A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Phase 3
Recruiting
- Conditions
- Sickle Cell DiseaseThalassemia
- 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
- 480
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.
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 or C. Participants greater than or equal to (≥) 12 years old with sickle cell disease Etavopivat C Participants will receive an oral dose of Etavopivat A or C. Participants ≥ 12 years old with sickle cell disease transfusion dependent Etavopivat A Participants will receive an oral dose of Etavopivat A or C. Participants ≥ 12 years old with sickle cell disease transfusion dependent Etavopivat C Participants will receive an oral dose of Etavopivat A or C. Participants ≥ 12 years old with transfusion-dependent thalassaemia Etavopivat A Participants will receive an oral dose of Etavopivat A or C. Participants ≥ 12 years old with transfusion-dependent thalassaemia Etavopivat C Participants will receive an oral dose of Etavopivat A or C. Participants ≥ 12 years old with non-transfusion dependent thalassaemia Etavopivat A Participants will receive an oral dose of Etavopivat A or C. Participants ≥ 12 years old with non-transfusion dependent thalassaemia Etavopivat C Participants will receive an oral dose of Etavopivat A or C. Participants ≥ 2 years 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 or C and participants \< 12 years of age will receive an oral dose of Etavopivat B. Participants ≥ 2 years 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 or C and participants \< 12 years of age will receive an oral dose of Etavopivat B. Participants ≥ 2 years to less than (<) 12 years old with sickle cell disease Etavopivat C 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.
- 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) up to end of study (up to week 316) Measured as number of events.
Number of adverse reactions, reported for each indication and age group separately Baseline (week 0 of FLORAL) up to end of study (up to week 316) 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) up to end of treatment (up to week 312) Measured as count.
Change in VOCs, reported for each age group separately Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312) Measured as count.
Change in hemoglobin (Hb) concentration, reported for each age group separately Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312) Measured as grams per deciliter (g/dL).
Annualised number of hospitalisations, reported for each age group separately Baseline (week 0 of FLORAL) up to end of treatment (up to week 312) Measured as count.
Average length of stay of hospitalisations, reported for each age group separately Baseline (week 0 of FLORAL) up to end of treatment (up to week 312) Measured as days.
Change in Hb concentration Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312) Measured as g/dL.
Number of red blood cell (RBC) units transfused, reported for each indication separately Baseline (week 0 of FLORAL) up to end of treatment (up to week 312) Measured as units.
Change in RBC units transfused, reported for each indication separately Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312) Measured as units.
Trial Locations
- Locations (97)
Başkent Üniversitesi Adana Dr. Turgut Noyan Uygulama ve Araştırma Merkezi
Adana, Turkey (Türkiye)
King's College Hospital - Paediatric Research
🇬🇧London, United Kingdom
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 D.C., District of Columbia, United States
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