Gene therapy for hemophilia b shows promise in Long-Term study
NCT ID NCT05360706
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This study follows 9 adults with severe or moderately severe hemophilia B who received a one-time gene therapy (AAV5-hFIX) to help their bodies produce clotting factor IX. Researchers are checking if the treatment remains safe and effective for up to 10 years after the initial dose. The goal is to see if this gene therapy can reduce or prevent bleeding episodes over the long term.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- gene therapy (AAV5-hFIX) to produce clotting factor IX
- What this could lead to
- If successful, this could show that a single gene therapy treatment provides long-lasting control of bleeding episodes, reducing or eliminating the need for regular factor IX infusions.
- What could go wrong
- This is a small extension study with only 9 participants, so results may not apply to everyone. Long-term safety concerns, such as liver effects or loss of effectiveness over time, are still being monitored.
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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Amsterdam UMC - Locatie AMC
Amsterdam, Netherlands
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Erasmus MC
Rotterdam, Netherlands
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Groningen UMC
Groningen, Netherlands
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Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitae
Frankfurt, Germany
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Universitair Medisch Centrum Utrecht
Utrecht, Netherlands
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Vivantes Klinikum im Friedrichshain
Berlin, Germany
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 single gene shot free hemophilia b patients from regular infusions?
- 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