Study of Biostate® in Children With Hemophilia A

NCT01229007 | Completed Phase 3 DMC

• 3 other identifiers: CSLCT-BIO-08-532009-015112-181495
• Study Type: interventional
• Target: 35
• Locations: 6 countries
• Sites: 8

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.

Conditions

Hemophilia A

Keywords

Hemophilia A; Children

Outcome Measures

Primary Outcomes (16)

• Subjective assessment of Haemostatic efficacy

  Over minimum of 50 exposure days

• Incremental recovery of FVIII

  Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1

• Half-life of FVIII

  Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• Number of infusions per bleeding event

  1 day

• Number of infusions per month

  1 month

• Number of infusions per year

  1 year

• Dose (IU/kg) per bleeding event

  1 day

• Dose (IU/kg) per month

  1 month

• Dose (IU/kg) per year

  1 year

Secondary Outcomes (6)

• Frequency of adverse events (AEs)

  6 months

• Severity of AEs per subject

  6 months

• Severity of AEs per infusion

  6 months

• Relatedness of AEs per subject

  6 months

• Relatedness of AEs per infusion

  6 months

Study Arms (1)

Biostate

EXPERIMENTAL

Biological: Biostate

Interventions

Biostate 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.

Biostate

Eligibility Criteria

• Age: Up to 12 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• CSL Behring: lead
• Parexel: collaborator

Study Sites (8)

Study site

Homyel, 246040, Belarus

Location

Study site

Minsk, 223040, Belarus

Location

Study Site

Tbilisi, 0179, Georgia

Location

Study Site

Guatemala City, 01010, Guatemala

Location

Study Site

Beirut, Lebanon

Location

Study site

Monterrey, 64000, Mexico

Location

Study Site

Dnipropetrovsk, Ukraine

Location

Study Site

Lviv, Ukraine

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Program Director Clinical R&D

  CSL Behring

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 1, 2010
• First Posted: October 27, 2010
• Study Start: August 1, 2010
• Primary Completion: July 1, 2014
• Study Completion: July 1, 2014
• Last Updated: August 24, 2017

Record last verified: 2014-07

Locations

GU (1); UA (2); LE (1); ME (1); BE (2); GE (1)