A Study of Efficacy and Safety of Sepranolone (UC1010) in Patients With Premenstrual Dysphoric Disorder (PMDD)

Study Overview
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Brief Summary
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The objective of this phase IIb study is to evaluate the effect and safety of Sepranolone (UC1010) on premenstrual symptoms in women with Premenstrual Dysphoric Disorder (PMDD). Patients will be taking Sepranolone or Placebo (blinded to patient and study doctor) during the two weeks preceding the menstruation of three menstrual cycles. Effect (change from baseline) will be assessed by comparison of symptoms recorded daily by the patients using a validated rating scale for assessment of PMDD symptoms. Sepranolone is an endogenous steroid.

Detailed Description
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The objective of this phase IIb study is to evaluate the effect and safety of two doses of Sepranolone (UC1010) on premenstrual symptoms in women with Premenstrual Dysphoric Disorder (PMDD) in comparison to placebo, administrated during the luteal phase of three consecutive menstrual cycles. Effect will be assessed by comparison of symptoms recorded daily by the patients using a validated rating scale for assessment of PMDD symptoms and impairment of symptoms on daily life of the patients. The scale is Daily Record of Severity of Problems (DRSP). Preceding the treatment period, the diagnosis of PMDD will be established in the study participants by at lest two menstrual cycles of prospective DRSP ratings by the patients. This period will also constitute the baseline data. The effect of study medication will be assessed as the change in symptoms from baseline to during treatment. The Total symptom score of the DRSP will constitute the primary variable and the late luteal phase ratings in the respective menstrual cycles, before and during treatment, will be used. The study will also include a follow-up (no treatment) cycle before patients final visit. The study is conducted in four European countries (Sweden, Germany, Poland and United Kingdom) and will include up to 225 patients.

Official Title
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A Phase II, Randomised, Double-blind, Placebo-controlled, Parallel Group, Multicentre Study Investigating Efficacy and Safety of Sepranolone (UC1010) in Patients With Premenstrual Dysphoric Disorder (PMDD)

Conditions
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Premenstrual Dysphoric Disorder

Intervention / Treatment
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* Drug: Sepranolone (UC1010) low dose
* Drug: Sepranolone (UC1010) high dose
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Main Inclusion Criteria: have PMDD according to DSM-5 verified in two menstrual cycles have a regular menstrual cycle of 24-35 days cycle, use double barrier contraception, non-hormonal IUD, be truly sexually abstinent, or subject or her partner has been surgically sterilized, Main Exclusion Criteria: steroid hormonal treatment or treatment with psychopharmaceuticals during previous three months treatments for premenstrual syndrome (PMS) or PMDD history of or significant medical condition ongoing be pregnant or plan a pregnancy within the study period

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: Sepranolone (UC1010) low dose<br>Sepranolone (UC1010) low dose administered subcutaneously (SC) during the luteal phase | Drug: Sepranolone (UC1010) low dose<br>* Subcutaneous (SC) administration<br>|
| Experimental: Sepranolone (UC1010) high dose<br>Sepranolone (UC1010) high dose administered subcutaneously (SC) during the luteal phase | Drug: Sepranolone (UC1010) high dose<br>* Subcutaneous (SC) administration<br>|
| Placebo Comparator: Placebo<br>Placebo administered subcutaneously (SC) during the luteal phase | Drug: Placebo<br>* Subcutaneous (SC) adminstration<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Effect on DRSP Total symptom score | Change in DRSP Total symptom score (Daily Record of Severity of Problems) measured during the luteal phase (i.e. the week before menstruation) before and during treatment | Baseline luteal phase score (average from two menstrual cycles) vs. luteal phase score during treatment. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Treatment-Emergent Adverse Events | Recording of adverse events | Starting from first dose given until 1 months after treatment stop |