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

Phase 3

Terminated

• Conditions: Hemophilia A
• Interventions: Biological: Biostate

• Registration Number: NCT01445197
• Lead Sponsor: CSL Behring

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2011-09-30
• Study First Submitted QC: 2011-09-30
• Study First Posted: 2011-10-03
• Study Start: 2012-12
• Primary Completion: 2013-12
• Study Completion: 2013-12
• Status Verified: 2017-10
• Last Update Submitted: 2017-10-02
• Last Update Posted: 2017-10-03

Recruitment & Eligibility

• Status: TERMINATED
• Sex: Male
• Target Recruitment: 1

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

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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).

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Biostate	Biostate	-

Primary Outcome Measures

Name	Time	Method
Response to immune tolerance induction (ITI) treatment	30 months	Number of subjects who achieve complete, partial, and no response (ITI failure) to treatment.

Secondary Outcome Measures

Name	Time	Method
Time to complete response (success)	Up to 65 months
Number of bleeding events per patient	Up to 65 months
FVIII inhibitor titre	Up to 65 months
Time to inhibitor titer <0.6 BU/mL for the first time	Up to 65 months
Severity of bleeding events per patient	Up to 65 months
Catheter-related complications	Up to 65 months	Number of line infections
Thromboembolic complications	Up to 65 months	Number of patients with clinical symptoms or increased markers of coagulation activation
Frequency of bleeding events	Up to 65 months

Trial Locations

Locations (1)

Study Site; 🇷🇺 Barnaul, Russian Federation