Trial Outcomes & Findings for Once-A-Day Prophylaxis for Youth and Young Adults With Severe Hemophilia A (NCT NCT00717626)

NCT ID: NCT00717626

Last Updated: 2020-10-08

Results Overview

Tool developed to assess joint health in persons with hemophilia (PWH) through several assessments of 9 items in the 6 index joints (elbows, knees, and ankles). It has been internationally validated and has demonstrated inter-observer and test-retest reliability supporting its use as an effective physical examination assessment tool for PWH. Range: 0 - 124 ( Best score = 0, Worst score = 124)

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

14 participants

Primary outcome timeframe

4 months, 12 months

Results posted on

2020-10-08

Participant Flow

Participant milestones

Participant milestones
Measure	Daily Administration of Low Dose FVIII Low dose daily prophylaxis using FVIII products (e.g.Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS) Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS: Starting at the 4-month visit, subjects will receive 250 units per day (if their weight is \< 50 kg.) or 500 units per day (weight ≥ 50 kg.) of their usual preparation of factor VIII.
Overall Study STARTED	14
Overall Study COMPLETED	14
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Once-A-Day Prophylaxis for Youth and Young Adults With Severe Hemophilia A

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Daily Administration of Low Dose FVIII n=14 Participants Low dose daily prophylaxis using FVIII products (e.g.Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS) Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS: Starting at the 4-month visit, subjects will receive 250 units per day (if their weight is \< 50 kg.) or 500 units per day (weight ≥ 50 kg.) of their usual preparation of factor VIII.
Age, Categorical <=18 years	4 Participants n=5 Participants
Region of Enrollment Canada	14 participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	14 Participants n=5 Participants

PRIMARY outcome

Timeframe: 4 months, 12 months

Population: Mean of differences between start of intervention (4 months) and end of study (12 months)

Tool developed to assess joint health in persons with hemophilia (PWH) through several assessments of 9 items in the 6 index joints (elbows, knees, and ankles). It has been internationally validated and has demonstrated inter-observer and test-retest reliability supporting its use as an effective physical examination assessment tool for PWH. Range: 0 - 124 ( Best score = 0, Worst score = 124)

Outcome measures

Outcome measures
Measure	Daily Administration of Low Dose FVIII n=14 Participants Low dose daily prophylaxis using FVIII products (e.g.Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS) Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS: Starting at the 4-month visit, subjects will receive 250 units per day (if their weight is \< 50 kg.) or 500 units per day (weight ≥ 50 kg.) of their usual preparation of factor VIII.
Hemophilia Joint Health Score (HJHS) 2.0	3.77 score on a scale Interval 0.63 to 6.91

PRIMARY outcome

Timeframe: 4 months, 12 months

Population: Mean of differences between start of intervention (4 months) and end of study (12 months)

Tool for assessment of 8 areas of musculoskeletal (MSK) function in patients with hemophlia. Range: 0 - 32 ( Best score = 0, Worst score = 32)

Outcome measures

Outcome measures
Measure	Daily Administration of Low Dose FVIII n=14 Participants Low dose daily prophylaxis using FVIII products (e.g.Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS) Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS: Starting at the 4-month visit, subjects will receive 250 units per day (if their weight is \< 50 kg.) or 500 units per day (weight ≥ 50 kg.) of their usual preparation of factor VIII.
Functional Independence Score in Hemophilia (FISH)	0.31 score on a scale Interval -0.41 to 1.02

PRIMARY outcome

Timeframe: 4 months, 12 months

Population: Mean of differences between start of intervention (4 months) and end of study (12 months)

Canadian Haemophilia Outcomes-Kids Life Assessment Tool is a validated 35-item disease specific measure of Quality of Life in boys with hemophilia ages 4 - 17 yrs. Range: 0 - 100 ( Best score = 100, Worst score = 0) The Adult Haemo-QoL Questionnaire is a validated disease specific measure of Quality of Life in adults with hemophilia ages \> 18 yrs. It is a 41-item questionnaire that considers how hemophilia affects 4 specific areas of their life. These areas include day-to-day activities, moods \& feelings, work or school life, family life \& social life, and hemophilia treatment. Range: 0 - 100 ( Best score = 0, Worst score = 100) The two measures were transformed into a Z-score to enable comparison across both HRQoL scores. A score of 0 represents no difference between the means, whereas a positive z-score would indicate an improvement in HRQoL between baseline and end of study.

