A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
- Conditions
- Sickle Cell Disease, Thalassemia
- Interventions
- Registration Number
- NCT06609226
- Lead Sponsor
- Novo Nordisk A/S
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 325
- 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 rechallenged 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., no changes to the dose except for changes to weight or age reasons).
- Have been compliant with the treatment regimen at the discretion of the investigator during participation of the parent study.
- 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 or temporary treatment discontinuation.
- Use of any of the following within the timeframes prior to the transfer visit as stated:
- Use of voxelotor 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 within the parent study or anticipated need for such agents 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 trial, for the duration of FLORAL.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Participants greater than or equal to (≥) 12 years old with sickle cell disease Etavopivat A Participants will receive an oral dose of Etavopivat A. Participants ≥ 12 years old with sickle cell disease on chronic red blood cell (RBC) transfusions Etavopivat A Participants will receive an oral dose of Etavopivat A. Participants ≥ 12 years old with transfusion-dependent thalassaemia Etavopivat A Participants will receive an oral dose of Etavopivat A. Participants ≥ 12 years old with non-transfusion dependent thalassaemia Etavopivat A Participants will receive an oral dose of Etavopivat A. Participants ≥ 11 months to less than (<) 12 years old with sickle cell disease Etavopivat A Participants ≥ 12 years of age will receive an oral dose of Etavopivat A and participants \< 12 years of age will receive an oral dose of Etavopivat B. Participants ≥ 11 months to less than (<) 12 years old with sickle cell disease Etavopivat B Participants ≥ 12 years of age will receive an oral dose of Etavopivat A and participants \< 12 years of age will receive an oral dose of Etavopivat B.
- Primary Outcome Measures
Name Time Method Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately Baseline (week 0 of FLORAL) to end of study (week 264, or earlier) Measured as number of events.
Number of adverse reactions, reported for each indication and age group separately Baseline (week 0 of FLORAL) to end of study (week 264, or earlier) Measured as number of adverse reactions.
- Secondary Outcome Measures
Name Time Method Annualised vaso-occlusive crisis (VOC) rates, reported for each age group separately Baseline (week 0 of FLORAL) to end of treatment at week 260, or earlier Measured as count.
Change in VOCs, reported for each age group separately Baseline (of parent study) to end of treatment at week 260, or earlier Measured as count.
Change in hemoglobin (Hb) concentration, reported for each age group separately Baseline (of parent study) to end of treatment at week 260, or earlier Measured as grams per deciliter (g/dL).
Annualised number of hospitalisations, reported for each age group separately Baseline (week 0 of FLORAL) to end of treatment at week 260, or earlier Measured as count.
Average length of stay of hospitalisations, reported for each age group separately Baseline (week 0 of FLORAL) to end of treatment at week 260, or earlier Measured as days.
Change in Hb concentration Baseline (of parent study) to end of treatment at week 260, or earlier Measured as g/dL.
Number of red blood cell (RBC) units transfused, reported for each indication separately Baseline (week 0 of FLORAL) to end of treatment at week 260, or earlier Measured as units.
Change in RBC units transfused, reported for each indication separately Baseline (of parent study) to end of treatment at week 260, or earlier Measured as units.
Trial Locations
- Locations (93)
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
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, 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
🇺🇸Saint Louis, Missouri, United States
Jacobi Medical Center
🇺🇸Bronx, New York, United States
Montefiore Medical Center
🇺🇸Bronx, New York, United States
NYC Health+Hospitals
🇺🇸Brooklyn, New York, 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
Columbia University Medical Center_New York_0
🇺🇸New York, New York, United States
Weill Cornell Med Coll-NYPH
🇺🇸New York, New York, 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
UTHSC-Memphis
🇺🇸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
Providence Hematolgy
🇨🇦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
Cairo University
🇪🇬Cairo, Egypt, Egypt
Abu El-Reesh El-Mounira Children University Hospital
🇪🇬Cairo, Egypt, Egypt
Faculty of Medicine Ain Shams Medical Research Institute (MASRI)
🇪🇬Cairo, Egypt
Ap-Hp-Hopital Henri Mondor
🇫🇷Créteil, France
Hospices Civils de Lyon-Hopital Edouard Herriot
🇫🇷Lyon Cedex 03, 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, 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
🇬🇷Larissa, Thessaly, Greece
Hippokration Hospital
🇬🇷Athens, Greece
General University Hospital of Patras
🇬🇷Patra, 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
🇮🇹Orbassano, Torino, Italy
Azienda Ospedale Universita Padova
🇮🇹Padova, Italy
Fondazione IRCCS Policlinico San Matteo
🇮🇹Pavia, Italy
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
Gertrude's Children's Hospital, Nairobi
🇰🇪Nairobi, Kenya
KEMRI Siaya Clinical Research Annex Siaya County Referral Hospital, Siaya
🇰🇪Siaya, Kenya
American University of Beirut Medical Centre
🇱🇧Hamra, Lebanon
Chronic Care Center
🇱🇧Hazmieh, 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, Oman
Prince Mohammad Bin Naser Hospital
🇸🇦Jizan, Saudi Arabia
King Khalid University Hospital
🇸🇦Riyadh, Saudi Arabia
Hospital Universitario de Cruces
🇪🇸Baracaldo, Spain
Hospital Vall d'Hebron
🇪🇸Barcelona, Spain
Hospital Universitario La Paz
🇪🇸Madrid, Spain
Baskent Universitesi Adana
🇹🇷Adana, Turkey
Hacettepe University Hematology
🇹🇷Ankara, Turkey
Mersin University Medical Faculty Hospital, Hematology
🇹🇷Mersin, Turkey
Mersin University Medical Faculty Pediatric Hematology
🇹🇷Mersin, Turkey
Kings College Hospital
🇬🇧London, United Kingdom
Imperial College London
🇬🇧London, United Kingdom
Manchester Royal Infirmary_Manchester_0
🇬🇧Manchester, United Kingdom
Royal Hallamshire Hospital
🇬🇧Sheffield, United Kingdom