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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.
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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Factor VIII | Experimental | escalating dose Factor VIII |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS) | Biological | escalating dose prophylaxis |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Bleeding Rate | Number of index hemarthorses (bleeds into ankles, elbows or knees) per patient per year | 6 months |
| Annualized Factor Use | annual factor usage per subject |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Brian M Feldman, MD | The Hospital for Sick Children | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Hospital for Sick Children | Toronto | Ontario | M5G 1X8 | Canada |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 29731369 | Derived | 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. |
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Between June 26, 1997, and Jan 30, 2007, 56 boys were enrolled from 12 Canadian centres.
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| ID | Title | Description |
|---|---|---|
| FG000 | Factor VIII | escalating dose Factor VIII Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose prophylaxis Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Factor VIII | escalating dose Factor VIII Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose prophylaxis Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | Posted | Count of Participants | Participants | 6 months |
|
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Factor VIII | escalating dose Factor VIII Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose prophylaxis Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Brian Feldman | The Hospital for Sick Children | 416-813-5828 | brian.feldman@sickkids.ca |
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| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| D005169 | Factor VIII |
| ID | Term |
|---|---|
| D001779 | Blood Coagulation Factors |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
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| Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS) | Biological | escalating dose |
|
|
| 12 months |
| 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 |
| 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 |
| 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 |
| Complications Arising From Indwelling Venous Catheter | collect information on any complications relating to indwelling venous catheters that some subject use. | 6 months |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
|
| Secondary | Annualized Bleeding Rate | Number of index hemarthorses (bleeds into ankles, elbows or knees) per patient per year | Posted | Median | Inter-Quartile Range | episodes per participant per year | 6 months |
|
|
|
| Secondary | Annualized Factor Use | annual factor usage per subject | Posted | Median | Inter-Quartile Range | IU/kg/year | 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 | Posted | Count of Participants | Participants | 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. | Posted | Median | Inter-Quartile Range | units on a scale | 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 | Median CCPES Score at study-end | Posted | Median | Full Range | units on a scale | 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. | 25 participants had a central venous access device (CVAD) during the study | Posted | Count of Participants | Participants | 6 months |
|
|
|
| 0 |
| 56 |
| 0 |
| 56 |
| 0 |
| 56 |
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| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D011498 |
| Protein Precursors |
| D001685 | Biological Factors |
| Title | Measurements |
|---|---|
|
| Right Elbow |
|
| Left Knee |
|
| Right Knee |
|