Canadian Hemophilia Prophylaxis Study (CHPS)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Brian Feldman, The Hospital for Sick Children
ClinicalTrials.gov Identifier:
NCT01085344
First received: March 1, 2010
Last updated: December 17, 2013
Last verified: December 2013
  Purpose

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

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Moderate Term Musculoskeletal Outcomes With Escalating Dose Prophylaxis: the Canadian Hemophilia Prophylaxis Study Follow-up Study

Resource links provided by NLM:


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

Primary Outcome Measures:
  • the frequency of development of target joint bleeding [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • #of bleeding episodes [ Time Frame: 6 months ] [ Designated as safety issue: No ]
  • #of factor infusions [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    number of infusions and reason for infusion to access cost of treatment

  • circulating inhibitor [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
    check for presence of inhitibors

  • physical disability as measured by the CHAQ [ Time Frame: 6 nibths ] [ Designated as safety issue: No ]
    complete the Child Health Assessment Questionaire at each 6 month visit

  • joint damage as determined by the physiotherapy score [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    Complete the modified Colarado Physiotherapy Assessment at each visit

  • complications arising from indwelling venous catheter [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
    collect information on any complications relating to indwelling venous catheters that some subject use.


Enrollment: 56
Study Start Date: July 1997
Estimated Study Completion Date: December 2014
Primary Completion Date: December 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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)

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).

  Eligibility

Ages Eligible for Study:   12 Months to 30 Months
Genders 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.)
  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 ClinicalTrials.gov identifier: NCT01085344

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

No publications provided

Responsible Party: Brian Feldman, Division Head, Rheumatology, The Hospital for Sick Children
ClinicalTrials.gov Identifier: NCT01085344     History of Changes
Other Study ID Numbers: 0019970022
Study First Received: March 1, 2010
Last Updated: December 17, 2013
Health Authority: Canada: Health Canada

Keywords provided by The Hospital for Sick Children:
hemophilia, prophylaxis, cost effectiveness

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
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 18, 2014