Assess Safety and Efficacy of Vilaprisan in Subjects With Uterine Fibroids

Study Overview
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Brief Summary
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The primary objective of this study is to describe the efficacy of vilaprisan in subjects with uterine fibroids compared to ulipristal. The secondary objective of this study is to evaluate the efficacy and safety of different treatment regimens of vilaprisan in subjects with uterine fibroids.

Official Title
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A Randomized, Parallel-group, Multicenter Study to Assess the Efficacy and Safety of Vilaprisan in Subjects With Uterine Fibroids

Conditions
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Uterine Fibroids

Intervention / Treatment
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* Drug: Vilaprisan (BAY1002670)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Women, 18 years or older at the time of Visit 1 Diagnosis of uterine fibroid(s) documented by ultrasound at screening with at least 1 fibroid with largest diameter more than 30 mm and less than 120 mm Heavy menstrual bleeding (HMB) >80.00 mL documented by menstrual pictogram (MP) in a bleeding episode period during the screening period Use of an acceptable non-hormonal method of contraception An endometrial biopsy performed during the screening period, without significant histological disorder such as endometrial hyperplasia (including simple hyperplasia) or other significant endometrial pathology Exclusion Criteria: Pregnancy or lactation (less than 3 months since delivery, abortion, or lactation before start of treatment) Hypersensitivity to any ingredient of the study drugs Hemoglobin values ≤6 g/dL or any condition requiring immediate blood transfusion (subjects with hemoglobin values ≤10.9 g/dL will be recommended to use iron supplementation) Any diseases, conditions, or medications that can compromise the function of the body systems and could result in altered absorption, excessive accumulation, impaired metabolism, or altered excretion of the study drug including Abuse of alcohol, drugs, or medicines (eg: laxatives) Undiagnosed abnormal genital bleeding Any diseases or conditions that might interfere with the conduct of the study or the interpretation of the results

Ages Eligible for Study
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Minimum Age: 18 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: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Group A1: Vilaprisan (3/1 regimen)<br>Orally, 2 mg, once daily, 3 months treatment followed by 1 menstrual bleeding episode | Drug: Vilaprisan (BAY1002670)<br>* Orally, 2 mg, once daily<br>|
| Experimental: Group A2: Vilaprisan (6/2 regimen)<br>Orally, 2 mg, once daily, 6 months treatment followed by 2 menstrual bleeding episodes | Drug: Vilaprisan (BAY1002670)<br>* Orally, 2 mg, once daily<br>|
| Experimental: Group A3/B (3/2 regimen)<br>Orally, 2 mg, once daily, 3 months treatment followed by 2 menstrual bleeding episodes With protocol version 5.0 the blinded study arms A3 and B were converted to one open-label study arm called A3/B. | Drug: Vilaprisan (BAY1002670)<br>* Orally, 2 mg, once daily<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Amenorrhea (yes/no) | Defined as menstrual blood loss (MBL) < 2 mL based on the menstrual pictogram (MP) during last 28 days. For the primary analysis of the primary variable, the amenorrhea rates after 12 weeks of treatment in Groups A1, A2 and A3 will be compared to the rate from Group B. | At 3 months (at the end of treatment perid 1) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Total volume of menstrual blood loss | Assessed by menstrual pictogram (MP). Volume of menstrual blood loss will be normalized by 28 days. | After approximately 1 year in subgroup 1 and after approximately 2 years in subgroup 2 | 
| Number of bleeding days | From day 1 of the first treatment period until the day before the next treatment period after the last treatment period would start again normalized to 28 days. | After approximately 1 year in subgroup 1 and after approximately 2 years in subgroup 2 | 
| Amenorrhea (yes/no) | Defined as menstrual blood loss (MBL) < 2 mL based on the menstrual pictogram (MP) during last 28 days of the treatment period. | At 6 months, at 12 months, at 15 months, at 18 months, at 21 months and at 24 months | 
| Absence of bleeding (spotting allowed) | Absence of bleeding defined as no bleeding (spotting allowed) during the last 28 days of the treatment; based on the UF-DBD (Uterine Fibroid Daily Symptom Diary). | Up to 24 months | 
| Time to onset of controlled bleeding | Onset of controlled bleeding is defined by the first day, for which the menstrual blood loss (assessed by MP) for all subsequent 28-day periods up to the end of the treatment period is less than 80.00 mL. | Quarterly up to 24 months | 
| HMB (Heavy Menstrual Bleeding) responder rate | Percentage of subjects with blood loss < 80.00 mL per 28 days and 50% reduction compared to baseline [assessed by MP] | By treatment period up to 24 months | 
| Percent change in volume of largest fibroid compared to baseline | Measured by MRI (magnetic resonance imaging). | At baseline, at 12 months and at 24 months | 
| Endometrial histology | E.g., benign endometrium, presence or absence of hyperplasia or malignancy | Up to 24 months | 
| Endometrial thickness |  | Up to 24 months |