Assess Safety and Efficacy of Vilaprisan in Subjects With Uterine Fibroids

Study Overview
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Brief Summary
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The study was performed to assess the efficacy and safety of Vilaprisan in subjects with uterine fibroids compared to standard of care

Official Title
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An Open-label, Parallel-group, Randomized, Multicenter Study to Assess the Safety and Efficacy of Vilaprisan in Subjects With Uterine Fibroids Versus Standard of Care

Conditions
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Uterine Fibroids

Intervention / Treatment
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* Drug: Vilaprisan (BAY1002670)
* Drug: Vilaprisan (BAY1002670)
* Drug: Vilaprisan (BAY1002670)
* Other: Standard of care
* Drug: Vilaprisan (BAY1002670)
* Drug: Vilaprisan (BAY1002670)
* Drug: Vilaprisan (BAY1002670)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 18 years or older Diagnosis of uterine fibroid(s) documented by ultrasound at screening AND/OR during a uterine preserving procedure within 3 months prior to screening in subjects with high risk for recurrence At least one symptom of uterine fibroid(s) - bleeding, pelvic pressure/pain Good general health Normal or clinically insignificant cervical smear An endometrial biopsy performed during the screening period, without significant histological disorder Use of an acceptable nonhormonal method of contraception starting at Visit 1 until the end of the study 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 drug Any condition requiring immediate blood transfusion 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 Any diseases or conditions that might interfere with the conduct of the study or the interpretation of the results Abuse of alcohol, drugs, or medicines (eg, laxatives) Use of other treatments that might interfere with the conduct of the study or the interpretation of the results Undiagnosed abnormal genital bleeding

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: A1(3/1 regimen)<br>2.0 mg treatment of 12 weeks, each separated by 1 bleeding break | Drug: Vilaprisan (BAY1002670)<br>* 2 mg of Vilaprisan once daily for 12 weeks, 4 treatment periods of 12 weeks, each separated by 1 bleeding episode (3/1 regimen).<br>Drug: Vilaprisan (BAY1002670)<br>* 2 mg of Vilaprisan once daily for 12 weeks, 8 treatment periods of 12 weeks, each separated by 1 bleeding episode (3/1 regimen)<br>|
| Experimental: A2(6/2 regimen)<br>2.0 mg treatment period of 24 weeks, each separated by 2 bleeding break | Drug: Vilaprisan (BAY1002670)<br>* 2 mg of Vilaprisan once daily for 24 weeks, 2 treatment periods of 24 weeks, each separated by 2 bleeding episodes (6/2 regimen).<br>Drug: Vilaprisan (BAY1002670)<br>* 2 mg of Vilaprisan once daily for 24 weeks, 4 treatment periods of 24 weeks, each separated by 2 bleeding episodes (6/2 regimen)<br>|
| Experimental: A3(3/2 regimen)<br>2.0 mg treatment period of 12 weeks, each separated by 2 bleeding break | Drug: Vilaprisan (BAY1002670)<br>* 2 mg of Vilaprisan once daily for 12 weeks, 4 treatment periods of 12 weeks, each separated by 2 bleeding episodes (3/2 regimen).<br>Drug: Vilaprisan (BAY1002670)<br>* 2 mg of Vilaprisan once daily for 12 weeks, 6 treatment periods of 12 weeks, each separated by 2 bleeding episodes (3/2 regimen)<br>|
| Other: B(Standard of care)<br>Standard of care as determined by the investigators, this could be watch & wait or non-hormonal medical treatment | Other: Standard of care<br>* Standard of care as determined by the investigators (including watch and wait, symptomatic nonhormonal)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage Change in Bone Mineral Density (BMD) of Lumbar Spine | The percentage change in BMD (measured by dual-energy X-ray absorptiometry (DEXA) scan) of lumbar spine from baseline to about one year after start of treatment (SoT) in all randomized and treated participants with measurements at those 2 time points in each treatment group. | From baseline to about 1 year after start of treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Bleeding Days | Number of bleeding days were defined from Day 1 of the first treatment period until the day before a new treatment period would start again following the last treatment period for that respective treatment group. Number to be normalized by 28 days | Treatment phase: approximately 1 year | 
| Number of Participants With Endometrial Histology Findings by Endometrial Biopsy Main Results (Majority Read, Main Diagnosis) | Number of participants with endometrial histology findings, e.g. benign endometrium, presence or absence of hyperplasia or malignancy | Up to 3 years (from study treatment start to end of study) | 
| Change From Baseline in Endometrial Thickness | Ultrasound examinations were performed. Endometrial thickness was measured in the medio-sagittal section as double-layer in millimeters. Summary statistics for change from baseline (worst measurement during baseline period) in endometrial thickness was provided in below table. | Treatment phase: approximately 1 year, follow-up phase: up to 2 years | 
| Percentage Change From Baseline in BMD Measured at Lumbar Spine (Other Time Points Not Mentioned as Primary Safety Variable) and Hip/Femoral Neck | Percentage change in BMD (measured by dual-energy X-ray absorptiometry (DEXA) scan) of lumbar spine (other time points not mentioned as primary safety analysis), hip, and femoral neck from baseline was analyzed using the same statistical methods as used for the primary variable. | Treatment phase: approximately 1 year, follow-up phase: up to 2 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Uterine Fibroid