Testing etavopivat in children with sickle cell disease
Treatments studied
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a new drug, etavopivat, to see how it works in children with sickle cell disease. It may help reduce painful crises and other complications.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must be between 6 months and 18 years old.
- They must have a confirmed diagnosis of sickle cell disease (like HbSS or HbSβ0-thalassemia).
- Their hemoglobin level must be between 5.5 and 10.5 g/dL.
- They must have had at least 2 sickle cell pain crises in the last year, or another serious complication like hospitalization or protein in urine.
- They cannot be on regular blood transfusions or have had a blood transfusion in the last 30 days.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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This study looks at the long-term safety and benefits of taking etavopivat for people with sickle cell disease or thalassaemia who have already been helped by it in a previous trial. It aims to see if staying on the treatment continues to be safe and effective.
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