PGL2001 Proof of Concept Study in Symptomatic Endometriosis

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Endometriosis

Intervention / Treatment
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* Drug: PGL2001 + Primolut-Nor 5
* Drug: Placebo + Primolut-Nor 5


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 45 Years

Sexes Eligible for Study
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Female

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: PGL2001<br>PGL2001 + NETA followed by NETA-only follow-up period | Drug: PGL2001 + Primolut-Nor 5<br>* 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.<br>|
| Placebo Comparator: Placebo<br>Placebo + NETA followed by NETA-only follow-up period | Drug: Placebo + Primolut-Nor 5<br>* 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.<br>|

What is the study measuring?
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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 |