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 (NCT03020160) enrolled 48 people in total across two groups: 7 participants in a smaller "pharmacokinetic run-in" group (used to study how the body processes the medicine, emicizumab) and 41 participants in the main "expansion" group. All 48 participants completed at least 24 weeks in the study. The trial was primarily measuring how often bleeds occurred per year — known as the "annualised bleeding rate" — in people receiving emicizumab, a medicine being studied for haemophilia A. A secondary measure looked at participants' self-reported quality of life using a standard questionnaire. The reported data shows that, in the main expansion group of 41 participants, the annualised rate of bleeds that required treatment was 2.4 per year. When all bleeds were counted (whether treated or not), the reported rate was 4.5 per year. Breaking this down further, the reported rate for treated bleeds that happened without an obvious cause (called spontaneous bleeds) was 0.6 per year; for treated joint bleeds it was 1.7 per year; and for treated bleeds in so-called "target joints" — joints that had bled repeatedly before the study — it was 1.0 per year. The reported data also shows a secondary quality-of-life finding. Adult participants completed a haemophilia-specific questionnaire (scored from 0 to 100, where lower scores reflect better quality of life) at the start and again at week 25. The reported average change in total score was −13.62 points, meaning scores were, on average, about 13.6 points lower at week 25 than at the start. The questionnaire's designers had defined a drop of 7 or more points as a "clinically meaningful" change, though what this means for any individual was not reported in the data provided. 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 your screening appointment
- You have been diagnosed with severe hemophilia A from birth, or hemophilia A where your body has developed antibodies (called inhibitors) that block the usual treatment
- You are already using a medication called rFVIIa to treat bleeding episodes, or you are willing to switch to it during the trial
- You have had a blood test to measure your inhibitor levels done during screening, with results available before the study drug is first given
- If you do not have inhibitors and previously completed a treatment called immune tolerance induction (ITI), it must have been successfully finished at least 5 years before screening, with no sign of inhibitors returning since then (confirm with trial site)
- Your blood counts, liver function, and kidney function are all at an acceptable level
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 undergoing or planning to start ITI therapy (though people for whom ITI has not worked may still be eligible after a 72-hour waiting period)
- You have a history of drug or alcohol misuse within the past 48 weeks, based on the doctor's assessment
- You are considered at high risk for a serious condition affecting small blood vessels (called TMA), for example due to your own or your family's medical history
- You have been treated for blood clots in the past 12 months, are currently being treated for them, or show signs of having them (with a limited exception for previous clots related to a medical catheter)
- You have other conditions, such as certain immune system diseases, that may currently raise your risk of bleeding or clotting
- You have had a significant allergic reaction to a monoclonal antibody treatment (a type of laboratory-made protein medication) or to any ingredient in the study drug
- You have HIV with a specific immune cell count below 200 cells per microliter, indicating a weakened immune system
- You are currently taking or planning to take medications that affect the immune system (such as interferon), not including HIV antiviral medications
- You have another disease, condition, or abnormal test result that the doctor believes could interfere with the study or put you at additional risk
- You are pregnant, breastfeeding, or planning to become pregnant during the study
- You have had a positive pregnancy blood test within 7 days before starting the study drug
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
Expansion Part: Annualized Bleeding Rate (ABR) for Treated Bleeds; Expansion Part: Annualized Bleeding Rate (ABR) for All Bleeds; Expansion Part: Annualized Bleeding Rate (ABR) for Treated Spontaneous Bleeds; Expansion Part: Annualized Bleeding Rate (ABR) for Treated Joint Bleeds; Expansion Part: Annualized Bleeding Rate (ABR) for Treated Target Joint Bleeds
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.