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

Last updated: April 9, 2025
Sponsor: Novo Nordisk A/S
Overall Status: Active - Recruiting

Phase

3

Condition

Thalassemia

Treatment

Etavopivat B

Etavopivat A

Clinical Study ID

NCT06609226
NN7535-7822
2024-510805-27
U1111-1301-8130
  • All Genders

Study 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

Inclusion

Inclusion Criteria:

  • Participant must have ongoing participation in an etavopivat parent study fortreatment of sickle cell disease (SCD) or thalassaemia and have completed at least atreatment period of the parent study.

  • Participant must have derived clinical benefit from treatment with etavopivat, asdetermined by the investigator.

  • Any participant with dose reduction or temporary discontinuation will need to berechallenged 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 during participation in the parent study (i.e., nochanges to the dose except for changes to weight or age reasons).

  • Have been compliant with the treatment regimen at the discretion of the investigatorduring participation of the parent study.

Exclusion

Exclusion Criteria:

  • Any disorder, except for conditions associated with SCD or thalassaemia, which inthe investigator's opinion might jeopardise participant's safety or compliance withthe protocol.

  • Participant withdrew or had permanent treatment discontinuation from an etavopivatclinical study.

  • Participants on permanent dose reduction or temporary treatment discontinuation.

  • Use of any of the following within the timeframes prior to the transfer visit asstated:

  • Use of voxelotor within participation of the parent study or anticipated need forthis agent during this study.

  • Use of an experimental selectin antagonist (e.g., monoclonal antibody or smallmolecule) within the parent study or anticipated need for such agents during thisstudy.

  • Use of erythropoietin or other haematopoietic growth factor treatment within theparent study or anticipated need for such agents during this study.

  • Receiving or use of concomitant medications that are strong inducers of cytochromeP450 (CYP) 3A4 within 2 weeks of the transfer visit or anticipated need for suchagents during the study.

  • Current participation in a study that is not a designated parent study, or plannedparticipation in any other clinical trial, for the duration of FLORAL.

Study Design

Total Participants: 325
Treatment Group(s): 2
Primary Treatment: Etavopivat B
Phase: 3
Study Start date:
January 10, 2025
Estimated Completion Date:
November 30, 2029

Connect with a study center

  • Providence Hematolgy

    Vancouver, British Columbia V6E 1M7
    Canada

    Site Not Available

  • The Hospital for Sick Children

    Toronto, Ontario M5G 1X8
    Canada

    Site Not Available

  • University Health Network - Toronto General Hospital

    Toronto, Ontario M5G 2C4
    Canada

    Site Not Available

  • CHU Ste-Justine

    Montreal, Quebec H3T 1C5
    Canada

    Site Not Available

  • Alexandria University Hospital

    Alexandria, Egypt, 21131
    Egypt

    Site Not Available

  • Zagazig University Hospital

    Alsharkia, Egypt, 44519
    Egypt

    Site Not Available

  • Faculty of Medicine Ain Shams Medical Research Institute (MASRI)

