Canadian Hemophilia Prophylaxis Study

NCT ID: NCT01085344

Last Updated: 2019-12-05

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 56 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 1997-06-26
• Study Completion Date: 2014-12-31

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).

Conditions

Severe Hemophilia A

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Factor VIII

escalating dose Factor VIII

Group Type: EXPERIMENTAL

Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)

Intervention Type: BIOLOGICAL

escalating dose prophylaxis

Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)

Intervention Type: BIOLOGICAL

escalating dose

Interventions

Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)

escalating dose prophylaxis

Intervention Type: BIOLOGICAL

Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)

escalating dose

Intervention Type: BIOLOGICAL

Other Intervention Names

Recombinant Factor VIII (antihemophilic agent); recombinant factor VIII (antihemophilic agent)

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.)

• Minimum Eligible Age: 12 Months
• Maximum Eligible Age: 30 Months
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

The Hospital for Sick Children

OTHER

Sponsor Role: lead

Responsible Party

Brian Feldman

Division Head, Rheumatology

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Brian M Feldman, MD

Role: PRINCIPAL_INVESTIGATOR

The Hospital for Sick Children

Locations

The Hospital for Sick Children

Toronto, Ontario, Canada

Countries

Canada

References

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.

Reference Type: DERIVED

PMID: 29731369 (View on PubMed)

Other Identifiers

0019970022

Identifier Type: -

Identifier Source: org_study_id