Study of Biostate for Treatment of Children With Hemophilia A Complicated by Antibody Development

Hemophilia A Clinical Trial

Official title:

A Multicentre, Interventional, Non-randomized, Open-label, Single-group Phase III Study to Evaluate Plasma-Derived Antihaemophilic Factor/Von Willebrand Factor Concentrate (Biostate®) for Immune Tolerance Induction in Male Paediatric Subjects With Haemophilia A (≤ 2%) Who Have Developed High-titre Antibodies to Factor VIII (Factor VIII Inhibitors)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01445197
• Other study ID #: CSLCT-BIO-10-67
• Secondary ID: 2010-020113-85
• Status: Terminated
• Phase: Phase 3
• First received: September 30, 2011
• Last updated: October 2, 2017
• Start date: December 2012
• Est. completion date: December 2013

Study information

• Verified date: October 2017
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a clinical study to investigate how well Biostate works in treatment of male patients below the age of 12 years who have a clotting factor deficiency that is aggravated by the development of antibodies. The antibodies are directed against the clotting factor that is given for replacement therapy and usually make therapy unsuccessful. The treatment used in this study is called immune tolerance therapy.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 1
• Est. completion date: December 2013
• Est. primary completion date: December 2013
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A to 11 Years

Eligibility

Inclusion Criteria:

• Male subjects diagnosed with haemophilia A (= 2% FVIII level in the absence of factor replacement, according to their medical history).

• Age 28 days to <12 years.

• Subject is eligible for immune tolerance induction (ITI) therapy

Exclusion Criteria:

• The subject has received ITI previously.

• Subjects with a historical peak inhibitor titre of = 200 BU/mL.

• Concomitant treatment with drugs with immunosuppressive side effects (eg, systemic corticosteroids), azathioprine, cyclophosphamide, high dose immunoglobulin or the use of a protein A column or plasmapheresis and interferons.

• High risk of cardiovascular, cerebrovascular, or other thromboembolic events (excluding catheter thrombosis) as judged by the investigator.

• Subjects who are human immunodeficiency virus (HIV)-1 or HIV-2 positive (as reported in the medical records or determined at screening).

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Biological:
• Biostate
200 IU/kg administered daily

Locations

Country	Name	City	State
Austria	Study Site	Vienna
Germany	Study Site	Frankfurt
Germany	Study Site	Hamburg
Greece	Study Site	Athens
Greece	Study Site	Thessaloniki
Italy	Study Site	Milano
Russian Federation	Study Site	Barnaul

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Countries where clinical trial is conducted

Austria,  Germany,  Greece,  Italy,  Russian Federation, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Response to immune tolerance induction (ITI) treatment	Number of subjects who achieve complete, partial, and no response (ITI failure) to treatment.	30 months
Secondary	FVIII inhibitor titre		Up to 65 months
Secondary	Time to complete response (success)		Up to 65 months
Secondary	Time to inhibitor titer <0.6 BU/mL for the first time		Up to 65 months
Secondary	Thromboembolic complications	Number of patients with clinical symptoms or increased markers of coagulation activation	Up to 65 months
Secondary	Frequency of bleeding events		Up to 65 months
Secondary	Number of bleeding events per patient		Up to 65 months
Secondary	Severity of bleeding events per patient		Up to 65 months
Secondary	Catheter-related complications	Number of line infections	Up to 65 months