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

Recruitment Status
RECRUITING
(See Contacts and Locations)Verified February 2026 by Novo Nordisk A/S
Sponsor
Novo Nordisk A/S
Information Provided by (Responsible Party)
Novo Nordisk A/S
Clinicaltrials.gov Identifier
NCT06609226
Other Study ID Numbers:
NN7535-7822
First Submitted
September 19, 2024
First Posted
September 23, 2024
Last Update Posted
March 23, 2026
Last Verified
February 2026

ClinicalTrials.gov processed this data on March 2026. Link to the current ClinicalTrials.gov record .

History of Changes

Study Details

Study Description

Condition or DiseaseIntervention/Treatment
Sickle Cell DiseaseThalassemia
Drug: Etavopivat ADrug: Etavopivat ADrug: Etavopivat ADrug: Etavopivat ADrug: Etavopivat A

Study Design

Study TypeInterventional
Actual Enrollment480 participants
Design AllocationNon-Randomized
Interventional ModelParallel Assignment
MaskingNone (Open Label)
Primary PurposeTreatment
Official TitleAn 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
Study Start DateJanuary 9, 2025
Actual Primary Completion Date4yrs 7mos from now
Actual Study Completion Date4yrs 7mos from now

Groups and Cohorts

Group/CohortIntervention/Treatment
Participants greater than or equal to (≥) 12 years old with sickle cell disease
Participants will receive an oral dose of Etavopivat A or C.
Drug: Etavopivat A
Participants will receive an oral dose of Etavopivat A.
Participants ≥ 12 years old with sickle cell disease transfusion dependent
Participants will receive an oral dose of Etavopivat A or C.
Drug: Etavopivat A
Participants will receive an oral dose of Etavopivat A.
Participants ≥ 12 years old with transfusion-dependent thalassaemia
Participants will receive an oral dose of Etavopivat A or C.
Drug: Etavopivat A
Participants will receive an oral dose of Etavopivat A.
Participants ≥ 12 years old with non-transfusion dependent thalassaemia
Participants will receive an oral dose of Etavopivat A or C.
Drug: Etavopivat A
Participants will receive an oral dose of Etavopivat A.
Participants ≥ 2 years to less than (<) 12 years old with sickle cell disease
Participants ≥ 12 years of age will receive an oral dose of Etavopivat A or C and participants \< 12 years of age will receive an oral dose of Etavopivat B.
Drug: Etavopivat A
Participants will receive an oral dose of Etavopivat A.

Outcome Measures

Primary Outcome Measures
  1. Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately
    Measured as number of events.
  2. Number of adverse reactions, reported for each indication and age group separately
    Measured as number of adverse reactions.
Secondary Outcome Measures
  1. Annualised vaso-occlusive crisis (VOC) rates, reported for each age group separately
    Measured as count.
  2. Change in VOCs, reported for each age group separately
    Measured as count.
  3. Change in hemoglobin (Hb) concentration, reported for each age group separately
    Measured as grams per deciliter (g/dL).
  4. Annualised number of hospitalisations, reported for each age group separately
    Measured as count.
  5. Average length of stay of hospitalisations, reported for each age group separately
    Measured as days.
  6. Change in Hb concentration
    Measured as g/dL.
  7. Number of red blood cell (RBC) units transfused, reported for each indication separately
    Measured as units.
  8. Change in RBC units transfused, reported for each indication separately
    Measured as units.

Eligibility Criteria

Ages Eligible for Study(Child, Adult, Older Adult)
Sexes Eligible for StudyAll
Accepts Healthy VolunteersNo
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.

