Plain-English translation of NCT06198712 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Sickle Cell Disease research guide →Phase 2 — Testing in a bigger group (up to a few hundred people) to see if the treatment actually works and is still safe.
This trial is testing a medication called , which is taken once daily by mouth, in children and teens with sickle cell disease. The study will look at how the medication works in the body, how safe it is, and whether it helps reduce sickle cell symptoms and complications. Researchers will follow participants for nearly 2 years to gather this information.
Sickle cell disease causes pain, organ damage, and serious complications that can require hospitalization. This medication represents a new treatment approach, and researchers need to understand how it works in younger patients and whether it can help reduce the number of painful episodes and other health problems.
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You will take the medication by mouth once every day for 96 weeks (about 2 years), then return for a final visit 4 weeks after that. During the study, you will visit the clinic regularly so doctors can check your blood work, monitor how you are feeling, and watch for any side effects. The research team will track how often you have pain episodes, how long the medication stays in your body, and whether the treatment is helping your sickle cell disease.
AI-generated summary from trial data · Jun 6, 2026 · Not medical advice
Canada
France
Kenya