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

This study is currently recruiting participants.
Verified May 2013 by CSL Behring
Sponsor:
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT01445197
First received: September 30, 2011
Last updated: May 12, 2013
Last verified: May 2013
History of Changes
  Purpose

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 Intervention Phase
Hemophilia A
 Biological: Biostate
 Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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)

Resource links provided by NLM:

MedlinePlus related topics: Hemophilia
U.S. FDA Resources

Further study details as provided by CSL Behring:

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


Secondary Outcome Measures:
  • FVIII inhibitor titre [ Time Frame: Up to 65 months ] [ Designated as safety issue: No ]
  • Time to complete response (success) [ Time Frame: Up to 65 months ] [ Designated as safety issue: No ]
  • Time to inhibitor titer <0.6 BU/mL for the first time [ Time Frame: Up to 65 months ] [ Designated as safety issue: No ]
  • Thromboembolic complications [ Time Frame: Up to 65 months ] [ Designated as safety issue: Yes ]
    Number of patients with clinical symptoms or increased markers of coagulation activation

  • Frequency of bleeding events [ Time Frame: Up to 65 months ] [ Designated as safety issue: Yes ]
  • Number of bleeding events per patient [ Time Frame: Up to 65 months ] [ Designated as safety issue: Yes ]
  • Severity of bleeding events per patient [ Time Frame: Up to 65 months ] [ Designated as safety issue: Yes ]
  • Catheter-related complications [ Time Frame: Up to 65 months ] [ Designated as safety issue: Yes ]
    Number of line infections


Estimated Enrollment: 20
Study Start Date: December 2012
Estimated Study Completion Date: November 2015
Arms Assigned Interventions
Experimental: Biostate Biological: Biostate
200 IU/kg administered daily

  Eligibility

Ages Eligible for Study:   up to 11 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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).
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01445197

Contacts
Contact: Clinical trial Registration Coordinator clinicaltrials@cslbehring.com

Locations
Austria
Study Site Recruiting
Vienna, Austria
Contact: Use central contact            
Germany
Study Site Recruiting
Hamburg, Germany
Contact: Use central contact            
Greece
Study Site Recruiting
Athens, Greece
Contact: Use central contact            
Study Site Recruiting
Thessaloniki, Greece
Contact: use central contact            
Italy
Study Site Recruiting
Milano, Italy
Contact: Use central contact            
Russian Federation
Study Site Recruiting
Barnaul, Russian Federation
Contact: Use central contact            
Sponsors and Collaborators
CSL Behring
Investigators
Principal Investigator: Carmen Escuriola-Ettingshausen Klinikum der Johann Wolfgang Goethe Universität
  More Information

No publications provided

Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT01445197     History of Changes
Other Study ID Numbers: CSLCT-BIO-10-67, 2010-020113-85
Study First Received: September 30, 2011
Last Updated: May 12, 2013
Health Authority: Austria: Federal Office for Safety in Health Care
Germany: Paul-Ehrlich-Institut
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Greece: Ministry of Health and Welfare
Italy: Ministry of Health
Russia: Ministry of Health of the Russian Federation

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
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 22, 2013