Phase 2 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 have been diagnosed with severe or moderately severe hemophilia B, meaning your Factor IX (FIX) activity level is very low (2% or less), or your genetic test confirms you have severe hemophilia B
- You are currently taking regular preventive Factor IX treatment (called prophylaxis) and have experience using Factor IX therapy, as defined by the trial guidelines
- You have taken part in either a lead-in period of this study or a related separate study for at least 6 months while on your regular Factor IX preventive treatment, so that your bleeding rate data could be recorded
Who may not be able to join:
- You have had a history of your body developing resistance (called inhibitors) to Factor IX treatment on two or more separate occasions
- A specific blood test at the start of the trial shows you have inhibitor levels above the normal range
- A blood test shows you already have antibodies to a specific virus carrier (AAV8) used in the treatment
- You have a significant liver condition such as liver scarring (cirrhosis), high blood pressure in the liver (portal hypertension), an enlarged spleen, or a condition causing brain problems related to the liver
- Tests show advanced liver scarring or significant fat build-up in the liver, as defined by the trial guidelines
- An ultrasound scan shows signs of liver scarring or high liver blood pressure, either at the start of the trial or within the 6 months before
- You have a history of blood clots in your arteries or veins, as defined by the trial guidelines
- You have had an allergic reaction to steroid medications before, or you have a health condition that requires you to take steroids regularly
- You have previously received any gene therapy using an AAV virus carrier, or you plan to receive any other AAV-based gene therapy during the trial
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: Clinical Trial Management, Regeneron Pharmaceuticals
Phone: 844-734-6643
Australian sites
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
3 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Occurrence of Treatment-Emergent Adverse Events (TEAEs); Severity of TEAEs; Coagulation Factor IX (FIX) functional activity measured using the chromogenic substrate assay; Change in FIX functional activity in plasma, measured using the chromogenic substrate assay; Annualized Bleeding Rate (ABR) following sustained FIX functional activity among participants receiving the RDE; Occurrence of Serious Adverse Events (SAEs); Severity of SAEs; Occurrence of Adverse Event of Special Interests (AESIs); Severity of AESIs; Occurrence of clinically meaningful Adverse Events (AEs); Severity of clinically m...
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.