Safety and efficacy of turoctocog alfa inprevention and treatment of bleeds in paediatricpreviously untreated patients with Haemophilia A

Phase 1

Conditions: Haemophilia A
MedDRA version: 19.0Level: LLTClassification code 10018937Term: Haemophilia ASystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2011-001033-16-AT

Lead Sponsor: ovo Nordisk A/S

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: Male

Target Recruitment: 60

Inclusion Criteria

- 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 factor products (previous exposure to blood components is acceptable)

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

Exclusion Criteria

- 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;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;Primary end point(s): Incidence rate of FVIII inhibitors (=0.6 BU/mL) ;Timepoint(s) of evaluation of this end point: Will be evaluated for the main phase of the trial

Secondary Outcome Measures

Name	Time	Method
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/mL);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 combinedmain and extension phase