Study to Gather Information How Well Three Different Doses of BAY1817080 Given Twice Daily Over 12 Weeks Work in Comparison to an Inactive Pill (Placebo) and Elagolix in Women Suffering From Pain Related to a Condition Where the Tissue That Usually Grows Inside the Womb Grows Outside of the Womb

Endometriosis Clinical Trial

— SCHUMANN  

Official title:

A Randomized, Double-blind, Open for Active Comparator, Parallel-group, Multicenter Phase 2b Study to Assess the Efficacy and Safety of Three Different Doses of P2X3 Antagonist (BAY1817080) Versus Placebo and Elagolix 150 mg in Women With Symptomatic Endometriosis

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04614246
• Other study ID #: 20584
• Secondary ID: 2020-003131-16
• Status: Terminated
• Phase: Phase 2
• Start date: January 29, 2021
• Est. completion date: May 3, 2022

Study information

• Verified date: July 2023
• Source: Bayer
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to assess safety and efficacy of BAY 1817080 compared to elagolix and placebo in women with symptomatic endometriosis. Study details include:

• Study duration: 155 up to 285 days

• Treatment duration: 84 days

• Visit frequency: 3 laboratory every 2 weeks for participants on BAY 1817080 or placebo

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 215
• Est. completion date: May 3, 2022
• Est. primary completion date: January 17, 2022
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Participant must be = 18 years of age at the time of signing the informed consent

• Visually-confirmed endometriosis: detection of endometriotic lesions during laparoscopy or laparotomy (with or without pathological diagnosis) within 10 years but no less than 8 weeks from Visit 1a (surgically diagnosed endometriosis). For Japan only and limited to no more than half of all randomized Japanese participants: the diagnosis can be based on previous imaging (i.e. endometriosis lesion detected by ultrasound or MRI). If the participant was diagnosed by ultrasound, the lesion must be visualized again by ultrasound at the screening visit. If the participant was diagnosed by MRI, the diagnosis must have been made within 12 months before Visit 1a (clinically diagnosed endometriosis).

• Both sub-criteria regarding pain symptoms must be fulfilled:

• At Visit 1a, participant presents self-reported moderate to severe pain which - based on the judgement of the investigator - carries a reasonable likelihood to translate into a severity of pain symptoms sufficient to fulfil the eligibility criterion and be caused by endometriosis, and

• During the screening period at least 24 daily ESD entries during the 28 consecutive days starting on the first day with menstrual bleeding at or after Visit 1a and entries in the ESD item 1a ('worst pain' on the daily numerical rating scale) sum up to 98 or more.

• Willingness to use standardized rescue pain medications for EAPP (i.e. ibuprofen, acetaminophen and tramadol) and not use any prophylactic pain medication, according to investigator's instruction

• Ability to swallow the study intervention, i.e., the different kinds of tablets, as complete units

• Good general health (except for findings related to endometriosis) as proven by medical history, physical and gynecological examinations and laboratory test results

• Normal or clinically insignificant cervical cytology not requiring further follow-up:

• A cervical cytology sample has to be obtained during screening, or

• A documented normal result has to be available from cervical cytology conducted within 12 months prior to Visit 1a.

• Human papilloma virus (HPV) testing in participants with atypical squamous cells of unknown significance (ASCUS) will be used as an adjunctive test automatically. Participants with ASCUS can be included if they are negative for high-risk HPV strains.

Exclusion Criteria:

• Current pregnancy or less than 3 months since delivery, abortion or stop of lactation before Visit 1a

• Hypersensitivity to any ingredient of the study intervention and/or the standardized rescue medications

• Known osteoporosis

• History of a low trauma fracture

• Contraindications for elagolix or the standardized rescue medications

• Current malignancy or history of cancer (exception: basal cell or squamous cell carcinoma of the skin) within the last 5 years prior to Visit 1a

• Any other diseases or conditions that according to the investigator can compromise the function of the body systems and could result in altered absorption, excessive accumulation, impaired metabolism, or altered excretion of the study intervention (e.g. extremely low body weight, chronic bowel disease, Crohn's disease and ulcerative colitis)

• Menopause or signs of menopausal transition, such as absence of regular menstrual cycles based on investigator's judgment (absence of information regarding menstrual bleeding pattern e.g. due to long term use of hormonal contraception is not an exclusion criterion)

• Any disease or condition that may worsen during the study period according to the assessment and opinion of the investigator

• Abnormal uterine bleeding in terms of regularity or heaviness (with the exception of heavy menstrual bleeding that does not require treatment)

• Any findings that require further diagnostic procedures to avoid harm to the participant (e.g. ovarian tumors of uncertain origin or pelvic masses of unclear etiology)

• Any serious or unstable diseases or medical conditions, including psychiatric disorders, that might interfere with the conduct of the study or the interpretation of the result, including for example:

