Hemophilia A
Conditions
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.
Interventions
BIOLOGICALBiostate
200 IU/kg administered daily
Sponsors
CSL Behring
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
28 Days to 11 Years
Healthy volunteers
No
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).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Response to immune tolerance induction (ITI) treatment | 30 months | Number of subjects who achieve complete, partial, and no response (ITI failure) to treatment. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | Number of patients with clinical symptoms or increased markers of coagulation activation |
| FVIII inhibitor titre | Up to 65 months | — |
| Number of bleeding events per patient | Up to 65 months | — |
| Severity of bleeding events per patient | Up to 65 months | — |
| Catheter-related complications | Up to 65 months | Number of line infections |
| Frequency of bleeding events | Up to 65 months | — |
Countries
Austria, Germany, Greece, Italy, Russia
Outcome results
None listed