Phase 4
N=56
Canadian Hemophilia Prophylaxis Study
Severe Hemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT01085344 ↗Enrolled (actual)
56
Serious AEs
0.0%
Results posted
Dec 2019
Primary outcome: Primary: Number of Participants Who Developed Target Joint Bleeding — 17 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS) (Biological)
- Age
- Pediatric · 0+ yrs
- Sex
- Male
- Sponsor
- The Hospital for Sick Children
- Primary completion
- Dec 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Who Developed Target Joint Bleeding |
17 | — |
| SECONDARY Annualized Bleeding Rate |
0.95 | — |
| SECONDARY Annualized Factor Use |
3600 | — |
| SECONDARY Number of Patients Who Developed an Inhibitor to FVIII |
5 | — |
| SECONDARY Physical Disability as Measured by the CHAQ |
— | — |
| SECONDARY Joint Damage as Determined by the Physiotherapy Score |
1; 1; 0; 0; 0; 0 | — |
| SECONDARY Complications Arising From Indwelling Venous Catheter |
— | — |
Summary
Primary prophylaxis given less frequently initially, with the infusion frequency increased if needed (Escalating Dose Prophylaxis), is likely to be less expensive and associated with fewer complications than standard prophylaxis while reducing disability to a greater degree than intermittent therapy.
Eligibility Criteria
Inclusion Criteria
- Severe hemophilia A (factor level less than 2%).
- Age greater than 1 year and less than or equal to 2.5 years.
- Normal joints using the World Federation of Hemophilia orthopedic scale.
- Normal radiographs of joints in which bleeding has occurred using the World Federation of Hemophilia radiographic scale.
- Platelet count of > 150,000.
- Informed consent to participate.
Exclusion Criteria
- Three or more clinically determined bleeds into any single elbow, knee or ankle.
- Presence or past history of a circulating inhibitor (level ≥ 0.5 Bethesda Units).
- Family judged to be non-compliant by the local hemophilia clinic director.
- Competing risk (symptomatic HIV infection, juvenile rheumatoid arthritis, metabolic bone disease, or other diseases known to cause or mimic arthritis.)
Data sourced from ClinicalTrials.gov (NCT01085344). 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.