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RECRUITING

NCT06609226

PHASE3

A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

Sponsor: Novo Nordisk A/S

View on ClinicalTrials.gov

Summary

Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin. Haemoglobin is the protein that carries oxygen through the body. This study is looking into how safe treatment with etavopivat is and how well it works over a long period of time. The study will last for up to 264 weeks, but it will end earlier if etavopivat is approved in the participant's country.

Official title: An Open-label, Multi-centre, Rollover Study to Characterise Long-term Safety and Efficacy of Etavopivat in Adults, Adolescents and Children Who Have Sickle Cell Disease or Thalassaemia and Have Completed a Treatment Period in an Etavopivat Study

Key Details

Gender

All

Age Range

2 Years - Any

Study Type

INTERVENTIONAL

Enrollment

480

Start Date

2025-01-10

Completion Date

2030-12-30

Last Updated

2026-03-24

Healthy Volunteers

Conditions

Sickle Cell Disease Thalassemia

Interventions

DRUG

Etavopivat A

Participants will receive an oral dose of Etavopivat A.

DRUG

Etavopivat B

Participants will receive an oral dose of Etavopivat B.

DRUG

Etavopivat C

Participants will receive an oral dose of Etavopivat C.

Inclusion Criteria: * Participant must have ongoing participation in an etavopivat parent study for treatment of sickle cell disease (SCD) or thalassaemia and have completed at least a treatment period of the parent study. * Participant must have derived clinical benefit from treatment with etavopivat, as determined by the investigator. * Any participant with dose reduction or temporary discontinuation will need to be successfully rechallenged to the full dose of etavopivat before transferring. * Participants on hydroxyurea (HU), crizanlizumab or l-glutamine oral powder (Endari®) treatment at the time of consent may be eligible if they have been on a stable dose in the parent study as defined at the investigator's discretion. Necessary adjustments related to weight or age are accepted. Participants with temporary dose reductions or pauses due to medical reasons may still be considered to have a stable dose, as determined by the investigator, who will assess the impact of these adjustments based on clinical context and the participant's overall health status. Exclusion Criteria: * Any disorder, except for conditions associated with SCD or thalassaemia, which in the investigator's opinion might jeopardise participant's safety or compliance with the protocol. * Participant withdrew or had permanent treatment discontinuation from an etavopivat clinical study. * Participants on permanent dose reduction (greater than \[\>\] 28 days or more) or ongoing temporary treatment discontinuation. * Use of any of the following within the timeframes prior to the transfer visit as stated: * Use of haemoglobin S (HbS) polymerisation inhibitors within participation of the parent study or anticipated need for this agent during this study. * Use of an experimental selectin antagonist (e.g., monoclonal antibody or small molecule) within the parent study or anticipated need for such agents during this study. * Use of erythropoietin or other haematopoietic growth factor treatment for more than 4 consecutive weeks during the parent study or anticipated need of such agents for a maintenance treatment during this study. * Receiving or use of concomitant medications that are strong inducers of cytochrome P450 (CYP) 3A4 within 2 weeks of the transfer visit or anticipated need for such agents during the study. * Current participation in a study that is not a designated parent study, or planned participation in any other clinical study, for the duration of FLORAL.

Locations (103)

Univ of Alabama Birmingham

Birmingham, Alabama, United States

Phoenix Children's Hsptl

Phoenix, Arizona, United States

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California, United States

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California, United States

UCSF Oakland Benioff ChildHosp

Oakland, California, United States

UCSF Oakland Benioff ChildHosp

Oakland, California, United States

Children's Hosp Of Orange

Orange, California, United States

University Of California Irvine

Orange, California, United States

University of Connecticut

Farmington, Connecticut, United States

Children's National Medical Center

Washington D.C., District of Columbia, United States

Foundation for Sickle Cell Disease Research

Hollywood, Florida, United States

Univ of Miami/SCCC

Miami, Florida, United States

Emory University School of Medicine

Atlanta, Georgia, United States

Children's Healthcare Atlanta

Atlanta, Georgia, United States

Center for Blood Disorders Augusta University

Augusta, Georgia, United States

Univer Of Illinois at Chicago

Chicago, Illinois, United States

Children's Hosp-New Orleans

New Orleans, Louisiana, United States

Boston Medical Center

Boston, Massachusetts, United States

Washington University-St.Louis

St Louis, Missouri, United States

NYC Health+Hospitals

Brooklyn, New York, United States

Columbia University Medical Center_New York_0

New York, New York, United States

Columbia University Medical Center_New York_0

New York, New York, United States

Weill Cornell Med Coll-NYPH

New York, New York, United States

Jacobi Medical Center

The Bronx, New York, United States

Montefiore Medical Center

The Bronx, New York, United States

Atrium Levine Children's/Atrium Health

Charlotte, North Carolina, United States

Duke University_Durham

Durham, North Carolina, United States

East Carolina Univ-Greenville

Greenville, North Carolina, United States

East Carolina University_Greenville

Greenville, North Carolina, United States

Atrium Health-Wake Forest Bapt

Winston-Salem, North Carolina, United States

Cincinnati Child's Hsp Med Ctr

Cincinnati, Ohio, United States

Neuro-Behavioral Clinical Research

North Canton, Ohio, United States

Medical University Of South Carolina_Charleston

Charleston, South Carolina, United States

Methodist University Hospital

Memphis, Tennessee, United States

Texas Children's Hospital_Houston

Houston, Texas, United States

UT Health University of Texas

Houston, Texas, United States

Virginia Comm Univ Medical Ctr

Richmond, Virginia, United States

Mary Bridge Children's Health

Tacoma, Washington, United States

Versiti, CCBD_Milwaukee

Milwaukee, Wisconsin, United States

St Pauls Hospital

Vancouver, British Columbia, Canada

The Hospital for Sick Children

Toronto, Ontario, Canada

University Health Network - Toronto General Hospital

Toronto, Ontario, Canada

CHU Ste-Justine

Montreal, Quebec, Canada

Alexandria University Hospital

Alexandria, Egypt, Egypt

Zagazig University Hospital

Alsharkia, Egypt, Egypt

Faculty of Medicine Ain Shams Medical Research Institute (MASRI)

