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 (NCT03417245) enrolled 120 people in total — 40 in a group who used clotting factor medication only when a bleed occurred (called "on-demand" treatment), and 80 in a group who received monthly injections of fitusiran 80 mg as a preventive (prophylaxis) treatment. The trial was primarily measuring the "annualised bleeding rate" — that is, how many treated bleeding episodes each person would be expected to have over the course of a year. By the end of the study, 37 people in the on-demand group and 79 in the fitusiran group had completed the trial. The reported data shows that, for the main outcome during the core measurement period, the estimated number of treated bleeds per person per year was approximately 31.0 in the on-demand group and approximately 3.1 in the fitusiran group. A second way of looking at the same primary outcome — using straightforward observed counts rather than a statistical model — recorded approximately 21.8 treated bleeds per person per year in the on-demand group and 0.0 in the fitusiran group. For secondary outcomes covering the full treatment period (including the first four weeks of fitusiran use), the estimated figures were approximately 31.4 and 4.1 bleeds per person per year respectively, while the directly observed figures were approximately 25.2 and 1.8. When looking only at bleeds that happened for no clear reason (called spontaneous bleeds), the estimated rate during the core period was approximately 22.0 per person per year in the on-demand group and approximately 1.8 in the fitusiran group; the directly observed figures were approximately 16.1 and 0.0 respectively. 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 37003278) ↗
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:
- You are male and at least 12 years old
- You have been diagnosed with severe hemophilia A or B, confirmed by a blood test showing very low levels of the relevant clotting factor
- You do not have inhibitors (antibodies that block clotting factor treatments), confirmed by a specific blood test at the screening visit
- You have been using clotting factor treatment on-demand (only when a bleed occurs, not as a regular preventive routine) for at least the last 6 months
- You have not used bypassing agents to treat bleeding episodes in the last 6 months
- You have not had immune tolerance induction therapy in the last 3 years
- You have had at least 6 bleeding episodes that needed clotting factor treatment in the past 6 months
- You are willing to follow the study requirements and sign a consent form (or assent form if applicable)
Who may not be able to join:
- You have another bleeding disorder in addition to hemophilia, such as Von Willebrand's disease, other clotting factor deficiencies, or a platelet disorder
- Your antithrombin activity level is below 60% at the screening blood test (confirm with trial site)
- You have a condition that makes your blood more likely to clot than normal (a thrombophilic disorder)
- You have significant liver disease
- You have an active hepatitis C infection
- You are HIV positive with a specific immune cell count below 200 cells per microlitre
- You have a history of blood clots in your arteries or veins
- Your kidneys are not functioning well enough (confirm with trial site)
- You have a history of allergies to multiple medicines, or a known allergic reaction to a specific type of drug ingredient used in this trial (confirm with trial site)
- You have had problems tolerating injections under the skin in the past
- You have any other health condition that the trial doctor believes would make participation unsafe or unsuitable for you
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, MD, 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
3 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period; Observed Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period
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.