A Phase 4 interventional study of Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS) and Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS) in Severe Hemophilia A, sponsored by The Hospital for Sick Children. Completed at 1 site in Canada. Open to male participants aged 12 Months to 30 Months. Per ClinicalTrials.gov, last updated 2019-12-05.
Sponsored by The Hospital for Sick Children · Phase 4, Interventional, and Treatment
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).
866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.
This study's enrollment of 56 is above the median of 28 across 512 interventional studies indexed under Hemophilia A.
Browse Hemophilia A studies →The Hospital for Sick Children is the lead sponsor of 568 studies on the registry; 81 are open to participants now.
Of its 7 completed or terminated interventional studies of FDA-regulated products, 0 (0%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
escalating dose Factor VIII
Biological: Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)
escalating dose prophylaxis
Also known as: Recombinant Factor VIII (antihemophilic agent)
escalating dose
Also known as: recombinant factor VIII (antihemophilic agent)
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.
Time frame: 6 months
Annualized Bleeding Rate
Number of index hemarthorses (bleeds into ankles, elbows or knees) per patient per year
Time frame: 6 months
Annualized Factor Use
annual factor usage per subject
Time frame: 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
Time frame: 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.
Time frame: 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
Time frame: 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.
Time frame: 6 months
Between June 26, 1997, and Jan 30, 2007, 56 boys were enrolled from 12 Canadian centres.
| Milestone | Factor VIII |
|---|---|
| Started | 56 |
| Completed | 50 |
| Not completed | 6 |
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.
| Participants | Factor VIII |
|---|---|
| Number of Participants Who Developed Target Joint Bleeding | 17 |
Number of index hemarthorses (bleeds into ankles, elbows or knees) per patient per year
| episodes per participant per year | Factor VIII |
|---|---|
| Annualized Bleeding Rate | 0.95 (0.44 to 1.35) |
annual factor usage per subject
| IU/kg/year | Factor VIII |
|---|---|
| Annualized Factor Use | 3600 (3200 to 4700) |
The number of patients who developed an inhibitor for FVIII, defined as \>= 0.5 Bethesda Units
| Participants | Factor VIII |
|---|---|
| Number of Patients Who Developed an Inhibitor to FVIII | 5 |
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.
| units on a scale | Factor VIII |
|---|---|
| Physical Disability as Measured by the CHAQ | 0 (0 to 0) |
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
| units on a scale | Factor VIII |
|---|---|
| Left Ankle | 1 (0 to 12) |
| Right Ankle | 1 (0 to 12) |
| Left Elbow | 0 (0 to 7) |
| Right Elbow | 0 (0 to 10) |
| Left Knee | 0 (0 to 5) |
| Right Knee | 0 (0 to 9) |
collect information on any complications relating to indwelling venous catheters that some subject use.
| Participants | Factor VIII |
|---|---|
| Complications Arising From Indwelling Venous Catheter | 0 |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Factor VIII | 0/56 (0%) | 0/56 (0%) | 0/56 (0%) |
| Age, Continuous(years) | Factor VIII |
|---|---|
| Median | 1.6 (1.2 to 2.0) |
| Sex: Female, Male(Participants) | Factor VIII |
|---|---|
| Female | 0 |
| Male | 56 |
| Race/Ethnicity, Customized(Participants) | Factor VIII |
|---|---|
| Race or Ethnic Group — White European | 44 |
| Race or Ethnic Group — Mixed Aboriginal-Canadian | 5 |
| Race or Ethnic Group — Mixed Latin-American | 3 |
| Race or Ethnic Group — Asian | 2 |
| Race or Ethnic Group — African Heritage | 1 |
| Race or Ethnic Group — Middle Eastern | 1 |
| Region of Enrollment(Participants) | Factor VIII |
|---|---|
| Canada | 56 |
This study is completed, as verified in Nov 2019. You cannot join it, but the record below documents what was studied.
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