A Multi-Center, Parallel Design, Randomized, Double-Blind Study to Evaluate the Safety and Efficacy of 6 and 12 mg Proellex® (Telapristone Acetate) Administered Orally in the Treatment of Premenopausal Women With Confirmed Symptomatic Uterine Fibroids

Uterine Fibroids Clinical Trial

Official title:

A Phase 2, Multi-Center, Parallel Design, Randomized, Double-Blind Study to Evaluate the Safety and Efficacy of 6 and 12 mg Proellex® (Telapristone Acetate) Administered Orally in the Treatment of Premenopausal Women With Confirmed Symptomatic Uterine Fibroids

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02301897
• Other study ID #: ZPU-203
• Status: Completed
• Phase: Phase 2
• Start date: March 16, 2015
• Est. completion date: June 2, 2017

Study information

• Verified date: May 2019
• Source: Repros Therapeutics Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The primary objective of this study is to determine the safety and efficacy of two oral doses of Proellex administered for up to 2 courses of treatment (18 weeks each), each separated by an Off-Drug Interval (ODI), to premenopausal women with symptomatic uterine fibroids.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 43
• Est. completion date: June 2, 2017
• Est. primary completion date: June 2, 2017
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 18 Years to 47 Years

Eligibility

Inclusion Criteria:

• Is a pre-menopausal female, between 18 and 47 years

• Participant has a history of at least 3 regular menstrual cycles in which menorrhagia (heavy bleeding) is due to uterine fibroids

• Confirmed uterine fibroid(s) at screening

• Visit 1 historical pictorial blood loss assessment chart of >120 milliliters (mL) of blood loss, to ensure participants have a menstrual blood loss of at least 80 mL by Alkaline Hematin Assay (during the baseline assessment period).

• A Body Mass Index (BMI) between 18 and 45 inclusive

Exclusion Criteria:

• Participant is pregnant or lactating or is attempting or expecting to become pregnant during the entire study period

• Received an investigational drug in the 30 days prior to the screening for this study

• Has an intrauterine device in place

• Observation or history of abnormal endometrial biopsy including the presence of endometrial intraepithelial neoplasia

• Participants with abnormally high liver enzymes or liver disease

Related Conditions & MeSH terms

• Leiomyoma
• Uterine Fibroids

Intervention

Drug:
• Telapristone Acetate
Telapristone acetate, orally, once daily for 18 weeks (Course 1) and repeated for an additional 18 weeks (Course 2) following the ODI.
• Placebo
Matching placebo, orally, once daily for 18 weeks (Course 1) and repeated for an additional 18 weeks (Course 2) following the ODI.

