Brief Title
Canadian Hemophilia Prophylaxis Study
Official Title
Moderate Term Musculoskeletal Outcomes With Escalating Dose Prophylaxis: the Canadian Hemophilia Prophylaxis Study Follow-up Study
Brief 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.
Detailed Description
There are 2 specific study objectives. The first is to estimate the incidence of target joint
bleeding in patients with severe hemophilia A treated (for primary prophylaxis) with
Escalating Dose Prophylactic factor replacement. The second objective is to obtain accurate
estimates of the direct and indirect costs associated with this protocol for use in a
cost-effectiveness model (comparing Escalating Dose with standard prophylaxis and with
intermittent therapy).
Study Phase
Phase 4
Study Type
Interventional
Primary Outcome
Number of Participants Who Developed Target Joint Bleeding
Secondary Outcome
Annualized Bleeding Rate
Condition
Severe Hemophilia A
Intervention
Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)
Study Arms / Comparison Groups
Factor VIII
Description: escalating dose Factor VIII
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
56
Start Date
June 26, 1997
Completion Date
December 2014
Primary Completion Date
December 2012
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.)
Gender
Male
Ages
12 Months - 30 Months
Accepts Healthy Volunteers
No
Contacts
Brian M Feldman, MD, ,
Location Countries
Canada
Location Countries
Canada
Administrative Informations
NCT ID
NCT01085344
Organization ID
0019970022
Responsible Party
Principal Investigator
Study Sponsor
The Hospital for Sick Children
Study Sponsor
Brian M Feldman, MD, Principal Investigator, The Hospital for Sick Children
Verification Date
November 2019