Study of Biostate® in Children With Hemophilia A

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

35 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-08-31

Study Completion Date

2014-07-31

Brief Summary

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The objective of this study is to assess the efficacy and safety of a Von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, and to investigate the pharmacokinetics of Biostate in children with haemophilia A.

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Detailed Description

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Conditions

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Hemophilia A

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Biostate

Group Type EXPERIMENTAL

Biostate

Intervention Type BIOLOGICAL

1 dose of 50 IU FVIII/kg body weight of Biostate administered intravenously on Day 1 in the PK component, followed by the Efficacy component for continuation of Biostate therapy, as required for a minimum of 50 exposure days.

Interventions

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Biostate

1 dose of 50 IU FVIII/kg body weight of Biostate administered intravenously on Day 1 in the PK component, followed by the Efficacy component for continuation of Biostate therapy, as required for a minimum of 50 exposure days.

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Male subjects between 0 and \<12 years of age.
* Diagnosed with severe haemophilia A (FVIII:C \<1%), and pre-treated for a minimum of 20 to 50 exposure days.
* Have evidence of vaccination against hepatitis A and B (or presence of antibodies against hepatitis A and B due to either a previous infection or prior immunisation), as documented in the medical notes at enrolment.
* The subject and/or legal guardian understand(s) the nature of the study and has/have given written informed consent to participate in the study and is/are willing to comply with the protocol.

Exclusion Criteria

* For all subjects at Day 1: Are actively bleeding.
* Have received an infusion of any FVIII product, cryoprecipitate, whole blood, plasma or desmopressin acetate in the 4 days prior to their dosing within the PK component.
* Have a known history of, or who are suspected of having FVIII inhibitors.
* Have received aspirin or other non-steroidal anti-inflammatory drugs (NSAIDs) within 7 days of administration of the IMP.
* Have an impaired liver function ie, bilirubin \>1.5 x upper limit of normal (ULN) and/or aspartate/alanine aminotransferase (AST/ALT) \>2.5 x ULN (referring to limits of the laboratory that performs the determination) at Screening.
* Are human immunodeficiency virus \[HIV\]-1/-2 antibody positive with a viral load of \>200/µL.
* Suffer from an acute or chronic medical condition, other than haemophilia A, which may, in the opinion of the Investigator, affect the conduct of the study.
* Suffering from von Willebrand disease (VWD) with von Willebrand factor: ristocetin cofactor (VWF:RCo) level \<50 IU/dL at Screening.
* Have a known or suspected hypersensitivity or previous evidence of severe side effects to a plasma-derived FVIII product or to human albumin.
* Have participated in a clinical study or used an investigational compound in another study (eg, a new chemical entity not registered for clinical use) in the 3 months preceding the first day of IMP administration, or are planning to enter such a study during the study period.
* Unwillingness and/or inability to comply with the study requirements.

Maximum Eligible Age

12 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No