Canadian Hemophilia Prophylaxis Study

Severe Hemophilia A Clinical Trial

— CHPS  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01085344
• Other study ID #: 0019970022
• Status: Completed
• Phase: Phase 4
• Start date: June 26, 1997
• Est. completion date: December 2014

Study information

• Verified date: November 2019
• Source: The Hospital for Sick Children
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 56
• Est. completion date: December 2014
• Est. primary completion date: December 2012
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Months to 30 Months

Eligibility

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.)

Related Conditions & MeSH terms

• Hemophilia A
• Severe Hemophilia A

Intervention

Biological:
• Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)
escalating dose prophylaxis
• Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)
escalating dose

Locations

Country	Name	City	State
Canada	The Hospital for Sick Children	Toronto	Ontario

Sponsors (1)

Lead Sponsor	Collaborator
The Hospital for Sick Children

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	6 months
Secondary	Annualized Bleeding Rate	Number of index hemarthorses (bleeds into ankles, elbows or knees) per patient per year	6 months
Secondary	Annualized Factor Use	annual factor usage per subject	12 months
Secondary	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	6 months
Secondary	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.	through study completion, a median of 10 years
Secondary	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	through study completion, a median of 10 years
Secondary	Complications Arising From Indwelling Venous Catheter	collect information on any complications relating to indwelling venous catheters that some subject use.	6 months