Locations

Country	Name	City	State
United States	Atlanta Women's Research Institute, Inc.	Atlanta	Georgia
United States	Augusta University	Augusta	Georgia
United States	Alabama Clinical Therapeutics, LLC	Birmingham	Alabama
United States	DCT-HCWC, LLC dba Discovery Clinical Trials	Dallas	Texas
United States	KO Clinical Research, LLC	Fort Lauderdale	Florida
United States	Advances in Health	Houston	Texas
United States	The Women's Hospital of Texas Clinical Research Center	Houston	Texas
United States	The Jackson Clinic, PA	Jackson	Tennessee
United States	Volunteer Research Group	Knoxville	Tennessee
United States	South Florida Clinical Research Institute	Margate	Florida
United States	Southern Clinical Research Associates, LLC	Metairie	Louisiana
United States	Clinical Research Partners, LLC	Richmond	Virginia
United States	WR-Mount Vernon Clinical Research, LLC	Sandy Springs	Georgia
United States	MomDoc Women's Health Research	Scottsdale	Arizona
United States	Palmetto Clinical Research	Summerville	South Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Repros Therapeutics Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage of Participants in Amenorrhea at the End of Treatment Course 1	Amenorrhea was defined as no bleeding intensity score greater than 1 using the Daily Diary Card during the 28 days leading up to the last day of dosing at Week 18. Bleeding intensity was graded on a 5-point scale where: 0=no bleeding to 4=heavy bleeding.	At the end of 18-weeks Treatment Course 1
Secondary	Percentage of Participants in Amenorrhea at the End of Treatment Course 2	Amenorrhea was defined as no bleeding intensity score greater than 1 using the Daily Diary Card during the 28 days leading up to the last day of dosing at Week 18. Bleeding intensity was graded on a 5-point scale where: 0=no bleeding to 4=heavy bleeding.	At the end of 18-weeks Treatment Course 2
Secondary	Change in Pictorial Blood Loss Assessment Chart (PBAC) Score From Baseline to the End of Treatment Courses 1 and 2, ODI Course 1 and the Course 2 Week 28 Follow-up (FU) Visit	Uterine bleeding was assessed with the use of the PBAC, a validated self-reporting method to estimate menstrual blood loss. Participants recorded daily the number of tampons and towels used and the degree to which individual items were soiled with blood (plus small or large clots). Pictorial scores range from score 1 for slightly stained tampon/towel, 5 for a partially stained tampon/towel, 10 for a completely saturated tampon, 20 for a completely saturated towel, and 5 for each episode of flooding and for each blood clot larger than a quarter in size. Total score can range from 0 (no bleeding) to >500. Higher scores indicate more bleeding. Lower scores indicate less bleeding. A negative change from Baseline indicates improvement (reduction in bleeding).	Baseline (No treatment period) to the end of 18-weeks Treatment Course 1, the end of ODI Course 1, the end of 18-weeks Treatment Course 2 and the Course 2 Week 28 FU Visit
Secondary	Percentage Change in PBAC Score From Baseline to the End of Treatment Courses 1 and 2, ODI Course 1 and the Course 2 Week 28 FU Visit	Uterine bleeding was assessed with the use of the PBAC, a validated self-reporting method to estimate menstrual blood loss. Participants recorded daily the number of tampons and towels used and the degree to which individual items were soiled with blood (plus small or large clots). Pictorial scores range from score 1 for slightly stained tampon/towel, 5 for a partially stained tampon/towel, 10 for a completely saturated tampon, 20 for a completely saturated towel, and 5 for each episode of flooding and for each blood clot larger than a quarter in size. Total score can range from 0 (no bleeding) to >500. Higher scores indicate more bleeding. Lower scores indicate less bleeding. A negative percentage change from Baseline indicates improvement (reduction in bleeding).	Baseline (No treatment period) to the end of 18-weeks Treatment Course 1, the end of ODI Course 1, the end of 18-weeks Treatment Course 2 and the Course 2 Week 28 FU Visit
Secondary	Percentage Change in Uterine Fibroid System Quality of Life Survey System Severity (UFS-SSS) Score From Baseline to the End of Treatment Courses 1 and 2, On-drug Course 2 and the Course 2 Week 24 FU Visit	UFS-SSS is an 8 question assessment tool used to measure symptom severity and has been validated as a three month look back questionnaire. Each question was answered on a 5-point scale where 1=Not at all to 5=A very great deal. The sum of total scores was transformed to a range of 0=no symptoms (best) to 100=most severe symptoms (worst). A negative percentage change from Baseline indicates improvement.	Baseline (No treatment period) to the end of 18-weeks Treatment Course 1, On-drug Course 2, the end of 18-weeks Treatment Course 2 and the Course 2 Week 24 FU Visit
Secondary	Percentage Change in the Individual UFS-SSS Subscale Score Question 1 From Baseline to the End of Treatment Courses 1 and 2, On-drug Course 2 and the Course 2 Week 24 FU Visit	UFS-SSS is an 8 question assessment tool used to measure symptom severity and has been validated as a three month look back questionnaire. The participant answered UFS-SSS subscale question 1: During the previous 3 months how distressed were you by "heavy bleeding during your menstrual period"? using a 5-point scale where 1=Not at all to 5=A very great deal. A negative percentage change from Baseline indicates improvement.	Baseline (No treatment period) to the end of 18-weeks Treatment Course 1, On-drug Course 2, the end of 18-weeks Treatment Course 2 and the Course 2 Week 24 FU Visit
