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

Sponsor:	The Hospital for Sick Children
Information provided by:	The Hospital for Sick Children
ClinicalTrials.gov Identifier:	NCT00717626

Purpose

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 II

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Single Group Assignment, Efficacy Study
Official Title:	Pilot Study of Once-A-Day Prophylaxis for Youth and Young Adults With Severe Hemophilia A

Resource links provided by NLM:

Genetics Home Reference related topics: hemophilia Help Me Understand Genetics

MedlinePlus related topics: Hemophilia

Drug Information available for: Octocog alfa Moroctocog Alfa Factor VIII

U.S. FDA Resources

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 ]

Estimated Enrollment:	25
Study Start Date:	July 2008
Estimated Study Completion Date:	December 2009
Estimated Primary Completion Date:	July 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Experimental	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 < 75 kg.) or 500 units per day (weight ≥ 75 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.

Eligibility

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

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00717626

Contacts

Contact: Brian Feldman, MD

(416)813-5677

brian.feldman@sickkids.ca

Locations

Canada, Ontario
The Hospital for Sick Children	Recruiting
Toronto, Ontario, Canada
Contact: Brian Feldman (416)813-5677 brian.feldman@sickkids.ca
St. Michael's Hospital	Recruiting
Toronto, Ontario, Canada
Contact: Jerome Teitel
Canada, Quebec
Hospital Sainte-Justine	Recruiting
Montreal, Quebec, Canada
Contact: Georges Rivard

Sponsors and Collaborators

The Hospital for Sick Children

Investigators

Principal Investigator:

Brian Feldman, MD

The Hospital for Sick Children

More Information

No publications provided

Responsible Party:	The Hospital for Sick Children ( Brian Feldman/Principal Investigator )
Study ID Numbers:	1000012140
Study First Received:	July 14, 2008
Last Updated:	September 19, 2008
ClinicalTrials.gov Identifier:	NCT00717626 History of Changes
Health Authority:	Canada: Health Canada

Keywords provided by The Hospital for Sick Children:

Hemophilia
Prophylaxis
Youth
Young Adults
Feasibility

Additional relevant MeSH terms:

Hemorrhagic Disorders
Blood Coagulation Disorders, Inherited
Genetic Diseases, Inborn
Coagulation Protein Disorders

Hematologic Diseases
Blood Coagulation Disorders
Hemophilia A

ClinicalTrials.gov processed this record on February 08, 2010