Trial results
AI generated results 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
According to the results reported on ClinicalTrials.gov, this trial enrolled 30 children with haemophilia B (a condition where the blood does not clot properly due to low levels of a protein called Factor IX). There were two age groups: 15 children under 6 years old, and 15 children aged 6 to under 12 years old. The trial was measuring whether a treatment called rFIXFc caused the immune system to develop "inhibitors" — antibodies that can block the treatment from working — and also tracked how often bleeding episodes occurred and how much of the treatment was used. Of the 30 children who started, 13 in the younger group and 14 in the older group completed the study. The reported data shows that the primary thing being measured — whether any child developed a confirmed inhibitor — was reported as 0% in both age groups, meaning no confirmed inhibitors were recorded in either group. For bleeding episodes, the reported average number of bleeds per child per year was 1.09 in the younger group and 2.13 in the older group. When looking only at spontaneous (unprompted) joint bleeds, the reported average was 0.00 per year in both groups. In terms of how caregivers rated the response to the first injection given to treat a bleed, around 89.5% of first injections in the younger group and 88.2% in the older group were rated as having some level of response. Doctors' overall assessments rated the treatment regimen as "excellent" for approximately 85% of responses in the younger group and 90% in the older group. The reported data also shows the average annual amount of the treatment used: roughly 3,042 international units per kilogram (a standard measure of dosing) in the younger group and 3,186 in the older group for routine prevention of bleeds, with smaller additional amounts used for treating bleeds when they occurred. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 36848635) ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 3 Haemophilia Trial, Completed
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:
- The patient must have severe Hemophilia B, meaning their body produces very little or none of the blood-clotting protein called Factor IX (2% or less of the normal amount)
- The patient must be male and younger than 12 years old, and weigh at least 13 kilograms (about 28.6 pounds)
- The patient must have a documented history of at least 50 previous treatments with a Factor IX product
- The patient must have no history of, and currently show no signs of, developing antibodies (called inhibitors) that fight against Factor IX treatments
Who may not be able to join:
- The patient has other blood-clotting disorders in addition to Hemophilia B
- The patient has ever had a severe allergic reaction (anaphylaxis) to any Factor IX product or to intravenous immunoglobulin (a medicine given through a drip into a vein)
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: Medical Director, Bioverativ Therapeutics Inc.
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
2 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Occurence of Factor IX (FIX) Inhibitor Development
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.