Safety and Efficacy Study to Evaluate Proellex in the Treatment of Premenopausal Women With Symptomatic Endometriosis

• Sponsor: Repros Therapeutics Inc. (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT00556075
• Collaborator: (none)
• Enrollment: 67
• Locations: 17
• Arms: 3
• Duration (Months): 20
• Patients Per Site: 3.9
• Patients Per Site Per Month: 0.2

Study Details

Study Description

Brief Summary

The purpose of this study is to determine whether Proellex is safe and effective for the treatment of symptomatic endometriosis.

Condition or Disease	Intervention/Treatment	Phase
Endometriosis	Drug: Proellex 25 mg; Drug: Placebo; Drug: Proellex 50 mg	Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 67 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Triple (Participant, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A Phase II, Three-Arm, Parallel Design, Dose-Ranging Placebo-Controlled, Randomized, Double-Blind, Multicenter Study Evaluating the Safety and Efficacy of Proellex in the Treatment of Premenopausal Women With Symptomatic Endometriosis
• Study Start Date: Nov 1, 2007
• Actual Primary Completion Date: May 1, 2009
• Actual Study Completion Date: Jul 1, 2009

Arms and Interventions

Arm	Intervention/Treatment
Placebo Comparator: Placebo Placebo once daily	Drug: Placebo; 1 capsule daily for 4 months
Experimental: 25 mg Proellex 25 mg once daily	Drug: Proellex 25 mg; 1 capsule daily for 4 months
Experimental: 50 mg Proellex 50 mg once daily	Drug: Proellex 50 mg; 2 capsules daily for 4 months

Outcome Measures

Primary Outcome Measures

1. Difference Between the 50 mg Proellex Group and Placebo Group in the Month 4 Subject Diary Composite Pain Score [4 months]

2. Difference Between the 25 mg and 50 mg Proellex Groups and Placebo Group in the Month 4 Subject Diary Composite Pain Score [4 months]

Secondary Outcome Measures

1. Difference Between Each Treatment Group in the Subject Diary Composite Pain Score at the Monthly Visits [monthly]

2. Time to Pain-free Assessments Using the Composite Pain Score as Determined by Data Recorded in the Subject Diaries [days]

3. Duration of Pain-free Assessments Using the Composite Pain Score as Determined by Data Recorded in the Subject Diaries [days]

4. The Number of Days With Pain as Determined by Data Recorded in the Subject Diaries, Analyzed Between Treatment Groups at the Monthly Visits [days]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 48 Years
• Sexes Eligible for Study: Female
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Premenopausal women aged 18-48 inclusive

• Endometriosis documented by laparoscopic or surgical assessment within the past ten (10) years

• Clinical symptoms of endometriosis for at least the past three (3) months

• Endometriosis symptoms

• Must be sexually active unless sexually inactive for endometriosis-related dyspareunia

• Must have a regular or steady menstrual cycle lasting from 24 to 36 days and a detectable ovulation during the baseline period using an ovulation monitoring kit (for timing of endometrial biopsy)

• Other inclusion criteria may apply

Exclusion Criteria:

• Six (6) months or more without a menstrual period, or

• Prior hysterectomy or

• Prior bilateral oophorectomy

• Diagnosis of osteopenia

• Present history or condition that causes non-endometriosis-related dyspareunia

• Presence of excessive bleeding or menorrhagia

• Abnormal screening endometrial biopsy

• Other exclusion criteria may apply

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Advanced Clinical Therapeutics, LLC	Tucson	Arizona	United States	85712
2	Lynn Institute of the Ozarks	Little Rock	Arkansas	United States	72205
3	Medical Center for Clinical Research	San Diego	California	United States	92108
4	Compass Clinical Research	San Ramon	California	United States	94583
5	Altus Research	Lake Worth	Florida	United States	33461
6	Comprehensive Clinical Trials	West Palm Beach	Florida	United States	33409
7	Medical Associates	Dubuque	Iowa	United States	52002
8	Physicians for Women	Cary	North Carolina	United States	27511
9	Wake Research Associates	Raleigh	North Carolina	United States	27612
10	Rapid Medical Research, Inc.	Cleveland	Ohio	United States	44122
11	HWC Women's Research Center	Miamisburg	Ohio	United States	45342
12	SC Clinical Research Center, LLC	Columbia	South Carolina	United States	29201
13	Gaffney Pharmaceutical Research	Gaffney	South Carolina	United States	29340
14	Greenville Pharma Research	Greenville	South Carolina	United States	29615
15	Advanced Research Associates	Corpus Christi	Texas	United States	78414
16	Clinical Trials of Texas/Institute for Women's Health	San Antonio	Texas	United States	78229
17	Clinical Trials of Texas/Seven Oaks Women's Center	San Antonio	Texas	United States	78229

Sponsors and Collaborators

• Repros Therapeutics Inc.

