Trial Outcomes & Findings for An Extension Study of 12 mg Proellex® (Telapristone Acetate) Administered Orally in the Treatment of Premenopausal Women With Confirmed Symptomatic Uterine Fibroids (NCT NCT02811159)

NCT ID: NCT02811159

Last Updated: 2019-06-27

Results Overview

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.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 20 participants
• Primary outcome timeframe: At the end of 18 weeks Treatment Course 1
• Results posted on: 2019-06-27

Participant Flow

Participants who had successfully completed either study ZPV-201 [NCT02323646] or ZPU-203 [NCT02301897] were enrolled in this study.

Participant milestones

Measure	Telapristone Acetate 12 mg Telapristone acetate 12 mg, orally, once daily for two 18-weeks courses (Treatment Courses 1 and 2) separated by an off-drug interval (ODI).
Treatment Course 1 STARTED	20
Treatment Course 1 COMPLETED	19
Treatment Course 1 NOT COMPLETED	1
Treatment Course 2 STARTED	4
Treatment Course 2 COMPLETED	4
Treatment Course 2 NOT COMPLETED	0

Reasons for withdrawal

Measure	Telapristone Acetate 12 mg Telapristone acetate 12 mg, orally, once daily for two 18-weeks courses (Treatment Courses 1 and 2) separated by an off-drug interval (ODI).
Treatment Course 1 Lost to Follow-up	1

Baseline Characteristics

Safety population included all study participants who were randomized, received study drug and had some post-baseline safety data.

Baseline characteristics by cohort

Measure	Telapristone Acetate 12 mg n=20 Participants Telapristone acetate 12 mg, orally, once daily for two 18-weeks courses (Treatment Courses 1 and 2) separated by an off-drug interval (ODI).
Age, Continuous	43.3 years STANDARD_DEVIATION 3.62 • n=20 Participants • Safety population included all study participants who were randomized, received study drug and had some post-baseline safety data.
Sex: Female, Male Female	20 Participants n=20 Participants • Safety population included all study participants who were randomized, received study drug and had some post-baseline safety data.
Sex: Female, Male Male	0 Participants n=20 Participants • Safety population included all study participants who were randomized, received study drug and had some post-baseline safety data.

PRIMARY outcome

Timeframe: At the end of 18 weeks Treatment Course 1

Population: Intent-to-treat (ITT) population included all participants who received study drug.

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.

Outcome measures

Measure	Telapristone Acetate 12 mg n=20 Participants Telapristone acetate 12 mg, orally, once daily for two 18-weeks courses (Treatment Courses 1 and 2) separated by an off-drug interval (ODI).
Percentage of Participants in Amenorrhea	35.00 percentage of participants

PRIMARY outcome

Timeframe: Baseline to the end of 18-weeks Treatment Course 1

Population: ITT population included all participants who received study drug.

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 the 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.

Outcome measures

Measure	Telapristone Acetate 12 mg n=20 Participants Telapristone acetate 12 mg, orally, once daily for two 18-weeks courses (Treatment Courses 1 and 2) separated by an off-drug interval (ODI).
Percentage Change From Baseline in Total Uterine Fibroid System Quality of Life Survey System Severity (UFS-SSS) Score	-7.21 percent change Standard Deviation 20.046

PRIMARY outcome

Timeframe: Baseline to the end of 18-weeks Treatment Course 1

Population: ITT population included all participants who received study drug.

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.

Outcome measures

Measure	Telapristone Acetate 12 mg n=20 Participants Telapristone acetate 12 mg, orally, once daily for two 18-weeks courses (Treatment Courses 1 and 2) separated by an off-drug interval (ODI).
Percentage Change From Baseline in the Individual UFS-SSS Subscale Score Question 1	-10.50 percent change Standard Deviation 26.253

PRIMARY outcome

Timeframe: Baseline to the end of 18-weeks Treatment Course 1

Population: ITT population included all participants who received study drug.

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.

Outcome measures

Measure	Telapristone Acetate 12 mg n=20 Participants Telapristone acetate 12 mg, orally, once daily for two 18-weeks courses (Treatment Courses 1 and 2) separated by an off-drug interval (ODI).
Percentage Change From Baseline in the Individual UFS-SSS Subscale Score Question 2	-5.83 percent change Standard Deviation 18.157

PRIMARY outcome

Timeframe: Baseline to the end of 18-weeks Treatment Course 1

Population: ITT population included all participants who received study drug.

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.

Outcome measures

Measure	Telapristone Acetate 12 mg n=20 Participants Telapristone acetate 12 mg, orally, once daily for two 18-weeks courses (Treatment Courses 1 and 2) separated by an off-drug interval (ODI).
Percentage Change From Baseline in the Individual UFS-SSS Subscale Score Question 3	-8.75 percent change Standard Deviation 23.333

PRIMARY outcome

Timeframe: Baseline to the end of 18-weeks Treatment Course 1

Population: ITT population included all participants who received study drug.

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.

Outcome measures

Measure	Telapristone Acetate 12 mg n=20 Participants Telapristone acetate 12 mg, orally, once daily for two 18-weeks courses (Treatment Courses 1 and 2) separated by an off-drug interval (ODI).
Percentage Change From Baseline in the Individual UFS-SSS Subscale Score Question 4	-3.75 percent change Standard Deviation 16.771

PRIMARY outcome

Timeframe: Baseline to the end of 18-weeks Treatment Course 1

Population: ITT population included all participants who received study drug.

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.

