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Canadian Hemophilia Prophylaxis Study (CHPS)

Primary Purpose

Severe Hemophilia A

Status
Completed
Phase
Phase 4
Locations
Canada
Study Type
Interventional
Intervention
Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)
Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)
Sponsored by
The Hospital for Sick Children
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Severe Hemophilia A focused on measuring hemophilia, prophylaxis, cost effectiveness

Eligibility Criteria

12 Months - 30 Months (Child)MaleDoes not accept healthy volunteers

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.)

Sites / Locations

  • The Hospital for Sick Children

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Factor VIII

Arm Description

escalating dose Factor VIII

Outcomes

Primary Outcome Measures

Number of Participants Who Developed Target Joint Bleeding
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

Annualized Bleeding Rate
Number of index hemarthorses (bleeds into ankles, elbows or knees) per patient per year
Annualized Factor Use
annual factor usage per subject
Number of Patients Who Developed an Inhibitor to FVIII
The number of patients who developed an inhibitor for FVIII, defined as >= 0.5 Bethesda Units
Physical Disability as Measured by the CHAQ
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.
Joint Damage as Determined by the Physiotherapy Score
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
Complications Arising From Indwelling Venous Catheter
collect information on any complications relating to indwelling venous catheters that some subject use.

Full Information

First Posted
March 1, 2010
Last Updated
November 18, 2019
Sponsor
The Hospital for Sick Children
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1. Study Identification

Unique Protocol Identification Number
NCT01085344
Brief Title
Canadian Hemophilia Prophylaxis Study
Acronym
CHPS
Official Title
Moderate Term Musculoskeletal Outcomes With Escalating Dose Prophylaxis: the Canadian Hemophilia Prophylaxis Study Follow-up Study
Study Type
Interventional

2. Study Status

Record Verification Date
November 2019
Overall Recruitment Status
Completed
Study Start Date
June 26, 1997 (Actual)
Primary Completion Date
December 2012 (Actual)
Study Completion Date
December 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
The Hospital for Sick Children

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.
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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Severe Hemophilia A
Keywords
hemophilia, prophylaxis, cost effectiveness

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
56 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Factor VIII
Arm Type
Experimental
Arm Description
escalating dose Factor VIII
Intervention Type
Biological
Intervention Name(s)
Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)
Other Intervention Name(s)
Recombinant Factor VIII (antihemophilic agent)
Intervention Description
escalating dose prophylaxis
Intervention Type
Biological
Intervention Name(s)
Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)
Other Intervention Name(s)
recombinant factor VIII (antihemophilic agent)
Intervention Description
escalating dose
Primary Outcome Measure Information:
Title
Number of Participants Who Developed Target Joint Bleeding
Description
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.
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Annualized Bleeding Rate
Description
Number of index hemarthorses (bleeds into ankles, elbows or knees) per patient per year
Time Frame
6 months
Title
Annualized Factor Use
Description
annual factor usage per subject
Time Frame
12 months
Title
Number of Patients Who Developed an Inhibitor to FVIII
Description
The number of patients who developed an inhibitor for FVIII, defined as >= 0.5 Bethesda Units
Time Frame
6 months
Title
Physical Disability as Measured by the CHAQ
Description
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.
Time Frame
through study completion, a median of 10 years
Title
Joint Damage as Determined by the Physiotherapy Score
Description
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
Time Frame
through study completion, a median of 10 years
Title
Complications Arising From Indwelling Venous Catheter
Description
collect information on any complications relating to indwelling venous catheters that some subject use.
Time Frame
6 months

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
12 Months
Maximum Age & Unit of Time
30 Months
Accepts Healthy Volunteers
No
Eligibility 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.)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Brian M Feldman, MD
Organizational Affiliation
The Hospital for Sick Children
Official's Role
Principal Investigator
Facility Information:
Facility Name
The Hospital for Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada

12. IPD Sharing Statement

Citations:
PubMed Identifier
29731369
Citation
Feldman BM, Rivard GE, Babyn P, Wu JKM, Steele M, Poon MC, Card RT, Israels SJ, Laferriere N, Gill K, Chan AK, Carcao M, Klaassen RJ, Cloutier S, Price VE, Dover S, Blanchette VS. Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort. Lancet Haematol. 2018 Jun;5(6):e252-e260. doi: 10.1016/S2352-3026(18)30048-6. Epub 2018 May 3.
Results Reference
derived

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Canadian Hemophilia Prophylaxis Study

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