Collecting stem cells for gene therapy in sickle cell disease
Treatments studied
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This study tests a new drug called motixafortide to help collect your own blood stem cells for future gene therapy. It is for adults with severe sickle cell disease who are willing to donate their stem cells and meet certain health requirements.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be 18 years or older with severe sickle cell disease (like needing regular blood transfusions or being eligible for a transplant).
- Have a working central line (a special IV), or be able to have one placed if needed for the procedure.
- Have good kidney, liver, and heart function, and no active infections or recent experimental treatments.
- Be willing to donate your own blood stem cells for future gene therapy research.
- Not have had a previous stem cell transplant or gene therapy, and be able to stop certain medications (like hydroxyurea) for 30 days before the study.
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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