Neoplasms

All Conditions

Skin and Connective Tissue Diseases

Leiomyoma

Myofibroma

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Neoplasms, Connective Tissue

Connective Tissue Diseases

2.0 mg treatment of 12 weeks, each separated by 1 bleeding break

2.0 mg treatment period of 24 weeks, each separated by 2 bleeding break

2.0 mg treatment period of 12 weeks, each separated by 2 bleeding break

Standard of care as determined by the investigators, this could be watch \& wait or non-hormonal medical treatment

2 mg of Vilaprisan once daily for 12 weeks, 4 treatment periods of 12 weeks, each separated by 1 bleeding episode (3/1 regimen).

Vilaprisan (BAY1002670)

2 mg of Vilaprisan once daily for 24 weeks, 2 treatment periods of 24 weeks, each separated by 2 bleeding episodes (6/2 regimen).

2 mg of Vilaprisan once daily for 12 weeks, 4 treatment periods of 12 weeks, each separated by 2 bleeding episodes (3/2 regimen).

Standard of care as determined by the investigators (including watch and wait, symptomatic nonhormonal)

Standard of care

2 mg of Vilaprisan once daily for 12 weeks, 8 treatment periods of 12 weeks, each separated by 1 bleeding episode (3/1 regimen)

2 mg of Vilaprisan once daily for 24 weeks, 4 treatment periods of 24 weeks, each separated by 2 bleeding episodes (6/2 regimen)

2 mg of Vilaprisan once daily for 12 weeks, 6 treatment periods of 12 weeks, each separated by 2 bleeding episodes (3/2 regimen)

Bayer Study Director

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

Assess Safety and Efficacy of Vilaprisan in Subjects With Uterine Fibroids

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

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.vivli.org 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 member section of the portal.

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.

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

Number of participants with endometrial histology findings, e.g. benign endometrium, presence or absence of hyperplasia or malignancy

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.

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.

Bayer

4 treatment periods of 12 weeks, each separated by 1 bleeding episode (3/1 regimen)

Vilaprisan 2 mg A1 (3/1 Regimen) - Treatment Emergent AEs

2 treatment periods of 24 weeks, separated by 2 bleeding episodes

Vilaprisan 2 mg A2 (6/2 Regimen) - Treatment Emergent AEs

3 treatment periods of 12 weeks, each separated by 2 bleeding episodes

Vilaprisan 2 mg A3 (3/2 Regimen) - Treatment Emergent AEs

Standard of care symptomatic nonhormonal medical treatment as determined by the investigators and/or watch and wait

Standard of Care B - Treatment Emergent AEs

Vilaprisan 2 mg A1 (3/1 Regimen) - Post Treatment AEs

Vilaprisan 2 mg A2 (6/2 Regimen) - Post Treatment AEs

Vilaprisan 2 mg A3 (3/2 Regimen) - Post Treatment AEs

Standard of Care B - Post Treatment AEs

4 treatment periods of 12 weeks, each separated by 1 bleeding episode

Vilaprisan 2 mg A1 (3/1 Regimen)

Vilaprisan 2 mg A2 (6/2 Regimen)

Vilaprisan 2 mg A3 (3/2 Regimen)

B (Standard of Care)

Total

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

Lumbar spine

Femoral neck

The baseline data of BMD measured by dual-energy X-ray absorptiometry (DEXA) scan for lumbar spine, hip and femoral neck is provided in below table.

Safety analysis set (SAF) was analyzed. SAF: All participants randomized to vilaprisan treatment groups who took at least 1 dose of study drug and all participants randomized to group B (standard of care) treatment group were included in the SAF. Participants were analyzed as treated. Some data in baseline was missing.

Baseline bone mineral density (BMD) of lumbar spine, hip and femoral neck

Ultrasound examinations were performed. Endometrial thickness was measured in the medio-sagittal section as double-layer in millimeters. Baseline data (Last non-missing value before reference start date) of endometrial thickness is provided in below table.