Secondary	Percentage Change in the Individual UFS-SSS Subscale Score Question 2 From Baseline to the End of Treatment Courses 1 and 2, On-drug Course 2 and the Course 2 Week 24 FU Visit	UFS-SSS is an 8 question assessment tool used to measure symptom severity and has been validated as a three month look back questionnaire. The participant answered UFS-SSS subscale question 2: During the previous 3 months how distressed were you by "passing blood clots during your menstrual period"? using a 5-point scale where 1=Not at all to 5=A very great deal. A negative percentage change from Baseline indicates improvement.	Baseline (No treatment period) to the end of 18-weeks Treatment Course 1, On-drug Course 2, the end of 18-weeks Treatment Course 2 and the Course 2 Week 24 FU Visit
Secondary	Percentage Change in the Individual UFS-SSS Subscale Score Question 3 From Baseline to the End of Treatment Courses 1 and 2, On-drug Course 2 and the Course 2 Week 24 FU Visit	UFS-SSS is an 8 question assessment tool used to measure symptom severity and has been validated as a three month look back questionnaire. The participant answered UFS-SSS subscale question 3: During the previous 3 months how distressed were you by "fluctuation in the duration of your menstrual period compared to your previous cycle"? using a 5-point scale where 1=Not at all to 5=A very great deal. A negative percentage change from Baseline indicates improvement.	Baseline (No treatment period) to the end of 18-weeks Treatment Course 1, On-drug Course 2, the end of 18-weeks Treatment Course 2 and the Course 2 Week 24 FU Visit
Secondary	Percentage Change in the Individual UFS-SSS Subscale Score Question 4 From Baseline to the End of Treatment Courses 1 and 2, On-drug Course 2 and the Course 2 Week 24 FU Visit	UFS-SSS is an 8 question assessment tool used to measure symptom severity and has been validated as a three month look back questionnaire. The participant answered UFS-SSS subscale question 4: During the previous 3 months how distressed were you by "fluctuation in the length of your monthly cycle compared to your previous cycles"? using a 5-point scale where 1=Not at all to 5=A very great deal. A negative percentage change from Baseline indicates improvement.	Baseline (No treatment period) to the end of 18-weeks Treatment Course 1, On-drug Course 2, the end of 18-weeks Treatment Course 2 and the Course 2 Week 24 FU Visit
Secondary	Percentage Change in the Individual UFS-SSS Subscale Score Question 5 From Baseline to the End of Treatment Courses 1 and 2, On-drug Course 2 and the Course 2 Week 24 FU Visit	UFS-SSS is an 8 question assessment tool used to measure symptom severity and has been validated as a three month look back questionnaire. The participant answered UFS-SSS subscale question 5: During the previous 3 months how distressed were you by "feeling tightness or pressure in your pelvic area"? using a 5-point scale where 1=Not at all to 5=A very great deal. A negative percentage change from Baseline indicates improvement.	Baseline (No treatment period) to the end of 18-weeks Treatment Course 1, On-drug Course 2, the end of 18-weeks Treatment Course 2 and the Course 2 Week 24 FU Visit
Secondary	Percentage Change in the Individual UFS-SSS Subscale Score Question 6 From Baseline to the End of Treatment Courses 1 and 2, On-drug Course 2 and the Course 2 Week 24 FU Visit	UFS-SSS is an 8 question assessment tool used to measure symptom severity and has been validated as a three month look back questionnaire. The participant answered UFS-SSS subscale question 6: During the previous 3 months how distressed were you by "frequent urination during the daytime hours"? using a 5-point scale where 1=Not at all to 5=A very great deal. A negative percentage change from Baseline indicates improvement.	Baseline (No treatment period) to the end of 18-weeks Treatment Course 1, On-drug Course 2, the end of 18-weeks Treatment Course 2 and the Course 2 Week 24 FU Visit
Secondary	Percentage Change in the Individual UFS-SSS Subscale Score Question 7 From Baseline to the End of Treatment Courses 1 and 2, On-drug Course 2 and the Course 2 Week 24 FU Visit	UFS-SSS is an 8 question assessment tool used to measure symptom severity and has been validated as a three month look back questionnaire. The participant answered UFS-SSS subscale question 7: During the previous 3 months how distressed were you by "frequent nighttime urination"? using a 5-point scale where 1=Not at all to 5=A very great deal. A negative percentage change from Baseline indicates improvement.	Baseline (No treatment period) to the end of 18-weeks Treatment Course 1, On-drug Course 2, the end of 18-weeks Treatment Course 2 and the Course 2 Week 24 FU Visit
Secondary	Percentage Change in the Individual UFS-SSS Subscale Score Question 8 From Baseline to the End of Treatment Courses 1 and 2, On-drug Course 2 and the Course 2 Week 24 FU Visit	UFS-SSS is an 8 question assessment tool used to measure symptom severity and has been validated as a three month look back questionnaire. The participant answered UFS-SSS subscale question 8: During the previous 3 months how distressed were you by "feeling fatigued"? using a 5-point scale where 1=Not at all to 5=A very great deal. A negative percentage change from Baseline indicates improvement.	Baseline (No treatment period) to the end of 18-weeks Treatment Course 1, On-drug Course 2, the end of 18-weeks Treatment Course 2 and the Course 2 Week 24 FU Visit
Secondary	Percentage Change in Total Uterine Fibroid Volume From Baseline to the End of Treatment Courses 1 and 2, On-drug Course 2 and the Course 2 Off-drug	The total uterine fibroid volume was measured by Magnetic Resonance Imaging (MRI). A negative percentage change from Baseline indicates improvement.	Baseline (No treatment period) to the end of 18-weeks Treatment Course 1, On-drug Course 2, the end of 18-weeks Treatment Course 2 and the Course 2 Off-drug