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

This study has been completed.
Information provided by (Responsible Party):
Brian Feldman, The Hospital for Sick Children Identifier:
First received: July 14, 2008
Last updated: December 17, 2013
Last verified: December 2013

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.

Condition Intervention Phase
Hemophilia A
Drug: Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Pilot Study of Once-A-Day Prophylaxis for Youth and Young Adults With Severe Hemophilia A

Resource links provided by NLM:

Further study details as provided by The Hospital for Sick Children:

Primary Outcome Measures:
  • 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. [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Clinical efficacy [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Estimate the effect size and variability of effect and resource utilization of once-daily prophylaxis. [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Enrollment: 14
Study Start Date: July 2008
Study Completion Date: April 2011
Primary Completion Date: April 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 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.

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.


Ages Eligible for Study:   12 Years to 24 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00717626

Canada, Ontario
St. Michael's Hospital
Toronto, Ontario, Canada
The Hospital for Sick Children
Toronto, Ontario, Canada
Canada, Quebec
Hospital Sainte-Justine
Montreal, Quebec, Canada
Sponsors and Collaborators
The Hospital for Sick Children
Principal Investigator: Brian Feldman, MD,MSc,FRCPC The Hospital for Sick Children
  More Information

No publications provided

Responsible Party: Brian Feldman, Division Head, Rheumatology, The Hospital for Sick Children Identifier: NCT00717626     History of Changes
Other Study ID Numbers: 1000012140
Study First Received: July 14, 2008
Last Updated: December 17, 2013
Health Authority: Canada: Health Canada

Keywords provided by The Hospital for Sick Children:
Young Adults

Additional relevant MeSH terms:
Hemophilia A
Hemophilia B
Blood Coagulation Disorders
Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Hematologic Diseases
Hemorrhagic Disorders processed this record on March 01, 2015