    Cairo, 0
    Egypt

    Site Not Available

  • Abu El-Reesh El-Mounira Children University Hospital

    Cairo, Egypt, 4241317
    Egypt

    Site Not Available

  • Cairo University

    Cairo, Egypt, 11956
    Egypt

    Site Not Available

  • Ap-Hp-Hopital Henri Mondor

    Créteil, 94000
    France

    Site Not Available

  • Hospices Civils de Lyon-Hopital Edouard Herriot

    Lyon Cedex 03, 69437
    France

    Site Not Available

  • Ap-Hp-Hopital Robert Debre

    Paris, 75019
    France

    Site Not Available

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

    Berlin, 13353
    Germany

    Site Not Available

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

    Berlin, 13353
    Germany

    Site Not Available

  • Universitätsklinikum Freiburg, Kinder- und Jugendklinik

    Freiburg, 79106
    Germany

    Site Not Available

  • Universitätsklinikum Freiburg, Zentrum für Kinder- und Jugendmedizin

    Freiburg, 79106
    Germany

    Site Not Available

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

    AccrA, 0
    Ghana

    Site Not Available

  • Kintampo Health Research Centre (KHRC), Kintampo, Ghana

    Kintampo, 200
    Ghana

    Site Not Available

  • General Hospital Of Larissa Koutlibaneio And Triantafylleio

    Larissa, Thessaly 41221
    Greece

    Site Not Available

  • Hippokration Hospital

    Athens, 11527
    Greece

    Active - Recruiting

  • General University Hospital of Patras

    Patra, 26504
    Greece

    Active - Recruiting

  • 'Ippokrateio' General Hospital of Thessaloniki

    Thessaloniki, 54642
    Greece

    Site Not Available

  • All India Institute of Medical Sciences (AIIMS), Raipur

    Raipur, Chhattisgarh 492099
    India

    Site Not Available

  • Nirmal Hospital Pvt. Ltd.

    Gujarat, 395002
    India

    Site Not Available

  • Suretech Hospital and Research Centre Ltd.

    Maharashtra, 440012
    India

    Site Not Available

  • All India Institute of Medical Sciences_Delhi

    New Delhi, 110029
    India

    Site Not Available

  • Azienda Ospedaliera Universitaria San Luigi Gonzaga

    Orbassano, Torino 10043
    Italy

    Active - Recruiting

  • Azienda Ospedale Universita Padova

    Padova, 35128
    Italy

    Active - Recruiting

  • Fondazione IRCCS Policlinico San Matteo

    Pavia, 27100
    Italy

    Site Not Available

  • KEMRI-Walter-Reed Kericho

    Kericho, 20200
    Kenya

    Site Not Available

  • Ahero Clinical Trials Unit, Kisumu, Kenya

    Kisumu, 40100
    Kenya

    Site Not Available

  • KEMRI Kondele Children Hospital, Kisumu

    Kisumu, 40100
    Kenya

    Site Not Available

  • Kombewa Clinical Research Centre

    Kisumu, 1932
    Kenya

    Site Not Available

  • Gertrude's Children's Hospital, Nairobi

    Nairobi, 00000
    Kenya

    Site Not Available

  • KEMRI Siaya Clinical Research Annex Siaya County Referral Hospital, Siaya

    Siaya, 0000
    Kenya

    Site Not Available

  • American University of Beirut Medical Centre

    Hamra,
    Lebanon

    Site Not Available

  • Chronic Care Center

    Hazmieh, 21211
    Lebanon

    Site Not Available

  • Hospital Nini

    Tripoli, 1434
    Lebanon

    Site Not Available

  • University College Hospital Paediatric Haematology and Oncology Unit, Ibadan

    Ibadan, Oyo State 0000
    Nigeria

    Site Not Available

  • University of Abuja Teaching Hospital, Gwagwalada, Abuja

    Abuja, 228
    Nigeria

    Site Not Available

  • University of Nigeria Teaching Hospital, Enugu

    Enugu, 00000
    Nigeria

    Site Not Available

  • Barau Dikko Teaching Hospital, Kaduna

    Kaduna, 800212
    Nigeria

    Site Not Available

  • Aminu Kano Teaching Hospital (AKTH)

