Alphanate in Immune Tolerance Induction Therapy



Status:Recruiting
Conditions:Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:Any - 8
Updated:11/7/2018
Start Date:January 3, 2018
End Date:October 2022
Contact:Donna Babiar
Email:donna.babiar@grifols.com
Phone:+1 919-316-2098

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A Multicenter Phase 2 Open-Label, Single-Arm, Prospective, Interventional Study of Plasma-Derived Factor VIII/VWF (Alphanate®) in Immune Tolerance Induction Therapy in Subjects With Congenital Hemophilia A

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.

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.

Inclusion Criteria:

- The subject has a documented diagnosis of severe congenital hemophilia A with FVIII:C
<1% of normal.

- The subject is a male up to 8 years (and at least 2 years of age if in India) at the
Baseline Visit.

- The subject's documented historical peak inhibitor titer is ≥10 BU and ≤200 BU.

- The subject has an inhibitor titer >0.6 BU and <10 BU at Screening.

- The subject has had a delay ≤24 months from the date of diagnosis of the inhibitor to
the start of the subject's ITI treatment.

Exclusion Criteria:

- The subject has acquired factor VIII (FVIII) deficiency.

- The subject has previously received ITI treatment.

- The subject has a recent (within 1 month) history of central line infection at the
time of Screening.

- The subject has a high risk of cardiovascular, cerebrovascular, or thromboembolic
event as judged by the investigator.

- The subject is currently undergoing treatment with immunosuppressive drugs (eg,
systemic corticosteroids), azathioprine, cyclophosphamide, high dose immunoglobulin,
interferon, or the use of a protein A column or plasmapheresis and is unwilling to
discontinue these treatments starting at the screening visit.

- The subject has a known infection with human immunodeficiency virus (HIV) or has
clinical signs and symptoms consistent with current HIV infection.

- The subject has a known previous infection with hepatitis B virus (HBV) or hepatitis C
virus (HCV) or has clinical signs and symptoms consistent with current HBV or HCV
infection.

- The subject has significant proteinuria, has a history of acute renal failure or
severe renal impairment (blood urea nitrogen or creatinine >2 times the upper limit of
normal), or is receiving dialysis at Screening.

- The subject has a value of aspartate transaminase or alanine aminotransferase >2 times
the upper limit of normal at Screening.

- The subject has clinical evidence of any significant acute or chronic disease that, in
the opinion of the investigator, may interfere with successful completion of the trial
or place the subject at undue medical risk.

- The subject has a history of anaphylaxis or severe systemic reaction to any plasma
derived or other blood products.
We found this trial at
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1900 9th Avenue
Seattle, Washington 98101
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201 Dowman Dr
Atlanta, Georgia 30303
(404) 727-6123
Phone: 404-785-8329
Emory University Emory University, recognized internationally for its outstanding liberal artscolleges, graduate and professional schools,...
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2401 Gillham Rd
Kansas City, Missouri 64108
(816) 234-3000
Principal Investigator: Shannon Carpenter
Children's Mercy Hospital Children's Mercy Hospitals and Clinics continues redefining pediatric medicine throughout the Midwest...
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101 Manning Drive
Chapel Hill, North Carolina 27517
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Hamilton, Ontario
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Lexington, Kentucky
859) 257-9000
University of Kentucky The University of Kentucky is a public, land grant university dedicated to...
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Minneapolis, Minnesota 55404
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125 Paterson Street
New Brunswick, New Jersey 08901
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