Safety analysis set (SAF) was analyzed. SAF: All participants randomized to vilaprisan treatment groups who took at least 1 dose of study drug and all participants randomized to group B (standard of care) treatment group were included in the SAF. Participants were analyzed as treated. Some data in baseline were missing.

Endometrial thickness

Participants in Full analysis set (FAS) was analyzed. The FAS consisted of all randomized subjects, excluding randomized subjects who did not start treatment Period 1 (never received study drug) due to the study being temporarily paused, including 1264 participants.

Only two patients are in safety follow up due to local requirement.

Therapeutic Area Head

Safety analysis set (SAF) was analyzed. SAF: All participants randomized to vilaprisan treatment groups who took at least 1 dose of study drug and all participants randomized to group B (standard of care) treatment group were included in the SAF. Participants were analyzed as treated. Overall number of participants analyzed represents number of participants without missing data.

Percentage Change in Bone Mineral Density (BMD) of Lumbar Spine

Number of Bleeding Days

Adequate endometrial tissue

Benign Endometrium

Hyperplasia WHO 2014, no atypia

Hyperplasia WHO 2014, atypia

Malignant Neoplasm

Endometrial Polyps

Number of Participants With Endometrial Histology Findings by Endometrial Biopsy Main Results (Majority Read, Main Diagnosis)

Baseline

Treatment phase (change from baseline)

Follow up phase (change from baseline)

Change From Baseline in Endometrial Thickness

Lumbar spine - Month 6 on treatment

Lumbar spine - End of treatment

Lumbar spine - Month 6 post treatment

Lumbar spine - Month 12 post treatment

Lumbar spine - End of follow up

Hip - Month 6 on treatment

Hip - Month 12 on treatment

Hip - End of treatment

Hip - Month 6 post treatment

Hip - Month 12 post treatment

Hip - End of follow up

Femoral neck - Month 6 on treatment

Femoral neck - Month 12 on treatment

Femoral neck - End of treatment

Femoral neck - Month 6 post treatment

Femoral neck - Month 12 post treatment

Femoral neck - End of follow up

SAF was analyzed. Percentage change from baseline in BMD of lumbar spine, hip and femoral neck was presented by time interval in below table.

Percentage Change From Baseline in BMD Measured at Lumbar Spine (Other Time Points Not Mentioned as Primary Safety Variable) and Hip/Femoral Neck

Overall Study

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Renal Insufficiency

Endometriosis

Genital Diseases, Female

Genital Diseases

Urogenital Diseases

Female Urogenital Diseases

Pregnancy Complications

Female Urogenital Diseases and Pregnancy Complications

Subjects with moderate renal impairment with an estimated glomerular filtration rate (eGFR) of 30 to 59 mL/min/1.73 m\*2 according to the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula.

Subjects with severe renal impairment not on dialysis with an eGFR \<30 mL/min/1.73 m\*2 (CKD-EPI formula).

Subjects with an eGFR ≥90 mL/min/1.73 m\*2 (CKD-EPI formula) who are matched based on sex, age, race and weight.

Single oral dose (1 x 2 mg immediate-release, film-coated tablet)

The purpose of the study is to evaluate the pharmacokinetics of vilaprisan in subjects with moderate to severe renal impairment compared with matched subjects with normal renal function.

This is a multiple-center, open-label, non-randomized, single-dose study in 3 parallel groups of subjects with moderately or severely impaired renal function or normal renal function matched with regard to sex, age, race and weight. PK blood and urine sampling for determination of vilaprisan concentrations in plasma and urine, respectively, will be preformed at pre-defined time points up to 14 days post-dose. Safety and tolerability will be assessed through adverse events, clinical laboratory tests, vital signs, 12-lead electrocardiograms and physical examinations.

