Study of Biostate for Treatment of Children With Hemophil... | NCT01445197

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

NCT01445197

Sponsor

CSL Behring

Status

Terminated

Last Update Posted

Oct 3, 2017Actual

Enrollment

1Actual

Phase

Phase 3

Conditions

Hemophilia A

Interventions

Biostate

Countries

Austria
Germany
Greece
Italy
Russia

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
2010-020113-85	EudraCT Number

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)

Status Module

Record Verification Date

Oct 2017

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Dec 2012

Primary Completion Date

Dec 2013Actual

Completion Date

Dec 2013Actual

First Submitted Date

Sep 30, 2011

First Submission Date that Met QC Criteria

Sep 30, 2011

First Posted Date

Oct 3, 2011Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Oct 2, 2017

Last Update Posted Date

Oct 3, 2017Actual

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

1Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Biostate	Experimental		Biological: Biostate

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Biostate	Biological	200 IU/kg administered daily	Biostate

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Response to immune tolerance induction (ITI) treatment	Number of subjects who achieve complete, partial, and no response (ITI failure) to treatment.	30 months

Secondary Outcomes

Measure	Description	Time Frame
FVIII inhibitor titre		Up to 65 months
Time to complete response (success)		Up to 65 months
Time to inhibitor titer <0.6 BU/mL for the first time

Other Outcomes

Not provided

Eligibility Module

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).

Accepts Healthy Volunteers

Sex

Male

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

28 Days

Maximum Age

11 Years

Standard Ages

Child

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Carmen Escuriola-Ettingshausen	Haemophilia Centre Rhein Main, Frankfurt - Mörfelden	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Study Site		Vienna			Austria
Study Site

Condition Browse Module

MeSH Terms

ID	Term
D006467	Hemophilia A

Ancestor Terms

ID	Term
D025861	Blood Coagulation Disorders, Inherited
D001778	Blood Coagulation Disorders
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases

Browse Leaves

Not provided

Browse Branches

Not provided