An 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
Novo Nordisk A/S
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.
Eligibility
- Age range
- 2+ years
- Sex
- All
- Healthy volunteers
- No
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…
Interventions
- DrugEtavopivat A
Participants will receive an oral dose of Etavopivat A.
- DrugEtavopivat B
Participants will receive an oral dose of Etavopivat B.
- DrugEtavopivat C
Participants will receive an oral dose of Etavopivat C.
Locations (103)
- Univ of Alabama BirminghamBirmingham, Alabama
- Phoenix Children's HsptlPhoenix, Arizona
- Children's Hospital Los Angeles - EndocrinologyLos Angeles, California
- Children's Hospital Los Angeles - EndocrinologyLos Angeles, California
- UCSF Oakland Benioff ChildHospOakland, California
- UCSF Oakland Benioff ChildHospOakland, California