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 108 children with haemophilia A — a condition where the blood does not clot properly due to low levels of a protein called Factor VIII. The trial tested a treatment called recombinant Factor VIII Fc fusion protein (rFVIIIFc), a manufactured version of that clotting protein. Of the 108 who enrolled, 103 went on to receive the treatment. Participants were placed into one of three treatment approaches: an "on-demand" (episodic) regimen where the treatment was given when a bleed occurred (81 participants), a preventive (prophylactic) regimen given regularly to try to prevent bleeds (89 participants), and an immune tolerance induction (ITI) regimen for participants who had developed antibodies against Factor VIII treatment (15 participants). Some participants moved between regimens during the study. The trial's main goal was to measure how many children developed "inhibitors" — antibodies that can block the treatment from working. The reported data shows that the primary outcome — confirmed inhibitor development — was recorded in approximately 31% of participants who had received at least 10 treatment days and had at least one inhibitor test performed. For the secondary outcomes, the reported average number of bleeding episodes per person per year (called the annualised bleeding rate, or ABR) was 2.24 for the episodic group, 1.49 for the prophylactic group, and 0.00 for the ITI group. The reported average number of spontaneous joint bleeds per person per year was 0.00 across all three groups. When parents or caregivers rated how each injection responded to a bleed using a four-point scale, the reported data shows the majority of rated responses fell into the "excellent" or "good" categories across all regimens, though the specific breakdown figures across each regimen and rating category are listed in the full data. The reported average annual dose of the treatment was approximately 198 IU/kg per person per year for the episodic group, around 5,384 IU/kg for the prophylactic group, and approximately 67,310 IU/kg for the ITI group. 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 38266154) ↗
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:
- A parent or legal guardian must be able to understand the study's purpose and risks, and be willing to sign a consent form allowing the use of their child's health information.
- The participant must weigh at least 3.5 kg (about 7.7 lbs) at the time of screening.
- The participant must have severe Hemophilia A, meaning their body produces less than 1% of the normal amount of a clotting protein called Factor VIII, as shown in their medical records or confirmed during screening.
Who may not be able to join:
- Anyone who has previously received blood products, Factor VIII replacement treatments, or a specific product called rFVIIIFc at any point before or during screening.
- Anyone who has ever had a positive test result showing their body developed resistance (called "inhibitors") to Factor VIII treatment.
- Anyone who has had an allergic reaction to the specific treatment used in this trial (rFVIIIFc).
- Anyone who has another blood clotting disorder in addition to Hemophilia A.
- Anyone who has another serious ongoing health condition that the study doctor feels would make participation unsafe.
- Anyone currently taking chemotherapy or medications that suppress the immune system.
⚠️ Important: Always verify eligibility with the trial site directly before applying. Note: There may be additional criteria not listed here — the trial site can provide the full details.
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
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
4 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
Can't join this trial?
Data last synced from ClinicalTrials.gov: 27 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.