Trial Outcomes & Findings for Extension Study of Proellex in Women Who Have Previously Completed Study ZPU 003 (NCT NCT00958334)

Last Updated: 2019-06-18

Results Overview

An ODI is defined as a time period of less than 3 months during which a return to menses occurs. All statistical endpoints will use the baseline of ZPU-003 Ext for 14-month data and baseline of ZPU-003 for 17-month data.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

65 participants

Primary outcome timeframe

Baseline to 17 months

Results posted on

2019-06-18

Participant Flow

ZPU-003 EXT was an open-label extension study of ZPU-003 (NCT00882258) and all patients received active medication (25 mg of Proellex); results were summarized according to the following groups - subjects who previously received active drug, by dose (12.5 mg and 25 mg) and subjects who previously received placebo in the double-blind ZPU-003 study.

Participant milestones

Participant milestones
Measure	Proellex 25 mg Participants received Proellex 25 mg by oral administration once per day in NCT00882258. In this extension study participants receive 25 mg Proellex by oral administration of two 12.5 mg capsules. PI discretion to decrease dose to 12.5mg if warranted.	Proellex 12.5 mg Then 25 mg Participants received Proellex 12.5 mg. capsule by oral administration once per day in NCT00882258. In this extension study participants receive 25 mg Proellex by oral administration of two 12.5 mg capsules.	Placebo, Then 25 mg Proellex Participants received Placebo capsule by oral administration once per day in NCT00882258. In this extension study participants receive 25 mg Proellex by oral administration of two 12.5 mg capsules.
Overall Study STARTED	22	22	21
Overall Study COMPLETED	8	5	8
Overall Study NOT COMPLETED	14	17	13

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Extension Study of Proellex in Women Who Have Previously Completed Study ZPU 003

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Proellex 25 mg n=22 Participants Participants received Proellex 25 mg by oral administration once per day in NCT00882258. In this extension study participants receive 25 mg Proellex by oral administration of two 12.5 mg capsules. PI discretion to decrease dose to 12.5mg if warranted.	Proellex 12.5 mg Then 25 mg n=22 Participants Participants received Proellex 12.5 mg. capsule by oral administration once per day in NCT00882258. In this extension study participants receive 25 mg Proellex by oral administration of two 12.5 mg capsules.	Placebo, Then 25 mg Proellex n=21 Participants Participants received Placebo capsule by oral administration once per day in NCT00882258. In this extension study participants receive 25 mg Proellex by oral administration of two 12.5 mg capsules.	Total n=65 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	22 Participants n=5 Participants	22 Participants n=7 Participants	21 Participants n=5 Participants	65 Participants n=4 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Continuous	41.5 years STANDARD_DEVIATION 5.55 • n=5 Participants	42.6 years STANDARD_DEVIATION 5.38 • n=7 Participants	40.6 years STANDARD_DEVIATION 6.48 • n=5 Participants	41.6 years STANDARD_DEVIATION 5.78 • n=4 Participants
Sex: Female, Male Female	22 Participants n=5 Participants	22 Participants n=7 Participants	21 Participants n=5 Participants	65 Participants n=4 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Region of Enrollment United States	22 Participants n=5 Participants	22 Participants n=7 Participants	21 Participants n=5 Participants	65 Participants n=4 Participants

