A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Recruiting · NCT06609226 · Interventional (participants receive a specific treatment) · Lead sponsor: Novo Nordisk A/S
View the official record on ClinicalTrials.gov →Interventions studied
Etavopivat AEtavopivat BEtavopivat C
What this trial is about
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.
Who can take part
Age range
2 Years and older
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 3
Study type
Interventional (participants receive a specific treatment)
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.
Where it is running
60 locations listed across 20 US states.
- 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
- University of Abuja Teaching Hospital, Gwagwalada, Abuja - Abuja, Nigeria
- Ghana Institute of Clinical Genetics, Korle Bu Teaching Hospital (KBTH) - Accra, Ghana
- Baskent Universitesi Adana - Adana, Turkey (Türkiye)
- Başkent Üniversitesi Adana-Hematoloji - Adana, Turkey (Türkiye)
- Alexandria University Hospital - Alexandria, Egypt, Egypt
- Zagazig University Hospital - Alsharkia, Egypt, Egypt
- Hacettepe University Hematology - Ankara, Turkey (Türkiye)
- Hacettepe Üniversitesi Hastanesi- Hematoloji - Ankara, Turkey (Türkiye)
- Hippokration Hospital - Athens, Greece
- Victoria Hospital, Bangalore - Bangalore, India
- Hospital Universitario de Cruces - Barakaldo, Spain
- Hospital Vall d'Hebron - Barcelona, Spain
- Charité - Campus Virchow-Klinikum - Klinik für Pädiatrie mit Schwerpunkt Onkologie und Hämatologie - Berlin, Germany
- 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
- Universitätsklinikum Freiburg - Kinder- und Jugendklinik - Freiburg im Breisgau, Germany
- Nirmal Hospital Pvt. Ltd. - Gujarat, India
- American University of Beirut Medical Centre - Hamra, Lebanon
- Chronic Care Center - Hazmiyeh, Lebanon
Showing 60 of 106 listed sites. See the official record for the complete list.
Read the full protocol, contacts and eligibility on ClinicalTrials.gov →