E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pre-menopausal women 18 years and older with confirmed endometriosis and moderate to severe endometriosis-associated pelvic pain (EAPP), otherwise in good general health. |
|
E.1.1.1 | Medical condition in easily understood language |
Women, 18 years or older with symptomatic endometriosis |
|
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 | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10014778 |
E.1.2 | Term | Endometriosis |
E.1.2 | System Organ Class | 10038604 - Reproductive system and breast disorders |
|
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 assess the efficacy and safety of two doses of vilaprisan compared to placebo in women with symptomatic endometriosis |
|
E.2.2 | Secondary objectives of the trial |
The secondary objective of this study is to evaluate the safety and tolerability of two different doses of vilaprisan in women with symptomatic endometriosis |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Signed and dated informed consent
2. Pre-menopausal women 18 years (inclusive) and above at the time of Visit 1
3. Women with endometriosis confirmed by laparoscopy or laparotomy OR diagnosed based on imaging
4. Moderate to severe endometriosis-associated pelvic pain (EAPP)
5. Adherence to screening period diary entries
6. Willingness to use only standardized pain medication if needed
7. Good general health (except for findings related to endometriosis)
8. Normal or clinically insignificant cervical cytology not requiring further follow-up
9. An endometrial biopsy performed at the screening phase without significant histological disorder
10. Use of an acceptable non-hormonal method of contraception
11. Willingness / ability to comply with electronic diary entry for the duration of study participation
|
|
E.4 | Principal exclusion criteria |
1. Pregnancy or lactation (less than 3 months since delivery, abortion, or lactation before Visit 1)
2. Hypersensitivity to any ingredient of the study treatments
3. Laboratory values outside the inclusion range before randomization, and considered clinically relevant
4. Any diseases or conditions 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 abnormal liver parameters
5. Any diseases or conditions that might interfere with the conduct of the study or the interpretation of the results
6. Undiagnosed abnormal genital bleeding
7. Abuse of alcohol, drugs, or medicines (e.g. laxatives) as evaluated by the investigator
8. Use of other treatments that might interfere with the conduct of the study or the interpretation of the results
9. Endometriosis-specific treatments for symptom relief except rescue pain medication according to protocol
10. Simultaneous participation in another clinical trial with investigational medicinal product(s). Participation in another trial prior to study entry that might have an impact on the study objectives, at the discretion of the investigator
11. Inability to cooperate with the study procedures for any reason
12. Previous assignment to treatment (e.g. randomization) during this study (allowing previously randomized subjects to be re-included into the study may lead to bias)
13. Hypersensitivity to any ingredient of standardized pain medication
14. Wish for pregnancy during the study
15. Regular use of pain medication due to other underlying diseases
16. Non-responsiveness of EAPP to GnRH-a
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
Change in the subject’s 7-day mean ‘worst pain’ from baseline to Month 3 of the first 24-week treatment period measured on a daily NRS.
|
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
The proportion of subjects with an improvement on dysmenorrhea AND the proportion of subjects with an improvement on non-menstrual pelvic pain, from baseline and no increase in rescue pain medication use.
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
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 | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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 | 3 |
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 | 6 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 27 |
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 |
Austria |
Bulgaria |
Canada |
China |
Czech Republic |
Denmark |
Finland |
Germany |
Hungary |
Italy |
Japan |
Poland |
Portugal |
Spain |
Sweden |
Switzerland |
Taiwan |
United States |
|
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
|
|
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 | 7 |
E.8.9.1 | In the Member State concerned days | 9 |
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 | 8 |
E.8.9.2 | In all countries concerned by the trial days | 26 |