Clinical trial NCT02131662: Bay1002670, Fibroids, Safety and Efficacy EU,US,Can, Jap
Official title: A Randomized, Parallel-group, Double-blind, Placebo Controlled, Multi-center Study to Assess the Efficacy and Safety of Different Doses of BAY1002670 in Subjects With Uterine Fibroids Over 3 Months
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Leiomyoma
Keywords: Uterine fibroids
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2014-05-06
Enrollment: 309
Sex: FEMALE
Minimum age (years): 18
Maximum age (years): 50
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The study is performed to assess the efficacy and safety of different doses of BAY1002670 in subjects with uterine fibroids. The dose-response relationship will be evaluated. Further, the study aims to establish a population pharmacokinetic/pharmacodynamic relationship for BAY1002670 in subjects with uterine fibroids. To assess the efficacy of BAY1002670 the interchangeability of menstrual pictogram and alkaline hematin method for the judgement of menstrual blood loss will be assessed.


Eligibility criteria: Inclusion Criteria:

* Signed and dated informed consent
* Diagnosis of uterine fibroid(s) documented by transvaginal or abdominal ultrasound at screening with at least 1 fibroid with largest diameter 3.0 cm
* 18 to 50 years of age at the time of screening
* Heavy menstrual bleeding \>80 mL documented by MP during the bleeding episode following the screening visit
* Normal or clinically insignificant cervical smear not requiring further follow-up
* An endometrial biopsy performed at the screening visit 1 (Visit 1), without significant histological disorder such as endometrial hyperplasia or other significant endometrial pathology
* Use of a non-hormonal barrier method of contraception starting at the bleeding episode following the screening visit 1 (Visit 1) until the end of the study
* Good general health (except for findings related to uterine fibroids)

Exclusion Criteria:

* Pregnancy or lactation
* Uterine fibroid with largest diameter \>10.0 cm
* Hypersensitivity to any ingredient of the study drug
* Laboratory values outside inclusion range before randomization and considered as clinically relevant
* Hemoglobin values \<6 g/dL or any condition requiring immediate blood transfusion (subjects with hemoglobin values \<10.9 g/dL will receive iron supplementation)
* 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