• history of hysterectomy and/or bilateral oophorectomy

• any conditions considered to contribute significantly to pelvic pain by the investigator, e.g. fibromyalgia, uterine fibroids, irritable bowel syndrome or other bowel disorders

• any other underlying diseases requiring regular use of pain medication (e.g. migraine)

• history of or current anxiety or depression unless stable with or without medical treatment = 6 months before Visit 1a

• Major surgery scheduled during the study period

• Non-responsiveness of EAPP to earlier treatment with GnRH-agonists or GnRH-antagonists, based on the judgement of the investigator

• SARS-CoV-2- positive virus RNA test within 4 weeks prior to Visit 1a reported by participant, regardless of whether the participant had symptoms

• History of COVID-19 infection with persistent/ongoing symptoms

• Contact with SARS-CoV-2- positive or COVID-19 patient within the last 4 weeks prior to Visit 1a

• Intake of medication prohibited due to potential drug-drug interaction

• Use of other treatments that might interfere with the conduct of the study or the interpretation of the results, including:

• hormonal medications

• other treatments intended for endometriosis/pelvic pain during participation in the study, including the use of herbal products or traditional Chinese medicine for symptom relief, with the exception of the standardized rescue pain medications

• Simultaneous participation in another clinical trial with investigational medicinal product(s). Participation in another trial 3 months prior to Visit 1a that might have an impact on the study objectives, at the discretion of the investigator

• Previous assignment to study intervention (randomization) in this study (allowing previously randomized participants to be re-included into the study may lead to bias)

• Laboratory values outside the inclusion range (specified in the laboratory manual) and considered clinically relevant

Related Conditions & MeSH terms

• Endometriosis

Intervention

Drug:
• BAY1817080
Tablet, oral administration
• Elagolix
Tablet, oral administration
• Placebo
Tablet, oral administration

