Transplant with defibrotide for sickle cell disease
Treatments studied
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a stem cell transplant from a half-matched donor using a strong conditioning regimen and a drug called defibrotide to protect organs. It aims to cure sickle cell disease in patients who have had serious complications like stroke, chest syndrome, or frequent pain episodes.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have sickle cell disease (like SS, SC, or beta thalassemia) and have had a serious complication such as a stroke, chest crisis, or frequent pain.
- You are willing to use a half-matched (haploidentical) donor, like a parent, child, or sibling, and do not have a fully matched sibling donor.
- You are not currently on strong blood thinners or allergic to defibrotide.
- You are not pregnant or breastfeeding and have no active uncontrolled infections.
- You have not had a prior stem cell transplant and have no severe liver damage.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
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