Outcome measures

Measure	Telapristone Acetate 12 mg n=20 Participants Telapristone acetate 12 mg, orally, once daily for two 18-weeks courses (Treatment Courses 1 and 2) separated by an off-drug interval (ODI).
Percentage Change From Baseline in the Individual UFS-SSS Subscale Score Question 5	-4.17 percent change Standard Deviation 33.278

PRIMARY outcome

Timeframe: Baseline to the end of 18-weeks Treatment Course 1

Population: ITT population included all participants who received study drug.

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.

Outcome measures

Measure	Telapristone Acetate 12 mg n=20 Participants Telapristone acetate 12 mg, orally, once daily for two 18-weeks courses (Treatment Courses 1 and 2) separated by an off-drug interval (ODI).
Percentage Change From Baseline in the Individual UFS-SSS Subscale Score Question 6	-7.33 percent change Standard Deviation 22.675

PRIMARY outcome

Timeframe: Baseline to the end of 18-weeks Treatment Course 1

Population: ITT population included all participants who received study drug.

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 night time 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.

Outcome measures

Measure	Telapristone Acetate 12 mg n=20 Participants Telapristone acetate 12 mg, orally, once daily for two 18-weeks courses (Treatment Courses 1 and 2) separated by an off-drug interval (ODI).
Percentage Change From Baseline in the Individual UFS-SSS Subscale Score Question 7	6.67 percent change Standard Deviation 35.210

PRIMARY outcome

Timeframe: Baseline to the end of 18-weeks Treatment Course 1

Population: ITT population included all participants who received study drug.

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.

Outcome measures

Measure	Telapristone Acetate 12 mg n=20 Participants Telapristone acetate 12 mg, orally, once daily for two 18-weeks courses (Treatment Courses 1 and 2) separated by an off-drug interval (ODI).
Percentage Change From Baseline in the Individual UFS-SSS Subscale Score Question 8	-6.67 percent change Standard Deviation 16.579

PRIMARY outcome

Timeframe: Baseline to the end of 18-weeks Treatment Course 1

Population: ITT population Included all participants who received study drug.

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).

Outcome measures

Measure	Telapristone Acetate 12 mg n=20 Participants Telapristone acetate 12 mg, orally, once daily for two 18-weeks courses (Treatment Courses 1 and 2) separated by an off-drug interval (ODI).
Change From Baseline in Pictorial Blood Loss Assessment Chart (PBAC) Score Baseline	139.50 score on a scale Standard Deviation 196.111
Change From Baseline in Pictorial Blood Loss Assessment Chart (PBAC) Score Change from Baseline to Week 18 Course 1	-81.10 score on a scale Standard Deviation 239.790

PRIMARY outcome

Timeframe: Baseline to the end of 18-weeks Treatment Course 1

Population: ITT population Included all participants who received study drug. MRI data was only available for 3 participants.

The total uterine fibroid volume was measured by Magnetic Resonance Imaging (MRI). A negative percentage change from Baseline indicates improvement.

Outcome measures

Measure	Telapristone Acetate 12 mg n=3 Participants Telapristone acetate 12 mg, orally, once daily for two 18-weeks courses (Treatment Courses 1 and 2) separated by an off-drug interval (ODI).
Percentage Change From Baseline in Total Uterine Fibroid Volume	0.00 percent change Standard Deviation 0.000

Adverse Events

Telapristone Acetate 12 mg

Serious events: 1 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Measure	Telapristone Acetate 12 mg n=20 participants at risk Telapristone acetate 12 mg, orally, once daily for two 18-weeks courses (Treatment Courses 1 and 2) separated by an off-drug interval (ODI).
Investigations Liver function test abnormal	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.

Other adverse events

Measure	Telapristone Acetate 12 mg n=20 participants at risk Telapristone acetate 12 mg, orally, once daily for two 18-weeks courses (Treatment Courses 1 and 2) separated by an off-drug interval (ODI).
Gastrointestinal disorders Abdominal pain	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Gastrointestinal disorders Diverticulitis	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Gastrointestinal disorders Dyspepsia	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Gastrointestinal disorders Flatulence	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Gastrointestinal disorders Nausea	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
General disorders Hot flush	10.0% 2/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
General disorders Chills	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
General disorders Fatigue	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Infections and infestations Upper respiratory tract infection	10.0% 2/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Infections and infestations Urinary tract infection	10.0% 2/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Infections and infestations Acute sinusitis	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Infections and infestations Bronchitis	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Infections and infestations Sinusitis	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Infections and infestations Vulvovaginal mycotic infection	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Investigations Blood cholesterol increased	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Investigations Blood pressure increased	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Musculoskeletal and connective tissue disorders Back pain	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Nervous system disorders Disorientation	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Nervous system disorders Dizziness	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Nervous system disorders Headache	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Renal and urinary disorders Pollakiuria	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Reproductive system and breast disorders Endometrial hypertrophy	10.0% 2/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Reproductive system and breast disorders Menometrorrhagia	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.
Skin and subcutaneous tissue disorders Urticaria	5.0% 1/20 • From first dose of study drug through 30 days after the last dose of study drug (approximately 43 weeks) Safety population consisted of all study participants who were randomized, received study drug and had some post-baseline safety data.

Additional Information

Therapeutic Area, Head

Allergan

Phone: 714-246-4500

Email: clinicaltrials@allergan.com

Results disclosure agreements

• Principal investigator is a sponsor employee A disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 90 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
• Publication restrictions are in place

Restriction type: OTHER