Study of Biostate® in Children With Hemophilia A

Hemophilia A Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01229007
• Other study ID #: CSLCT-BIO-08-53
• Secondary ID: 2009-015112-1814
• Status: Completed
• Phase: Phase 3
• First received: October 1, 2010
• Last updated: August 23, 2017
• Start date: August 2010
• Est. completion date: July 2014

Study information

• Verified date: July 2014
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 35
• Est. completion date: July 2014
• Est. primary completion date: July 2014
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A to 12 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Biological:
• 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.

Locations

Country	Name	City	State
Belarus	Study site	Gomel
Belarus	Study site	Minsk
Georgia	Study Site	Tbilisi
Guatemala	Study Site	Guatemala
Lebanon	Study Site	Beirut
Mexico	Study site	Monterrey
Ukraine	Study Site	Dnipropetrovsk
Ukraine	Study Site	Lviv

Sponsors (2)

Lead Sponsor	Collaborator
CSL Behring	Parexel

Countries where clinical trial is conducted

Belarus,  Georgia,  Guatemala,  Lebanon,  Mexico,  Ukraine, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Subjective assessment of Haemostatic efficacy		Over minimum of 50 exposure days
Primary	Incremental recovery of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Primary	Half-life of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Primary	Area under the concentration curve (AUC) of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Primary	Mean residence time (MRT) of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Primary	Volume of distribution at steady state (Vss) of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Primary	Maximum Plasma Concentration (Cmax) of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Primary	Minimum Plasma Concentration (Cmin) of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Primary	Time the maximum concentration occurs (tmax) of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Primary	Total clearance of the drug from the body (CL=dose/AUC) of FVIII		Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Primary	Number of infusions per bleeding event		1 day
Primary	Number of infusions per month		1 month
Primary	Number of infusions per year		1 year
Primary	Dose (IU/kg) per bleeding event		1 day
Primary	Dose (IU/kg) per month		1 month
Primary	Dose (IU/kg) per year		1 year
Secondary	Frequency of adverse events (AEs)		6 months
Secondary	Severity of AEs per subject		6 months
Secondary	Severity of AEs per infusion		6 months
Secondary	Relatedness of AEs per subject		6 months
Secondary	Relatedness of AEs per infusion		6 months
Secondary	Development of FVIII inhibitors		Samples taken at screening visit, on day 2, on months 1 and 3, and at final visit

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