One-Shot gene therapy aims to fix bleeding disorder
NCT ID NCT07548411
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial is testing a single-dose gene therapy called GS1191-0445 in 7 Chinese adults with severe hemophilia A. The therapy uses a harmless virus to deliver a working gene to the liver, so the body can produce its own clotting factor. The main goal is to check safety, but researchers will also see if it reduces bleeding episodes and the need for factor infusions.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- GS1191-0445 injection (gene therapy)
- What this could lead to
- If it works, this could allow people with severe hemophilia A to produce their own clotting factor, reducing or eliminating the need for regular infusions.
- What could go wrong
- This is a very early, small trial (only 7 people) focused on safety. The gene therapy may not produce enough clotting factor, could cause side effects like liver issues or blood clots, and long-term effects are unknown.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
Tianjin, Tianjin Municipality, 300020, China
-
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei, 430022, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- A Once-a-Week shot could transform hemophilia Care—Even for those with inhibitors
- Can a new clotting factor offer better bleed protection for severe hemophilia?
- New clotting factor aims to cut bleeding episodes in severe hemophilia a
- New clotting factor aims to stop bleeding in severe hemophilia a
- New clotting factor shows promise for hemophilia a bleeds
- Blood test may predict bleeding risk in hemophilia a patients on emicizumab