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:
- You are a male between 18 and 65 years old
- You have a confirmed diagnosis of Hemophilia B with very low levels of the clotting factor FIX (2% or less)
- You have received FIX replacement treatment products on at least 100 separate occasions in the past
- In the past year, you have had at least 3 or more bleeding episodes and/or long-term joint damage in one or more joints that needed treatment with FIX products
- You agree to use reliable barrier contraception and not donate sperm for 52 weeks after receiving the study treatment
- You are willing to take part voluntarily, follow the study rules, complete all planned visits, and provide blood or other biological samples for testing
Who may not be able to join:
- You have had an allergic reaction to any ingredient in the study drug, including immunosuppressant medicines
- You are unable to tolerate immunosuppressant or steroid medicines
- Your blood tests show you have developed antibodies that work against FIX (called "inhibitors"), or you have a history of this
- You have ever had or currently have cancer of any kind
- You currently have an active autoimmune disease (where the body attacks itself)
- You have serious heart problems, such as angina, heart attack, heart failure, significant heart defects, heart valve disease, or irregular heartbeat
- You have liver disease or a history of liver disease, such as scarring of the liver, fluid build-up in the belly, or problems with blood flow through the liver
- You have tested positive for Hepatitis B or Hepatitis C, or you are currently being treated for either of these conditions
- You have diabetes that is not well controlled despite medication
- You have blood pressure that is too high or too low and cannot be controlled
- Your blood count (haemoglobin) is below a certain level, suggesting anaemia (confirm with trial site)
- Your platelet count (cells that help blood clot) is below a certain level (confirm with trial site)
- Your liver function blood tests are above acceptable levels (confirm with trial site)
- Your kidney function blood test is above the normal upper limit
- Your blood protein (albumin) level is below the normal lower limit
- You have tested positive for HIV or syphilis
- You have high levels of certain antibodies against the virus used to deliver the gene therapy (AAV5 neutralising antibody titre above 1:640) (confirm with trial site)
- You have previously taken part in a gene therapy clinical trial, used an experimental FIX drug within the last month, or joined any other drug or device trial within the last 3 months
- You have any surgery planned within 52 weeks after receiving the study treatment
- You lost more than 400 mL of blood in the 3 months before joining the study
- You have epilepsy, a history of mental illness such as schizophrenia, depression, mania, or anxiety, or any condition that affects your ability to make decisions
- You have a history of drug abuse or alcohol dependency
- The study doctors believe you are unlikely to follow the study requirements or complete all follow-up visits
- You have any other significant health condition or reason that the study doctors consider makes it unsafe or unsuitable for you to participate
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, China
Phone: +86 022-23909240
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 adverse events; Number of participants with clinically significant change from baseline in vital signs; Number of participants with clinically significant change from baseline in physical examination findings; Number of participants with clinical laboratory abnormalities; Antibody against AAV capsid protein
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.