Clinical trial NCT01445197: 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
Overall status: TERMINATED
Phase: PHASE3
Conditions: Hemophilia A
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2011-10-03
Enrollment: 1
Sex: MALE
Minimum age (years): 28
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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).

Primary outcomes:
measure: Response to immune tolerance induction (ITI) treatment
timeFrame: 30 months
Secondary outcomes:
measure: FVIII inhibitor titre
timeFrame: Up to 65 months
measure: Time to complete response (success)
timeFrame: Up to 65 months
measure: Time to inhibitor titer <0.6 BU/mL for the first time
timeFrame: Up to 65 months
measure: Thromboembolic complications
timeFrame: Up to 65 months
measure: Frequency of bleeding events
timeFrame: Up to 65 months
measure: Number of bleeding events per patient
timeFrame: Up to 65 months
measure: Severity of bleeding events per patient
timeFrame: Up to 65 months
measure: Catheter-related complications
timeFrame: Up to 65 months
Other outcomes:
Locations:
city: Vienna
country: Austria
facility: Study Site
city: Frankfurt
country: Germany
facility: Study Site
city: Hamburg
country: Germany
facility: Study Site
city: Athens
country: Greece
facility: Study Site
city: Thessaloniki
country: Greece
facility: Study Site
city: Milano
country: Italy
facility: Study Site
city: Barnaul
country: Russian Federation
facility: Study Site
Study officials:
affiliation: Haemophilia Centre Rhein Main, Frankfurt - Mörfelden
name: Carmen Escuriola-Ettingshausen
role: PRINCIPAL_INVESTIGATOR
References: