Brief Title
Study of Biostate for Treatment of Children With Hemophilia A Complicated by Antibody Development
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)
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.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Response to immune tolerance induction (ITI) treatment
Secondary Outcome
FVIII inhibitor titre
Condition
Hemophilia A
Intervention
Biostate
Study Arms / Comparison Groups
Biostate
Description:
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
1
Start Date
December 2012
Completion Date
December 2013
Primary Completion Date
December 2013
Eligibility Criteria
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).
Gender
Male
Ages
28 Days - 11 Years
Accepts Healthy Volunteers
No
Contacts
Carmen Escuriola-Ettingshausen, ,
Location Countries
Austria
Location Countries
Austria
Administrative Informations
NCT ID
NCT01445197
Organization ID
CSLCT-BIO-10-67
Secondary IDs
2010-020113-85
Responsible Party
Sponsor
Study Sponsor
CSL Behring
Study Sponsor
Carmen Escuriola-Ettingshausen, Principal Investigator, Haemophilia Centre Rhein Main, Frankfurt - Mörfelden
Verification Date
October 2017