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Completed

NCT Number: NCT01085344

Canadian Hemophilia Prophylaxis 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.

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Key information

Conditions

Severe Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemophilia A Hemorrhagic Disorders

Age range

12 month–30 month

Sex eligibility

Male

Study type

Interventional

Phase

Phase 4

Primary location

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.)

Treatment and study plan

Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)

Biological

escalating dose prophylaxis

Other names: Recombinant Factor VIII (antihemophilic agent)

Primary outcomes

  1. 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.

Secondary outcomes

  1. Annualized Bleeding Rate

    Time frame: 6 months

    Number of index hemarthorses (bleeds into ankles, elbows or knees) per patient per year

  2. Annualized Factor Use

    Time frame: 12 months

    annual factor usage per subject

  3. 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

  4. 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.

  5. 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

  6. Complications Arising From Indwelling Venous Catheter

    Time frame: 6 months

    collect information on any complications relating to indwelling venous catheters that some subject use.

Sponsors and collaborators

Lead sponsor

The Hospital for Sick Children

Other

Registry information

Official study title

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

Acronym: CHPS

Important dates

Study start
1997
Primary completion
2012
Study completion
2014
First posted
Mar 11, 2010
Registry last updated
Dec 5, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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