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

Primary Purpose

Hemophilia A

Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Biostate
Sponsored by
CSL Behring
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A

Eligibility Criteria

28 Days - 11 Years (Child)MaleDoes not accept 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).

Sites / Locations

  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Biostate

Arm Description

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

Full Information

First Posted
September 30, 2011
Last Updated
October 2, 2017
Sponsor
CSL Behring
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1. Study Identification

Unique Protocol Identification Number
NCT01445197
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)
Study Type
Interventional

2. Study Status

Record Verification Date
October 2017
Overall Recruitment Status
Terminated
Study Start Date
December 2012 (undefined)
Primary Completion Date
December 2013 (Actual)
Study Completion Date
December 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
CSL Behring

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
1 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Biostate
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
Biostate
Intervention Description
200 IU/kg administered daily
Primary Outcome Measure Information:
Title
Response to immune tolerance induction (ITI) treatment
Description
Number of subjects who achieve complete, partial, and no response (ITI failure) to treatment.
Time Frame
30 months
Secondary Outcome Measure Information:
Title
FVIII inhibitor titre
Time Frame
Up to 65 months
Title
Time to complete response (success)
Time Frame
Up to 65 months
Title
Time to inhibitor titer <0.6 BU/mL for the first time
Time Frame
Up to 65 months
Title
Thromboembolic complications
Description
Number of patients with clinical symptoms or increased markers of coagulation activation
Time Frame
Up to 65 months
Title
Frequency of bleeding events
Time Frame
Up to 65 months
Title
Number of bleeding events per patient
Time Frame
Up to 65 months
Title
Severity of bleeding events per patient
Time Frame
Up to 65 months
Title
Catheter-related complications
Description
Number of line infections
Time Frame
Up to 65 months

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
28 Days
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
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).
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Carmen Escuriola-Ettingshausen
Organizational Affiliation
Haemophilia Centre Rhein Main, Frankfurt - Mörfelden
Official's Role
Principal Investigator
Facility Information:
Facility Name
Study Site
City
Vienna
Country
Austria
Facility Name
Study Site
City
Frankfurt
Country
Germany
Facility Name
Study Site
City
Hamburg
Country
Germany
Facility Name
Study Site
City
Athens
Country
Greece
Facility Name
Study Site
City
Thessaloniki
Country
Greece
Facility Name
Study Site
City
Milano
Country
Italy
Facility Name
Study Site
City
Barnaul
Country
Russian Federation

12. IPD Sharing Statement

Learn more about this trial

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

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