E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
Uterine Fibroids, heavy menstrual bleeding |
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E.1.1.2 | Therapeutic area | Diseases [C] - Female diseases of the urinary and reproductive systems and pregancy complications [C13] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10016628 |
E.1.2 | Term | Fibroids |
E.1.2 | System Organ Class | 100000004864 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10046784 |
E.1.2 | Term | Uterine fibroids |
E.1.2 | System Organ Class | 100000004864 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10022794 |
E.1.2 | Term | Intramural leiomyoma of uterus |
E.1.2 | System Organ Class | 100000004864 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective of this study is to show superiority of vilaprisan in the treatment of heavy menstrual bleeding (HMB) in subjects with uterine fibroids compared to placebo |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives of this study are to additionally evaluate the efficacy and safety of vilaprisan in subjects with uterine fibroids |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Women, 18 years or older in good General health 2. 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 3. Heavy menstrual bleeding (HMB) in at least 2 bleeding periods during the Screening period each with blood loss volume of >80.00 mL documented by alkaline hematin (AH) method 4. An endometrial biopsy performed during the Screening period without significant histological disorder such as endometrial hyperplasia (including simple hyperplasia) or other significant endometrial pathology 5. Use of an acceptable non-hormonal method of contraception (ie, either male condom, cap, diaphragm or sponge, each in combination with spermicide) starting at Visit 1 until the end of the study |
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E.4 | Principal exclusion criteria |
1. Pregnancy or lactation (less than 3 month since delivery, abortion, or lactation before start of Treatment) 2. Hypersensitivity to any ingredient of the study drug 3. Any condition requiring immediate blood transfusion 4. Laboratory values outside inclusion range before randomization and considered as clinically relevant. 5. 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 6. Any diseases or conditions that might interfere with the conduct of the study or the interpretation of the results 7. Abuse of alcohol, drugs, or medicines (eg, laxatives) 8. Use of other treatments that might interfere with the conduct of the study or the interpretation of the results 9. Undiagnosed abnormal genital bleeding |
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E.5 End points |
E.5.1 | Primary end point(s) |
Amenorrhea (yes/no), defined as menstrual blood loss (MBL) <2 mL during the last 28 days of treatment |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
The last 28 days of treatment period 1 and treatment period 2 |
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E.5.2 | Secondary end point(s) |
1. HMB response defined as blood loss <80.00 mL during the last 28 days of treatment and >50% reduction compared to baseline 2. Time to onset of amenorrhea. Onset of amenorrhea is defined by the first day for which the menstrual blood loss for all subsequent 28-day periods up to the end of the treatment period is <2 mL 3. Time to onset of controlled bleeding. Onset of controlled bleeding is defined by the first day for which the menstrual blood loss for all subsequent 28-day periods up to the end of the treatment period is <80.00 mL 4. Absence of bleeding (spotting allowed) during the last 28 days of the treatment; based on the UF-DBD 5. Endometrial histology (e.g. benign endometrium, presence or absence of hyperplasia or malignancy) 6. Endometrial thickness |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. The last 28 days of treatment period 1 and treatment period 2 2. In treatment period 1and in treatment period 2 3. In treatment period 1 and in treatment period 2 4. The last 28 days of treatment period 1 and treatment period 2 5. Up to 36 weeks 6. Up to 36 weeks |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 4 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 8 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Bulgaria |
China |
Czech Republic |
Israel |
Malaysia |
New Zealand |
Singapore |
South Africa |
United States |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | 5 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 4 |
E.8.9.2 | In all countries concerned by the trial days | 5 |