NCT00717626

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

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: The Hospital for Sick Children · Academic / Other
• Sex: Male
• Age: 12 Years – 24 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Hemophilia is an important and costly disorder; if left untreated, it may have serious consequences. The greatest impact of hemophilia occurs from recurrent bleeding into joints. The consequences of joint bleeding include pain associated with acute bleeding and later chronic arthropathy. Half of affected children with severe hemophilia have joint damage; joint damage is more frequent with increased bleeding. The prevention and treatment of bleeding is very expensive and therefore finding a cost-effective treatment is of high priority. Worldwide, two major treatment strategies have been used to prevent arthropathy - on demand therapy and factor prophylaxis. The goal of prophylaxis is to convert the severe to the moderate phenotype by providing circulating factor activity of greater than 1%. Patients with greater than 1% circulating factor VIII activity rarely have spontaneous hemarthroses. Therefore, the goal of providing circulating factor at this level is to eliminate spontaneous hemarthroses. The term primary prophylaxis suggests using preventative factor VIII replacement from a very early age. The term secondary prophylaxis is used to describe the application of prophylaxis at a later disease stage. In this study, secondary prophylaxis will be used. Once-daily prophylaxis is a novel application of hemophilia factor prophylaxis for youth and young adults. Before embarking on a costly definitive trial we feel that it is necessary to demonstrate that subjects will be willing to enroll and will be compliant with the therapy. Moreover, we need to establish an estimate of the effect of once-daily prophylaxis on bleeding rates, quality of life, and joint damage progression in order to design a definitive trial.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
DRUG	Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS	Starting at the 4-month visit, subjects will receive 250 units per day (if their weight is \< 50 kg.) or 500 units per day (weight ≥ 50 kg.) of their usual preparation of factor VIII.

Timeline

• Start date: 2008-07-01
• Primary completion: 2011-04-01
• Completion: 2011-04-01
• First posted: 2008-07-17
• Last updated: 2020-10-08
• Results posted: 2020-10-08

Locations

3 sites across 1 country: Canada