Pharmacokinetics and Safety of Vilaprisan in Renal Impairment

An Open-label, Single-dose Study to Evaluate the Pharmacokinetics and Safety of Vilaprisan in Subjects With Decreased Renal Function in Comparison With Matched Subjects With Normal Renal Function

Area under the concentration versus time curve from zero to the last data point above the lower limit of quantitation \[AUC(0-tlast)\], if AUC cannot be estimated in all subjects.

In subjects with normal and moderately reduced renal function.

In subjects with normal and moderately reduced renal function.

In subjects with normal, moderately, and severely reduced renal function.

2 treatment periods of 12 weeks without a break

Placebo

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.The secondary objectives of this study are to additionally evaluate the efficacy and safety of vilaprisan in subjects with uterine fibroids.

Assessment of Safety and Efficacy of Vilaprisan in Subjects With Uterine Fibroids

A Randomized, Parallel-group, Double-blind Placebo-controlled, Multicenter Study to Assess the Efficacy and Safety of Vilaprisan in Subjects With Uterine Fibroids

Defined as menstrual blood loss (MBL) \<2 mL (based on menstrual pictogram)

Defined as blood loss \<80.00 mL during the last 28 days of treatment and \>50% reduction compared to baseline

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 \<2mL.

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.

Symptoms and General Pathology

Hemorrhage

Menorrhagia

Uterine Hemorrhage

Uterine Diseases

Menstruation Disturbances

To evaluate the safety and efficacy of vilaprisan in Japanese subjects with uterine fibroids and heavy menstrual bleeding (HMB).

A Study to Assess the Safety and Efficacy of Vilaprisan in Japanese Subjects With Uterine Fibroids and Heavy Menstrual Bleeding

An Open-label, Parallel-group, Randomized, Multicenter Study to Assess the Safety and Efficacy of Vilaprisan in Japanese Subjects With Uterine Fibroids and Heavy Menstrual Bleeding

Availability of this study's data will be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, time point 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.

Vilaprisan (2 mg) in treatment period 1 for 12 weeks and in treatment period 2 for 12 weeks, separated by 1 bleeding episode.

Vilaprisan (2 mg) in treatment period 1 for 12 weeks and in treatment period 2 for 12 weeks without a break.

Placebo in treatment period 1 for 12 weeks, and vilaprisan (2 mg) in treatment period 2 for 12 weeks, separated by 1 bleeding episode.

Vilaprisan (2 mg) in treatment period 1 for 12 weeks and placebo in treatment period 2 for 12 weeks, separated by 1 bleeding episode.

2 mg of Vilaprisan once daily up to 2 x 12 weeks

Matching placebo was administered to group B1 and B2.

The primary objective of this study was to show superiority in the treatment of HMB of vilaprisan in subjects with uterine fibroids compared to placebo.

The secondary objectives of this study were to additionally evaluate the efficacy and safety of vilaprisan in subjects with uterine fibroids.

Blinding will be applied to Treatment Groups A1, B1, and B2; Treatment Group A2 will be open-label

Assess Safety and Efficacy of Vilaprisan in Subjects With Uterine Fibroids (ASTEROID 3)

A Randomized, Parallel-group, Double-blind and Open-label Placebo-controlled, Multicenter Study to Assess the Efficacy and Safety of Vilaprisan in Subjects With Uterine Fibroids

Amenorrhea was defined as menstrual blood loss (MBL) \<2 mL during the last 28 days of treatment measured by the alkaline hematin (AH) method.

HMB response was defined as MBL \<80 mL during the last 28 days of treatment and \>50% reduction from baseline based on AH-method.

Onset of amenorrhea was defined by the first day for which the MBL for all subsequent 28-day periods up to the end of a treatment period was \< 2 mL (amenorrhea defined similar to primary endpoint).

Onset of controlled bleeding was defined by the first day for which the MBL for all subsequent 28-day periods up to the end of a treatment period was \<80.00 mL based on AH-method.

