YOLT-204 for sickle cell disease in children and teens
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a new medicine called YOLT-204 for young people with sickle cell disease. It aims to see if the drug can reduce severe pain episodes and other serious complications.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 3 to 17 years old.
- You have a specific type of sickle cell disease (HbSS or HbSβ0).
- You have had at least two severe sickle cell crises (such as severe pain, acute chest syndrome, or spleen problems) in the last year despite standard care.
- If you take L-glutamine, your dose has been stable for at least 3 months; if you take hydroxyurea, you have stopped it at least 8 weeks ago.
- Your major organs (heart, lungs, liver, kidneys) are working well enough based on blood tests and heart ultrasound.
- You or your guardian must sign a consent form and follow the study schedule.
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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