Primary outcomes:
measure: Percentage of Subjects With Amenorrhea, Defined as no Scheduled or Unscheduled Bleeding/Spotting After the End of the Initial Bleeding Episode Until End of Treatment
timeFrame: After end of the initial bleeding episode until the end of treatment, up to 12 weeks
Secondary outcomes:
measure: Change in Volume of Menstrual Blood Loss Per 28 Days From Baseline During Treatment by Reference Period (Assessed by Alkaline Hematin Method)
timeFrame: From baseline to end of follow-up
measure: Time to Onset of Controlled Bleeding
timeFrame: During treatment period
measure: Change in Volume of Largest Fibroid Compared to Baseline Measured by MRI
timeFrame: From baseline to end of follow-up period
Other outcomes:
measure: Exposure-response Analysis of Vilaprisan - Percentage of Subjects Achieving Maximum Effect (Emax) of Induced Amenorrhea
timeFrame: From start of the study treatment to Day 84 (treatment period)
measure: Steady-state Exposure Achieving Half-maximal Effect (EAUC50) of Induced Amenorrhea During Treatment Period of Vilaprisan
timeFrame: From start of the study treatment to Day 84 (treatment period)
measure: Exposure-response Analysis of Vilaprisan - Predicted Percentage of Subjects Below 90% of the Maximum Probability of Induced Amenorrhea
timeFrame: From start of the study treatment to Day 84 (treatment period)
measure: Assessment of MP to Identify Subjects With Heavy Menstrual Bleeding (HMB)
timeFrame: At baseline
measure: Percentage of Subjects With Amenorrhea (Defined as MBL < 2 mL) During the Last 28 Days of Treatment
timeFrame: Last 28 Days of Treatment
measure: Percentage of Subjects With HMB Response During the Last 28 Days of Treatment
timeFrame: Last 28 Days of Treatment
measure: Estimated Dose-response Curve Based on Amenorrhea - E0 and Emax
timeFrame: After end of the initial bleeding episode until the end of treatment
measure: Estimated Dose-response Curve Based on Amenorrhea - ED50
timeFrame: After end of the initial bleeding episode until the end of treatment
measure: Estimated Dose-response Curve Based on Amenorrhea - δ
timeFrame: After end of the initial bleeding episode until the end of treatment
Locations:
city: Mesa
country: United States
state: Arizona
city: Tucson
country: United States
state: Arizona
city: San Diego
country: United States
state: California
city: San Diego
country: United States
state: California
city: Denver
country: United States
state: Colorado
city: Southington
country: United States
state: Connecticut
city: Washington
country: United States
state: District of Columbia
city: Gainesville
country: United States
state: Florida
city: Jacksonville
country: United States
state: Florida
city: Plantation
country: United States
state: Florida
city: South Miami
country: United States
state: Florida
city: Sandy Springs
country: United States
state: Georgia
city: Naperville
country: United States
state: Illinois
city: Newburgh
country: United States
state: Indiana
city: Marrero
country: United States
state: Louisiana
city: Lincoln
country: United States
state: Nebraska
city: Moorestown
country: United States
state: New Jersey
city: Neptune City
country: United States
state: New Jersey
city: New Brunswick
country: United States
state: New Jersey
city: New York
country: United States
state: New York
city: Durham
country: United States
state: North Carolina
city: Raleigh
country: United States
state: North Carolina
city: Cleveland
country: United States
state: Ohio
city: Columbus
country: United States
state: Ohio
city: Portland
country: United States
state: Oregon
city: Philadelphia
country: United States
state: Pennsylvania
city: Philadelphia
country: United States
state: Pennsylvania
city: Pittsburgh
country: United States
state: Pennsylvania
city: Pittsburgh
country: United States
state: Pennsylvania
city: Chattanooga
country: United States
state: Tennessee
city: Dallas
country: United States
state: Texas
city: Frisco
country: United States
state: Texas
city: Houston
country: United States
state: Texas
city: Houston
country: United States
state: Texas
city: Norfolk
country: United States
state: Virginia
city: Seattle
country: United States
state: Washington
city: Bruxelles - Brussel
country: Belgium
city: Bruxelles - Brussel
country: Belgium
city: Charleroi
country: Belgium
city: Leuven
country: Belgium
city: Liege
country: Belgium
city: Pleven
country: Bulgaria
city: Sofia
country: Bulgaria
city: Sofia
country: Bulgaria
city: Sofia
country: Bulgaria
city: Stara Zagora
country: Bulgaria
city: Winnipeg
country: Canada
state: Manitoba
city: Hamilton
country: Canada
state: Ontario
city: Ottawa
country: Canada
state: Ontario
city: Pointe-Claire
country: Canada
state: Quebec
city: Quebec
country: Canada
city: Ceske Budejovice
country: Czechia
city: Hradec Kralove
country: Czechia
city: Olomouc
country: Czechia
city: Pisek
country: Czechia
city: Praha 2
country: Czechia
city: Praha
country: Czechia
city: Espoo
country: Finland
city: Helsinki
country: Finland
city: Joensuu
country: Finland
city: Kuopio
country: Finland
city: Oulu
country: Finland
city: Karlsruhe
country: Germany
state: Baden-Württemberg
city: Erlangen
country: Germany
state: Bayern
city: Geseke
country: Germany
state: Nordrhein-Westfalen
city: Bernburg
country: Germany
state: Sachsen-Anhalt
city: Blankenburg
country: Germany
state: Sachsen-Anhalt
city: Dresden
country: Germany
state: Sachsen
city: Lübeck
country: Germany
state: Schleswig-Holstein
city: Berlin
country: Germany
city: Ilsede
country: Germany
city: Debrecen
country: Hungary
city: Debrecen
country: Hungary
city: Kecskemet
country: Hungary
city: Szeged
country: Hungary
city: Szentes
country: Hungary
city: Matsudo
country: Japan
state: Chiba
city: Iizuka
country: Japan
state: Fukuoka
city: Koriyama
country: Japan
state: Fukushima
city: Sapporo
country: Japan
state: Hokkaido
city: Sapporo
country: Japan
state: Hokkaido
city: Kanazawa
country: Japan
state: Ishikawa
city: Omura
country: Japan
state: Nagasaki
city: Numazu
country: Japan
state: Shizuoka
city: Kita
country: Japan
state: Tokyo
city: Nakano-ku
country: Japan
state: Tokyo
city: Tacchikawa
country: Japan
state: Tokyo
city: Kumamoto
country: Japan
city: Nagano
country: Japan
city: Nesttun
country: Norway
city: Oslo
country: Norway
city: Sellebakk
country: Norway
city: Trondheim
country: Norway
city: Trondheim
country: Norway
city: Aravaca
country: Spain
state: Madrid
city: Barcelona
country: Spain
city: Barcelona
country: Spain
city: Sevilla
country: Spain
city: Sevilla
country: Spain
city: Valencia
country: Spain
city: Göteborg
country: Sweden
city: Stockholm
country: Sweden
city: Uppsala
country: Sweden
city: Örebro
country: Sweden
city: Bern
country: Switzerland
Study officials:
affiliation: Bayer
name: Bayer Study Director
role: STUDY_DIRECTOR
References:
citation: Ren X, Xia J. An algorithm for computing profile likelihood based pointwise confidence intervals for nonlinear dose-response models. PLoS One. 2019 Jan 25;14(1):e0210953. doi: 10.1371/journal.pone.0210953. eCollection 2019.
citation: Bradley LD, Singh SS, Simon J, Gemzell-Danielsson K, Petersdorf K, Groettrup-Wolfers E, Ren X, Zvolanek M, Seitz C. Vilaprisan in women with uterine fibroids: the randomized phase 2b ASTEROID 1 study. Fertil Steril. 2019 Feb;111(2):240-248. doi: 10.1016/j.fertnstert.2018.10.012. Epub 2018 Dec 7.