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 (NCT04759131) enrolled 74 children with haemophilia A who were treated with a medicine called BIVV001. Thirty-eight children were under 6 years old and 36 were aged 6 to under 12 years. The trial was measuring two main things: whether the children's immune systems developed "inhibitors" (antibodies that can block the medicine from working) and how often bleeding episodes occurred while on treatment. The reported data shows that for the primary outcome — inhibitor development — zero participants in either age group developed these blocking antibodies during the study. For bleeding episodes, the trial tracked what is called an "annualised bleeding rate," meaning the estimated average number of bleeding episodes per person per year. For bleeds that required treatment, the reported figures were approximately 0.48 episodes per year in the under-6 group and 1.33 episodes per year in the 6-to-under-12 group. When all bleeding episodes were counted (both treated and untreated), the reported figures were approximately 2.78 per year in the younger group and 2.85 per year in the older group. A separate sensitivity analysis of the same data reported slightly different figures for the older group (0.75 treated bleeds per year; 2.32 total bleeds per year), while the younger group's figures remained the same. The reported data also shows bleeding broken down by type — spontaneous (no clear cause), traumatic (a known cause), and unknown. The reported treated bleed rates by type were approximately 0.17 (spontaneous), 0.28 (traumatic), and 0.03 (unknown) episodes per year in the under-6 group, and 0.14 (spontaneous), 0.59 (traumatic), and 0.59 (unknown) episodes per year in the 6-to-under-12 group. Two participants in the younger group did not complete the study; the data does not report the reasons. 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 40701256) ↗
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 child must be under 12 years old at the time of signing the consent form.
- The child must have severe hemophilia A, confirmed by a blood test showing very low or absent clotting Factor VIII, or by a known genetic result that causes severe hemophilia A.
- The child must have already been treated for hemophilia A (either regularly to prevent bleeding, or when needed to stop bleeding) — at least 150 previous treatment days if aged 6 to under 12, or at least 50 previous treatment days if under 6 years old.
- The child must weigh at least 10 kilograms (approximately 22 pounds).
Who may not be able to join:
- Children who have previously had a severe allergic reaction or anaphylaxis to any Factor VIII product.
- Children who have a history of developing "inhibitors" — which are antibodies that block Factor VIII from working properly — or who have shown signs that Factor VIII treatments stopped working as expected. (Note: a family history of inhibitors alone would not automatically exclude the child.)
- Children who test positive for inhibitors at the screening appointment before the trial begins.
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 Sciences & Operations, Sanofi
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
Number of Participants With Neutralising Antibodies (Development of Inhibitors) Directed Against Factor VIII
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.