Clinical trial NCT01629563: PGL4001 Efficacy Assessment in Reduction of Symptoms Due to Uterine Leiomyomata
Official title: A Phase III, Multicentre, Randomized, Double-blind Clinical Study, Investigating the Efficacy and Safety of Repeated 12-week Courses of Daily 5mg or 10mg Doses of PGL4001 for the Long-term Management of Symptomatic Uterine Fibroids
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Uterine Fibroids
Lead sponsor: PregLem SA
Sponsor class: INDUSTRY
First posted: 2012-06-27
Enrollment: 451
Sex: FEMALE
Minimum age (years): 18
Maximum age (years): 50
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Percentage of Subjects Who Are in Amenorrhea at the End of All Four Treatment Courses
timeFrame: 18 months study duration per subject (4 3-month intermittent treatment courses)
Secondary outcomes:
measure: Percentage of Subjects Who Were in Amenorrhea at the End of Treatment Course 4
timeFrame: After 18 months
measure: Percentage of Subjects With Controlled Bleeding at the End of All 4 Treatment Courses
timeFrame: After 18 months
measure: Percentage of Change From Baseline to End of Treatment Course 4 in the Total Volume of the 3 Largest Fibroids
timeFrame: After 18 months
measure: Percentage of Change From Baseline to End of Treatment Course 4 in Quality of Life (Uterine Fibroid Symptom Severity (UFSQoL)
timeFrame: After 18 months
measure: Percentage of Change From Baseline to End of Treatment Course 4 in Quality of Life -Uterine Fibroid Health Related Quality of Life (HRQL)
timeFrame: 18 months
measure: Percentage of Change From Baseline to End of Treatment Course 4 in Pain Using a Visual Analogue Scale (VAS)
timeFrame: After 18 months
Other outcomes:
Locations:
city: Brussels
country: Belgium
facility: Cliniques Universitaires Saint-Luc Gynécologie-Obstétrique
city: Leuven
country: Belgium
facility: UZ Leuven Campus Gasthuisberg
city: Liège
country: Belgium
facility: CHU de Liège, CHR de la Citadelle Gynécologie-Obstétrique
city: Yvoir
country: Belgium
facility: CHU Mont-Godinne
city: Brno
country: Czechia
facility: Centrum ambulantni gynekologie a primarni pece
city: Brno
country: Czechia
facility: FN Brno Gynekologicko - porodnická klinika
city: Hradec Kralove
country: Czechia
facility: Sanatorium SANUS
city: Jihlava
country: Czechia
facility: Nemocnice Jihlava Gynekologicko - porodnicke oddeleni
city: Olomouc
country: Czechia
facility: G-CENTRUM Olomouc s.r.o.
city: Olomouc
country: Czechia
facility: FN Olomouc, Porodnicko-Gynekologicka klinika
city: Praha 3
country: Czechia
facility: Femina Sana, s.r.o.
city: Le Kremlin Bicêtre
country: France
facility: Hôpital Bicêtre - APHP
city: Paris
country: France
facility: Hôpital Bichat, Service de Gynécologie Obstétrique
city: Tours
country: France
facility: CHU Bretonneau Service de Gynécologie Obstétrique
city: Hamburg
country: Germany
facility: Private practice
city: Hamburg
country: Germany
facility: Private practice
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover Klinik für Frauenheilkunde und Geburtshilfe
city: Hessen
country: Germany
facility: Frauenarztpraxis
city: Magdeburg
country: Germany
facility: Praxis für Frauenheilkunde, Klinische Forschung und Weiterbildung
city: München
country: Germany
facility: Technische Universität München
city: Bekescsaba
country: Hungary
facility: Rethy Pal Korhaz és Rendelointezet Szuleszeti es Nogyogyaszati Osztaly
city: Budapest
country: Hungary
facility: Institution Robert Karoly Maganklinika
city: Debrecen
country: Hungary
facility: Szent Anna Szuleszeti, Nogyogyaszati es Ultrahang Maganrendelo
city: Nyiregyhaza
country: Hungary
facility: Josa Andras Oktatokorhaz
city: Szekesfehervar
country: Hungary
facility: Fejer Megyei Szent Gyorgy Korhaz Szuleszeti es Nogyogyaszati Osztaly
city: Szentes
country: Hungary
facility: Szuleszeti es Nogyogyaszati Osztaly
city: Szolnok
country: Hungary
facility: Sandor Dent Bt.
