Safety and efficacy of turoctocog alfa in prevention and treatment of bleeds in previously untreated children with Haemophilia A
- Conditions
- Haemophilia AMedDRA version: 20.0 Level: LLT Classification code 10018937 Term: Haemophilia A System Organ Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2011-001033-16-DK
- Lead Sponsor
- ovo Nordisk A/S
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 60
- Informed consent obtained before any trial-related activities (trial-related activities are any procedure that would not have been performed during normal management of the patient)
- Male patients diagnosed with congenital severe haemophilia A (FVIII = 1%)
- Age < 6 years
- No prior use of purified clotting FVIII products (previous exposure, equal to or less than 5 ED to blood components, e.g. cryoprecipitate, fresh frozen plasma is accepted) including commercially available NovoEight® /Novoeight®
Are the trial subjects under 18? yes
Number of subjects for this age range: 60
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- Known or suspected allergy to hamster protein or intolerance to trial product(s) or related products
- Previous participation in this trial defined as withdrawal after administration of trial product
- Congenital or acquired coagulation disorders other than haemophilia A
- Any history of FVIII inhibitor
- Ongoing treatment or planned treatment during the trial with immunomodulatory agents (e.g. intravenous immunoglobulin (IVIG)), routine systemic corticosteroids)
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate safety of turoctocog alfa in paediatric previously untreated patients (PUP) with haemophilia A;<br> Secondary Objective: - To evaluate efficacy of turoctocog alfa in treatment of bleeds in paediatric PUP with haemophilia A<br> - To evaluate preventive effect of turoctocog alfa on bleeds in paediatric PUP with haemophilia A<br> ;Primary end point(s): Incidence rate of FVIII inhibitors (=0.6 BU) ;Timepoint(s) of evaluation of this end point: will be evaluated for the main phase of the trial
- Secondary Outcome Measures
Name Time Method <br> Secondary end point(s): - Haemostatic effect of turoctocog alfa on treatment of bleeds assessed on a predefined four point scale: Excellent, Good, Moderate and None<br> - Annualized bleeding rate<br> - Incidence rate of FVIII inhibitors (=0.6 BU)<br> ;Timepoint(s) of evaluation of this end point: Will be evaluated for the main phase of trial, for the extension phase of trial, and for the combined main and extension phases