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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01445197
Recruitment Status : Terminated
First Posted : October 3, 2011
Last Update Posted : October 3, 2017
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Hemophilia A Biological: Biostate Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Study Start Date : December 2012
Actual Primary Completion Date : December 2013
Actual Study Completion Date : December 2013

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arm Intervention/treatment
Experimental: Biostate Biological: Biostate
200 IU/kg administered daily

Primary Outcome Measures :
  1. Response to immune tolerance induction (ITI) treatment [ Time Frame: 30 months ]
    Number of subjects who achieve complete, partial, and no response (ITI failure) to treatment.

Secondary Outcome Measures :
  1. FVIII inhibitor titre [ Time Frame: Up to 65 months ]
  2. Time to complete response (success) [ Time Frame: Up to 65 months ]
  3. Time to inhibitor titer <0.6 BU/mL for the first time [ Time Frame: Up to 65 months ]
  4. Thromboembolic complications [ Time Frame: Up to 65 months ]
    Number of patients with clinical symptoms or increased markers of coagulation activation

  5. Frequency of bleeding events [ Time Frame: Up to 65 months ]
  6. Number of bleeding events per patient [ Time Frame: Up to 65 months ]
  7. Severity of bleeding events per patient [ Time Frame: Up to 65 months ]
  8. Catheter-related complications [ Time Frame: Up to 65 months ]
    Number of line infections

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 11 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01445197

Study Site
Vienna, Austria
Study Site
Frankfurt, Germany
Study Site
Hamburg, Germany
Study Site
Athens, Greece
Study Site
Thessaloniki, Greece
Study Site
Milano, Italy
Russian Federation
Study Site
Barnaul, Russian Federation
Sponsors and Collaborators
CSL Behring
Principal Investigator: Carmen Escuriola-Ettingshausen Haemophilia Centre Rhein Main, Frankfurt - Mörfelden

Responsible Party: CSL Behring Identifier: NCT01445197     History of Changes
Other Study ID Numbers: CSLCT-BIO-10-67
2010-020113-85 ( EudraCT Number )
First Posted: October 3, 2011    Key Record Dates
Last Update Posted: October 3, 2017
Last Verified: October 2017

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Immunologic Factors
Physiological Effects of Drugs