Phase 2
N=14
Once-A-Day Prophylaxis for Youth and Young Adults With Severe Hemophilia A
Hemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT00717626 ↗Enrolled (actual)
14
Serious AEs
0.0%
Results posted
Oct 2020
Primary outcome: Primary: Hemophilia Joint Health Score (HJHS) 2.0 — 3.77 score on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS (Drug)
- Age
- Pediatric, Adult · 12+ yrs
- Sex
- Male
- Sponsor
- The Hospital for Sick Children
- Primary completion
- Apr 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Hemophilia Joint Health Score (HJHS) 2.0 |
3.77 | — |
| PRIMARY Functional Independence Score in Hemophilia (FISH) |
0.31 | — |
| PRIMARY HRQoL Transformed Z-Score (Includes the Canadian Haemophilia Outcomes-Kids Life Assessment Tool (CHO-KLAT) and Haemo-QoL A) |
— | — |
| PRIMARY Treatment Satisfaction Questionnaire for Medication (TSQM) . Version II |
-6.41; -4.70; -5.13 | — |
| PRIMARY The Previous Day Physical Activity Recall |
-0.15; -0.69; -0.15; 0; -0.92; 2.31 | — |
| PRIMARY Total Number of Index Joint Bleeds |
0; 0; 0 | — |
| SECONDARY Mean Yearly Factor Consumption of FVIII Product on Low Dose Daily Prophylaxis |
211528 | — |
Summary
The primary objective of this study is to test the feasibility of a large-scale clinical trial of once-daily prophylaxis. The secondary objectives are to collect clinical efficacy outcomes so that we can better plan a large-scale study; we will estimate the effect size and variability of effect and resource utilization of once-daily prophylaxis to allow us to set a sample size for a definitive trial.
Eligibility Criteria
Inclusion Criteria
- Severe hemophilia A (<1% factor VIII)
- Age 12 to 24 years inclusive
- Male
- No current factor VIII inhibitor (an inhibitor will be defined as ≥ 0.6 Bethesda Units) within the past year
- Able to participate in a home infusion program with adequate peripheral venous access as assessed ny the treating investigator
Exclusion Criteria
- Important co-morbidities (Acquired Immunodeficiency Syndrome or symptomatic HIV infection, symptomatic hepatitis B or C infection)
- Other concomitant acquired or congenital bleeding disorder (e.g. von Willebrand's Disease)
- Receiving factor VIII replacement through central venous catheter
Data sourced from ClinicalTrials.gov (NCT00717626). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.