Assess Safety and Efficacy of Vilaprisan in Subjects With Endometriosis (VILLENDO)
Primary Purpose
Endometriosis
Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Vilaprisan (BAY1002670)
Matching Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Endometriosis
Eligibility Criteria
Inclusion Criteria:
- Signed and dated informed consent
- Pre-menopausal women 18 years (inclusive) and above at the time of Visit 1
- Women with endometriosis confirmed by laparoscopy or laparotomy OR diagnosed based on imaging
- Moderate to severe endometriosis-associated pelvic pain (EAPP)
- Adherence to screening period diary entries
- Willingness to use only standardized pain medication if needed
- Good general health (except for findings related to endometriosis)
- Normal or clinically insignificant cervical cytology not requiring further follow-up
- An endometrial biopsy performed at the screening phase without significant histological disorder
- Use of an acceptable non-hormonal method of contraception
- Willingness / ability to comply with electronic diary entry for the duration of study participation
Exclusion Criteria:
- Pregnancy or lactation (less than 3 months since delivery, abortion, or lactation before Visit 1)
- Hypersensitivity to any ingredient of the study treatments
- Laboratory values outside the inclusion range before randomization, and considered clinically relevant
- 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 elevated liver enzymes
- Any diseases or conditions that might interfere with the conduct of the study or the interpretation of the results
- Undiagnosed abnormal genital bleeding
- Abuse of alcohol, drugs, or medicines (e.g. laxatives) as evaluated by the investigator
- Use of other treatments that might interfere with the conduct of the study or the interpretation of the results
- Endometriosis-specific treatments for symptom relief except rescue pain medication according to protocol
- 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
- Inability to cooperate with the study procedures for any reason
- 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)
- Hypersensitivity to any ingredient of standardized pain medication
- Wish for pregnancy during the study
- Regular use of pain medication due to other underlying diseases
- Non-responsiveness of EAPP to GnRH-a (Gonadotropin-releasing hormone agonists)
Sites / Locations
- Office of Dr. James A. Simon, MD
- Helix Biomedics, LLC
- Solutions Through Advanced Research, Inc.
- Southern Clinical Research Associates LLC
- Unified Women's Clinical Research - Morehead City
- Unified Women's Clinical Research
- Kepler Universitätsklinikum Campus IV
- Medizinische Universität Graz
- KABEG Landeskrankenhaus Villach
- Universitätsklinikum AKH Wien
- Queen's University
- Ottawa Hospital-Riverside Campus
- Clinique OVO
- Gynekologie MEDA s.r.o.
- GynCare MUDr. Michael Svec s.r.o.
- VL-Medi Oy
- Satakunnan keskussairaala
- A.O.U.I. Verona
- Tokeidai Memorial Clinic
- Ishikawa Prefectural Central Hospital
- Japanese Red Cross Kumamoto Hospital
- Toyama Prefectural Central Hospital
- Centrum Medyczne Chodzki
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Placebo Comparator
Arm Label
Vilaprisan (BAY1002670) 2 mg
Vilaprisan (BAY1002670) 4 mg
Placebo group
Arm Description
Premenopausal women 18 years and older with endometriosis with randomized ratio = 1:1:1 Vilaprisan: 2 mg
Premenopausal women 18 years and older with endometriosis with randomized ratio = 1:1:1 Vilaprisan: 4 mg
Premenopausal women 18 years and older with endometriosis with randomized ratio = 1:1:1
Outcomes
Primary Outcome Measures
Mean Worst Pelvic Pain (Measured on a Numerical Rating Scale [NRS], Recorded in the Daily Endometriosis Symptom Diary [ESD])
Pain intensity was assessed on 11-point (0-10) NRS by ESD item 1. In ESD item 1, participants were asked to rate the worst pain in the target area during the past 24 hours, where 0= no pain and 10= worst imaginable pain and responses were recorded in ESD. Mean 'worst pelvic pain' was calculated as the sum of the participant's daily assessments of the ESD item 1 ("worst pain" during the last 24 hours) during a study period divided by number of days with pain assessment in that study period. This was summarized by study period. No inferential statistical analysis was performed.
Secondary Outcome Measures
Mean Worst Pelvic Pain (Measured on a Numerical Rating Scale [NRS], Recorded in the Daily Endometriosis Symptom Diary [ESD]) on Days With/Without Vaginal Bleeding
Pain intensity was assessed on 11-point (0-10) NRS by ESD item 1. In ESD item 1, participants were asked to rate the worst pain in the target area during the past 24 hours, where 0= no pain and 10= worst imaginable pain and responses were recorded in ESD. Mean 'worst pelvic pain' on bleeding/non-bleeding days was calculated as the sum of the participant's daily assessments of the ESD item 1 ("worst pain" during the last 24 hours) on bleedings/non-bleeding days during a study period divided by number of bleeding/non-bleeding days with pain assessment in that study period. This was summarized by study period. No inferential statistical analysis was performed.
