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

Brief Title

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

Official Title

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

Brief 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.

Study Phase

Phase 2

Study Type

Interventional

Primary Outcome

To test the feasibility of a large-scale clinical trial of once-daily prophylaxis. As such, the primary outcome will be related to process measures.

Secondary Outcome

 Clinical efficacy

Condition

Hemophilia A

Intervention

Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS

Study Arms / Comparison Groups

Description:

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

Drug

Estimated Enrollment

Start Date

July 2008

Completion Date

April 2011

Primary Completion Date

April 2011

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

Gender

Male

Ages

12 Years - 24 Years

Accepts Healthy Volunteers

No

Contacts

Brian Feldman, MD,MSc,FRCPC, ,

Location Countries

Canada

Location Countries

Canada

Administrative Informations

NCT ID

NCT00717626

Organization ID

1000012140

Responsible Party

Principal Investigator

Study Sponsor

The Hospital for Sick Children

Study Sponsor

Brian Feldman, MD,MSc,FRCPC, Principal Investigator, The Hospital for Sick Children

Verification Date

December 2013