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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
-
10 people
The number who actually took part.
- Started
-
Dec 2020
- Finished
-
Apr 2026
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 years and older
- Sex
-
Male participants only
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Males ≥ 18 years of age with hemophilia A and documented prior residual FVIII activity ≤ 1 IU/dL including, but not limited to, at the time of detected inhibitors, at the time of signing the informed consent. 2. History of a positive inhibitor result with the first positive result at least 12 month prior to Screening. Part A: Demonstrated no immunological tolerance to exogenous FVIII. Part B: Demonstrated tolerance to exogenous FVIII and negative FVIII inhibitor screening titer \< 0.6 BU. 3. Prophylactic or on-demand hemophilia therapy in the last 12 months. Bleeding, inhibitor \& hemophilia therapy Hx over previous 12 months. 4. Sexually active participants must agree to use an acceptable method of effective contraception. Participants must agree to contraception use for at least 12 weeks post-infusion. 5. Willing to abstain from consumption of alcohol for at least the first 52 weeks following BMN 270 infusion. Exclusion Criteria: 1. Detectable pre-existing antibodies to the AAV5 capsid. 2. Any evidence of active infection or any immunosuppressive disorder; patients with HIV infection and undetectable viral load are not excluded. 3. Currently undergoing, or plan to receive during the study, immune tolerance induction therapy or prophylaxis with FVIII (Part A only). 4. Significant renal dysfunction or liver dysfunction, infection or history of hepatic malignancy. 5. Evidence of any bleeding disorder not related to hemophilia A.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Hemophilia A with anti factor viii are added.
Genom att skicka in godkänner du våra Användarvillkor
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
-
Chaim Sheba Medical Center
Ramat Gan, Israel
-
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
-
Ege University School of Medicine
Izmir, Turkey (Türkiye)
-
Hemocentro Da UNICAMP
Campinas, Brazil
-
Kaohsiung Medical University - Chung-Ho Memorial Hospital
Kaohsiung City, Taiwan
-
Kyung Hee University Hospital at Gangdong
Seoul, South Korea
-
National Taiwan University Hospital
Taipei, Taiwan
-
Royal Free Hospital
London, United Kingdom
-
Taichung Veterans General Hospital
Taichung, Taiwan
More trials for these conditions
Other studies related to the condition(s) this trial covers.