Health Related Quality of Life of Youth and Young Adults With Hemophilia A

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01034904
Recruitment Status : Completed
First Posted : December 18, 2009
Last Update Posted : December 10, 2013
Information provided by (Responsible Party):
CSL Behring

Brief Summary:
The relevance of the study will be in establishing whether Health Related Quality of Life (HRQoL) measures obtained repeatedly during hemophilia care are sensitive to significant changes in health or personal circumstances related to the transition from youth to adulthood. This may support the future use of HRQoL measures as part of routine care in order to identify important changes that are not detectible by other clinical means.

Condition or disease
Hemophilia A

Study Type : Observational
Actual Enrollment : 48 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Prospective Non-Interventional Study to Describe Health Related Quality of Life of Youth and Young Adults With Moderate or Severe Hemophilia A Using Recombinant Factor VIII (Helixate FS).
Study Start Date : October 2009
Primary Completion Date : July 2013
Study Completion Date : November 2013

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia
U.S. FDA Resources

Patients who have moderate or severe Hemophilia A, living in Canada and who are using Helixate FS either on-demand or prophylaxis

Primary Outcome Measures :
  1. Health Related Quality of Life (HRQoL) [ Time Frame: Every 6 months over 2 to 3 years ]

Secondary Outcome Measures :
  1. Sensitivity of HRQoL measures to significant life events [ Time Frame: 3 years ]

Information from the National Library of Medicine

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Ages Eligible for Study:   14 Years to 30 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The study is focused on youth (14.0 to 18.0 years of age) and young adults (18.01 to 29.99 years of age), living in Canada, who have moderate and severe Hemophilia A, and who are using Helixate FS either on-demand or as prophylaxis treatment.

Inclusion Criteria:

  • Moderate or severe Hemophilia A (factor level ≤5%)
  • Receiving Helixate FS as on-demand treatment or prophylaxis therapy at time of informed consent
  • Patient age 14 to 29 years at the time of recruitment
  • Complete informed consent process and documentation; (Patients under age 18 must assent in addition to consent of legal guardian)
  • Willing/able to follow study protocol, including keeping a bleeding and treatment diary
  • Able to communicate clearly in either English or French - both written and verbal communication is essential.

Exclusion Criteria:

  • Known inhibitor at time of recruitment (> 0,5 Bethesda units) or immune tolerance therapy at time of recruitment
  • HIV positive
  • Symptomatic Hepatitis B or C infection or active treatment for hepatitis B or C
  • Inability to comply with study protocol
  • Hypersensitivity to Helixate FS or to any ingredients in the formulation or component of the container
  • Hypersensitivity to mouse or hamster protein
  • Unsuitable to participate in study for any other reason as assessed by investigator

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01034904

Canada, Ontario
Sick Kids Hospital
Toronto, Ontario, Canada, M5G 1X8
Canada, Quebec
CHU Sainte-Justine
Montreal, Quebec, Canada, H3T 1C5
Sponsors and Collaborators
CSL Behring
Principal Investigator: Jean St-Louis, MD St. Justine's Hospital

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: CSL Behring Identifier: NCT01034904     History of Changes
Other Study ID Numbers: CE1250_5002_CA
First Posted: December 18, 2009    Key Record Dates
Last Update Posted: December 10, 2013
Last Verified: December 2013

Keywords provided by CSL Behring:
Quality of Life
Hemophilia A
Helixate FS

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII