Trial Outcomes & Findings for Canadian Hemophilia Prophylaxis Study (NCT NCT01085344)

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

56 participants

Primary outcome timeframe

6 months

Results posted on

2019-12-05

Participant Flow

Between June 26, 1997, and Jan 30, 2007, 56 boys were enrolled from 12 Canadian centres.

Participant milestones

Participant milestones
Measure	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
Overall Study STARTED	56
Overall Study COMPLETED	50
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Canadian Hemophilia Prophylaxis Study

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Factor VIII n=56 Participants escalating dose Factor VIII Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose prophylaxis Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose
Age, Continuous	1.6 years n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	56 Participants n=5 Participants
Race/Ethnicity, Customized Race or Ethnic Group · White European	44 Participants n=5 Participants
Race/Ethnicity, Customized Race or Ethnic Group · Mixed Aboriginal-Canadian	5 Participants n=5 Participants
Race/Ethnicity, Customized Race or Ethnic Group · Mixed Latin-American	3 Participants n=5 Participants
Race/Ethnicity, Customized Race or Ethnic Group · Asian	2 Participants n=5 Participants
Race/Ethnicity, Customized Race or Ethnic Group · African Heritage	1 Participants n=5 Participants
Race/Ethnicity, Customized Race or Ethnic Group · Middle Eastern	1 Participants n=5 Participants
Region of Enrollment Canada	56 Participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Factor VIII n=56 Participants escalating dose Factor VIII Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose prophylaxis Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose
Number of Participants Who Developed Target Joint Bleeding	17 Participants

SECONDARY outcome

Timeframe: 6 months

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

Outcome measures

Outcome measures
Measure	Factor VIII n=56 Participants escalating dose Factor VIII Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose prophylaxis Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose
Annualized Bleeding Rate	0.95 episodes per participant per year Interval 0.44 to 1.35

SECONDARY outcome

Timeframe: 12 months

annual factor usage per subject

Outcome measures

Outcome measures
Measure	Factor VIII n=56 Participants escalating dose Factor VIII Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose prophylaxis Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose
Annualized Factor Use	3600 IU/kg/year Interval 3200.0 to 4700.0

SECONDARY outcome

Timeframe: 6 months

The number of patients who developed an inhibitor for FVIII, defined as \>= 0.5 Bethesda Units

Outcome measures

Outcome measures
Measure	Factor VIII n=56 Participants escalating dose Factor VIII Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose prophylaxis Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose
Number of Patients Who Developed an Inhibitor to FVIII	5 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Factor VIII n=56 Participants escalating dose Factor VIII Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose prophylaxis Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose
Physical Disability as Measured by the CHAQ	0 units on a scale Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: through study completion, a median of 10 years

Population: Median CCPES Score at study-end

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

Outcome measures

Outcome measures
Measure	Factor VIII n=56 Participants escalating dose Factor VIII Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose prophylaxis Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose
Joint Damage as Determined by the Physiotherapy Score Left Ankle	1 units on a scale Interval 0.0 to 12.0
Joint Damage as Determined by the Physiotherapy Score Right Ankle	1 units on a scale Interval 0.0 to 12.0
Joint Damage as Determined by the Physiotherapy Score Left Elbow	0 units on a scale Interval 0.0 to 7.0
Joint Damage as Determined by the Physiotherapy Score Right Elbow	0 units on a scale Interval 0.0 to 10.0
Joint Damage as Determined by the Physiotherapy Score Left Knee	0 units on a scale Interval 0.0 to 5.0
Joint Damage as Determined by the Physiotherapy Score Right Knee	0 units on a scale Interval 0.0 to 9.0

SECONDARY outcome

Timeframe: 6 months

Population: 25 participants had a central venous access device (CVAD) during the study

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

Outcome measures

Outcome measures
Measure	Factor VIII n=25 Participants escalating dose Factor VIII Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose prophylaxis Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS): escalating dose
Complications Arising From Indwelling Venous Catheter	0 Participants

Adverse Events

Factor VIII

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Brian Feldman

The Hospital for Sick Children

Phone: 416-813-5828

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place