Absence of bleeding was defined as no scheduled or unscheduled bleeding (spotting allowed) during the last 28 days of a treatment period based on subjects' daily responses to the Uterine Fibroid Daily Bleeding Diary (UF-DBD).

Number of participants with endometrial histology findings, e.g. benign endometrium, Malignant Neoplasm, Hyperplasia WHO 2014, no atypia or Hyperplasia WHO 2014, atypia and Endometrial Polyps.

Ultrasound examinations were performed. Endometrial thickness was measured in the medio-sagittal section as double-layer in millimeters. Summary statistics for change from baseline in endometrial thickness was provided in below table.

For TEAEs: participant will be counted for both treatment groups if she received different treatments (vilaprisan or placebo) in the two treatment periods. SAF population was used.

For Post-treatment AEs: Mistakes in the administration of study drug might have affected the assignment to treatment. In general, participants who received a different study drug to the one they were randomized to, were assigned to the treatment most often received. SAF population was used.

Participants who received the treatment of Vilaprisan in the study - Treatment emergent AEs.

Vilaprisan - Treatment Emergent AEs

Participants who received placebo in the study - Treatment emergent AEs.

Placebo - Treatment Emergent AEs

Vilaprisan (2 mg), 2 treatment periods of 12 weeks, separated by 1 bleeding episode - Post treatment AEs.

Vilaprisan (A1) - Post Treatment AEs

Vilaprisan (2 mg), 2 treatment periods of 12 weeks without a break - Post treatment AEs.

Vilaprisan (A2) - Post Treatment AEs

Placebo, 1 treatment period of 12 weeks, and Vilaprisan (2 mg), 1 treatment period of 12 weeks, separated by 1 bleeding episode - Post treatment AEs.

Placebo+ Vilaprisan (B1) - Post Treatment AEs

Vilaprisan (2 mg), 1 treatment period of 12 weeks, and placebo, 1 treatment period of 12 Weeks, separated by 1 bleeding episode - Post treatment AEs.

Vilaprisan+ Placebo (B2) - Post Treatment AEs

Vilaprisan (A1)

Vilaprisan (A2)

Placebo+Vilaprisan (B1)

Vilaprisan+Placebo (B2)

Ultrasound examinations were performed. Endometrial thickness was measured in the medio-sagittal section as double-layer in millimeters. Baseline data of endometrial thickness is provided in the table.

Participants in safety analysis set (SAF) with endometrial thickness measured and analyzed.

SAF consisted of all randomized participants in FAS who took at least 1 dose of study drug. The discrepancy of group B1 and B2 between subject dispostion and baseline characteristics was caused by mistakes in the administration of study drug.

Three participants who were assigned to B1, erroneously received vilaprisan treatment instead of placebo in Treatment Period (TP) 1. Two participants who were assigned to B1, erroneously received placebo instead of vilaprisan in TP2.

Vilaprisan (A1) and Vilaprisan+Placebo (B2) combined vs. Placebo+Vilaprisan (B1) in treatment period 1

FAS was analysed. FAS consists of all randomized participants, excluding randomized participants who did not start treatment period 1 due to the study being temporarily on hold, including 84 participants. Participants were analyzed as randomized.

Number of Participants With Amenorrhea

Treatment period 1

Treatment period 2

Number of Participants With Heavy Menstrual Bleeding (HMB) Response

Treatment Period 1

Treatment Period 2

Treatment periods 1 and 2 combined

FAS population was analysed. In treatment period 2, participants who did not start the respective treatment period or had less than 28 days of treatment are included among censored observations.

For treatment arm Vilaprisan (A2), the combined data of treatment period 1 and treatment period 2 were entered.

Time to Onset of Amenorrhea

Time to Onset of Controlled Bleeding

Number of Participants With Absence of Bleeding (Spotting Allowed)

Participants in safety analysis set with adequate tissue were analyzed.

Treatment phase

Follow-up phase

Participants in SAF with endometrial thickness measured and analyzed.

