Gene therapy for adults with severe hemophilia A
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a new gene therapy called SPK-8011QQ for adults with severe or moderately severe hemophilia A. The goal is to see if a single infusion can help your body produce its own factor VIII, reducing or stopping the need for regular factor VIII injections.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 18 or older
- You are male
- You have severe or moderately severe hemophilia A (factor VIII level 3% or less)
- You have been on regular preventive treatment (prophylaxis) or had at least 5 bleeds needing treatment in the last 6 months
- You have had at least 150 exposures to factor VIII products (like recombinant or plasma-derived factor VIII)
- You have never had a serious allergic reaction to any factor VIII product
- You do not have a current inhibitor (antibody that blocks factor VIII) and have not had one in the past unless you completed immune tolerance therapy successfully over 5 years ago
- You have good liver and kidney function, and no HIV or active hepatitis B or C (unless controlled)
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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