Locations

Country	Name	City	State
Austria	Medizinische Universität Graz	Graz	Steiermark
Austria	Universitätsklinikum AKH Wien	Wien
Belgium	CU Saint-Luc/UZ St-Luc	Bruxelles - Brussel
Belgium	Ziekenhuis Oost-Limburg	Genk
Belgium	AZ Jan Palfijn Gent	Gent	Oost-Vlaanderen
Belgium	UZ Gent	Gent
Bulgaria	MC Asklepii OOD	Dupnitza
Bulgaria	Medical Center Panaceya	Sofia
Bulgaria	Spec. Hospital for Active Treatment of Oncological Diseases	Sofia
Bulgaria	MHAT Niamed	Stara Zagora
Bulgaria	SHOGAT Prof Dimitar Stamatov	Varna
Canada	Clinique OVO	Montreal	Quebec
Canada	The Ottawa Hospital	Ottawa	Ontario
Canada	The Ottawa Hospital	Ottawa	Ontario
Canada	The Ottawa Hospital | The Ottawa Hospital Research Institute - Neurology - Ottawa Stroke Program	Ottawa	Ontario
Canada	Alpha Recherche Clinique LB9	Quebec
China	Beijing Obstetrics and Gynecology Hospital,Capital Med.Uni.	Beijing
China	Peking University First Hospital	Beijing
China	First Affiliated Hospital of Guangzhou Medical University	Guangzhou	Guangdong
China	Guangzhou Women and Children's Medical Center	Guangzhou	Guangdong
China	Women's Hospital School of Medicine Zhejiang University	Hangzhou	Zhejiang
China	The Second Affiliated Hospital of Nanjing Medical university	Nanjing	Jiangsu
China	The Second Affiliated Hospital of Nanjing Medical university	Nanjing	Jiangsu
China	Wuhan Tongji Reproductive Medicine Hospital	Wuhan	Hubei
Czechia	Fakultni nemocnice Brno	Brno
Czechia	Gynekologie Meda s.r.o. - Lidicka	Brno - stred
Czechia	GYN-MIKA s.r.o.	Ceske Budejovice
Czechia	Centrum gynekologicke rehabilitace	Pisek
Czechia	GynCare MUDr. Michael Svec s.r.o.	Plzen
Czechia	GYNERA	Praha 5
Czechia	Gynekologie Studentsky dum s.r.o.	Praha 6
Czechia	Fakultní nemocnice Bulovka	Praha 8
Czechia	Dr. Smrhova-Kovacs	Tabor
Estonia	Parnu Hospital	Parnu
Estonia	Clinic Elite	Tartu
Finland	HUS / Naistenklinikka	Helsinki
Finland	Lääkärikeskus Gyneko	Oulu
Germany	Charité - Campus Virchow-Klinikum (CVK)	Berlin
Germany	emovis GmbH	Berlin
Germany	Praxis f. Gynäkologie und Geburtshilfe	Bernburg	Sachsen-Anhalt
Germany	Universitätsklinikum Heidelberg	Heidelberg	Baden-Württemberg
Germany	Praxisklinik am Rosengarten	Mannheim	Baden-Württemberg
Greece	ARETAIEION University Hospital	Athens
Greece	University General Hospital of Patras | Univ Obs & Gynae Cli	Patra
Hungary	Cortex Study Center	Budapest
Hungary	SZTE ÁOK Szent Györgyi Albert Klinikai Kozpont	Szeged
Italy	A.O.U. Careggi	Firenze	Toscana
Italy	IRCCS Ospedale Policlinico San Martino	Genova	Liguria
Italy	Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico	Milano	Lombardia
Italy	Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Roma	Lazio
Italy	ASST Sette Laghi	Varese	Lombardia
Italy	A.O.U.I. Verona	Verona	Veneto
Japan	Fujisawa City Hospital	Fujisawa	Kanagawa
Japan	Aiiku Ladies Clinic	Funabashi	Chiba
Japan	Hitachi General Hospital	Hitachi	Ibaraki
Japan	Ibaraki Prefectural Central Hospital	Kasama	Ibaraki
Japan	Japanese Red Cross Kumamoto Hospital	Kumamoto
Japan	Kurashiki Medical Clinic	Kurashiki	Okayama
Japan	Omi Medical Center	Kusatsu	Shiga
Japan	Nagasaki University Hospital	Nagasaki
Japan	Saiseikai Nagasaki Hospital	Nagasaki
Japan	Kashiwazaki ladies clinic	Saitama	Tokyo
Japan	Nishikawa Women's Health Clinic	Sapporo	Hokkaido
Japan	Social Medical Corporation Tokeidai Memorial Hospital	Sapporo	Hokkaido
Japan	Teine Keijinkai Hospital	Sapporo	Hokkaido
Japan	NTT Medical Center Tokyo	Shinagawa-ku	Tokyo
Japan	JCHO Tokuyama Central Hospital	Shunan	Yamaguchi
Japan	Suita Tokushukai Hospital	Suita	Osaka
Japan	Toyama Rosai Hospital	Uozu	Toyama
Japan	Tokyo Bay Urayasu Ichikawa Medical Center	Urayasu	Chiba
Japan	Yokosuka Kyosai Hospital	Yokosuka	Kanagawa
Japan	Sou Clinic	Yotsukaido	Chiba
Latvia	I.Dinsbergas Physician Practice	Riga
Latvia	I.Vasaraudzes Private Practice	Riga
Latvia	Latvian Maritime Medicine Center	Riga
Latvia	Vitols and Vitols Ltd	Riga
Lithuania	JSC Gyvenk Silciau Medical Center Maxmeda	Vilnius
Lithuania	JSC Seimos gydytojas family medical center	Vilnius
Lithuania	V. Jonaitienes private gynecology clinic	Vilnius
Norway	Kirkeparken Spesialistpraksis	Fredrikstad
Norway	Oslo Universitetssykehus HF, Ullevål	Oslo
Norway	Sykehuset i Vestfold HF, Tønsberg	Tønsberg
Poland	Gabinet Ginekologiczny Janusz Tomaszewski	Bialystok
Poland	MICS Centrum Medyczne Torun	Bydgoszcz
Poland	CLINICAL MEDICAL RESEARCH Sp. z o. o.	Katowice
Poland	NZOZ MEDEM Wilk Sp. j.	Katowice
Poland	Vita Longa Sp. z o.o.	Katowice
Poland	Prywatny Gabinet Lekarski Ginekologia, Poloznictwo i Ultr.	Lodz
Poland	NZOZ NOVITA, Specjalistyczne Gabinety Lekarskie S.C	Lublin
Poland	Specjalistyczny Gabinet Ginekologiczno-Polozniczy	Lublin
Poland	VitroLive Sp. z o.o.	Szczecin
Poland	Medical Concierge Centrum Medyczne	Warszawa
Poland	MTZ Clinical Research Sp z o.o.	Warszawa
Slovakia	ULMUS, s r.o.	Hlohovec
Slovakia	GA Lucenec s.r.o	Lucenec
Slovakia	Virina sano, s.r.o. Gynekologicko porodnicka ambulancia	Velky Krtis
Spain	Hospital de la Santa Creu i de Sant Pau | Ginecologia	Barcelona
Spain	Hospital de Basurto	Bilbao	Vizcaya
Spain	Hospital Universitario 12 de Octubre	Madrid
Spain	Hospital General Universitario de Valencia	Valencia
United States	Bosque Womens Care	Albuquerque	New Mexico
United States	Medisense, Inc.	Atlanta	Georgia
United States	Augusta University Medical Center	Augusta	Georgia
United States	Clinical Inquest Center, Ltd.	Beavercreek	Ohio
United States	Accel Research Sites - Cahaba Medical Care	Birmingham	Alabama
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	Helix Biomedics, LLC	Boynton Beach	Florida
United States	SUNY Downstate Medical Center	Brooklyn	New York
United States	Chattanooga Medical Research, LLC	Chattanooga	Tennessee
United States	ClinSearch, LLC	Chattanooga	Tennessee
United States	Paramount Research Solutions-College Park	College Park	Georgia
United States	Discovery Clinical Trials - Dallas	Dallas	Texas
United States	Wayne State University Physicians Group	Detroit	Michigan
United States	Aventiv Research - Dublin	Dublin	Ohio
United States	Centricity Research formerly Aventiv - Dublin	Dublin	Ohio
United States	Carolina Women's Research & Wellness Center	Durham	North Carolina
United States	Diagnamics, Inc.	Encinitas	California
United States	HWC Women's Research Center	Englewood	Ohio
United States	Alliance for Multispecialty Research, LLC - Fort Meyers	Fort Myers	Florida
United States	Advanced Women's Health Institute	Greenwood Village	Colorado
United States	Biopharma Informatic, Inc.	Houston	Texas
United States	Centex Studies, Inc.	Houston	Texas
United States	Centex Studies, Inc.	Houston	Texas
United States	UT Health Women's Research Center at Memorial City	Houston	Texas
United States	Clinical Research Prime	Idaho Falls	Idaho
United States	Essential Women's Health Associates	Las Vegas	Nevada
United States	University Hospitals Landerbrook Health Center	Mayfield Heights	Ohio
United States	Medical Research Center of Memphis, LLC	Memphis	Tennessee
United States	Southern Clinical Research Associates LLC	Metairie	Louisiana
United States	Mobile Ob-Gyn, PC	Mobile	Alabama
United States	International Clinical Research-Tennessee LLC.	Murfreesboro	Tennessee
United States	Suncoast Clinical Research Center, Inc.	New Port Richey	Florida
United States	Eastern Virginia Medical School	Norfolk	Virginia
United States	Eastern Virginia Medical School | OB/GYN Clinical Research Center	Norfolk	Virginia
United States	Tidewater Clinical Research, Inc.	Norfolk	Virginia
United States	Virginia Physicians For Women	North Chesterfield	Virginia
United States	A Premier Medical Research of Florida, LLC	Orange City	Florida
United States	Advances in Health	Pearland	Texas
United States	Precision Trials, AZ, LLC	Phoenix	Arizona
United States	West Coast OB/GYN Associates	San Diego	California
United States	Physician Care Clinical Research	Sarasota	Florida
United States	Seattle Clinical Research Center	Seattle	Washington
United States	Journey Medical Research	Snellville	Georgia
United States	Palmetto Clinical Research	Summerville	South Carolina
United States	Continental Clinical Solutions, LLC	Towson	Maryland
United States	Office of Dr. James A. Simon, MD	Washington	District of Columbia
United States	Lyndhurst Clinical Research	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Bayer

