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NCT06609226PHASE3Recruiting

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

Novo Nordisk A/S

Start Date

1/10/2025

Completion Date

12/30/2030

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.

Eligibility Criteria

Age Range: 2 years to No maximum

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.

Interventions

DRUG

Etavopivat A

DRUG

Etavopivat B

DRUG

Etavopivat C

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Conditions

Sickle Cell DiseaseThalassemia

Locations

Univ of Alabama Birmingham

Birmingham, Alabama 35233

United States

Phoenix Children's Hsptl

Phoenix, Arizona 85016

United States

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California 90027

United States

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California 90027

United States

UCSF Oakland Benioff ChildHosp

Oakland, California 94609

United States

UCSF Oakland Benioff ChildHosp

Oakland, California 94609

United States

Children's Hosp Of Orange

Orange, California 92868

United States

University Of California Irvine

Orange, California 92868

United States

University of Connecticut

Farmington, Connecticut 06030

United States

Children's National Medical Center

Washington D.C., District of Columbia 20010

United States

Foundation for Sickle Cell Disease Research

Hollywood, Florida 33023

United States

Univ of Miami/SCCC

Miami, Florida 33136

United States

Emory University School of Medicine

Atlanta, Georgia 30303

United States

Children's Healthcare Atlanta

Atlanta, Georgia 30329

United States

Center for Blood Disorders Augusta University

Augusta, Georgia 30912

United States

Univer Of Illinois at Chicago

Chicago, Illinois 60612

United States

Children's Hosp-New Orleans

New Orleans, Louisiana 70118

United States

Boston Medical Center

Boston, Massachusetts 02118

United States

Washington University-St.Louis

St Louis, Missouri 63110

United States

NYC Health+Hospitals

Brooklyn, New York 11203

United States

Columbia University Medical Center_New York_0

New York, New York 10032

United States

Columbia University Medical Center_New York_0

New York, New York 10032

United States

Weill Cornell Med Coll-NYPH

New York, New York 10065

United States

Jacobi Medical Center

The Bronx, New York 10461

United States

Montefiore Medical Center

The Bronx, New York 10467

United States

Atrium Levine Children's/Atrium Health

Charlotte, North Carolina 28204

United States

Duke University_Durham

Durham, North Carolina 27705

United States

East Carolina Univ-Greenville

Greenville, North Carolina 27834

United States

East Carolina University_Greenville

Greenville, North Carolina 27834

United States

Atrium Health-Wake Forest Bapt

Winston-Salem, North Carolina 27157

United States

Cincinnati Child's Hsp Med Ctr

Cincinnati, Ohio 45229

United States

Neuro-Behavioral Clinical Research

North Canton, Ohio 44720

United States

Medical University Of South Carolina_Charleston

Charleston, South Carolina 29425

United States

Methodist University Hospital

Memphis, Tennessee 38104

United States

Texas Children's Hospital_Houston

Houston, Texas 77030

United States

UT Health University of Texas

Houston, Texas 77030

United States

Virginia Comm Univ Medical Ctr

Richmond, Virginia 23298

United States

Mary Bridge Children's Health

Tacoma, Washington 98405

United States

Versiti, CCBD_Milwaukee

Milwaukee, Wisconsin 53226

United States

St Pauls Hospital

Vancouver, British Columbia V6Z1Y6

Canada

The Hospital for Sick Children

Toronto, Ontario M5G 1X8

Canada

University Health Network - Toronto General Hospital

Toronto, Ontario M5G 2C4

Canada

CHU Ste-Justine

Montreal, Quebec H3T 1C5

Canada

Alexandria University Hospital

Alexandria, Egypt, 21131

Egypt

Zagazig University Hospital

Alsharkia, Egypt, 44519

Egypt

Faculty of Medicine Ain Shams Medical Research Institute (MASRI)

Cairo, 1181

Egypt

Cairo University

Cairo, Egypt, 11956

Egypt

Abu El-Reesh El-Mounira Children University Hospital

Cairo, Egypt, 4241317

Egypt

Ap-Hp-Hopital Henri Mondor

Créteil, 94000

France

Hospices Civils de Lyon-Hopital Edouard Herriot

Lyon, 69437

France

Ap-Hp-Hopital Robert Debre

Paris, 75019

France

Ap-Hp-Hopital Robert Debre

Paris, 75019

France

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

Berlin, 13353

Germany

Universitätsklinikum Freiburg - Kinder- und Jugendklinik

Freiburg im Breisgau, 79106

Germany

Kintampo Health Research Centre (KHRC)