Investigators

• Study Director: Andre vanAs, MD, PhD, Repros Therapeutics Inc.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Repros Therapeutics Inc.
• ClinicalTrials.gov Identifier: NCT00556075
• Other Study ID Numbers: ZPE-201
• First Posted: Nov 9, 2007
• Last Update Posted: Aug 25, 2014
• Last Verified: Aug 1, 2014

• Keywords provided by Repros Therapeutics Inc.: Endometriosis; Pelvic pain; Oral progesterone blocker
• Additional relevant MeSH terms: Endometriosis

Study Results

Participant Flow

Arm/Group Title	C Placebo	A 25 mg	B 50 mg
Arm/Group Description	Placebo: 1 capsule daily for 4 months	Proellex 25 mg: 1 capsule daily for 4 months	Proellex 50 mg: 2 capsules daily for 4 months
Period Title: Overall Study
STARTED	22	22	23
COMPLETED	9	13	15
NOT COMPLETED	13	9	8

Baseline Characteristics

Arm/Group Title	Placebo	25 mg	50 mg	Total
Arm/Group Description	Placebo: 1 capsule daily for 4 months	Proellex 25 mg: 1 capsule daily for 4 months	Proellex 50 mg: 2 capsules daily for 4 months	Total of all reporting groups
Overall Participants	22	22	23	67
Age (Count of Participants)
<=18 years	0 0%	0 0%	0 0%	0 0%
Between 18 and 65 years	22 100%	22 100%	23 100%	67 100%
>=65 years	0 0%	0 0%	0 0%	0 0%
Sex: Female, Male (Count of Participants)
Female	22 100%	22 100%	23 100%	67 100%
Male	0 0%	0 0%	0 0%	0 0%
Region of Enrollment (participants) [Number]
United States	22 100%	22 100%	23 100%	67 100%

Outcome Measures

1. Primary Outcome

Title	Difference Between the 50 mg Proellex Group and Placebo Group in the Month 4 Subject Diary Composite Pain Score
Time Frame	4 months

Outcome Measure Data

Analysis Population Description

Zero participants were analyzed because no data were collected due to early termination

Arm/Group Title	Placebo	25 mg	50 mg
Arm/Group Description	Placebo: 1 capsule daily for 4 months	Proellex 25 mg: 1 capsule daily for 4 months	Proellex 50 mg: 2 capsules daily for 4 months
Measure Participants	0	0	0

2. Primary Outcome

Title	Difference Between the 25 mg and 50 mg Proellex Groups and Placebo Group in the Month 4 Subject Diary Composite Pain Score
Time Frame	4 months

Outcome Measure Data

Analysis Population Description

Zero participants were analyzed because no data were collected due to early termination

Arm/Group Title	Placebo	25 mg	50 mg
Arm/Group Description	Placebo: 1 capsule daily for 4 months	Proellex 25 mg: 1 capsule daily for 4 months	Proellex 50 mg: 2 capsules daily for 4 months
Measure Participants	0	0	0

3. Secondary Outcome

Title	Difference Between Each Treatment Group in the Subject Diary Composite Pain Score at the Monthly Visits
Time Frame	monthly

Outcome Measure Data

Analysis Population Description

Zero participants were analyzed because no data were collected due to early termination

Arm/Group Title	Placebo	25 mg	50 mg
Arm/Group Description	Placebo: 1capsule daily for 4 months	Proellex 25 mg Proellex 25 mg: 1 capsule daily for 4 months	Proellex 50 mg Proellex 50 mg: 2 capsules daily for 4 months
Measure Participants	0	0	0

4. Secondary Outcome

Title	Time to Pain-free Assessments Using the Composite Pain Score as Determined by Data Recorded in the Subject Diaries
Time Frame	days

Outcome Measure Data

Analysis Population Description

Zero participants were analyzed because no data were collected due to early termination

Arm/Group Title	Placebo	25 mg	50 mg
Arm/Group Description	Placebo Placebo: 1 capsule daily for 4 months	Proellex 25 mg Proellex 25 mg: 1 capsule daily for 4 months	Proellex 50 mg Proellex 50 mg: 2 capsules daily for 4 months
Measure Participants	0	0	0

5. Secondary Outcome

Title	Duration of Pain-free Assessments Using the Composite Pain Score as Determined by Data Recorded in the Subject Diaries
Time Frame	days

Outcome Measure Data

Analysis Population Description

Zero participants were analyzed because no data were collected due to early termination

Arm/Group Title	Placebo	25 mg	50 mg
Arm/Group Description	Placebo: 1 capsule daily for 4 months	Proellex 25 mg: 1 capsule daily for 4 months	Proellex 50 mg: 2 capsules daily for 4 months
Measure Participants	0	0	0

