One-shot gene therapy aims to free hemophilia patients from constant infusions
NCT ID NCT04684940
First seen Jul 10, 2026 · Last updated Jul 10, 2026
Summary
This trial tests a gene therapy called valoctocogene roxaparvovec for people with severe hemophilia A who have developed inhibitors (antibodies that block standard treatment). The therapy uses a harmless virus to deliver a working copy of the gene for clotting factor VIII, so the body can produce its own factor. The study includes two groups: those with active inhibitors and those with a past history of inhibitors. The goal is to see if this single treatment is safe and can reduce or stop the need for regular 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
- valoctocogene roxaparvovec (gene therapy)
- What this could lead to
- If successful, this gene therapy could allow people with hemophilia A and inhibitors to produce their own clotting factor, reducing or eliminating the need for regular infusions.
- What could go wrong
- This is an early-phase trial with only 10 participants, so results may not apply to everyone. Gene therapy carries risks including immune reactions and long-term effects that are still 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
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Locations
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Chaim Sheba Medical Center
Ramat Gan, Israel
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Ege University School of Medicine
Izmir, Turkey (Türkiye)
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Hemocentro Da UNICAMP
Campinas, Brazil
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Kaohsiung Medical University - Chung-Ho Memorial Hospital
Kaohsiung City, Taiwan
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Kyung Hee University Hospital at Gangdong
Seoul, South Korea
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National Taiwan University Hospital
Taipei, Taiwan
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Royal Free Hospital
London, United Kingdom
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Taichung Veterans General Hospital
Taichung, Taiwan
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