Gene therapy for severe sickle cell disease (Drepamir study)
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial compares a new gene therapy to the standard care for people with severe sickle cell disease. It aims to see if the gene therapy is better at reducing painful crises and complications.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 12 and 35 years old.
- You must have a confirmed diagnosis of sickle cell disease (type HbSS).
- You must have had serious complications like frequent pain crises, acute chest syndrome, or priapism.
- Hydroxyurea (a common sickle cell treatment) didn't work well enough or caused side effects.
- You must not have a matched brother or sister for a bone marrow transplant.
- You must be willing to use effective contraception if sexually active.
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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