city: Catanzaro
country: Italy
facility: Dipartimento di Ostetricia e Ginecologia, Università degli Studi di Catanzaro "Magna Graecia"
city: Roma
country: Italy
facility: Policlinico Universitario "Agostino Gemelli"
city: Riga
country: Latvia
facility: Riga 1. hospital
city: Riga
country: Latvia
facility: Latvian Medical Marine Center
city: Riga
country: Latvia
facility: Medical Company "ARS"
city: Kaunas
country: Lithuania
facility: Saules Family Medicine Center
city: Vilnius
country: Lithuania
facility: Family Medicine Centre"Seimos Gydytojas"
city: Vilnius
country: Lithuania
facility: Private Clinic "Maxmeda"
city: Vilnius
country: Lithuania
facility: Private Clinic "Kardiolita"
city: Bucuresti
country: Romania
facility: Centrul Medical SANA SRL
city: Bucuresti
country: Romania
facility: Quantum Medical Center SRL Obstetrica-Ginecologie
city: Bucuresti
country: Romania
facility: Fortis Medical Center SRL Obstetrica Ginecologie
city: Bucuresti
country: Romania
facility: Spitalul Clinic Dr. I.Cantacuzino sectia Obstetrica-Ginecologie
city: Bucuresti
country: Romania
facility: Centrul Medical EUROMED SRL, Departamentul de Obtetrica/Ginecologie
city: Iasi
country: Romania
facility: Spitalul Clinic de Obstetrica
city: Kharkiv
country: Ukraine
facility: Kharkiv City Perinatal Center Gynaecological Department #1
city: Odessa
country: Ukraine
facility: Municipal Institution "Maternity Hospital #1" City Center of family planning
city: Zaporizhzhya
country: Ukraine
facility: Maternity Hospital#4 Department of Gynaecology
city: Edinburgh
country: United Kingdom
facility: MRC Centre for Reproductive Health University of Edinburgh
city: London
country: United Kingdom
facility: North Middlesex University Hospital NHS Trust
city: Newcastle upon Tyne
country: United Kingdom
facility: Women's Health, Royal Victoria Infirmary
city: Norwich
country: United Kingdom
facility: Norfolk & Norwich University Hospital
Study officials:
affiliation: PregLem SA
name: Pablo Arrigada, MD
role: STUDY_DIRECTOR
References:
citation: Donnez J, Donnez O, Matule D, Ahrendt HJ, Hudecek R, Zatik J, Kasilovskiene Z, Dumitrascu MC, Fernandez H, Barlow DH, Bouchard P, Fauser BC, Bestel E, Loumaye E. Long-term medical management of uterine fibroids with ulipristal acetate. Fertil Steril. 2016 Jan;105(1):165-173.e4. doi: 10.1016/j.fertnstert.2015.09.032. Epub 2015 Oct 23.
citation: Donnez J, Hudecek R, Donnez O, Matule D, Arhendt HJ, Zatik J, Kasilovskiene Z, Dumitrascu MC, Fernandez H, Barlow DH, Bouchard P, Fauser BC, Bestel E, Terrill P, Osterloh I, Loumaye E. Efficacy and safety of repeated use of ulipristal acetate in uterine fibroids. Fertil Steril. 2015 Feb;103(2):519-27.e3. doi: 10.1016/j.fertnstert.2014.10.038. Epub 2014 Dec 24.