Countries where clinical trial is conducted

United States,  Austria,  Belgium,  Bulgaria,  Canada,  China,  Czechia,  Estonia,  Finland,  Germany,  Greece,  Hungary,  Italy,  Japan,  Latvia,  Lithuania,  Norway,  Poland,  Slovakia,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Absolute Change in Mean Worst Endometriosis Associated Pelvic Pain (EAPP), Primary Per Protocol Set (pPPS)	The worst EAPP was measured daily on the 0-10 Numerical Rating Scale (NRS) by item 1 of the Endometriosis Symptom Diary (ESD). In question 1, participants were asked to rate the 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. The absolute change in mean worst EAPP was from baseline (last 28 days before the first intake of study drug) to end of intervention (last 28 days ending with the last intake of study drug planned on Day 84 [+3]). The time frame of 28 days captures a menstrual cycle on average.	change from baseline to week 12
Secondary	Least Squares Mean (SE) Changes in Worst EAPP From Baseline to Week 12, Per Protocol Set	The worst EAPP was measured daily on the 0-10 Numerical Rating Scale (NRS) by item 1 of the Endometriosis Symptom Diary (ESD). In question 1, participants were asked to rate the 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. The absolute change in mean worst EAPP was from baseline (last 28 days before the first intake of study drug) to end of intervention (last 28 days ending with the last intake of study drug planned on Day 84 [+3]). The time frame of 28 days captures a menstrual cycle on average.	change from baseline to week 12
Secondary	Number of Participants With Treatment-emergent Adverse Events (TEAE)	A treatment-emergent AE (TEAE) was defined as any event arising or worsening after the start of study drug administration until 14 days after the last study medication intake.	up to 14 days after the last study medication intake

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