Mean Number of Tablets of Rescue Pain Medication 1 (Ibuprofen 200 mg) Taken Daily for Endometriosis-associated Pelvic Pain (EAPP)
Mean number of tablets of rescue pain medication 1 (Ibuprofen 200 mg) taken daily for EAPP was calculated as the sum of the tablets taken for EAPP during a study period divided by the number of days in that study period. This was summarized by study period. No inferential statistical analysis was performed.
Mean Number of Tablets of Rescue Pain Medication 2 (Tramadol 50 mg) Taken Daily for Endometriosis-associated Pelvic Pain (EAPP)
Mean number of tablets of rescue pain medication 2 (Tramadol 50 mg) taken daily for EAPP was calculated as the sum of the tablets taken for EAPP during a study period divided by the number of days in that study period. This was summarized by study period. No inferential statistical analysis was performed.
The Number of Participants With Treatment Emergent Adverse Events (TEAEs)
An adverse event (AE) is any untoward medical occurrence (i.e. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a patient or clinical investigation subject after providing written informed consent for participation in the study. TEAE is defined as AE that is observed or reported after the first administration of study drug or if it starts before the first administration of study drug and the intensity/grade worsens on treatment) in this study.
Number of Participants With Clinical Significant Abnormal Endometrial Histology Findings
Number of participants with endometrial histology findings, e.g. hyperplasia, malignant neoplasm or endometrial polyps
Number of Participants With Clinical Significant Abnormal Ultrasound Examinations
Ultrasound examinations (evaluated for efficacy and safety) will be performed by a qualified expert in performing gynecologic ultrasound exams. If possible, the same examiner should conduct all examinations of a subject throughout the study and the same ultrasound machine (per site) should be used throughout the study. Preferably the safety evaluation should be performed by transvaginal ultrasound (TVU). However, if deemed appropriate, transabdominal or transrectal ultrasound examinations can be performed instead. The chosen method should be used consistently throughout the study.
Number of Participants With Clinical Significant Abnormal Bone Mineral Density Measurements
A Dual-energy X-ray absorptiometry (DEXA) scan of the lumbar spine (lumbar anterior-posterior, L1-L4) and the hip/femoral neck were performed.
Number of Participants With Clinical Significant Abnormal Laboratory Values
Clinical laboratory values including the values of hematology, general chemistry, urinalysis, coagulation, hormones, immunology and vitamins.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03573336
Brief Title
Assess Safety and Efficacy of Vilaprisan in Subjects With Endometriosis
Acronym
VILLENDO
Official Title
A Randomized, Double-blind, Parallel-group, Multicenter Phase 2b Study to Assess the Efficacy and Safety of Two Different Doses of Vilaprisan (BAY1002670) Versus Placebo in Women With Symptomatic Endometriosis
Study Type
Interventional
2. Study Status
Record Verification Date
April 2022
Overall Recruitment Status
Terminated
Why Stopped
Due to a change in the development program, the study was closed prematurely.
Study Start Date
July 4, 2018 (Actual)
Primary Completion Date
March 18, 2019 (Actual)
Study Completion Date
November 26, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bayer
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
The primary objective of this study was to assess the efficacy and safety of two doses of vilaprisan compared to placebo in women with symptomatic endometriosis.
The secondary objective of this study was to evaluate the safety and tolerability of two different doses of vilaprisan in women with symptomatic endometriosis.
With the implementation of protocol version 4.0 dated 11-Dec-2018, no new subjects were enrolled. The objectives above cannot be reached as only limited data is available from subjects recruited before the temporary pause.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Endometriosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
8 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Vilaprisan (BAY1002670) 2 mg
Arm Type
Experimental
Arm Description
Premenopausal women 18 years and older with endometriosis with randomized ratio = 1:1:1 Vilaprisan: 2 mg
Arm Title
Vilaprisan (BAY1002670) 4 mg
Arm Type
Experimental
Arm Description
Premenopausal women 18 years and older with endometriosis with randomized ratio = 1:1:1 Vilaprisan: 4 mg
Arm Title
Placebo group
Arm Type
Placebo Comparator
Arm Description
Premenopausal women 18 years and older with endometriosis with randomized ratio = 1:1:1
Intervention Type
Drug
Intervention Name(s)
Vilaprisan (BAY1002670)
Intervention Description
Intake orally, once daily
Intervention Type
Drug
Intervention Name(s)
Matching Placebo
Intervention Description
Intake orally, once daily
Primary Outcome Measure Information:
Title
Mean Worst Pelvic Pain (Measured on a Numerical Rating Scale [NRS], Recorded in the Daily Endometriosis Symptom Diary [ESD])
Description
Pain intensity was assessed on 11-point (0-10) NRS by ESD item 1. In ESD item 1, participants were asked to rate the worst pain in the target area during the past 24 hours, where 0= no pain and 10= worst imaginable pain and responses were recorded in ESD. Mean 'worst pelvic pain' was calculated as the sum of the participant's daily assessments of the ESD item 1 ("worst pain" during the last 24 hours) during a study period divided by number of days with pain assessment in that study period. This was summarized by study period. No inferential statistical analysis was performed.
