Once-A-Day Prophylaxis for Youth and Young Adults With Severe Hemophilia A
Recruitment status was Recruiting
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Hemophilia A |
Drug: Kogenate FS, Advate, or Humate-P, Recombinate, Helixate FS |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Pilot Study of Once-A-Day Prophylaxis for Youth and Young Adults With Severe Hemophilia A |
- To test the feasibility of a large-scale clinical trial of once-daily prophylaxis. As such, the primary outcome will be related to process measures. [ Time Frame: 12 months ] [ Designated as safety issue: No ]
- Clinical efficacy [ Time Frame: 12 months ] [ Designated as safety issue: No ]
- Estimate the effect size and variability of effect and resource utilization of once-daily prophylaxis. [ Time Frame: 12 months ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 25 |
| Study Start Date: | July 2008 |
| Estimated Study Completion Date: | December 2009 |
| Estimated Primary Completion Date: | July 2009 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: 1 |
Drug: 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 < 75 kg.) or 500 units per day (weight ≥ 75 kg.) of their usual preparation of factor VIII.
|
Detailed Description:
Hemophilia is an important and costly disorder; if left untreated, it may have serious consequences. The greatest impact of hemophilia occurs from recurrent bleeding into joints. The consequences of joint bleeding include pain associated with acute bleeding and later chronic arthropathy. Half of affected children with severe hemophilia have joint damage; joint damage is more frequent with increased bleeding. The prevention and treatment of bleeding is very expensive and therefore finding a cost-effective treatment is of high priority.
Worldwide, two major treatment strategies have been used to prevent arthropathy - on demand therapy and factor prophylaxis. The goal of prophylaxis is to convert the severe to the moderate phenotype by providing circulating factor activity of greater than 1%. Patients with greater than 1% circulating factor VIII activity rarely have spontaneous hemarthroses. Therefore, the goal of providing circulating factor at this level is to eliminate spontaneous hemarthroses. The term primary prophylaxis suggests using preventative factor VIII replacement from a very early age. The term secondary prophylaxis is used to describe the application of prophylaxis at a later disease stage. In this study, secondary prophylaxis will be used.
Once-daily prophylaxis is a novel application of hemophilia factor prophylaxis for youth and young adults. Before embarking on a costly definitive trial we feel that it is necessary to demonstrate that subjects will be willing to enroll and will be compliant with the therapy. Moreover, we need to establish an estimate of the effect of once-daily prophylaxis on bleeding rates, quality of life, and joint damage progression in order to design a definitive trial.
Eligibility| Ages Eligible for Study: | 12 Years to 24 Years |
| Genders Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
Contacts and Locations| Contact: Brian Feldman, MD | (416)813-5677 | brian.feldman@sickkids.ca |
| Canada, Ontario | |
| The Hospital for Sick Children | Recruiting |
| Toronto, Ontario, Canada | |
| Contact: Brian Feldman (416)813-5677 brian.feldman@sickkids.ca | |
| St. Michael's Hospital | Recruiting |
| Toronto, Ontario, Canada | |
| Contact: Jerome Teitel | |
| Canada, Quebec | |
| Hospital Sainte-Justine | Recruiting |
| Montreal, Quebec, Canada | |
| Contact: Georges Rivard | |
| Principal Investigator: | Brian Feldman, MD | The Hospital for Sick Children |
More Information
No publications provided
| Responsible Party: | Brian Feldman/Principal Investigator, The Hospital for Sick Children |
| ClinicalTrials.gov Identifier: | NCT00717626 History of Changes |
| Other Study ID Numbers: | 1000012140 |
| Study First Received: | July 14, 2008 |
| Last Updated: | September 19, 2008 |
| Health Authority: | Canada: Health Canada |
Keywords provided by The Hospital for Sick Children:
|
Hemophilia Prophylaxis Youth Young Adults Feasibility |
Additional relevant MeSH terms:
|
Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases Coagulation Protein Disorders Hemorrhagic Disorders |
Genetic Diseases, Inborn Factor VIII Coagulants Hematologic Agents Therapeutic Uses Pharmacologic Actions |
ClinicalTrials.gov processed this record on June 18, 2013