Gene therapy NGGT003 for severe hemophilia A
Part of Blood & lymphatic, Genetic & congenital clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Male and 18 years old or older
- Severe hemophilia A (factor VIII level below 1%)
- At least 150 days of treatment with factor VIII products in the past
- Had a bleed or needed factor VIII in the last 12 weeks
- No history of factor VIII inhibitors
- No major liver disease or other serious health issues
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This trial tests a gene therapy to help your body produce more of the clotting protein (factor VIII) it's missing. It may reduce or stop the need for regular factor infusions.
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.
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 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.
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 Phase 1 trial tests a gene therapy given with an “own blood stem cell” transplant to improve hemophilia A. It’s mainly to check safety and early signs of how well the therapy works in people with severe bleeding problems.
Hear when a new Hemophilia A trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.