NCT01445197

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.

Trial Health

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Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Dec 2012

Shorter than P25 for phase_3

Geographic Reach
5 countries

7 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 30, 2011

Completed
3 days until next milestone

First Posted

Study publicly available on registry

October 3, 2011

Completed
1.2 years until next milestone

Study Start

First participant enrolled

December 1, 2012

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2013

Completed
Last Updated

October 3, 2017

Status Verified

October 1, 2017

Enrollment Period

1 year

First QC Date

September 30, 2011

Last Update Submit

October 2, 2017

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

  • 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 (8)

  • 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

    Up to 65 months

  • Thromboembolic complications

    Up to 65 months

  • Frequency of bleeding events

    Up to 65 months

  • +3 more secondary outcomes

Study Arms (1)

Biostate

EXPERIMENTAL
Biological: Biostate

Interventions

BiostateBIOLOGICAL

200 IU/kg administered daily

Biostate

Eligibility Criteria

Age28 Days - 11 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • 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).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Study Site

Vienna, Austria

Location

Study Site

Frankfurt, Germany

Location

Study Site

Hamburg, Germany

Location

Study Site

Athens, Greece

Location

Study Site

Thessaloniki, Greece

Location

Study Site

Milan, Italy

Location

Study Site

Barnaul, Russia

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Carmen Escuriola-Ettingshausen

    Haemophilia Centre Rhein Main, Frankfurt - Mörfelden

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 30, 2011

First Posted

October 3, 2011

Study Start

December 1, 2012

Primary Completion

December 1, 2013

Study Completion

December 1, 2013

Last Updated

October 3, 2017

Record last verified: 2017-10

Locations

AU(1)DE(2)RU(1)GR(2)IT(1)