Contacts and Locations

Sponsors and CollaboratorsNovo Nordisk A/S
Locations
Univ of Alabama Birmingham | Birmingham Alabama, United States, 35233Phoenix Children's Hsptl | Phoenix Arizona, United States, 85016Children's Hospital Los Angeles - Endocrinology | Los Angeles California, United States, 90027Children's Hospital Los Angeles - Endocrinology | Los Angeles California, United States, 90027UCSF Oakland Benioff ChildHosp | Oakland California, United States, 94609UCSF Oakland Benioff ChildHosp | Oakland California, United States, 94609Children's Hosp Of Orange | Orange California, United States, 92868University Of California Irvine | Orange California, United States, 92868University of Connecticut | Farmington Connecticut, United States, 06030Children's National Medical Center | Washington D.C. District of Columbia, United States, 20010Foundation for Sickle Cell Disease Research | Hollywood Florida, United States, 33023Univ of Miami/SCCC | Miami Florida, United States, 33136Emory University School of Medicine | Atlanta Georgia, United States, 30303Children's Healthcare Atlanta | Atlanta Georgia, United States, 30329Center for Blood Disorders Augusta University | Augusta Georgia, United States, 30912Univer Of Illinois at Chicago | Chicago Illinois, United States, 60612Children's Hosp-New Orleans | New Orleans Louisiana, United States, 70118Boston Medical Center | Boston Massachusetts, United States, 02118Washington University-St.Louis | St Louis Missouri, United States, 63110NYC Health+Hospitals | Brooklyn New York, United States, 11203Columbia University Medical Center_New York_0 | New York New York, United States, 10032Columbia University Medical Center_New York_0 | New York New York, United States, 10032Weill Cornell Med Coll-NYPH | New York New York, United States, 10065Jacobi Medical Center | The Bronx New York, United States, 10461Montefiore Medical Center | The Bronx New York, United States, 10467Atrium Levine Children's/Atrium Health | Charlotte North Carolina, United States, 28204Duke University_Durham | Durham North Carolina, United States, 27705East Carolina Univ-Greenville | Greenville North Carolina, United States, 27834East Carolina University_Greenville | Greenville North Carolina, United States, 27834Atrium Health-Wake Forest Bapt | Winston-Salem North Carolina, United States, 27157Cincinnati Child's Hsp Med Ctr | Cincinnati Ohio, United States, 45229Neuro-Behavioral Clinical Research | North Canton Ohio, United States, 44720Medical University Of South Carolina_Charleston | Charleston South Carolina, United States, 29425Methodist University Hospital | Memphis Tennessee, United States, 38104Texas Children's Hospital_Houston | Houston Texas, United States, 77030UT Health University of Texas | Houston Texas, United States, 77030Virginia Comm Univ Medical Ctr | Richmond Virginia, United States, 23298Mary Bridge Children's Health | Tacoma Washington, United States, 98405Versiti, CCBD_Milwaukee | Milwaukee Wisconsin, United States, 53226St Pauls Hospital | Vancouver British Columbia, Canada, V6Z1Y6The Hospital for Sick Children | Toronto Ontario, Canada, M5G 1X8University Health Network - Toronto General Hospital | Toronto Ontario, Canada, M5G 2C4CHU Ste-Justine | Montreal Quebec, Canada, H3T 1C5Alexandria University Hospital | Alexandria, Egypt , Egypt, 21131Zagazig University Hospital | Alsharkia, Egypt , Egypt, 44519Faculty of Medicine Ain Shams Medical Research Institute (MASRI) | Cairo , Egypt, 1181Cairo University | Cairo, Egypt , Egypt, 11956Abu El-Reesh El-Mounira Children University Hospital | Cairo, Egypt , Egypt, 4241317Ap-Hp-Hopital Henri Mondor | Créteil , France, 94000Hospices Civils de Lyon-Hopital Edouard Herriot | Lyon , France, 69437Ap-Hp-Hopital Robert Debre | Paris , France, 75019Ap-Hp-Hopital