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NCT04728841Possibly a fitRecruiting

Gene therapy trial for Chinese hemophilia A

Hemophilia AGene Therapy

Part of Blood & lymphatic, Genetic & congenital clinical trials.

This trial tests a one-time gene therapy treatment (GS001) to help people with hemophilia A make more of the clotting factor they lack. If it works and is safe for you, it could improve how well blood clots form over time.

Summary written for real people, not researchers, by Clin2.

Phase
N/A
Enrollment
12 people
Ages
18 years and older
Study type
Interventional

Who can take part

  • You are an adult (18+) and male, and you can follow the study and sign consent
  • You have severe hemophilia A with very low factor VIII activity (≤1% at screening)
  • You must not have had dangerous allergic reactions to factor VIII products
  • You should not have a factor VIII inhibitor (a blocker) based on blood tests done more than once
  • Your blood tests and organ function (liver, kidneys, blood counts) must be within allowed ranges
  • You must agree to contraception and provide semen samples that test negative for the gene therapy vector

View the official record on ClinicalTrials.gov

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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