Canadian Hemophilia Prophylaxis Study (CHPS)

November 18, 2019 updated by: Brian Feldman, The Hospital for Sick Children

Moderate Term Musculoskeletal Outcomes With Escalating Dose Prophylaxis: the Canadian Hemophilia Prophylaxis Study Follow-up Study

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.

Study Overview

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).

Study Type

Interventional

Enrollment (Actual)

56

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • The Hospital for Sick Children

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 2 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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.)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Factor VIII
escalating dose Factor VIII
escalating dose prophylaxis
Other Names:
  • Recombinant Factor VIII (antihemophilic agent)
escalating dose
Other Names:
  • recombinant factor VIII (antihemophilic agent)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Annualized Bleeding Rate
Time Frame: 6 months
Number of index hemarthorses (bleeds into ankles, elbows or knees) per patient per year
6 months
Annualized Factor Use
Time Frame: 12 months
annual factor usage per subject
12 months
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
6 months
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.

through study completion, a median of 10 years
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
through study completion, a median of 10 years
Complications Arising From Indwelling Venous Catheter
Time Frame: 6 months
collect information on any complications relating to indwelling venous catheters that some subject use.
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Principal Investigator: Brian M Feldman, MD, The Hospital for Sick Children

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 26, 1997

Primary Completion (Actual)

December 1, 2012

Study Completion (Actual)

December 1, 2014

Study Registration Dates

First Submitted

March 1, 2010

First Submitted That Met QC Criteria

March 10, 2010

First Posted (Estimate)

March 11, 2010

Study Record Updates

Last Update Posted (Actual)

December 5, 2019

Last Update Submitted That Met QC Criteria

November 18, 2019

Last Verified

November 1, 2019

More Information

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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