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Main
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Note: This record shows only the 20 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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17 October 2012 |
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Main ID: |
NCT00212472 |
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Date of registration:
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13/09/2005 |
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Primary sponsor: |
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Public title:
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International Immune Tolerance Study
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Scientific title:
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An International Randomised Controlled Trial Of Immune Tolerance Induction |
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Date of first enrolment:
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July 2002 |
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Target sample size:
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134 |
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Recruitment status: |
Terminated |
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URL:
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http://clinicaltrials.gov/show/NCT00212472 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
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Countries of recruitment
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United States
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Contacts
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Name:
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Charles Hay, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Manchester Royal Infirmary |
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Name:
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Donna M DiMichele, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Weill Cornell Medical College-NY Presybetrian Hospital |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Severe hemophilia A (FVIII level <1%).
- A maximum historical inhibitor titer of between 5 BU and 200 BU that must be
confirmed once prior to the beginning of ITI.
- The inhibitor titer should be <10 BU at the start of ITI, confirmed once.
- The inhibitor must be present for <24 months when ITI begins.
- Maximum age of 7 at the start of ITI.
- Willingness to comply with the protocol.
Exclusion Criteria:
- Moderate or mild hemophilia A (FVIII level >1%).
- Spontaneous disappearance of the inhibitor prior to ITI.
- Historical maximum inhibitor titer <5 BU or > 200 BU before starting ITI.
- Inhibitor titer > 10 BU at the start of ITI.
- Inhibitor present for more than 24 months before starting ITI.
- Systemic immunomodulatory drug therapy during immune tolerance e.g. corticosteroids
(< 5 days every 2 months maximum dose 2 mg/kg or 60 mg/day), azathioprine,
cyclophosphamide, high-dose immunoglobulin or the use of a protein A column or
plasmapheresis.
- Age > 7 years at the start of ITI.
- Inability or unwillingness to comply with the protocol.
- Previous attempt at ITI.
Age minimum:
N/A
Age maximum:
7 Years
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Hemophilia A With Inhibitors
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Intervention(s)
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Drug: Factor VIII concentrates
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Other: High-dose treatment
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Other: Low-dose treatment
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Primary Outcome(s)
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A comparative assessment of morbidity between the two treatment arms including: number of intercurrent bleeds, infections and number of hospital in-patient days.
[Time Frame: Up to 69 months]
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Success-rate and partial success-rate
[Time Frame: Up to 69 months]
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The comparative cost-effectiveness of the two treatment arms
[Time Frame: Up to 69 months]
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The inhibitor recurrence (relapse) rate in the first twelve months after successful ITI.
[Time Frame: Up to 45 months]
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The time from the start of ITI to successful tolerance
[Time Frame: Up to 33 months]
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Secondary Outcome(s)
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The age at the time of inhibitor detection, success-rate and time to success,
[Time Frame: Up to 69 months]
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The dose-regimen, success rate and time to ITI,
[Time Frame: Up to 69 months]
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The effect of interim infections/immunisations, success rate and time to success,
[Time Frame: Up to 69 months]
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The effect of treatment interruption, success rate and time to success.
[Time Frame: Up to 69 months]
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The number of factor VIII treatment days between inhibitor detection and initiation of ITI, success of ITI.
[Time Frame: Up to 69 months]
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The peak historical inhibitor titre, success rate and time to ITI,
[Time Frame: Up to 69 months]
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The peak inhibitor titre after starting ITI, success rate and time to success,
[Time Frame: Up to 69 months]
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The starting inhibitor titre, success rate and time to ITI,
[Time Frame: Up to 69 months]
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The type of concentrate used (von Willebrand factor-containing, monoclonal or recombinant), success rate and time to success,
[Time Frame: Up to 69 months]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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