Time Frame
Screening period (up to a maximum of 75 days) + treatment period (up to a maximum of 168 days)
Secondary Outcome Measure Information:
Title
Mean Worst Pelvic Pain (Measured on a Numerical Rating Scale [NRS], Recorded in the Daily Endometriosis Symptom Diary [ESD]) on Days With/Without Vaginal Bleeding
Description
Pain intensity was assessed on 11-point (0-10) NRS by ESD item 1. In ESD item 1, participants were asked to rate the worst pain in the target area during the past 24 hours, where 0= no pain and 10= worst imaginable pain and responses were recorded in ESD. Mean 'worst pelvic pain' on bleeding/non-bleeding days was calculated as the sum of the participant's daily assessments of the ESD item 1 ("worst pain" during the last 24 hours) on bleedings/non-bleeding days during a study period divided by number of bleeding/non-bleeding days with pain assessment in that study period. This was summarized by study period. No inferential statistical analysis was performed.
Time Frame
Screening period (up to a maximum of 75 days) + treatment period (up to a maximum of 168 days)
Title
Mean Number of Tablets of Rescue Pain Medication 1 (Ibuprofen 200 mg) Taken Daily for Endometriosis-associated Pelvic Pain (EAPP)
Description
Mean number of tablets of rescue pain medication 1 (Ibuprofen 200 mg) taken daily for EAPP was calculated as the sum of the tablets taken for EAPP during a study period divided by the number of days in that study period. This was summarized by study period. No inferential statistical analysis was performed.
Time Frame
Screening period (up to a maximum of 75 days) + treatment period (up to a maximum of 168 days)
Title
Mean Number of Tablets of Rescue Pain Medication 2 (Tramadol 50 mg) Taken Daily for Endometriosis-associated Pelvic Pain (EAPP)
Description
Mean number of tablets of rescue pain medication 2 (Tramadol 50 mg) taken daily for EAPP was calculated as the sum of the tablets taken for EAPP during a study period divided by the number of days in that study period. This was summarized by study period. No inferential statistical analysis was performed.
Time Frame
Screening period (up to a maximum of 75 days) + treatment period (up to a maximum of 168 days)
Title
The Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Description
An adverse event (AE) is any untoward medical occurrence (i.e. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a patient or clinical investigation subject after providing written informed consent for participation in the study. TEAE is defined as AE that is observed or reported after the first administration of study drug or if it starts before the first administration of study drug and the intensity/grade worsens on treatment) in this study.
Time Frame
Up to 6 months
Title
Number of Participants With Clinical Significant Abnormal Endometrial Histology Findings
Description
Number of participants with endometrial histology findings, e.g. hyperplasia, malignant neoplasm or endometrial polyps
Time Frame
Up to 6 months
Title
Number of Participants With Clinical Significant Abnormal Ultrasound Examinations
Description
Ultrasound examinations (evaluated for efficacy and safety) will be performed by a qualified expert in performing gynecologic ultrasound exams. If possible, the same examiner should conduct all examinations of a subject throughout the study and the same ultrasound machine (per site) should be used throughout the study. Preferably the safety evaluation should be performed by transvaginal ultrasound (TVU). However, if deemed appropriate, transabdominal or transrectal ultrasound examinations can be performed instead. The chosen method should be used consistently throughout the study.
Time Frame
Up to 6 months
Title
Number of Participants With Clinical Significant Abnormal Bone Mineral Density Measurements
Description
A Dual-energy X-ray absorptiometry (DEXA) scan of the lumbar spine (lumbar anterior-posterior, L1-L4) and the hip/femoral neck were performed.
Time Frame
Up to 6 months
Title
Number of Participants With Clinical Significant Abnormal Laboratory Values
Description
Clinical laboratory values including the values of hematology, general chemistry, urinalysis, coagulation, hormones, immunology and vitamins.