Outcome measures

Outcome measures
Measure	Daily Administration of Low Dose FVIII n=14 Participants Low dose daily prophylaxis using FVIII products (e.g.Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS) Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS: Starting at the 4-month visit, subjects will receive 250 units per day (if their weight is \< 50 kg.) or 500 units per day (weight ≥ 50 kg.) of their usual preparation of factor VIII.
HRQoL Transformed Z-Score (Includes the Canadian Haemophilia Outcomes-Kids Life Assessment Tool (CHO-KLAT) and Haemo-QoL A)	0 score on a scale Interval -0.64 to 0.64

PRIMARY outcome

Timeframe: 4 months, 12 months

Population: Mean of differences between start of intervention (4 months) and end of study (12 months)

Treatment Satisfaction Questionnaire for Medication (TSQM) is an 11-item questionnaire used to evaluate patient satisfaction when starting a new medication and/or treatment. The areas of Effectiveness, Side-Effects, Convenience, and Global Satisfaction are assessed. A modified version (approved by developers) of the questionnaire with the deletion of side effects was used fro the purpose of the study. Effectiveness, Convenience, and Global Satisfaction all include the Range: 0 - 100 ( Best score = 0, Worst score = 100)

Outcome measures

Outcome measures
Measure	Daily Administration of Low Dose FVIII n=14 Participants Low dose daily prophylaxis using FVIII products (e.g.Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS) Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS: Starting at the 4-month visit, subjects will receive 250 units per day (if their weight is \< 50 kg.) or 500 units per day (weight ≥ 50 kg.) of their usual preparation of factor VIII.
Treatment Satisfaction Questionnaire for Medication (TSQM) . Version II TSQM Effectiveness	-6.41 score on a scale Interval -18.25 to 5.43
Treatment Satisfaction Questionnaire for Medication (TSQM) . Version II TSQM Convenience	-4.70 score on a scale Interval -14.08 to 4.68
Treatment Satisfaction Questionnaire for Medication (TSQM) . Version II TSQM Global Satisfaction	-5.13 score on a scale Interval -14.89 to 4.63

PRIMARY outcome

Timeframe: 4 months and 12 months

Population: Mean of differences between start of intervention (4 months) and end of study (12 months)

The Previous Day Physical Activity Recall is a well-validated assessment of habitual physical activity. Activity was reported on 1 weekend day, and 2 weekdays.The intensity of the activity was recorded in Metabolic Equivalents (METs) and categorized as Light (\< 3.0 METs), Moderate (3.0 - 5.9 METs), Hard (6.0 - 8.9 METs), Very Hard (10.0 METs) Range: 0 - 10 METs per activity ( Best score = 10, Worst score = 0) MPA = Moderate Physical Activity VPA = Vigorous Physical Activity

Outcome measures

Outcome measures
Measure	Daily Administration of Low Dose FVIII n=14 Participants Low dose daily prophylaxis using FVIII products (e.g.Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS) Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS: Starting at the 4-month visit, subjects will receive 250 units per day (if their weight is \< 50 kg.) or 500 units per day (weight ≥ 50 kg.) of their usual preparation of factor VIII.
The Previous Day Physical Activity Recall Weekend VPA	2.31 score on a scale Interval -1.76 to 6.38
The Previous Day Physical Activity Recall Weekday 1 MPA	-0.15 score on a scale Interval -2.24 to 1.94
The Previous Day Physical Activity Recall Weekday 1 VPA	-0.69 score on a scale Interval -3.65 to 2.26
The Previous Day Physical Activity Recall Weekday 2 MPA	-0.15 score on a scale Interval -2.54 to 2.24
The Previous Day Physical Activity Recall Weekday 2 VPA	0 score on a scale Interval -5.1 to 5.1
The Previous Day Physical Activity Recall Weekend MPA	-0.92 score on a scale Interval -4.06 to 2.21

PRIMARY outcome

Timeframe: 4, 8, and 12 months

The total number of index joint (elbows, knees, ankles) were extracted from the participant's (self-reported) diaries.

Outcome measures

Outcome measures
Measure	Daily Administration of Low Dose FVIII n=14 Participants Low dose daily prophylaxis using FVIII products (e.g.Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS) Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS: Starting at the 4-month visit, subjects will receive 250 units per day (if their weight is \< 50 kg.) or 500 units per day (weight ≥ 50 kg.) of their usual preparation of factor VIII.
Total Number of Index Joint Bleeds 4 months - Index joint bleeds	0 Bleeds Interval 0.0 to 5.0
Total Number of Index Joint Bleeds 8 months - Index joint bleeds	0 Bleeds Interval 0.0 to 5.0
Total Number of Index Joint Bleeds 12 months - Index joint bleeds	0 Bleeds Interval 0.0 to 12.0

SECONDARY outcome

Timeframe: 12 months

The mean yearly factor consumption of FVIII product on low dose daily prophylaxis was collected for comparison against standard prophylaxis (High dose alternate day treatment). Patient diaries indicating the amount of factor VIII product taken per daily infusion was collected on study.

Outcome measures

Outcome measures
Measure	Daily Administration of Low Dose FVIII n=14 Participants Low dose daily prophylaxis using FVIII products (e.g.Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS) Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS: Starting at the 4-month visit, subjects will receive 250 units per day (if their weight is \< 50 kg.) or 500 units per day (weight ≥ 50 kg.) of their usual preparation of factor VIII.
Mean Yearly Factor Consumption of FVIII Product on Low Dose Daily Prophylaxis	211528 International Units (IU) Interval 176125.79 to 246930.61

Adverse Events

Daily Administration of Low Dose FVIII

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