6. Secondary Outcome

Title	The Number of Days With Pain as Determined by Data Recorded in the Subject Diaries, Analyzed Between Treatment Groups at the Monthly Visits
Time Frame	days

Outcome Measure Data

Analysis Population Description

Zero participants were analyzed because no data were collected due to early termination

Arm/Group Title	Placebo	25 mg	50 mg
Arm/Group Description	Placebo: 1 capsule daily for 4 months	Proellex 25 mg: 1 capsule daily for 4 months	Proellex 50 mg: 2 capsules daily for 4 months
Measure Participants	0	0	0

Adverse Events

Arm/Group Title	Placebo		25 mg		50 mg
Arm/Group Description	Placebo: 1 capsule daily for 4 months		Proellex 25 mg: 1 capsule daily for 4 months		Proellex 50 mg: 2 capsules daily for 4 months
All Cause Mortality
	Placebo		25 mg		50 mg
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)		/ (NaN)
Serious Adverse Events
	Placebo		25 mg		50 mg
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/22 (4.5%)		0/22 (0%)		1/23 (4.3%)
Investigations
Elevated LFTs	0/22 (0%)	0	0/22 (0%)	0	1/23 (4.3%)	1
Nervous system disorders
Grand Mal Seizure	1/22 (4.5%)	1	0/22 (0%)	0	0/23 (0%)	0
Other (Not Including Serious) Adverse Events
	Placebo		25 mg		50 mg
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	18/22 (81.8%)		22/22 (100%)		22/23 (95.7%)
Gastrointestinal disorders
Adominal pain	5/22 (22.7%)		3/22 (13.6%)		8/23 (34.8%)
Nausea	3/22 (13.6%)		8/22 (36.4%)		11/23 (47.8%)
Vomiting	1/22 (4.5%)		3/22 (13.6%)		1/23 (4.3%)
General disorders
Fatigue	0/22 (0%)		2/22 (9.1%)		1/23 (4.3%)
Irritability	0/22 (0%)		0/22 (0%)		2/23 (8.7%)
Immune system disorders
Seasonal allergy	0/22 (0%)		0/22 (0%)		2/23 (8.7%)
Infections and infestations
Pharyngitis streptococcal	0/22 (0%)		4/22 (18.2%)		0/23 (0%)
Sinusitis	2/22 (9.1%)		2/22 (9.1%)		0/23 (0%)
URI	4/22 (18.2%)		1/22 (4.5%)		4/23 (17.4%)
UTI	1/22 (4.5%)		3/22 (13.6%)		1/23 (4.3%)
Vulvovaginal mycotic infection	2/22 (9.1%)		1/22 (4.5%)		3/23 (13%)
Injury, poisoning and procedural complications
Procedural pain	0/22 (0%)		0/22 (0%)		3/23 (13%)
Investigations
Bone density decreased	2/22 (9.1%)		4/22 (18.2%)		11/23 (47.8%)
Musculoskeletal and connective tissue disorders
Back pain	0/22 (0%)		3/22 (13.6%)		0/23 (0%)
Pain in extremity	1/22 (4.5%)		0/22 (0%)		3/23 (13%)
Nervous system disorders
Dizziness	0/22 (0%)		2/22 (9.1%)		4/23 (17.4%)
Headache	1/22 (4.5%)		4/22 (18.2%)		6/23 (26.1%)
Migraine	0/22 (0%)		0/22 (0%)		2/23 (8.7%)
Reproductive system and breast disorders
Cervical cyst	1/22 (4.5%)		1/22 (4.5%)		2/23 (8.7%)
Endometrial disorder	0/22 (0%)		2/22 (9.1%)		3/23 (13%)
Endometrial hypertrophy	1/22 (4.5%)		1/22 (4.5%)		3/23 (13%)
Ovarian cyst	16/22 (72.7%)		10/22 (45.5%)		15/23 (65.2%)
Parovarian cyst	0/22 (0%)		1/22 (4.5%)		2/23 (8.7%)
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain	0/22 (0%)		2/22 (9.1%)		1/23 (4.3%)
Vascular disorders
Hot flush	0/22 (0%)		4/22 (18.2%)		7/23 (30.4%)

Limitations/Caveats

The study was prematurely terminated for safety reasons (Proellex studies were put on clinical hold due to liver toxicity). Sponsor was unable to pay vendor, and was therefore not provided with complete study data.

More Information

Certain Agreements

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

Prior to publication, Investigator shall submit to the Sponsor a copy of any proposed publication. Sponsor shall have sixty (60) days to review the proposed publication for possible disclosure of Sponsor's Confidential Information and, upon request of Sponsor, Investigator shall delete any of Sponsor's Confidential Information or withhold submission of such publication to allow Sponsor to protect its intellectual property rights