Time Frame
Up to 6 months
10. Eligibility
Sex
Female
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Signed and dated informed consent
Pre-menopausal women 18 years (inclusive) and above at the time of Visit 1
Women with endometriosis confirmed by laparoscopy or laparotomy OR diagnosed based on imaging
Moderate to severe endometriosis-associated pelvic pain (EAPP)
Adherence to screening period diary entries
Willingness to use only standardized pain medication if needed
Good general health (except for findings related to endometriosis)
Normal or clinically insignificant cervical cytology not requiring further follow-up
An endometrial biopsy performed at the screening phase without significant histological disorder
Use of an acceptable non-hormonal method of contraception
Willingness / ability to comply with electronic diary entry for the duration of study participation
Exclusion Criteria:
Pregnancy or lactation (less than 3 months since delivery, abortion, or lactation before Visit 1)
Hypersensitivity to any ingredient of the study treatments
Laboratory values outside the inclusion range before randomization, and considered clinically relevant
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 elevated liver enzymes
Any diseases or conditions that might interfere with the conduct of the study or the interpretation of the results
Undiagnosed abnormal genital bleeding
Abuse of alcohol, drugs, or medicines (e.g. laxatives) as evaluated by the investigator
Use of other treatments that might interfere with the conduct of the study or the interpretation of the results
Endometriosis-specific treatments for symptom relief except rescue pain medication according to protocol
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
Inability to cooperate with the study procedures for any reason
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)
Hypersensitivity to any ingredient of standardized pain medication
Wish for pregnancy during the study
Regular use of pain medication due to other underlying diseases
Non-responsiveness of EAPP to GnRH-a (Gonadotropin-releasing hormone agonists)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Bayer Study Director
Organizational Affiliation
Bayer
Official's Role
Study Director
Facility Information:
Facility Name
Office of Dr. James A. Simon, MD
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20036
Country
United States
Facility Name
Helix Biomedics, LLC
City
Boynton Beach
State/Province
Florida
ZIP/Postal Code
33435
Country
United States
Facility Name
Solutions Through Advanced Research, Inc.
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32256
Country
United States
Facility Name
Southern Clinical Research Associates LLC
City
Metairie
State/Province
Louisiana
ZIP/Postal Code
70001
Country
United States
Facility Name
Unified Women's Clinical Research - Morehead City
City
Morehead City
State/Province
North Carolina
ZIP/Postal Code
28557
Country
United States
Facility Name
Unified Women's Clinical Research
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27103
Country
United States
Facility Name
Kepler Universitätsklinikum Campus IV
City
Linz
State/Province
Oberösterreich
ZIP/Postal Code
4020
Country
Austria
Facility Name
Medizinische Universität Graz
City
Graz
State/Province
Steiermark
ZIP/Postal Code
8036
Country
Austria
Facility Name
KABEG Landeskrankenhaus Villach
City
Villach
ZIP/Postal Code
9500
Country
Austria
Facility Name
Universitätsklinikum AKH Wien
City
Wien
ZIP/Postal Code
1090
Country
Austria
Facility Name
Queen's University
City
Kingston
State/Province
Ontario
ZIP/Postal Code
K7L 2V7
Country
Canada
Facility Name
Ottawa Hospital-Riverside Campus
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H 7W9
Country
Canada
Facility Name
Clinique OVO
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H4P 2S4
Country
Canada
Facility Name
Gynekologie MEDA s.r.o.
City
Brno
ZIP/Postal Code
602 00
Country
Czechia
Facility Name
GynCare MUDr. Michael Svec s.r.o.
City
Plzen
ZIP/Postal Code
326 00
Country
Czechia
Facility Name
VL-Medi Oy
City
Helsinki
ZIP/Postal Code
00510
Country
Finland
Facility Name
Satakunnan keskussairaala
City
Pori
ZIP/Postal Code
28500
Country
Finland
Facility Name
A.O.U.I. Verona
City
Verona
State/Province
Veneto
ZIP/Postal Code
37126
Country
Italy
Facility Name
Tokeidai Memorial Clinic
City
Sapporo
State/Province
Hokkaido
ZIP/Postal Code
060-0031
Country
Japan
Facility Name
Ishikawa Prefectural Central Hospital
City
Kanazawa
State/Province
Ishikawa
ZIP/Postal Code
920-8530
Country
Japan
Facility Name
Japanese Red Cross Kumamoto Hospital
City
Kumamoto
ZIP/Postal Code
861-8520
Country
Japan
Facility Name
Toyama Prefectural Central Hospital
City
Toyama
ZIP/Postal Code
930-8550
Country
Japan
Facility Name
Centrum Medyczne Chodzki
City
Lublin
ZIP/Postal Code
20-093
Country
Poland
12. IPD Sharing Statement
Plan to Share IPD
No
IPD Sharing Plan Description
Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.clinicalstudydatarequest.com to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the Study sponsors section of the portal.
Links:
URL
https://clinicaltrials.bayer.com/
Description
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URL
https://www.clinicaltrialsregister.eu/
Description
Click here to find information about studies related to Bayer Healthcare products conducted in Europe
Learn more about this trial
Assess Safety and Efficacy of Vilaprisan in Subjects With Endometriosis
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