PGL4001 Efficacy Assessment in Reduction of Symptoms Due to Uterine Leiomyomata

Phase 3

Completed

Conditions: Uterine Fibroids

Interventions: Drug: PGL4001 5 mg
Drug: PGL4001 10 mg

Registration Number: NCT01629563

Lead Sponsor: PregLem SA

Brief Summary: Phase III, multicentre, randomized, double-blind, parallel group, long-term study investigating the efficacy and safety of the 5mg and 10mg doses of PGL4001 for the treatment of uterine myoma.

Detailed Description: The target population is composed of pre-menopausal women with symptomatic uterine myoma(s) characterised by heavy bleeding.The main objective of this study is to assess the sustained efficacy and safety of long term on-off treatment with PGL4001 5 or 10mg doses on uterine bleeding, myoma size, pain and quality of life.

Recruitment & Eligibility

Status: COMPLETED

Sex: Female

Target Recruitment: 451

Inclusion Criteria

Be a pre-menopausal woman between 18 and 50 years inclusive.
Have a Body Mass Index (BMI) ≥ 18 and ≤ 40.
Have FSH levels ≤ 20 mIU/mL
Have excessive uterine bleeding due to myoma.
Have regular menstrual cycles
Have a myomatous uterus < 16 weeks with at least one myoma ≥ 3 cm in diameter.
If of childbearing potential the subject must be practicing a non-hormonal method of contraception.

Exclusion Criteria

Has a history of or current uterine, cervical, ovarian or breast cancer.
Has a history of or current endometrium atypical hyperplasia or adenocarcinoma.
Has a known severe coagulation disorder.
Has a history of or current treatment for myoma with a Selective Progesterone Receptor Modulator (SPRM).
Has abnormal hepatic function at study entry.
Has a positive pregnancy test, is nursing or planning a pregnancy during the course of the study.
Has a current (within twelve months) problem with alcohol or drug abuse.
Is currently enrolled in an investigational drug or device study or has participated in such a study within the last 30 days.

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Ulipristal acetate (PGL4001) 5mg	PGL4001 5 mg	All subjects will be asked to take a 150mg size tablet (PGL4001 5mg or matching placebo: placebo 5) orally daily for repeated periods 84 days. The first treatment course will start on the first 4 days of menstruation and will be orally administered, once daily (1 tablet of 150mg size), for 84 days. The following three treatment courses should be started in the first two days of a menstrual period.
Ulipristal acetate (PGL4001) 10mg	PGL4001 10 mg	All subjects will be asked to take a 300mg size tablet (PGL4001 10mg or matching placebo: placebo 10 ) orally daily for repeated periods 84 days. The first treatment course will start on the first 4 days of menstruation and will be orally administered, once daily (1 tablet of 300mg size), for 84 days. The following three treatment courses should be started in the first two days of a menstrual period.

Primary Outcome Measures

Name	Time	Method
Percentage of Subjects Who Are in Amenorrhea at the End of All Four Treatment Courses	18 months study duration per subject (4 3-month intermittent treatment courses)	Amenorrhoea was defined as no more than 1 day of spotting within a 35 day interval. Subjects need to be in amenorrhoea at the end of all four treatment courses, i.e for at least 4x35 days.

Secondary Outcome Measures

Name	Time	Method
Percentage of Subjects Who Were in Amenorrhea at the End of Treatment Course 4	After 18 months	Amenorrhoea was defined as no more than 1 day of spotting within a 35 day interval.
Percentage of Subjects With Controlled Bleeding at the End of All 4 Treatment Courses	After 18 months	Controlled bleeding was defined as no episodes of heavy bleeding and a maximum of 8 days of bleeding during the last 56 days of a treatment course. Subjects need to be in controlled bleeding at the end of all 4 treatment courses i.e. for at least 4x56 days.
Percentage of Change From Baseline to End of Treatment Course 4 in Pain Using a Visual Analogue Scale (VAS)	After 18 months	Pain was assessed using a Visual Analogue Scale (VAS) ranging from 0 to 100 with higher score indicating more severe pain. Subjects were exposed to 4 3-month intermittent courses.
Percentage of Change From Baseline to End of Treatment Course 4 in the Total Volume of the 3 Largest Fibroids	After 18 months	For the 3 largest myomas at baseline and the 3 largest myomas at the end of treatment course 4 identified by transvaginal ultrasound, length, height and depth were measured and the volume was estimated by applying the equation for the voulme of an ellipsoid (length x height x depht x π/6). Subjects were exposed to 4 3-month intermittent courses.
Percentage of Change From Baseline to End of Treatment Course 4 in Quality of Life (Uterine Fibroid Symptom Severity (UFSQoL)	After 18 months	Quality of Life was assessed using a validated questionnaire measuring uterine fibroid symptom severity (UFSQoL) where lower scores indicate fewer symtoms and where a level of 23 has been reported for healthy subject (scale 0-100). Subjects were exposed to 4 3-month intermittent courses.
Percentage of Change From Baseline to End of Treatment Course 4 in Quality of Life -Uterine Fibroid Health Related Quality of Life (HRQL)	18 months	Quality of Life was measured using a validated uterine fibroid symptom questionnaire. Total score for health related quality of Life (HRQL) range from 0 to 100 with higher scores indicating better Quality of Life. Subjects were exposed to 4 3-month intermittent courses.

Trial Locations

Locations (48): Cliniques Universitaires Saint-Luc Gynécologie-Obstétrique
🇧🇪
Brussels, Belgium
UZ Leuven Campus Gasthuisberg
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Leuven, Belgium
CHU de Liège, CHR de la Citadelle Gynécologie-Obstétrique
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Liège, Belgium
CHU Mont-Godinne
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Yvoir, Belgium
Centrum ambulantni gynekologie a primarni pece
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Brno, Czechia
FN Brno Gynekologicko - porodnická klinika
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Brno, Czechia
Sanatorium SANUS
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Hradec Kralove, Czechia
Nemocnice Jihlava Gynekologicko - porodnicke oddeleni
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Jihlava, Czechia
G-CENTRUM Olomouc s.r.o.
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Olomouc, Czechia
FN Olomouc, Porodnicko-Gynekologicka klinika
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Olomouc, Czechia
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Cliniques Universitaires Saint-Luc Gynécologie-Obstétrique
🇧🇪Brussels, Belgium