Study compares quality of life for boys on different hemophilia drugs
NCT ID NCT06191068
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This completed observational study looked at boys aged 7-18 with moderate or severe hemophilia A in Canadian treatment centres. Researchers compared quality of life and caregiver burden between those receiving an intravenous factor replacement product and those on a subcutaneous non-factor product. Participants and their caregivers filled out questionnaires at several time points to see if one treatment approach was linked to better well-being.
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Study facts
What this study's own registry entry says, in plain language.
- Participants
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190 people
The number who actually took part.
- Started
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Jul 2024
- Finished
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Nov 2025
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
This study will involve data collection on male patients 18 years of age or less with moderate or severe hemophilia A, on prophylaxis, with or without FVIII inhibitors, and their parents/guardians. Parents/guardians of boys \<18 years with moderate or severe hemophilia A will also be included for the objectives that involve the H-FIT 1.1.
- Ages
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1 month to 18 years
- Sex
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Male participants only
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Boys with moderate/severe hemophilia A, with or without FVIII inhibitors, age 7-18 years, * Group A: who have previously received prophylaxis with SHL or EHL CFCs at least once a week for the previous year who plan on switching or who have switched to emicizumab. * Group B: who are currently receiving prophylaxis at least once a week for the previous year with any form of treatment other than emicizumab who are not switching treatment regimens. * Group C: who have not been on long-term prophylaxis for the year prior to study commencement who begin prophylactic treatment with emicizumab (i.e., previously untreated patients or patients receiving treatment on demand). * Group D: boys with moderate/severe hemophilia A who have neutralizing alloantibodies to FVIII (FVIII inhibitors), defined as a level of ≥0.6 Bethesda Units (BU) using the Nijmegen modification of the Bethesda assay on two separate occasions within a 1-4 week period1, who are receiving a by-passing hemostatic agent such as a non-activated or activated plasma-derived, virus-inactivated prothrombin complex clotting factor concentrate (e.g., FEIBA), recombinant FVIIa (Niastase), or emicizumab. FVIII Inhibitors of \<5 BU are considered low titer and those ≥5 BU are considered high titer. * Parents/caregivers of boys with moderate/severe hemophilia A with or without FVIII inhibitors between the ages of 0-18 years. * Moderate or severe hemophilia A, defined as FVIII activity level ≤5%. * Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations. Exclusion Criteria: Candidates will be excluded from study entry if any of the following criteria exist at the time of screening, or at the time point specified in the individual criterion listed: * Boys with mild hemophilia A, defined as a FVIII activity between 5-40%. * Presence of significant comorbid diseases (e.g., HIV) as per the judgement of the clinical team. * Inability to read, write, and/or understand English or French. * Unable or unwilling to provide informed consent.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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The Hospital for Sick Children
Toronto, Ontario, Canada
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Other studies related to the condition(s) this trial covers.
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