Once-A-Day Prophylaxis for Youth and Young Adults With Severe Hemophilia A

• Status: Completed
• Trial end date: 2011-04-01
• Target enrollment: 0
• Participant gender: Male

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: The Hospital for Sick Children
• Treatments: Factor VIII

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