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

CSL Behring

Status and phase

Terminated

Phase 3

Conditions

Hemophilia A

Treatments

Biological: Biostate

Study type

Interventional

Funder types

Industry

Identifiers

NCT01445197

2010-020113-85 (EudraCT Number)

CSLCT-BIO-10-67

Details and patient eligibility

About

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.

Enrollment

1 patient

Sex

Male

Ages

28 days to 11 years old

Volunteers

No Healthy Volunteers

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).