CompletedNot Applicable

Canadian CHO-KLAT/H-FIT Study - Quality of Life of, and Burden of Caring for, Persons With Hemophilia

Canada190 participantsStarted 2024-07-02

Plain-language summary

The primary goal of this observational study is to determine if: * health-related quality of life of boys with moderate/severe hemophilia A who are followed in Canadian pediatric hemophilia treatment centres is significantly different for boys receiving an intravenously administered factor replacement product compared to a subcutaneously administered non-factor replacement product, as measured at the 3 month time-point. * Burden of caring for a boy with moderate/severe hemophilia A is significantly different for parents/caregivers of boys with moderate/severe hemophilia A receiving an intravenously administered factor replacement product compared to a subcutaneously administered non-factor replacement product, as measured at the 3 month time-point. Persons with hemophilia and their caregivers will complete questionnaires at baseline, three months, six months, and one week after six months.

Who can participate

Age range

1 Month – 18 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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%. * …

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Differences in health-related quality of life between boys receiving an intravenously administered factor product compared to subcutaneous non-factor product.

Timeframe: 3 months

Differences in burden of caring for boys receiving an intravenously administered factor product compared to subcutaneous non-factor product.

Timeframe: 3 months

Trial details

NCT IDNCT06191068

SponsorThe Hospital for Sick Children

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2025-11-30

View on ClinicalTrials.gov More Hemophilia trials

Plain-language summary

Who can participate

Age range

1 Month – 18 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Differences in health-related quality of life between boys receiving an intravenously administered factor product compared to subcutaneous non-factor product.

Timeframe: 3 months

Differences in burden of caring for boys receiving an intravenously administered factor product compared to subcutaneous non-factor product.

Timeframe: 3 months