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Study of Biostate® in Children With Hemophilia A

Primary Purpose

Hemophilia A

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Biostate

About this trial

This is an interventional treatment trial for Hemophilia A focused on measuring Hemophilia A, Children

Eligibility Criteria

undefined - 12 Years (Child)MaleDoes not accept healthy volunteers

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.

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Biostate

Arm Description

Outcomes

Primary Outcome Measures

Subjective assessment of Haemostatic efficacy

Incremental recovery of FVIII

Half-life of FVIII

Area under the concentration curve (AUC) of FVIII

Mean residence time (MRT) of FVIII

Volume of distribution at steady state (Vss) of FVIII

Maximum Plasma Concentration (Cmax) of FVIII

Minimum Plasma Concentration (Cmin) of FVIII

Time the maximum concentration occurs (tmax) of FVIII

Total clearance of the drug from the body (CL=dose/AUC) of FVIII

Number of infusions per bleeding event

Number of infusions per month

Number of infusions per year

Dose (IU/kg) per bleeding event

Dose (IU/kg) per month

Dose (IU/kg) per year

Secondary Outcome Measures

Frequency of adverse events (AEs)

Severity of AEs per subject

Severity of AEs per infusion

Relatedness of AEs per subject

Relatedness of AEs per infusion

Development of FVIII inhibitors

Full Information

NCT ID

NCT01229007

First Posted

October 1, 2010

Last Updated

August 23, 2017

Sponsor

CSL Behring

Collaborators

Parexel

1. Study Identification

Unique Protocol Identification Number

NCT01229007

Brief Title

Study of Biostate® in Children With Hemophilia A

Official Title

A Phase III, Open-Label, Multicentre Study to Evaluate Efficacy, Pharmacokinetics, and Safety of Biostate® in Paediatric Subjects With Haemophilia A

Study Type

Interventional

2. Study Status

Record Verification Date

July 2014

Overall Recruitment Status

Completed

Study Start Date

August 2010 (undefined)

Primary Completion Date

July 2014 (Actual)

Study Completion Date

July 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

CSL Behring

Collaborators

Parexel

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hemophilia A

Keywords

Hemophilia A, Children

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

35 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Biostate

Arm Type

Experimental

Intervention Type

Biological

Intervention Name(s)

Biostate

Intervention Description

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.

Primary Outcome Measure Information:

Title

Subjective assessment of Haemostatic efficacy

Time Frame

Over minimum of 50 exposure days

Title

Incremental recovery of FVIII

Time Frame