Cairo, Egypt

Cairo University

Cairo, Egypt, Egypt

Abu El-Reesh El-Mounira Children University Hospital

Cairo, Egypt, Egypt

Ap-Hp-Hopital Henri Mondor

Créteil, France

Hospices Civils de Lyon-Hopital Edouard Herriot

Lyon, France

Ap-Hp-Hopital Robert Debre

Paris, France

Ap-Hp-Hopital Robert Debre

Paris, France

Charité - Campus Virchow-Klinikum - Klinik für Pädiatrie mit Schwerpunkt Onkologie und Hämatologie

Berlin, Germany

Universitätsklinikum Freiburg - Kinder- und Jugendklinik

Freiburg im Breisgau, Germany

Kintampo Health Research Centre (KHRC)

Kintampo, Bono East, Ghana

Ghana Institute of Clinical Genetics, Korle Bu Teaching Hospital (KBTH)

Accra, Ghana

General Hospital Of Larissa Koutlibaneio And Triantafylleio - Thalassemia and SCD Unit

Larissa, Thessaly, Greece

Hippokration Hospital

Athens, Greece

General University Hospital of Patras

Pátrai, Greece

'Ippokrateio' General Hospital of Thessaloniki

Thessaloniki, Greece

All India Institute of Medical Sciences (AIIMS), Raipur

Raipur, Chhattisgarh, India

K.J Somaiya Hospital and Research Centre

Mumbai, Maharashtra, India

Victoria Hospital, Bangalore

Bangalore, India

Nirmal Hospital Pvt. Ltd.

Gujarat, India

Suretech Hospital and Research Centre Ltd.

Maharashtra, India

All India Institute of Medical Sciences_Delhi

New Delhi, India

Azienda Ospedaliera Universitaria San Luigi Gonzaga - S.C.D.O. Microcitemie e malattie rare ematologiche

Orbassano, Torino, Italy

Azienda Ospedale Universita Padova

Padova, Italy

Fondazione IRCCS Policlinico San Matteo

Pavia, Italy

Gertrude's Children's Hospital

Nairobi, Nairobi County, Kenya

KEMRI CRDR Siaya Clinical Research Annex, Country Referral Hospital

Siaya, Siaya County, Kenya

KEMRI-Walter-Reed Kericho

Kericho, Kenya

Kombewa Clinical Research Centre

Kisumu, Kenya

KEMRI Kondele Children Hospital, Kisumu

Kisumu, Kenya

Ahero Clinical Trials Unit

Kisumu, Kenya

American University of Beirut Medical Centre

Hamra, Lebanon

Chronic Care Center

Hazmiyeh, Lebanon

Hospital Nini

Tripoli, Lebanon

University of Nigeria Teaching Hospital (UNTH)

Ituku-Ozalla, Enugu State, Nigeria

University College Hospital Paediatric Haematology and Oncology Unit, Ibadan

Ibadan, Oyo State, Nigeria

University of Abuja Teaching Hospital, Gwagwalada, Abuja

Abuja, Nigeria

Barau Dikko Teaching Hospital, Kaduna

Kaduna, Nigeria

Aminu Kano Teaching Hospital (AKTH)

Kano, Nigeria

Lagos University Teaching Hospital, Lagos

Lagos, Nigeria

Sultan Qaboos University Hospital

Muscat, Sultanet of Oman/Muscat/Al Khoud, Oman

Prince Mohammad Bin Naser Hospital

Jizan, Saudi Arabia

King Khalid University Hospital

Riyadh, Saudi Arabia

Hospital Universitario de Cruces

Barakaldo, Spain

Hospital Vall d'Hebron

Barcelona, Spain

Hospital Universitario La Paz

Madrid, Spain

Baskent Universitesi Adana

Adana, Turkey (Türkiye)

Başkent Üniversitesi Adana-Hematoloji

Adana, Turkey (Türkiye)

Hacettepe University Hematology

Ankara, Turkey (Türkiye)

Hacettepe Üniversitesi Hastanesi- Hematoloji

Ankara, Turkey (Türkiye)

Mersin University Medical Faculty Pediatric Hematology

Mersin, Turkey (Türkiye)

Mersin Üniversitesi Tip Fakültesi Hastanesi- Çiftlikköy Yerleşkesi- Hematoloji

Mersin, Turkey (Türkiye)

Guy's Hospital - Haematology

London, United Kingdom

Guy's Hospital

London, United Kingdom

King's College Hospital - Paediatric Research

London, United Kingdom

Kings College Hospital - Haematology

London, United Kingdom

Imperial College London

London, United Kingdom

Manchester Royal Infirmary

Manchester, United Kingdom

Royal Hallamshire Hospital

Sheffield, United Kingdom

Clinical Research Directory

A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (103)