Gene therapy trial for hemophilia A using FVIII
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This Phase 1 study tests a gene therapy designed to help the body make factor VIII (FVIII), which is missing in hemophilia A. It may help reduce bleeding and the need for FVIII infusions, but it is an early-safety trial.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (or your child) can understand the study and sign consent, and you can follow study visits and instructions
- You are male age 2 or older with confirmed hemophilia A (FVIII level ≤2 IU/dL or ≤2% of normal)
- You have frequent bleeding—about at least 4 bleeding events per year that need FVIII treatment or preventative FVIII infusions
- Your blood tests show no FVIII “inhibitors” (the body’s antibodies that block FVIII), and you’ve never had them
- You have to use reliable birth-control/barrier protection until follow-up tests show three negative results for the gene therapy vector
- You do not have major liver problems, active hepatitis B or C, or HIV with CD4 count 200 or less (well-controlled HIV with undetectable viral load may be allowed)
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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This Phase 1/2 study tests a one-time gene therapy (ASC618) to help the body make clotting factor for people with hemophilia A. It may reduce bleeding and the need for factor infusions, but you must meet several safety and eligibility checks.
This early-phase trial tests a new gene therapy called NGGT003 for men with severe hemophilia A. It aims to see if the treatment is safe and can help reduce bleeding episodes.
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This early-phase study tests an FVIII gene therapy to help your body make more of the missing clotting factor in hemophilia A. It’s aimed at adults with severe low FVIII levels who have already used FVIII replacement products for a long time and have not developed an immune blocker (an inhibitor).
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.
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