Robert Debre | Paris , France, 75019Charité - Campus Virchow-Klinikum - Klinik für Pädiatrie mit Schwerpunkt Onkologie und Hämatologie | Berlin , Germany, 13353Universitätsklinikum Freiburg - Kinder- und Jugendklinik | Freiburg im Breisgau , Germany, 79106Kintampo Health Research Centre (KHRC) | Kintampo Bono East, Ghana, 27WF+98MGhana Institute of Clinical Genetics, Korle Bu Teaching Hospital (KBTH) | Accra , Ghana, 0General Hospital Of Larissa Koutlibaneio And Triantafylleio - Thalassemia and SCD Unit | Larissa Thessaly, Greece, 41221Hippokration Hospital | Athens , Greece, 11527General University Hospital of Patras | Pátrai , Greece, 26504'Ippokrateio' General Hospital of Thessaloniki | Thessaloniki , Greece, 54642All India Institute of Medical Sciences (AIIMS), Raipur | Raipur Chhattisgarh, India, 492099K.J Somaiya Hospital and Research Centre | Mumbai Maharashtra, India, 400022Victoria Hospital, Bangalore | Bangalore , India, 560002Nirmal Hospital Pvt. Ltd. | Gujarat , India, 395002Suretech Hospital and Research Centre Ltd. | Maharashtra , India, 440012All India Institute of Medical Sciences_Delhi | New Delhi , India, 110029Azienda Ospedaliera Universitaria San Luigi Gonzaga - S.C.D.O. Microcitemie e malattie rare ematologiche | Orbassano Torino, Italy, 10043Azienda Ospedale Universita Padova | Padova , Italy, 35128Fondazione IRCCS Policlinico San Matteo | Pavia , Italy, 27100Gertrude's Children's Hospital | Nairobi Nairobi County, Kenya, 00509KEMRI CRDR Siaya Clinical Research Annex, Country Referral Hospital | Siaya Siaya County, Kenya, 40600KEMRI-Walter-Reed Kericho | Kericho , Kenya, 20200Kombewa Clinical Research Centre | Kisumu , Kenya, 1932KEMRI Kondele Children Hospital, Kisumu | Kisumu , Kenya, 40100Ahero Clinical Trials Unit | Kisumu , Kenya, 40101American University of Beirut Medical Centre | Hamra , Lebanon, 11-0236Chronic Care Center | Hazmiyeh , Lebanon, 21211Hospital Nini | Tripoli , Lebanon, 1434University of Nigeria Teaching Hospital (UNTH) | Ituku-Ozalla Enugu State, Nigeria, 400001University College Hospital Paediatric Haematology and Oncology Unit, Ibadan | Ibadan Oyo State, Nigeria, 0000University of Abuja Teaching Hospital, Gwagwalada, Abuja | Abuja , Nigeria, 228Barau Dikko Teaching Hospital, Kaduna | Kaduna , Nigeria, 800212Aminu Kano Teaching Hospital (AKTH) | Kano , Nigeria, 3452Lagos University Teaching Hospital, Lagos | Lagos , Nigeria, 102215Sultan Qaboos University Hospital | Muscat Sultanet of Oman/Muscat/Al Khoud, Oman, 123Prince Mohammad Bin Naser Hospital | Jizan , Saudi Arabia, 82943King Khalid University Hospital | Riyadh , Saudi Arabia, 12372Hospital Universitario de Cruces | Barakaldo , Spain, 48903Hospital Vall d'Hebron | Barcelona , Spain, 08035Hospital Universitario La Paz | Madrid , Spain, 28046Baskent Universitesi Adana | Adana , Turkey (Türkiye), 01250Başkent Üniversitesi Adana-Hematoloji | Adana , Turkey (Türkiye), 01250Hacettepe University Hematology | Ankara , Turkey (Türkiye), 06230Hacettepe Üniversitesi Hastanesi- Hematoloji | Ankara , Turkey (Türkiye), 06230Mersin University Medical Faculty Pediatric Hematology | Mersin , Turkey (Türkiye), 33110Mersin Üniversitesi Tip Fakültesi Hastanesi- Çiftlikköy Yerleşkesi- Hematoloji | Mersin , Turkey (Türkiye), 33110Guy's Hospital - Haematology | London , United Kingdom, SE1 9RTGuy's Hospital | London , United Kingdom, SE1 9RTKing's College Hospital - Paediatric Research | London , United Kingdom, SE5 9RSKings College Hospital - Haematology | London , United Kingdom, SE5 9RSImperial College London | London , United Kingdom, W12 0NNManchester Royal Infirmary | Manchester , United Kingdom, M13 9WLRoyal Hallamshire Hospital | Sheffield , United Kingdom, S10 2JF
Investigators
Study Director: Clinical Transparency (dept. 2834), Novo Nordisk A/S