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Canadian Hemophilia Prophylaxis Study (CHPS)

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.
 
ClinicalTrials.gov Identifier: NCT01085344
Recruitment Status : Completed
First Posted : March 11, 2010
Results First Posted : December 5, 2019
Last Update Posted : December 5, 2019
Sponsor:
Information provided by (Responsible Party):
Brian Feldman, The Hospital for Sick Children

Brief Summary:
Primary prophylaxis given less frequently initially, with the infusion frequency increased if needed (Escalating Dose Prophylaxis), is likely to be less expensive and associated with fewer complications than standard prophylaxis while reducing disability to a greater degree than intermittent therapy.

Condition or disease Intervention/treatment Phase
Severe Hemophilia A Biological: Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS) Phase 4

Detailed Description:
There are 2 specific study objectives. The first is to estimate the incidence of target joint bleeding in patients with severe hemophilia A treated (for primary prophylaxis) with Escalating Dose Prophylactic factor replacement. The second objective is to obtain accurate estimates of the direct and indirect costs associated with this protocol for use in a cost-effectiveness model (comparing Escalating Dose with standard prophylaxis and with intermittent therapy).

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 56 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Moderate Term Musculoskeletal Outcomes With Escalating Dose Prophylaxis: the Canadian Hemophilia Prophylaxis Study Follow-up Study
Actual Study Start Date : June 26, 1997
Actual Primary Completion Date : December 2012
Actual Study Completion Date : December 2014


Arm Intervention/treatment
Experimental: Factor VIII
escalating dose Factor VIII
Biological: Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)
escalating dose prophylaxis
Other Name: Recombinant Factor VIII (antihemophilic agent)

Biological: Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)
escalating dose
Other Name: recombinant factor VIII (antihemophilic agent)




Primary Outcome Measures :
  1. Number of Participants Who Developed Target Joint Bleeding [ Time Frame: 6 months ]
    The number of participants who developed target joint bleeding during the study, which was defined as 3 bleeds into any 1 joint within a period of 3 months.


Secondary Outcome Measures :
  1. Annualized Bleeding Rate [ Time Frame: 6 months ]
    Number of index hemarthorses (bleeds into ankles, elbows or knees) per patient per year

  2. Annualized Factor Use [ Time Frame: 12 months ]
    annual factor usage per subject

  3. Number of Patients Who Developed an Inhibitor to FVIII [ Time Frame: 6 months ]
    The number of patients who developed an inhibitor for FVIII, defined as >= 0.5 Bethesda Units

  4. Physical Disability as Measured by the CHAQ [ Time Frame: through study completion, a median of 10 years ]

    complete the Child Health Assessment Questionnaire (CHAQ) at each 6 month visit. The CHAQ is a validated tool to measure a disability index, with a possible score range of 0-3, where 0 represents no disability and 3 represents maximal disability. The CHAQ is known to have a strong ceiling effect.

    The CHAQ was collected at each study visit (i.e. every 6 months for the duration each patient was on study). The reported score represents the median end of study score.


  5. Joint Damage as Determined by the Physiotherapy Score [ Time Frame: through study completion, a median of 10 years ]
    Complete the modified Colarado Physiotherapy Assessment every 6 months at each visit with a score range 0-30 for ankles and knees and 0-26 for elbows), measured at all study visits, which we modified by not assessing crepitus or the ankle joint circumference measurement. For each scale, 0 represents no joint damage, with 26/30 representing maximum possible joint damage. The reported score represents the median end of study score

  6. Complications Arising From Indwelling Venous Catheter [ Time Frame: 6 months ]
    collect information on any complications relating to indwelling venous catheters that some subject use.



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Ages Eligible for Study:   12 Months to 30 Months   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Severe hemophilia A (factor level less than 2%).
  • Age greater than 1 year and less than or equal to 2.5 years.
  • Normal joints using the World Federation of Hemophilia orthopedic scale.
  • Normal radiographs of joints in which bleeding has occurred using the World Federation of Hemophilia radiographic scale.
  • Platelet count of > 150,000.
  • Informed consent to participate.

Exclusion Criteria:

  • Three or more clinically determined bleeds into any single elbow, knee or ankle.
  • Presence or past history of a circulating inhibitor (level ≥ 0.5 Bethesda Units).
  • Family judged to be non-compliant by the local hemophilia clinic director.
  • Competing risk (symptomatic HIV infection, juvenile rheumatoid arthritis, metabolic bone disease, or other diseases known to cause or mimic arthritis.)

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 ClinicalTrials.gov identifier (NCT number): NCT01085344


Locations
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Canada, Ontario
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Sponsors and Collaborators
The Hospital for Sick Children
Investigators
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Principal Investigator: Brian M Feldman, MD The Hospital for Sick Children

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Brian Feldman, Division Head, Rheumatology, The Hospital for Sick Children
ClinicalTrials.gov Identifier: NCT01085344    
Other Study ID Numbers: 0019970022
First Posted: March 11, 2010    Key Record Dates
Results First Posted: December 5, 2019
Last Update Posted: December 5, 2019
Last Verified: November 2019
Keywords provided by Brian Feldman, The Hospital for Sick Children:
hemophilia, prophylaxis, cost effectiveness
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants