Can a single gene shot free hemophilia b patients from regular infusions?
NCT ID NCT05164471
First seen Jul 31, 2026 · Last updated Jul 31, 2026
Summary
This trial tests a gene therapy called FLT180a in adults with hemophilia B, a condition where the body lacks a clotting protein. The therapy delivers a working gene to help the body produce its own clotting factor. A small group of participants will receive a single dose and be followed for a year to see if it safely boosts clotting factor levels and reduces bleeding episodes.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- A gene therapy called verbrinacogene setparvovec (FLT180a) that delivers a working gene to help the body produce its own clotting factor IX.
- What this could lead to
- If successful, this could offer a one-time treatment that reduces or eliminates the need for regular clotting factor infusions in people with hemophilia B.
- What could go wrong
- This is an early-stage trial with a small number of participants, so results may not apply broadly. Gene therapy carries risks like immune reactions or the body not producing enough factor IX.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
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Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
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Glasgow Royal Infirmary
Glasgow, United Kingdom
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Guys Hospital
London, United Kingdom
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Royal Free London NHS Foundation Tust
London, United Kingdom
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Royal Victoria Infirmary
Newcastle, United Kingdom
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University of Michigan
Ann Arbor, Michigan, 48109, United States
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University of South Florida
Tampa, Florida, 33612, United States
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
- Gene Editing's lasting impact: a 10-Year safety watch
- Can a video call replace the clinic for hemophilia pain relief?
- Newborn screening study aims to catch rare diseases at birth
- Monthly shot could free kids with hemophilia from frequent infusions
- New shot aims to cut bleeding in hemophilia patients