PRIMARY outcome

Timeframe: Baseline to 17 months

Population: Intent to treat population

Outcome measures

Outcome measures
Measure	Proellex 25 mg n=22 Participants Participants received Proellex 25 mg by oral administration once per day in NCT00882258. In this extension study participants receive 25 mg Proellex by oral administration of two 12.5 mg capsules. PI discretion to decrease dose to 12.5mg if warranted.	Proellex 12.5 mg Then 25 mg n=22 Participants Participants received Proellex 12.5 mg. capsule by oral administration once per day in NCT00882258. In this extension study participants receive 25 mg Proellex by oral administration of two 12.5 mg capsules.	Placebo, Then 25 mg Proellex n=21 Participants Participants received Placebo capsule by oral administration once per day in NCT00882258. In this extension study participants receive 25 mg Proellex by oral administration of two 12.5 mg capsules.
The Change in Menorrhagia From the Baseline of ZPU-003 to the End of Each Off Drug Interval(ODI) Within the ZPU-003 Extension Study and the Baseline of ZPU-003 to the End of ZPU-003 Ext. Baseline	136.29 mL Standard Deviation 88.12	179.95 mL Standard Deviation 119.83	130.86 mL Standard Deviation 147.10
The Change in Menorrhagia From the Baseline of ZPU-003 to the End of Each Off Drug Interval(ODI) Within the ZPU-003 Extension Study and the Baseline of ZPU-003 to the End of ZPU-003 Ext. Change from baseline to end of ODI 1	-41.00 mL Standard Deviation 71.63	-63.57 mL Standard Deviation 100.83	—
The Change in Menorrhagia From the Baseline of ZPU-003 to the End of Each Off Drug Interval(ODI) Within the ZPU-003 Extension Study and the Baseline of ZPU-003 to the End of ZPU-003 Ext. Change from baseline to end of ODI 2	-38.60 mL Standard Deviation 69.30	-33.33 mL Standard Deviation 213.63	-9.90 mL Standard Deviation 125.88
The Change in Menorrhagia From the Baseline of ZPU-003 to the End of Each Off Drug Interval(ODI) Within the ZPU-003 Extension Study and the Baseline of ZPU-003 to the End of ZPU-003 Ext. Change from baseline to end of ODI 3	-7.00 mL Standard Deviation 69.63	-222.50 mL Standard Deviation 121.62	-31.29 mL Standard Deviation 175.46
The Change in Menorrhagia From the Baseline of ZPU-003 to the End of Each Off Drug Interval(ODI) Within the ZPU-003 Extension Study and the Baseline of ZPU-003 to the End of ZPU-003 Ext. Change from baseline to end of ODI 4	-48.00 mL Standard Deviation 11.31	—	—
The Change in Menorrhagia From the Baseline of ZPU-003 to the End of Each Off Drug Interval(ODI) Within the ZPU-003 Extension Study and the Baseline of ZPU-003 to the End of ZPU-003 Ext. Change from baseline to 17 months	1.43 mL Standard Deviation 94.09	-211.40 mL Standard Deviation 148.06	17.50 mL Standard Deviation 192.88

SECONDARY outcome

Timeframe: Baseline, 14 months, 17 months

Population: Intent to treat population

Patient assessed all menstrual products used for bleeding during the month-long time period between study visits. For the total menstrual pictogram score, a lower score indicated less menstrual bleeding, a higher score indicated more menstrual bleeding. Over the course of a menstrual cycle, each pad was scored on an ordinal scale from 1 to 5, each tampon scored either 1, 1.5, 3 or 8, and clots were scored 1, 3 or 5, based on apparent size. Pictogram scored assessments were converted to approximate mL of blood.

Outcome measures

Outcome measures
Measure	Proellex 25 mg n=22 Participants Participants received Proellex 25 mg by oral administration once per day in NCT00882258. In this extension study participants receive 25 mg Proellex by oral administration of two 12.5 mg capsules. PI discretion to decrease dose to 12.5mg if warranted.	Proellex 12.5 mg Then 25 mg n=22 Participants Participants received Proellex 12.5 mg. capsule by oral administration once per day in NCT00882258. In this extension study participants receive 25 mg Proellex by oral administration of two 12.5 mg capsules.	Placebo, Then 25 mg Proellex n=21 Participants Participants received Placebo capsule by oral administration once per day in NCT00882258. In this extension study participants receive 25 mg Proellex by oral administration of two 12.5 mg capsules.
Change From Baseline of ZPU-003 Ext to 14 Months and 17 Months in Subject's Menstrual Pictogram Scores (mL) (Subjects Evaluable for Menorrhagia Only) Change from baseline to month 14	-150.67 mL of Blood Standard Deviation 66.89	-248.67 mL of Blood Standard Deviation 174.37	-654.00 mL of Blood Standard Deviation NA SD not calculated due to small sample size.
Change From Baseline of ZPU-003 Ext to 14 Months and 17 Months in Subject's Menstrual Pictogram Scores (mL) (Subjects Evaluable for Menorrhagia Only) Change from baseline to month 17	-25.33 mL of Blood Standard Deviation 48.01	-230.00 mL of Blood Standard Deviation 240.02	—

