PGL2001 Proof of Concept Study in Symptomatic Endometriosis (AMBER)

A Phase II, Multicentre, Randomised, Two-Arm, Parallel Group, Double-Blind, Placebo Controlled Study of the Steroid Sulfatase Inhibitor PGL2001 With Concomitant Administration of NETA (Norethisterone Acetate) for the Treatment of Symptoms Related to Endometriosis

This is a Phase II, multicentre, randomised, two-arm, parallel group ,double-blind, placebo controlled, study to investigate the efficacy, safety, pharmacokinetics and pharmacodynamics of the steroid sulfatase inhibitor PGL2001 with concomitant, continuous NETA administration for the treatment of pain symptoms suggestive of endometriosis. The target population will be women of reproductive age with symptoms suggestive of endometriosis.

Study Overview

• Status: Completed
• Conditions: Endometriosis
• Intervention / Treatment: Drug: PGL2001 + Primolut-Nor 5; Drug: Placebo + Primolut-Nor 5

• Study Type: Interventional
• Enrollment (Actual): 162
• Phase: Phase 2

Contacts and Locations

Study Locations

• Hungary
  • Budapest, Hungary, 1036
    • Synexus Magyarorszag Kft.
  • Budapest, Hungary, 1082
    • Semmelweis Egyetem II. Szülészeti és Nőgyógyászati Klinika
  • Debrecen, Hungary, 4012
    • Debreceni Egyetem Orvos-es Egeszsegtudomanyi Centrum Szuleszeti es Nogyogyaszati Klinika
  • Pecs, Hungary, 7624
    • Pécsi Tudományegyetem Klinikai Központ Szülészeti és Nőgyógyászati Klinika
  • Szeged, Hungary, 6725
    • Szegedi Tudományegyetem Szent-Györgyi Albert Klinkai Központ Szülészeti és Nőgyógyászati Klinika
• Poland
  • Katowice, Poland, 40-954
    • Medica Pro Familia Sp. z o.o. S.K.A. - Oddział Katowie
  • Lublin, Poland, 20-496
    • Specjalistyczny Gabinet Ginekologiczno-Położniczy
  • Poznan, Poland, 60-535
    • Klinika Endokrynologii I Nieplodnosci Rozrodu, Ginekologiczno Polozniczy Szpital UM
  • Swidnik, Poland, 21040
    • Lubelskie Centrum Diagnostyczne
  • Szczecin, Poland, 71-074
    • VitroLive Sp. Z o.o
  • Warszawa, Poland, 01-826
    • NZOZ Lecznica Medea
  • Warszawa, Poland, 02-201
    • Private Practice
  • Warszawa, Poland, 02-507
    • Centralny Szpital Kliniczny MSWiA, Klinika Połoznictwa, Chorob Kobiecych i Ginekologii Onkologicznej
  • Łódź, Poland, 91-308
    • Medeor Plus Szpital Wielospecjalistyczny
  • Łódź, Poland, 93-338
    • Klinka Ginekologii Operacyjnej, Instytut-Centrum Zdrowia Matki Polki
• Romania
  • Baia Mare, Romania, 430032
    • Euromedica Hospital SA
  • Brasov, Romania, 500283
    • Centrul Medical de Diagnostic si Tratament Ambulator NEOMED SRL
  • Bucharest, Romania, 011475
    • Genesys Fertility Center SRL
  • Bucharest, Romania, 020762
    • Centrul Medical Euromed SRL
  • Bucharest, Romania, 022441
    • Spitalul Clinic "Nicolae Malaxa"

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 41 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Female

Description

Inclusion Criteria:

• Provide written informed consent prior to initiation of any study related procedures.
• Be a woman of reproductive age between 18 and 45 years inclusive.
• Present with clinical signs suggestive of endometriosis.
• Be suffering from non menstrual pelvic pain and dysmenorrhea suggestive of endometriosis for at least 3 months prior to screening visit.

Exclusion Criteria:

• Be pregnant or currently lactating.
• Have diseases or suspected diseases which may cause pelvic pain not due to endometriosis.
• Have had any surgical treatment for endometriosis within the last 12 months.
• Have documented significant adenomyosis.
• Have participated in another clinical trial within the 30 days prior to the screening visit.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: PGL2001; PGL2001 + NETA followed by NETA-only follow-up period	Drug: PGL2001 + Primolut-Nor 5; PGL2001(estradiol sulfamate, E2MATE, ZK190628), Tablets, 1mg. 4 mg weekly for 4 weeks followed by 2mg weekly for 12 weeks. Norethisterone acetate (NETA), Tablets, 5mg. Once daily for 16 weeks plus during follow-up.
Placebo Comparator: Placebo; Placebo + NETA followed by NETA-only follow-up period	Drug: Placebo + Primolut-Nor 5; Placebo, Tablets.4 tablets weekly for 4 weeks followed by 2 tablets weekly for 12 weeks. Norethisterone acetate (NETA), Tablets, 5mg.Once daily for 16 weeks plus during follow-up.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Non-menstrual pelvic pain.	Use of a Visual Analogue Scale (VAS).	Daily collection up to 16 weeks

Collaborators and Investigators

• Sponsor: PregLem SA

Study record dates

Study Major Dates

• Study Start: June 1, 2012
• Primary Completion (Actual): October 1, 2013
• Study Completion (Actual): May 1, 2014

Study Registration Dates

• First Submitted: June 19, 2012
• First Submitted That Met QC Criteria: June 28, 2012
• First Posted (Estimate): June 29, 2012

Study Record Updates

• Last Update Posted (Estimate): June 3, 2014
• Last Update Submitted That Met QC Criteria: June 2, 2014
• Last Verified: June 1, 2014

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Endometriosis
• Other Study ID Numbers: PGL11-007