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

NCT ID: NCT01445197

Last Updated: 2017-10-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-12-31

Study Completion Date

2013-12-31

Brief Summary

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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

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Conditions

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Hemophilia A

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Biostate

Group Type EXPERIMENTAL

Biostate

Intervention Type BIOLOGICAL

200 IU/kg administered daily

Interventions

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Biostate

200 IU/kg administered daily

Intervention Type BIOLOGICAL

Eligibility Criteria

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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).
Minimum Eligible Age

28 Days

Maximum Eligible Age

11 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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CSL Behring

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Carmen Escuriola-Ettingshausen

Role: PRINCIPAL_INVESTIGATOR

Haemophilia Centre Rhein Main, Frankfurt - Mörfelden

Locations

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Study Site

Vienna, , Austria

Site Status

Study Site

Frankfurt, , Germany

Site Status

Study Site

Hamburg, , Germany

Site Status

Study Site

Athens, , Greece

Site Status

Study Site

Thessaloniki, , Greece

Site Status

Study Site

Milan, , Italy

Site Status

Study Site

Barnaul, , Russia

Site Status

Countries

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Austria Germany Greece Italy Russia

Other Identifiers

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2010-020113-85

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CSLCT-BIO-10-67

Identifier Type: -

Identifier Source: org_study_id