Gene-Matched Family Stem Cell Transplant for Severe Sickle Cell
Treatments studied
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests whether a stem cell transplant from a partially gene-matched family member can cure severe sickle cell disease in children and young adults. The transplant uses a new approach that may work even when a perfectly matched sibling isn't available, and it might help patients whose sickle cell is causing serious complications like strokes, organ damage, or frequent pain crises.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 22 years old or younger
- You have sickle cell disease with serious complications (such as abnormal brain blood flow, stroke, repeated chest crisis, frequent pain episodes, priapism, regular blood transfusions, or organ damage)
- You have a family member willing to donate bone marrow or blood stem cells who shares at least half of your genetic markers
- You are healthy enough to undergo transplant (good heart, kidney, and lung function, no active serious infections, not pregnant)
- The donor is HIV-negative, not pregnant or breast-feeding, and can pass medical screening
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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