU/ml or 6 weeks postsurgical bilateral oophorectomy with or without hysterectomy

• SUI symptoms for at least 6 months duration

• Predominant SUI (MESA questionnaire)

• 24 hour pad weight >3 gms at baseline

• A minimum of 1 SUI episode per day, in the 3 day diary

• 3-15 SUI episodes per day, averaged over 3 days, in the 3 day diary

• Serum AST and ALT within normal limits

• Total bilirubin within normal limits

• Positive Bladder Stress Test during screening

• Subject agrees to not start any new treatment (medication or otherwise) that is known to affect lower urinary tract function throughout the treatment and follow up periods

• Subject agrees to maintain on a stable dose of any medication known to affect lower urinary tract function, including but not limited to anticholinergics, tricyclic antidepressants, or alpha-adrenergic blockers, throughout the treatment and follow-up period

Exclusion Criteria:

• Pelvic floor physical therapy in a clinical setting within 30 days prior to screening

• History of pelvic radiation treatment

• History of urethral diverticula

• History of urethral sling, anterior prolapse repair, ureteral bulking agents and/or other SUI procedure or surgery

• Known vesicoureteral reflux, vaginal prolapse beyond the introitus, or other significant urological findings, including pelvic floor abnormalities which, in the judgement of the investigator, could impact treatment

• Urinary incontinence of neurogenic etilogy

• Patient is morbidly obese (defined as 100 pounds over their ideal body weight, or body mass index 40 or greater)

• Chronic hepatitis

• Hepatic cirrhosis

• HIV and/or hepatitis A, B, or C

• Subjects taking systemic hormone products

• Subjects with a history of breast or endometrial cancer

• Myocardial infarction or arterial thromboembolic events within 6 months prior to Baseline, severe or unstable angina, New York Heart Association (NYHA) Class III or IV disease, serious uncontrolled cardiac arrhythmia grade II or higher according to NYHA, uncontrolled hypertension (systolic > 150 and/or diastolic > 100 mm Hg)

• Subjects with an entry measurement of > 5 mm endometrial stripe thickness

• Clinically confirmed urinary tract infection

• Any other condition which per investigators' judgment may increase subject risk

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	William Beaumont Hospital	Royal Oak	Michigan	United States	48073
2	Urologic Consultants of Southeastern Pennsylvania	Bala-Cynwyd	Pennsylvania	United States	19004
3	Penn Center for Continence and Pelvic Health, University of Pennsylvania	Philadelphia	Pennsylvania	United States	19104

Sponsors and Collaborators

• GTx

Investigators

• Principal Investigator: Kenneth M. Peters, MD, William Beaumont Hospitals

Study Documents (Full-Text)

• Study Protocol and Statistical Analysis Plan - Aug 7, 2015

More Information

Publications

None provided.

• Responsible Party: GTx
• ClinicalTrials.gov Identifier: NCT02658448
• Other Study ID Numbers: G201001
• First Posted: Jan 18, 2016
• Last Update Posted: Feb 1, 2021
• Last Verified: Jan 1, 2021

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Additional relevant MeSH terms: Urinary Incontinence; Enuresis; Urinary Incontinence, Stress

Study Results

Participant Flow

Arm/Group Title	GTx-024 3 mg
Arm/Group Description	GTx-024 softgel capsules will be administered once daily to a total dose of 3 mg for up to 12 weeks. GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 3 mg
Period Title: Overall Study
STARTED	19
COMPLETED	18
NOT COMPLETED	1

Baseline Characteristics

Arm/Group Title	GTx-024 3 mg
Arm/Group Description	GTx-024 softgel capsules will be administered once daily to a total dose of 3 mg for up to 12 weeks. GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 3 mg
Overall Participants	19
Age (years) [Mean (Full Range) ]
Mean (Full Range) [years]	61.4
Sex: Female, Male (Count of Participants)
Female	19 100%
Male	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%
Asian	0 0%
Native Hawaiian or Other Pacific Islander	0 0%
Black or African American	3 15.8%
White	16 84.2%
More than one race	0 0%
Unknown or Not Reported	0 0%
Region of Enrollment (Count of Participants)
United States	19 100%
Baseline mean number of stress leaks per day (number of leaks per day) [Mean (Full Range) ]
Mean (Full Range) [number of leaks per day]	5.17