    Kano, 3452
    Nigeria

    Site Not Available

  • Lagos University Teaching Hospital, Lagos

    Lagos, 102215
    Nigeria

    Site Not Available

  • Sultan Qaboos University Hospital

    Muscat, 123
    Oman

    Site Not Available

  • Prince Mohammad Bin Naser Hospital

    Jizan, 82943
    Saudi Arabia

    Site Not Available

  • King Khalid University Hospital

    Riyadh, 12372
    Saudi Arabia

    Site Not Available

  • Hospital Universitario de Cruces

    Baracaldo, 48903
    Spain

    Site Not Available

  • Hospital Vall d'Hebron

    Barcelona, 08035
    Spain

    Active - Recruiting

  • Hospital Universitario La Paz

    Madrid, 28046
    Spain

    Active - Recruiting

  • Baskent Universitesi Adana

    Adana, 01250
    Turkey

    Site Not Available

  • Hacettepe University Hematology

    Ankara, 06230
    Turkey

    Site Not Available

  • Mersin University Medical Faculty Hospital, Hematology

    Mersin, 33110
    Turkey

    Site Not Available

  • Mersin University Medical Faculty Pediatric Hematology

    Mersin, 33110
    Turkey

    Site Not Available

  • Imperial College London

    London, W12 0NN
    United Kingdom

    Site Not Available

  • Kings College Hospital

    London, SE5 9RS
    United Kingdom

    Site Not Available

  • Manchester Royal Infirmary_Manchester_0

    Manchester, M13 9WL
    United Kingdom

    Site Not Available

  • Royal Hallamshire Hospital

    Sheffield, S10 2JF
    United Kingdom

    Active - Recruiting

  • Univ of Alabama Birmingham

    Birmingham, Alabama 35233
    United States

    Active - Recruiting

  • Phoenix Children's Hsptl

    Phoenix, Arizona 85016
    United States

    Site Not Available

  • University Of California Irvine

    Irvine, California 92697
    United States

    Site Not Available

  • Children's Hospital Los Angeles - Endocrinology

    Los Angeles, California 90027
    United States

    Active - Recruiting

  • UCSF Oakland Benioff ChildHosp

    Oakland, California 94609
    United States

    Site Not Available

  • Children's Hosp Of Orange

    Orange, California 92868
    United States

    Site Not Available

  • University Of California Irvine

    Orange, California 92868
    United States

    Active - Recruiting

  • University of Connecticut

    Farmington, Connecticut 06030
    United States

    Site Not Available

  • Children's National Medical Center

    Washington, District of Columbia 20010
    United States

    Site Not Available

  • Foundation for Sickle Cell Disease Research

    Hollywood, Florida 33021
    United States

    Site Not Available

  • Univ Miami-Miller School Med

    Miami, Florida 33136
    United States

    Site Not Available

  • Univ of Miami/SCCC

    Miami, Florida 33136
    United States

    Site Not Available

  • Children's Healthcare Atlanta

    Atlanta, Georgia 30329
    United States

    Site Not Available

  • Emory University School of Medicine

    Atlanta, Georgia 30303
    United States

    Site Not Available

  • Center for Blood Disorders Augusta University

    Augusta, Georgia 30912
    United States

    Site Not Available

  • Children's Hosp-New Orleans

    New Orleans, Louisiana 70118
    United States

    Site Not Available

  • Boston Medical Center

    Boston, Massachusetts 02118
    United States

    Active - Recruiting

  • Washington University-St.Louis

    Saint Louis, Missouri 63110
    United States

    Site Not Available

  • Jacobi Medical Center

    Bronx, New York 10461
    United States

    Active - Recruiting

  • Montefiore Medical Center

    Bronx, New York 10467
    United States

    Site Not Available

  • NYC Health+Hospitals

    Brooklyn, New York 11203
    United States

    Active - Recruiting

  • Columbia University Medical Center_New York_0

    New York, New York 10032
    United States

    Site Not Available

  • Weill Cornell Med Coll-NYPH

    New York, New York 10065
    United States

    Site Not Available

  • Duke University_Durham

    Durham, North Carolina 27705
    United States

    Site Not Available

  • East Carolina University_Greenville

    Greenville, North Carolina 27834
    United States

    Site Not Available

  • Atrium Health-Wake Forest Bapt

    Winston-Salem, North Carolina 27157
    United States

    Site Not Available

  • Cincinnati Child's Hsp Med Ctr

    Cincinnati, Ohio 45229
    United States

    Active - Recruiting

  • Neuro-Behavioral Clinical Research

    North Canton, Ohio 44720
    United States

    Active - Recruiting

  • Medical University Of South Carolina_Charleston

    Charleston, South Carolina 29425
    United States

    Site Not Available

  • East Carolina Univ-Greenville

    Greenville, South Carolina 27858
    United States

    Site Not Available

  • East Carolina University_Greenville_0

    Greenville, South Carolina 27858
    United States

    Site Not Available

  • UTHSC-Memphis

    Memphis, Tennessee 38104
    United States

    Site Not Available

  • Texas Children's Hospital_Houston

    Houston, Texas 77030
    United States

    Site Not Available

  • UT Health University of Texas

    Houston, Texas 77030
    United States

    Site Not Available

  • Virginia Comm Univ Medical Ctr

    Richmond, Virginia 23298
    United States

    Site Not Available

  • Mary Bridge Children's Health

    Tacoma, Washington 98405
    United States

    Active - Recruiting

  • Versiti, CCBD_Milwaukee

    Milwaukee, Wisconsin 53226
    United States

    Site Not Available

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