Change From Baseline of Endometrial Thickness

Post-treatment Period 1

Follow-up Period

Vilaprisan in treatment period 1 for 12 weeks and in treatment period 2 for 12 weeks, separated by 1 bleeding episode.

Placebo in treatment period 1 for 12 weeks, and vilaprisan in treatment period 2 for 12 weeks, separated by 1 bleeding episode.

Vilaprisan in treatment period 1 for 12 weeks, and placebo in treatment period 2 for 12 weeks, separated by 1 bleeding episode.

The primary objective of this study was to show superiority of vilaprisan in the treatment of heavy menstrual bleeding (HMB) in subjects with uterine fibroids compared to placebo

The secondary objectives of this study were to additionally evaluate the efficacy and safety of vilaprisan in subjects with uterine fibroids

Assess Safety and Efficacy of Vilaprisan in Subjects With Uterine Fibroids (ASTEROID 4)

A Randomized, Parallel-group, Double-blind and Placebo-controlled, Multicenter Study to Assess the Efficacy and Safety of Vilaprisan in Subjects With Uterine Fibroids

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Assess Safety and Efficacy of Vilaprisan in Subjects With Uterine Fibroids	NCT03194646	Uterine Fibroid...	Vilaprisan (BAY... Vilaprisan (BAY... Vilaprisan (BAY... Standard of car... Vilaprisan (BAY... Vilaprisan (BAY... Vilaprisan (BAY...	18 Years -	Bayer
Pharmacokinetics and Safety of Vilaprisan in Renal Impairment	NCT03411980	Uterine Fibroid... Endometriosis	Vilaprisan (BAY...	18 Years - 79 Years	Bayer
Assessment of Safety and Efficacy of Vilaprisan in Subjects With Uterine Fibroids	NCT03699176	Uterine Fibroid...	Vilaprisan (BAY... Placebo	18 Years -	Bayer
A Study to Assess the Safety and Efficacy of Vilaprisan in Japanese Subjects With Uterine Fibroids and Heavy Menstrual Bleeding	NCT03476928	Uterine Fibroid...	Vilaprisan (BAY...	18 Years -	Bayer
A Study to Assess the Safety and Efficacy of Vilaprisan in Japanese Subjects With Uterine Fibroids and Heavy Menstrual Bleeding	NCT03476928	Uterine Fibroid...	Vilaprisan (BAY...	18 Years -	Bayer
Pharmacokinetics and Safety of Vilaprisan in Renal Impairment	NCT03411980	Uterine Fibroid... Endometriosis	Vilaprisan (BAY...	18 Years - 79 Years	Bayer
Assess Safety and Efficacy of Vilaprisan in Subjects With Uterine Fibroids (ASTEROID 3)	NCT03400943	Uterine Fibroid...	Vilaprisan (BAY... Placebo	18 Years -	Bayer
Assess Safety and Efficacy of Vilaprisan in Subjects With Uterine Fibroids (ASTEROID 3)	NCT03400943	Uterine Fibroid...	Vilaprisan (BAY... Placebo	18 Years -	Bayer
Assess Safety and Efficacy of Vilaprisan in Subjects With Uterine Fibroids	NCT03194646	Uterine Fibroid...	Vilaprisan (BAY... Vilaprisan (BAY... Vilaprisan (BAY... Standard of car... Vilaprisan (BAY... Vilaprisan (BAY... Vilaprisan (BAY...	18 Years -	Bayer
Assess Safety and Efficacy of Vilaprisan in Subjects With Uterine Fibroids (ASTEROID 4)	NCT03400956	Uterine Fibroid...	Vilaprisan (BAY... Placebo	18 Years -	Bayer
Effect of Hepatic Impairment on the Pharmacokinetics, Safety and Tolerability of BAY1002670 (Vilaprisan)	NCT03092999	Leiomyoma	Vilaprisan (BAY...	18 Years - 79 Years	Bayer

Spots Global Cancer Trial Database for vilaprisan (bay1002670)