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 113 people in total across four groups. The trial involved people with a bleeding disorder called haemophilia A who also had inhibitors (meaning their body resists a standard treatment). Three groups (Arms A, C, and D) received a medicine called emicizumab given by injection once a week, while one group (Arm B) received no preventive treatment at first, then later switched to emicizumab. The trial was primarily measuring how often bleeds occurred over the course of a year — a figure called the annualised bleed rate, or ABR, which is simply an estimate of how many bleeds a person might experience in a 12-month period. The reported data shows that for the main comparison — Arm A (emicizumab) versus Arm B (no preventive treatment) — the estimated number of bleeds requiring treatment per year was 2.9 for the emicizumab group and 23.3 for the no-treatment group. When all bleeds (treated and untreated) were counted, the reported figures were 5.5 per year for the emicizumab group and 28.3 per year for the no-treatment group. For joint bleeds specifically requiring treatment, the reported figures were 0.8 per year (emicizumab) versus 6.7 per year (no treatment). The reported data also shows comparisons within the same participants before and after starting emicizumab: people in Arm A who had previously used on-demand treatment reported a drop from 37.7 all bleeds per year to 4.1, and treated bleeds dropped from 21.6 to 1.7. For Arm C participants who had previously used a different type of regular preventive treatment, all bleeds per year went from a reported 24.3 down to 5.5 on emicizumab. 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 35939785) ↗
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 weigh at least 40 kilograms (about 88 pounds) at the time of screening
- You have been diagnosed with hemophilia A (present from birth, of any severity) and have a documented history of high-level inhibitors (proteins that block clotting factor treatment), measured at 5 Bethesda Units or more
- You have been treated with bypassing agents (special medications used when inhibitors are present) either as needed or as a regular preventive treatment for at least the past 24 weeks
- You have had 6 or more bleeding episodes in the last 24 weeks if you only use bypassing agents when bleeds occur, OR 2 or more bleeding episodes in the last 24 weeks if you use bypassing agents regularly to prevent bleeds
- Your blood counts, liver function, and kidney function are within an acceptable range
- If you are a woman who has not gone through menopause or had surgery to prevent pregnancy, you must agree to abstain from sex or use highly effective birth control during the study
Who may not be able to join:
- You have a bleeding disorder other than hemophilia A, whether you were born with it or developed it
- You are currently receiving, or plan to receive during the study, immune tolerance therapy or regular preventive treatment with Factor VIII (clotting factor), unless you have been on a specific combined regimen of both Factor VIII and bypassing agents for prevention
- You have been treated for a blood clotting condition (such as a blood clot in a vein or artery) in the past 12 months, are currently being treated for one, or currently show signs of one (with a limited exception for a past clot related to a catheter that no longer requires treatment)
- You have other health conditions, such as certain autoimmune diseases, that may increase your risk of bleeding or blood clots
- You have had a significant allergic reaction to a monoclonal antibody therapy (a type of laboratory-made protein treatment) or to any ingredient in the study drug (emicizumab)
- You have a known HIV infection with a CD4 cell count below 200 cells per microliter within the past 24 weeks (confirm with trial site)
- You are currently using or plan to use certain immune-modifying medications (such as interferon or rituximab) during the study, except for HIV antiretroviral therapy
- In your doctor's opinion, you are considered at high risk for a serious condition affecting small blood vessels called thrombotic microangiopathy — for example, if you or a family member have had this condition before
- You have another illness, treatment, or abnormal test result that, in the opinion of the study doctor, could interfere with the study or put you at risk
- You have a major surgery planned during the study (minor procedures such as tooth extractions are generally acceptable)
- You have previously received emicizumab (the study drug) in another research study, or have recently received certain other investigational (experimental) drugs within specified time periods
- You are a woman who is unwilling to use highly effective birth control for the required period specified in the study
- You have a significant abnormal finding on your screening tests that, in the study doctor's opinion, may make it unsafe for you to take the study drug
- You are currently pregnant, breastfeeding, or plan to become pregnant during the study
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 Trials, Hoffmann-La Roche
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
Model-Based Annualized Bleed Rate (ABR) for Treated Bleeds, Arm A: Emicizumab Versus Arm B: No Prophylaxis
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.