An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients With Sickle Cell Disease (HIBISCUS)

Who is this study for? Adults and adolescents with sickle cell disease
What treatments are being studied? FT-4202
Status: Active_not_recruiting
Location: See all (142) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY

This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of etavopivat and test how well etavopivat works compared to placebo to improve the amount of hemoglobin in the blood and to reduce the number of vaso-occlusive crises (times when the blood vessels become blocked and cause pain).

Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Maximum Age: 65
Healthy Volunteers: f
View:

• Provision of consent

• Patient has a confirmed diagnosis of sickle cell disease

• At least 2 episodes of vaso-occlusive crises in the past 12 months

• Hemoglobin ≥ 5.5 and ≤ 10.5 g/dL (≥ 55 and ≤ 105 g/L) during screening

• Patients taking hydroxyurea, must demonstrate a stable dose for at least 90 days prior to start of study treatment

• Patients on crizanlizumab or L-glutamine treatment at the time of consent must be on a stable dose for ≥ 12 months and must be ≥ 80% compliant with the planned regimen at the time of consent and meet the VOC eligibility criteria

• Female patients of childbearing potential must use highly effective methods of contraception, male patients are willing to use barrier methods of contraception

Locations
United States
Alabama
Univ of Alabama Birmingham
Birmingham
Arkansas
[Legal] Woodland International Research Group, LLC
Little Rock
Arizona
Phoenix Children's Hsptl
Phoenix
California
[Legal] Collaborative Neuroscience Network, LLC
Long Beach
Pacific Research Partners
Oakland
UCSF Oakland Benioff ChildHosp
Oakland
University Of California Irvine
Orange
UC Davis Medical Center
Sacramento
Connecticut
University of Connecticut
Farmington
Yale University
New Haven
Washington, D.c.
Howard University
Washington D.c.
Florida
Cornerstone Research Institute
Altamonte Springs
University of Florida
Gainesville
Foundation for Sickle Cell Disease Research
Hollywood
Advanced Pharma CR
Miami
University Of Miami
Miami
Arnold Palmer Children's Hospital
Orlando
Georgia
Children's Healthcare of Atlanta Investigational Medication Pharmacy
Atlanta
Emory University School of Medicine
Atlanta
Sonar Clinical Research
Atlanta
Center for Blood Disorders Augusta University
Augusta
iResearch Atlanta, LLC
Decatur
Illinois
University of Illinois at Chicago
Chicago
Louisiana
Children's Hosp-New Orleans
New Orleans
Massachusetts
Boston Medical Center
Boston
Massachusetts General Hospital_Boston
Boston
Maryland
University Of Maryland School of Medicine
Baltimore
Michigan
[Legal] Dr. Vince Clinical Research, LLC and Dr. Vince Clinical Research, P.A.
Detroit
Karmanos Cancer Institute
Detroit
Missouri
Washington University School of Medicine_St. Louis
St Louis
North Carolina
[Legal] The University of North Carolina at Chapel Hill
Chapel Hill
Levine Cancer Institute
Charlotte
Duke University Medical Center
Durham
East Carolina University
Greenville
Wake Forest Baptist Health
Winston-salem
Nebraska
University of Nebraska Medical Center
Omaha
New Jersey
Children's National Health Hospital
Washington
New York
NYC Health+Hospitals
Brooklyn
Queens Hospital Center
Jamaica
North Shore Long Island Jewish Medical Center
New Hyde Park
Columbia University Medical Center_New York
New York
Weill Cornell Med Coll-NYPH
New York
Jacobi Medical Center
The Bronx
Montefiore Medical Center
The Bronx
Ohio
[Legal] Children's Hospital Medical Center dba Cincinnati Children's
Cincinnati
University of Cincinnati
Cincinnati
The Ohio State University Medical Center
Columbus
Neuro-Behavioral Clinical Research
North Canton
Oklahoma
Jimmy Everest Cancer Hematology & Oncology
Oklahoma City
Lynn Institute of Tulsa
Tulsa
Pennsylvania
Children's Hosptl Philadelphia
Philadelphia
Thomas Jefferson University Hospital
Philadelphia
University of Pittsburgh Medical Center
Pittsburgh
Rhode Island
Rhode Island Hospital
Providence
South Carolina
Medical University of South Carolina
Charleston
Prisma Health-Upstate
Greenville
Tennessee
[Legal] St. Jude Children's Research Hospital
Memphis
Methodist University Hospital
Memphis
Texas
[Legal] Baylor College of Medicine
Houston
UT Health University of Texas
Houston
Virginia
Virginia Commonwealth University
Richmond
Washington
Mary Bridge Children's Health
Tacoma
Wisconsin
Versiti Blood Center of Wisconsin
Milwaukee
Other Locations
Canada
CHU Sainte-Justine Mother and Child University Hospital
Montreal
The Hospital for Sick Children
Toronto
University Health Network - Toronto General Hospital
Toronto
Providence Hematolgy
Vancouver
Egypt
[Legal] Alexandria Clinical Research LLC
Alexandria