Adverse Events

Proellex 25 mg

Serious events: 2 serious events

Other events: 19 other events

Deaths: 0 deaths

Proellex 12.5 mg Then 25 mg

Serious events: 1 serious events

Other events: 18 other events

Deaths: 0 deaths

Placebo, Then 25 mg Proellex

Serious events: 1 serious events

Other events: 19 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Proellex 25 mg n=22 participants at risk Participants received Proellex 25 mg by oral administration once per day in NCT00882258. In this extension study participants receive 25 mg Proellex by oral administration of two 12.5 mg capsules. PI discretion to decrease dose to 12.5mg if warranted.	Proellex 12.5 mg Then 25 mg n=22 participants at risk Participants received Proellex 12.5 mg. capsule by oral administration once per day in NCT00882258. In this extension study participants receive 25 mg Proellex by oral administration of two 12.5 mg capsules.	Placebo, Then 25 mg Proellex n=21 participants at risk Participants received Placebo capsule by oral administration once per day in NCT00882258. In this extension study participants receive 25 mg Proellex by oral administration of two 12.5 mg capsules.
Gastrointestinal disorders Colitis	4.5% 1/22 • Number of events 1 • 24 months ZPU-003 EXT was an open-label extension study of ZPU-003 (NCT00882258) and all patients received active medication (25 mg of Proellex); however, results were summarized according to the following groups - subjects who previously received active drug, by dose (12.5 mg and 25 mg) and subjects who previously received placebo in the double-blind ZPU-003 study.	0.00% 0/22 • 24 months ZPU-003 EXT was an open-label extension study of ZPU-003 (NCT00882258) and all patients received active medication (25 mg of Proellex); however, results were summarized according to the following groups - subjects who previously received active drug, by dose (12.5 mg and 25 mg) and subjects who previously received placebo in the double-blind ZPU-003 study.	0.00% 0/21 • 24 months ZPU-003 EXT was an open-label extension study of ZPU-003 (NCT00882258) and all patients received active medication (25 mg of Proellex); however, results were summarized according to the following groups - subjects who previously received active drug, by dose (12.5 mg and 25 mg) and subjects who previously received placebo in the double-blind ZPU-003 study.
Hepatobiliary disorders Autoimmune hepatitis	4.5% 1/22 • Number of events 1 • 24 months ZPU-003 EXT was an open-label extension study of ZPU-003 (NCT00882258) and all patients received active medication (25 mg of Proellex); however, results were summarized according to the following groups - subjects who previously received active drug, by dose (12.5 mg and 25 mg) and subjects who previously received placebo in the double-blind ZPU-003 study.	0.00% 0/22 • 24 months ZPU-003 EXT was an open-label extension study of ZPU-003 (NCT00882258) and all patients received active medication (25 mg of Proellex); however, results were summarized according to the following groups - subjects who previously received active drug, by dose (12.5 mg and 25 mg) and subjects who previously received placebo in the double-blind ZPU-003 study.	0.00% 0/21 • 24 months ZPU-003 EXT was an open-label extension study of ZPU-003 (NCT00882258) and all patients received active medication (25 mg of Proellex); however, results were summarized according to the following groups - subjects who previously received active drug, by dose (12.5 mg and 25 mg) and subjects who previously received placebo in the double-blind ZPU-003 study.
Vascular disorders Uterine Haemorrhage	0.00% 0/22 • 24 months ZPU-003 EXT was an open-label extension study of ZPU-003 (NCT00882258) and all patients received active medication (25 mg of Proellex); however, results were summarized according to the following groups - subjects who previously received active drug, by dose (12.5 mg and 25 mg) and subjects who previously received placebo in the double-blind ZPU-003 study.	4.5% 1/22 • Number of events 2 • 24 months ZPU-003 EXT was an open-label extension study of ZPU-003 (NCT00882258) and all patients received active medication (25 mg of Proellex); however, results were summarized according to the following groups - subjects who previously received active drug, by dose (12.5 mg and 25 mg) and subjects who previously received placebo in the double-blind ZPU-003 study.	0.00% 0/21 • 24 months ZPU-003 EXT was an open-label extension study of ZPU-003 (NCT00882258) and all patients received active medication (25 mg of Proellex); however, results were summarized according to the following groups - subjects who previously received active drug, by dose (12.5 mg and 25 mg) and subjects who previously received placebo in the double-blind ZPU-003 study.
Reproductive system and breast disorders Ovarian cyst ruptured	0.00% 0/22 • 24 months ZPU-003 EXT was an open-label extension study of ZPU-003 (NCT00882258) and all patients received active medication (25 mg of Proellex); however, results were summarized according to the following groups - subjects who previously received active drug, by dose (12.5 mg and 25 mg) and subjects who previously received placebo in the double-blind ZPU-003 study.	0.00% 0/22 • 24 months ZPU-003 EXT was an open-label extension study of ZPU-003 (NCT00882258) and all patients received active medication (25 mg of Proellex); however, results were summarized according to the following groups - subjects who previously received active drug, by dose (12.5 mg and 25 mg) and subjects who previously received placebo in the double-blind ZPU-003 study.	4.8% 1/21 • Number of events 1 • 24 months ZPU-003 EXT was an open-label extension study of ZPU-003 (NCT00882258) and all patients received active medication (25 mg of Proellex); however, results were summarized according to the following groups - subjects who previously received active drug, by dose (12.5 mg and 25 mg) and subjects who previously received placebo in the double-blind ZPU-003 study.

Other adverse events

Additional Information

Therapeutic Area Head

Allergan

Phone: 714-246-4500

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: OTHER