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

NCT06191068 | Completed

• 1 other identifier: 41238567
• Study Type: observational
• Target: 190
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Hemophilia

Keywords

health-related quality of life; quality of life; burden of care

Outcome Measures

Primary Outcomes (2)

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

  The correlation of the CHO-KLAT scores 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. The Canadian Hemophilia Outcomes-Kids' Life Assessment Tool v3.0 has 41 questions. Items 1 to 28 are scored on a 5-point scale from 1 (Never) to 5 (Always). Items 29 to 41 are scored on the same 5-point scale, with an additional Not Applicable option. The tool is scored using a summary score of all items from 0 to 100, with 100 representing the best quality of life.

  3 months

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

  The correlation of the H-FIT scores for parents/caregivers of 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. The Hemophilia Family Impact Tool v1.1 has 25 questions scored on a 5-point scale from 1 (never) to 5 (always). The questionnaire is scored as a summary score of all items from 0 to 100, with 100 representing the lowest family burden.

  3 months

Secondary Outcomes (1)

• Validity of the CHO-KLAT and H-FIT

  6 months

Eligibility Criteria

• Age: 1 Month - 18 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• The Hospital for Sick Children: lead
• Roche Pharma AG: collaborator

Study Sites (1)

The Hospital for Sick Children

Toronto, Ontario, Canada

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator, Staff Physician Division of Haematology/Oncology

Study Record Dates

• First Submitted: December 4, 2023
• First Posted: January 5, 2024
• Study Start: July 2, 2024
• Primary Completion: November 30, 2025
• Study Completion: November 30, 2025
• Last Updated: April 24, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)