Kintampo, Bono East 27WF+98M

Ghana

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

Accra, 0

Ghana

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

Larissa, Thessaly 41221

Greece

Hippokration Hospital

Athens, 11527

Greece

General University Hospital of Patras

Pátrai, 26504

Greece

'Ippokrateio' General Hospital of Thessaloniki

Thessaloniki, 54642

Greece

All India Institute of Medical Sciences (AIIMS), Raipur

Raipur, Chhattisgarh 492099

India

K.J Somaiya Hospital and Research Centre

Mumbai, Maharashtra 400022

India

Victoria Hospital, Bangalore

Bangalore, 560002

India

Nirmal Hospital Pvt. Ltd.

Gujarat, 395002

India

Suretech Hospital and Research Centre Ltd.

Maharashtra, 440012

India

All India Institute of Medical Sciences_Delhi

New Delhi, 110029

India

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

Orbassano, Torino 10043

Italy

Azienda Ospedale Universita Padova

Padova, 35128

Italy

Fondazione IRCCS Policlinico San Matteo

Pavia, 27100

Italy

Gertrude's Children's Hospital

Nairobi, Nairobi County 00509

Kenya

KEMRI CRDR Siaya Clinical Research Annex, Country Referral Hospital

Siaya, Siaya County 40600

Kenya

KEMRI-Walter-Reed Kericho

Kericho, 20200

Kenya

Kombewa Clinical Research Centre

Kisumu, 1932

Kenya

KEMRI Kondele Children Hospital, Kisumu

Kisumu, 40100

Kenya

Ahero Clinical Trials Unit

Kisumu, 40101

Kenya

American University of Beirut Medical Centre

Hamra, 11-0236

Lebanon

Chronic Care Center

Hazmiyeh, 21211

Lebanon

Hospital Nini

Tripoli, 1434

Lebanon

University of Nigeria Teaching Hospital (UNTH)

Ituku-Ozalla, Enugu State 400001

Nigeria

University College Hospital Paediatric Haematology and Oncology Unit, Ibadan

Ibadan, Oyo State 0000

Nigeria

University of Abuja Teaching Hospital, Gwagwalada, Abuja

Abuja, 228

Nigeria

Barau Dikko Teaching Hospital, Kaduna

Kaduna, 800212

Nigeria

Aminu Kano Teaching Hospital (AKTH)

Kano, 3452

Nigeria

Lagos University Teaching Hospital, Lagos

Lagos, 102215

Nigeria

Sultan Qaboos University Hospital

Muscat, Sultanet of Oman/Muscat/Al Khoud 123

Oman

Prince Mohammad Bin Naser Hospital

Jizan, 82943

Saudi Arabia

King Khalid University Hospital

Riyadh, 12372

Saudi Arabia

Hospital Universitario de Cruces

Barakaldo, 48903

Spain

Hospital Vall d'Hebron

Barcelona, 08035

Spain

Hospital Universitario La Paz

Madrid, 28046

Spain

Baskent Universitesi Adana

Adana, 01250

Turkey (Türkiye)

Başkent Üniversitesi Adana-Hematoloji

Adana, 01250

Turkey (Türkiye)

Hacettepe University Hematology

Ankara, 06230

Turkey (Türkiye)

Hacettepe Üniversitesi Hastanesi- Hematoloji

Ankara, 06230

Turkey (Türkiye)

Mersin University Medical Faculty Pediatric Hematology

Mersin, 33110

Turkey (Türkiye)

Mersin Üniversitesi Tip Fakültesi Hastanesi- Çiftlikköy Yerleşkesi- Hematoloji

Mersin, 33110

Turkey (Türkiye)

VM Medical Park Mersin Hastanesi- Hematoloji

Mersin, 33200

Turkey (Türkiye)

Evelina London Children's Hospital - Haemophilia

London, SE1 7EH

United Kingdom

Guy's Hospital - Haematology

London, SE1 9RT

United Kingdom

Guy's Hospital

London, SE1 9RT

United Kingdom

King's College Hospital - Paediatric Research

London, SE5 9RS

United Kingdom

Kings College Hospital - Haematology

London, SE5 9RS

United Kingdom

Imperial College London

London, W12 0NN

United Kingdom

Manchester Royal Infirmary

Manchester, M13 9WL

United Kingdom

Royal Hallamshire Hospital

Sheffield, S10 2JF

United Kingdom