Clinical Trial Details
— Status: Not yet recruiting
Administrative data
| NCT number |
NCT06191068 |
| Other study ID # |
41238567 |
| Secondary ID |
|
| Status |
Not yet recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
July 2024 |
| Est. completion date |
June 2025 |
Study information
| Verified date |
April 2024 |
| Source |
The Hospital for Sick Children |
| Contact |
n/a |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical Trial 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.
Description:
Hemophilia A is an inherited bleeding disorder that affects 1/5000 live male births. Advances
in hemophilia care through the development of recombinant standard and extended half-life
(SHL and EHL, respectively) clotting factor concentrates (CFCs) have improved disease
management; however, the burden of frequent administration of intravenous CFCs can contribute
to negative physical and psychosocial outcomes in both the patient and their family. A new
non-factor hemostatic agent (emicizumab) allows for subcutaneous injections, given on a
schedule that ranges from once weekly to once monthly. Given the very high cost of preventive
treatments ("prophylaxis") as advances in care of persons with hemophilia are made (e.g., EHL
CFCs, emicizumab), it is imperative that the potential health outcome benefits of these
treatments are assessed using relevant validated outcome measures. There are two
patient-reported outcome measures (PROMs) that have been previously validated for use in boys
with hemophilia using SHL or EHL CFCs and their parents: the Canadian Hemophilia
Outcomes-Kids Life Assessment Tool version 3 (CHO-KLAT 3.0) assessing health-related quality
of life for boys with hemophilia A ages 4-18, and the Hemophilia Family Impact Tool version
1.1 (H-FIT 1.1) assessing burden of caring for a child with hemophilia for parents of boys
with hemophilia A ages 0-18 years. However, their use in the context of emicizumab has not
been evaluated. As such, the primary aim of this study is to determine if health-related
quality of life of boys with moderate/severe hemophilia A who are followed in Canadian
pediatric HTCs is significantly different for boys receiving an intravenously administered
factor replacement product compared to a subcutaneously administered non-factor replacement
product, and to determine if 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.
Secondary aims include:
- To assess the validity of the CHO-KLAT 3.0/H-FIT 1.1 for use in the context of
emicizumab.
- To determine the measurement properties of the CHO-KLAT 3.0 and the H-FIT 1.1, including
the minimal clinical important difference, the test-retest reliability, and the standard
error of measurement (SEM);
- To determine parents' satisfaction with emicizumab using the Emicizumab Preference
(EmiPref) Survey;
- To translate the CHO-KLAT 3.0 and the H-FIT 1.1 to French (Canadian) and assess its
validity.
Lastly, the investigators will explore the HRQoL and impact of hemophilia on boys with
moderate/severe hemophilia A complicated by FVIII inhibitors, and their families. The
investigators will also explore the use of the EmiPref and Parental Needs Scale for Rare
Diseases (PNF-RD) in parents of boys with moderate/severe hemophilia to investigate their
relationship with the disease-specific CHO-KLAT 3.0 and H-FIT 1.1, respectively.
The CHO-KLAT 3.0 is a validated, child-centric HRQoL measure for use in boys with hemophilia
between the ages of 7-18 (self-report), and between the ages of 4-18 (parent-proxy report).
The H-FIT 1.0 was developed to determine the impact of caring for a child with hemophilia
from birth to 18 years on the family, and is especially relevant during the early years of
diagnosis and initiation of primary prophylaxis. The H-FIT 1.1 was adapted following
modifications during cognitive debriefing (ongoing). Additional tools that will be included
in this study are the Pediatric Quality of Life-Core Module (PedsQL-Core), the Pediatric
Quality of Life-Family Impact Module (PedsQL-FIM), the Parental Needs Scale for Rare Diseases
(PNS-RD), and the Emicizumab Preference Survey (EmiPref). The PedsQL-Core is a validated,
generic tool designed to measure the impact of having a chronic health condition on quality
of life and will be used to determine construct validity of the hemophilia-specific CHO-KLAT
3.0. The PedsQL-FIM is a validated, generic tool designed to measure the impact of caring for
a child with a chronic health condition on parents and the family and will be used to
determine construct validity of the hemophilia-specific H-FIT 1.1. The PNS-RD is a measure of
the supportive care needs of parents of children with rare diseases and its relationship with
the H-FIT 1.1 will be explored. The EmiPref is an industry (Roche)-developed tool used to
assess patients' treatment preference and will be used to examine patients' satisfaction with
emicizumab compared to previous treatments. These measures will be translated into French
(Canadian) prior to commencing data collection (Phase 1). Cognitive debriefing will be
completed with boys (n=8) and parents (n=8) at one French-speaking site in Quebec to confirm
the translated measures are clear and relevant. Questionnaires will be administered in a
random order to account for order effects.
For all groups, reliability data will also be collected with a repeat administration of the
CHO-KLAT 3.0 and H-FIT 1.1 within 7 days of the 6-month time point in study subjects in a
non-bleeding state (by self-report on the study case report form) for a minimum of 2 weeks
prior to the administration of the questionnaires, since reliability can only be determined
if the health status of the participant has not changed between administrations. Only one
parent/guardian will be requested to complete the questionnaires per family.
