Last updated on November 2018

Alphanate in Immune Tolerance Induction Therapy


Brief description of study

This is a multicenter, multinational, prospective, single-arm, nonrandomized, open-label study of approximately 25 male subjects with congenital hemophilia A who will receive their first (primary) immune tolerance induction (ITI) treatment with Alphanate.

The study consists of 2 phases:

  • An ITI Treatment Phase in which all eligible subjects will receive ITI treatment with Alphanate for a period of up to 33 months. Upon confirmation of complete immune tolerization, subjects will then enter a 12-month Prophylactic Phase. If, after 33 months of ITI, a subject has achieved partial immune tolerance, the subject will enter a 12-month Prophylactic Phase.
  • A 12-month Prophylactic Phase for all subjects who meet the criteria for complete or partial success to continue on a prophylactic dosing regimen of Alphanate.

Detailed Study Description

Male subjects <8 years of age presenting with an inhibitor titer >0.6 to <10 Bethesda Units (BU) will be screened before the planned start of ITI treatment. Subjects continuing to meet the entrance criteria will enter the ITI Treatment Phase and receive daily doses of Alphanate 100 IU/kg/day for up to 33 months, with a one-time option to increase to a dosing regimen of 200 IU/kg/day at any time after 90 days of ITI treatment.

Subjects will continue to receive their daily Alphanate dose for up to 33 months until the titer is negative (<0.6 BU) on 2 consecutive assessments and treatment success is confirmed by FVIII:C pharmacokinetic assessments, at which time they will enter the 12 month Prophylactic Phase.

In addition, subjects who have achieved partial immune tolerance at the completion of 33 months of ITI treatment will enter the 12-month Prophylactic Phase. Subjects who do not achieve partial immune tolerance at the completion of 33 months of ITI treatment will be discontinued as treatment failures.

The Prophylactic Phase begins with an 8 week taper period for subjects tolerized with 100 IU/kg/day or with a 12-week taper period for subjects tolerized with 200 IU/kg/day to bring the dose down in a step-wise manner to a prophylactic dose of Alphanate 50 IU/kg every other day or 3 times per week, at the investigator's discretion. During the Prophylactic Phase, subjects will be monitored monthly for the first 4 months and then every 2 months for the remaining 8 months to assess sustainability of immune tolerance.

Clinical Study Identifier: NCT03095287

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Emory University

Atlanta, GA United States
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University of Kentucky

Lexington, KY United States
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Robert Wood Johnson Medical Group

New Brunswick, NJ United States
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The Childrens Mercy Hospital

Kansas City, MO United States
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Recruitment Status: Open


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