Early Phase 1 Haemophilia Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- People who have been fully informed about the trial and have voluntarily signed a consent form agreeing to take part.
- Men aged 18 years or older.
- People diagnosed with hemophilia A where the body produces very low or no Factor VIII (a clotting protein), at 2% or less of normal levels.
- People who have previously been treated with Factor VIII products on at least 150 separate occasions.
- People who have had bleeding episodes or received Factor VIII treatments in the 12 weeks before joining, with this documented in their medical records.
- People with no history of serious allergic reactions (including anaphylaxis) to Factor VIII products or intravenous immunoglobulin.
- People who have no current Factor VIII inhibitors (antibodies that block the treatment from working) and no history of developing them after 150 or more treatment days, with no signs of reduced response to Factor VIII infusions.
- People who agree to use reliable barrier contraception from the time of signing the consent form until 52 weeks after receiving the trial treatment.
- People who are willing to follow the trial requirements and are motivated to participate in a gene therapy clinical trial.
Who may not be able to join:
- People who test positive for hepatitis B or hepatitis C infections.
- People currently receiving antiviral treatment for hepatitis B or C.
- People who have additional blood clotting disorders beyond hemophilia A.
- People who have taken immune-suppressing medications (other than corticosteroids) in the 30 days before joining.
- People who have received a vaccine in the 30 days before the screening visit.
- People with known liver problems, such as high blood pressure in the liver, an enlarged spleen, liver-related brain problems, significant liver scarring, liver nodules or cysts found on ultrasound, or abnormal liver-related blood test results that the trial doctors consider significant.
- People who have a major surgery planned during the 52-week trial period.
- People who have previously taken part in a gene therapy trial, or who have recently participated in another clinical trial involving an experimental drug (within a specific timeframe based on how long that drug stays in the body — confirm with trial site).
- People with alcohol or drug dependence, or those who are unable to avoid alcohol for the entire duration of the study.
- People with any other serious ongoing illness or condition that the trial doctors consider would make participation unsuitable.
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Lei Zhang, MD, Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
Phone: 0086-22-23909095
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Incidence of dose limiting toxicity (DLT) events; The incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Can't join this trial?
Data last synced from ClinicalTrials.gov: 21 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.