A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
NCT ID: NCT06609226
Last Updated: 2025-12-30
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
480 participants
INTERVENTIONAL
2025-01-10
2030-12-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Participants greater than or equal to (≥) 12 years old with sickle cell disease
Participants will receive an oral dose of Etavopivat A or C.
Etavopivat A
Participants will receive an oral dose of Etavopivat A.
Etavopivat C
Participants will receive an oral dose of Etavopivat C.
Participants ≥ 12 years old with sickle cell disease transfusion dependent
Participants will receive an oral dose of Etavopivat A or C.
Etavopivat A
Participants will receive an oral dose of Etavopivat A.
Etavopivat C
Participants will receive an oral dose of Etavopivat C.
Participants ≥ 12 years old with transfusion-dependent thalassaemia
Participants will receive an oral dose of Etavopivat A or C.
Etavopivat A
Participants will receive an oral dose of Etavopivat A.
Etavopivat C
Participants will receive an oral dose of Etavopivat C.
Participants ≥ 12 years old with non-transfusion dependent thalassaemia
Participants will receive an oral dose of Etavopivat A or C.
Etavopivat A
Participants will receive an oral dose of Etavopivat A.
Etavopivat C
Participants will receive an oral dose of Etavopivat C.
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.
Etavopivat A
Participants will receive an oral dose of Etavopivat A.
Etavopivat B
Participants will receive an oral dose of Etavopivat B.
Etavopivat C
Participants will receive an oral dose of Etavopivat C.
Interventions
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Etavopivat A
Participants will receive an oral dose of Etavopivat A.
Etavopivat B
Participants will receive an oral dose of Etavopivat B.
Etavopivat C
Participants will receive an oral dose of Etavopivat C.
Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
2 Years
ALL
No
Sponsors
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Novo Nordisk A/S
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Transparency (dept. 2834)
Role: STUDY_DIRECTOR
Novo Nordisk A/S
Locations
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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
Ghana Institute of Clinical Genetics, Korle Bu Teaching Hospital (KBTH)
Accra, , Ghana
Kintampo Health Research Centre (KHRC), Kintampo, Ghana
Kintampo, , 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
Padua, , 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
Ahero Clinical Trials Unit, Kisumu, Kenya
Kisumu, , Kenya
KEMRI Kondele Children Hospital, Kisumu
Kisumu, , Kenya
American University of Beirut Medical Centre
Hamra, , Lebanon
Chronic Care Center
Hazmiyeh, , Lebanon
Hospital Nini
Tripoli, , Lebanon
University College Hospital Paediatric Haematology and Oncology Unit, Ibadan
Ibadan, Oyo State, Nigeria
University of Abuja Teaching Hospital, Gwagwalada, Abuja
Abuja, , Nigeria
University of Nigeria Teaching Hospital, Enugu
Enugu, , 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)
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
Countries
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Central Contacts
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Other Identifiers
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NN7535-7822
Identifier Type: -
Identifier Source: org_study_id
U1111-1301-8130
Identifier Type: OTHER
Identifier Source: secondary_id
2024-510805-27
Identifier Type: OTHER
Identifier Source: secondary_id