CRISPR gene-edited bone marrow transplant for severe sickle cell
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This Phase 1/2 study tests a CRISPR-edited bone marrow (stem cell) transplant to treat severe sickle cell disease. It aims to reduce dangerous sickle-related complications by changing the patient’s stem cells before transplant.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 12 to 35 years old when you sign consent
- You have severe sickle cell complications in the last 2 years despite standard supportive care (examples include repeated lung episodes, frequent severe pain, blood pooling in the spleen, recurrent priapism, liver complications, or stubborn leg ulcers)
- You have enough body function for a transplant: good heart, lung, kidney, and liver test results, plus adequate overall function
- You have not had certain infections, HIV, hepatitis B/C, or recent cancer-related blood findings that would make the transplant unsafe
- You have not had a bone marrow transplant or any solid organ transplant
- You can follow study rules (including long-term contraception for 12 months after the stem cell infusion, if applicable)
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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