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

NCT00717626 | Completed Phase 2 Results DMC

• 1 other identifier: 1000012140
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 3

Brief Summary

The primary objective of this study is to test the feasibility of a large-scale clinical trial of once-daily prophylaxis. The secondary objectives are to collect clinical efficacy outcomes so that we can better plan a large-scale study; we will estimate the effect size and variability of effect and resource utilization of once-daily prophylaxis to allow us to set a sample size for a definitive trial.

Conditions

Hemophilia A

Keywords

Hemophilia; Prophylaxis; Youth; Young Adults; Feasibility

Outcome Measures

Primary Outcomes (6)

• Hemophilia Joint Health Score (HJHS) 2.0

  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)

  4 months, 12 months

• Functional Independence Score in Hemophilia (FISH)

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

  4 months, 12 months

• HRQoL Transformed Z-Score (Includes the Canadian Haemophilia Outcomes-Kids Life Assessment Tool (CHO-KLAT) and Haemo-QoL A)

  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.

  4 months, 12 months

• Treatment Satisfaction Questionnaire for Medication (TSQM) . Version II

  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)

  4 months, 12 months

• The Previous Day Physical Activity Recall

  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

  4 months and 12 months

• Total Number of Index Joint Bleeds

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

  4, 8, and 12 months

Secondary Outcomes (1)

• Mean Yearly Factor Consumption of FVIII Product on Low Dose Daily Prophylaxis

  12 months

Study Arms (1)

Daily administration of low dose FVIII

EXPERIMENTAL

Low dose daily prophylaxis using FVIII products (e.g.Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS)

Drug: Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS

Interventions

Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS DRUG

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.

Daily administration of low dose FVIII

Eligibility Criteria

• Age: 12 Years - 24 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Severe hemophilia A (\<1% factor VIII)
• Age 12 to 24 years inclusive
• Male
• No current factor VIII inhibitor (an inhibitor will be defined as ≥ 0.6 Bethesda Units) within the past year
• Able to participate in a home infusion program with adequate peripheral venous access as assessed ny the treating investigator

You may not qualify if:

• Important co-morbidities (Acquired Immunodeficiency Syndrome or symptomatic HIV infection, symptomatic hepatitis B or C infection)
• Other concomitant acquired or congenital bleeding disorder (e.g. von Willebrand's Disease)
• Receiving factor VIII replacement through central venous catheter

Sponsors & Collaborators

• The Hospital for Sick Children: lead

Study Sites (3)

St. Michael's Hospital

Toronto, Ontario, Canada

Location

The Hospital for Sick Children

Toronto, Ontario, Canada

Location

Hospital Sainte-Justine

Montreal, Quebec, Canada

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

F8 protein, human; Factor VIII

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Results Point of Contact

• Title: Dr. Brian Feldman
• Organization: The Hospital for Sick Children

brian.feldman@sickkids.ca

416-813-5828

Study Officials

• Brian Feldman, MD,MSc,FRCPC

  The Hospital for Sick Children

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Division Head, Rheumatology

Study Record Dates

• First Submitted: July 14, 2008
• First Posted: July 17, 2008
• Study Start: July 1, 2008
• Primary Completion: April 1, 2011
• Study Completion: April 1, 2011
• Last Updated: October 8, 2020
• Results First Posted: October 8, 2020

Record last verified: 2020-09

Locations

CA (3)