Survey of Inhibitors in Plasma-Product Exposed Toddlers



Status:Completed
Conditions:Anemia, Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:Any - 6
Updated:4/21/2016
Start Date:January 2010
End Date:May 2015

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Inhibitor Development in Previously Untreated Patients (PUPs) or Minimally Blood Component-Treated Patients (MBCTPs) When Exposed to Plasma-derived Von Willebrand Factor-Containing Factor VIII (VWF/FVIII) Concentrates and to Recombinant Factor VIII (rFVIII) Concentrates: An Independent, International, Multicentre, Prospective, Controlled, Randomised, Open Label, Clinical Trial

The primary objective of the study is to assess the immunogenicity of VWF/FVIII and of
rFVIII concentrates by determining the frequency of inhibitor development in previously
untreated patients (PUPs) or minimally blood component-treated (MBCTPs) in the first 50 EDs
or in the first 3 years from enrollment, whichever occurs first.

Patients meeting the enrollment criteria will be consecutively enrolled at each
participating centre, randomized to be treated exclusively with a single FVIII product
either plasma-derived or recombinant, and followed up until inhibitor development or until
50 exposure days (EDs) or 3 years from enrolment have elapsed, whichever comes first. Study
products, belonging to the class of rFVIII concentrates and to the class of plasma-derived
VWF/FVIII concentrates, will be provided for free to the patients for all the duration of
the study

Inclusion Criteria:

- Male subjects

- Any ethnicity

- Age <6 years

- Severe haemophilia A (FVIII:C <1%), as confirmed at enrolment by the central
laboratory.

o Those patients diagnosed locally as severe but subsequently found to have FVIII
levels >= 1% on testing at the central laboratory will be separately recorded in the
screening list.

- Previously untreated (0 EDs to any FVIII concentrates or blood products) or minimally
treated (<5 EDs) with blood components, namely whole blood, fresh frozen plasma,
packed red blood cells, platelets or cryoprecipitate.

o Patients not meeting these criteria will be separately recorded in the screening
list.

- Negative inhibitor measurement at both local and central laboratory at screening

- Ability to comply with study requirements

- Signed informed consent of legal tutors o Patients who will not accept to enter into
the study or to be randomized will be separately recorded.

Exclusion Criteria:

- Previous history of FVIII inhibitor

- Other congenital or acquired bleeding defects

- Plasma FVIII level >= 1%, as assayed at the central laboratory

o Those patients originally diagnosed locally as severe but subsequently found to
have FVIII levels ranging from 1% to 2% on testing at the central laboratory will be
separately recorded in the screening list.

- Concomitant congenital or acquired immunodeficiency

- Concomitant treatment with systemic immunosuppressive drugs

- Concomitant treatment with any investigational drug
We found this trial at
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2401 Gillham Rd
Kansas City, Missouri 64108
(816) 234-3000
Children's Mercy Hospital Children's Mercy Hospitals and Clinics continues redefining pediatric medicine throughout the Midwest...
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Kansas City, MO
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Aurora, CO
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Chicago, IL
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1500 East Duarte Road
Duarte, California 91010
626-256-HOPE (4673)
City of Hope National Medical Center City of Hope is dedicated to making a difference...
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Duarte, CA
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Fargo, ND
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Forth Worth, Texas 76104
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Forth Worth, TX
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Jackson, MS
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La Plata,
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Las Vegas, NV
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Los Angeles, California 90027
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Los Angeles, CA
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