[Legal] Alexandria Clinical Research LLC
Alexandria
Ain Shams University Hospital
Cairo
Al Kasr AL Aini Hospital
Cairo
Cairo University
Cairo
Sydnawy University Hospital
Zagazig
France
Ap-Hp-Hopital Henri Mondor
Créteil
Hospices Civils de Lyon-Hopital Edouard Herriot
Lyon
Centre Hospitalier Universitaire de Montpellier-Hopital Saint-Eloi
Montpellier
GHR Mulhouse Sud-Alsace
Mulhouse
Ap-Hp-Hopital Robert Debre
Paris
Saint-Denis Hospital Center
Saint-denis
Germany
Charité - Campus Virchow-Klinikum - Klinik für Pädiatrie mit Schwerpunkt Onkologie und Hämatologie
Berlin
Universitätsklinikum Frankfurt
Frankfurt Am Main
Universitätsklinikum Freiburg-Zentrum für Kinder- und Jugendmedizin
Freiburg Im Breisgau
Universitätsklinikum Hamburg-Eppendorf - University Medical Center Hamburg-Eppendorf
Hamburg
Uniklinik Heidelberg - Klinik für Pädiatrische Onkologie, Hämatologie, Immunologie und Pneumologie
Heidelberg
Universitätsklinikum Regensburg
Regensburg
Ghana
Ghana Institute of Clinical Genetics, Korle Bu Teaching Hospital (KBTH)
Accra
Kintampo Health Research Centre (KHRC), Kintampo, Ghana
Kintampo
Greece
Gen Hosp Athens Ippokrateio,B' Uni Clinic Internal Medicine
Athens
General Hospital Of Larissa Koutlibaneio And Triantafylleio - Thalassemia and SCD Unit
Larissa
General University Hospital of Patras
Pátrai
'Ippokrateio' General Hospital of Thessaloniki
Thessaloniki
India
Victoria Hospital, Bangalore
Bangalore
Srirama Chandra Bhanj Medical College & Hospital (S.C.B Medical College)
Cutack
Yashoda Hospital
Hyderabad
K.J Somaiya Hospital and Research Centre
Mumbai
All India Institute of Medical Sciences (AIIMS)
New Delhi
Italy
Azienda Ospedaliero Universitaria Policlinico G. Rodolico - San Marco
Catania
Azienda Ospedaliera Universitaria San Luigi Gonzaga - S.C.D.O. Microcitemie e malattie rare ematologiche
Orbassano
Azienda Ospedale Universita Padova
Padua
ARNAS Ospedali Civico Di Cristina Benfratelli - Presidio Ospedaliero Civio e Benfratelli
Palermo
Centro per lo Studio e la Cura delle Amiloidosi Sistemiche Fondazione IRCCS Policlinico San Matteo
Pavia
Ospedale Pediatrico Bambino Ges
Roma
Kenya
KEMRI Kondele Children Hospital, Kisumu
Kisumu
KEMRI Siaya Clinical Research Annex Siaya County Referral Hospital, Siaya
Kisumu
Kombewa Clinical Research Centre
Kisumu
Gertrude's Children's Hospital, Nairobi
Nairobi
Lebanon
Master Centre for Lebanon
Amman
American University of Beirut Medical center
Beirut
Hospital Nini
Tripoli
Nigeria
University of Abuja Teaching Hospital, Gwagwalada, Abuja
Abuja
Barau Dikko Teaching Hospital, Kaduna
Kaduna
Aminu Kano Teaching Hospital (AKTH)
Kano
Lagos University Teaching Hospital, Lagos
Lagos
University of Nigeria Teaching Hospital, Enugu
Lagos
Oman
Sultan Qaboos University Hospital
Muscat
Saudi Arabia
King Fahad Specialist Hospital-Dammam
Dammam
Prince Mohammad Bin Naser Hospital
Jizan
King Khaled University Hospital,King Saud Univ. Med. City
Riyadh
Spain
Complejo Hospitalario Universitario A Coruña
A Coruña
Hospital Germans Trias i Pujol
Badalona
Hospital Universitario de Cruces
Barakaldo
Hospital Sant Joan de Déu
Barcelona
Hospital Vall d'Hebron
Barcelona
Hospital Clinico San Carlos
Madrid
Hospital Gregorio Marañón
Madrid
Hospital Ramón y Cajal
Madrid
Hospital Universitario La Paz
Madrid
Hospital Universitario Virgen del Rocío
Seville
Turkey
Adana City Training and Research Hospital
Adana
Başkent Üniversitesi Adana Dr. Turgut Noyan Uygulama ve Araştırma Merkezi
Adana
Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi- Hematoloji
Adana
Hacettepe Üniversitesi Hastanesi- Hematoloji
Ankara
Ege Üniversitesi Tip Fakultesi Hastanesi - Nefroloji
Izmir
Mersin Üniversitesi Tip Fakültesi Hastanesi- Çiftlikköy Yerleşkesi- Hematoloji
Mersin
VM Medical Park Mersin Hastanesi- Kardiyoloji
Mersin
United Kingdom
Leicester Royal Infirmary - Haemostasis & Thrombosis Unit
Leicester
Guys and St Thomas NHS Foundation Trust / Evelina Childrens Hospital
London
Hammersmith Hospital - London
London
Kings College Hospital - Haematology
London
University Hospital Lewisham, Lewisham and Greenwich NHS Trust
London
Churchill Hospital
Oxford
Royal Hallamshire Hospital
Sheffield
Time Frame
Start Date: 2021-01-29
Completion Date: 2027-03-19
Participants
Target number of participants: 450
Treatments
Experimental: Double blind etavopivat Low Dose
Double blind etavopivat Low Dose
Experimental: Double blind etavopivat High Dose
Double blind etavopivat High Dose
Experimental: Double blind placebo
Double blind placebo
Experimental: Open label etavopivat
Open label etavopivat
Sponsors
Leads: Forma Therapeutics, Inc.
Collaborators: This study is currently undergoing a sponsor transition from Forma to Novo Nordisk. Sponsor field will be updated once the transition is complete.

This content was sourced from clinicaltrials.gov