Testing three medicines for high-risk sickle cell disease
Treatments studied
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This Phase 1 study tests whether nicotinamide, oral tetrahydrouridine, and decitabine can help people with high-risk sickle cell disease. It is mainly a safety-and-dosing study, but it also looks for signs the medicines may improve disease-related problems.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 18 years or older and able to sign consent
- You must have confirmed sickle cell disease by blood testing (hemoglobin pattern)
- You must not have a sickle cell emergency in the last 14 days (like hospitalization for pain or acute chest syndrome)
- You must weigh at least 40 kg
- You must have “high-risk” sickle cell symptoms even after at least 6 months of hydroxyurea, or you can’t or won’t take hydroxyurea
- You must not be pregnant or breastfeeding and must use required birth control
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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