Outcome Measures

1. Primary Outcome

Title	The Mean Percent Change in Number of Stress Incontinence Episodes/Day as Assessed by Patient Completion of the 3 Day Voiding Diary
Description	To describe the effect of 12 weeks of treatment of GTx-024 on the number of stress incontinence episodes/day as assessed by patient completion of the 3 day voiding diary
Time Frame	12 weeks

Outcome Measure Data

Analysis Population Description

all patients with at least one post enrollment assessment

Arm/Group Title	GTx-024 3 mg
Arm/Group Description	GTx-024 softgel capsules will be administered once daily to a total dose of 3 mg for up to 12 weeks. GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 3 mg
Measure Participants	18
Mean (Full Range) [percent reduction]	81

2. Secondary Outcome

Title	Stress Urinary Incontinence as Assessed by 24 Hour Pad Weight Test. Mean Percent Change in Pad Weight
Description	To describe the effect of 12 weeks of treatment of GTx-024 on SUI as assessed by 24 hour pad weight test. Mean percent reduction in pad weight
Time Frame	12 weeks

Outcome Measure Data

Analysis Population Description

all patients with at least one post enrollment assessment

Arm/Group Title	GTx-024 3 mg
Arm/Group Description	GTx-024 softgel capsules will be administered once daily to a total dose of 3 mg for up to 12 weeks. GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 3 mg
Measure Participants	18
Mean (Full Range) [percent]	71

3. Other Pre-specified Outcome

Title	Number of Participants With Adverse Events
Description	To describe the safety profile of GTx-024 3 mg PO daily in subjects with stress urinary incontinence.
Time Frame	12 weeks

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	GTx-024 3 mg
Arm/Group Description	GTx-024 softgel capsules will be administered once daily to a total dose of 3 mg for up to 12 weeks. GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 3 mg
Measure Participants	19
Count of Participants [Participants]	9 47.4%

Adverse Events

Time Frame	12 weeks
Arm/Group Title	GTx-024 3 mg
Arm/Group Description	GTx-024 softgel capsules will be administered once daily to a total dose of 3 mg for up to 12 weeks. GTx-024: GTx-024 softgel capsules will be administered once-daily to a total dose of 3 mg
All Cause Mortality
	GTx-024 3 mg
	Affected / at Risk (%)	# Events
Total	0/19 (0%)
Serious Adverse Events
	GTx-024 3 mg
	Affected / at Risk (%)	# Events
Total	0/19 (0%)
Other (Not Including Serious) Adverse Events
	GTx-024 3 mg
	Affected / at Risk (%)	# Events
Total	9/19 (47.4%)
Cardiac disorders
hypertension	1/19 (5.3%)	1
Endocrine disorders
hot flash	1/19 (5.3%)	1
Gastrointestinal disorders
nausea	1/19 (5.3%)	1
constipation	1/19 (5.3%)	1
General disorders
headache	1/19 (5.3%)	1
fatigue	1/19 (5.3%)	1
insomnia	1/19 (5.3%)	1
Infections and infestations
tooth abscess	1/19 (5.3%)	1
periodontitis	1/19 (5.3%)	1
thrush	1/19 (5.3%)	1
cold	1/19 (5.3%)	1
flu	1/19 (5.3%)	1
Investigations
elevated alanine aminotransferase	1/19 (5.3%)	1
low HDL	1/19 (5.3%)	1
elevated HDL	1/19 (5.3%)	1
low sex hormone binding globulin	1/19 (5.3%)	1
elevated INR	1/19 (5.3%)	1
Musculoskeletal and connective tissue disorders
muscle soreness	1/19 (5.3%)	1
arthritis	1/19 (5.3%)	1
pelvic floor muscle spasm	1/19 (5.3%)	2
abdominal muscle soreness	1/19 (5.3%)	1
osteoarthritis	1/19 (5.3%)	1
Renal and urinary disorders
urinary frequency	1/19 (5.3%)	1
proteinuria	1/19 (5.3%)	1
ketonuria	1/19 (5.3%)	1
bilirubinuria	1/19 (5.3%)	1
urinary tract infection	1/19 (5.3%)	1
cyctocele	1/19 (5.3%)	1
Reproductive system and breast disorders
vaginal itching	1/19 (5.3%)	1
vaginal bleeding	1/19 (5.3%)	1
breast cyst	1/19 (5.3%)	1
Skin and subcutaneous tissue disorders
acne	1/19 (5.3%)	1

Limitations/Caveats

Open label, small number of subjects

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.