Safety and efficacy of NNC 0155-0000-0004 inprevention and treatment of bleeds in previously untreated children with Haemophilia A

Phase 1

• Conditions: Haemophilia A; MedDRA version: 14.0Level: LLTClassification code 10018937Term: Haemophilia ASystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2011-001033-16-GB
• Lead Sponsor: ovo Nordisk A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-08-17
• Last Update Posted: 2 October 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 120

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 with congenital severe haemophilia A (baseline level 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: 120
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
- FVIII inhibitor (>= 0.6 BU/mL) at screening
- Ongoing treatment or planned treatment during the trial with immunomodulatory agents (e.g. intravenous immunoglobulin (IVIG)), routine systemic corticosteroids)
- Platelet count <50,000 platelets/µL

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 NNC 0155-0000-0004 in paediatric previously untreated patients (PUP) 1<br>with haemophilia A;Secondary Objective: - To evaluate efficacy of NNC 0155-0000-0004 in treatment of bleeds in paediatric PUP with<br>haemophilia A<br>- To evaluate preventive effect of NNC 0155-0000-0004 on bleeds in paediatric PUP with<br>haemophilia A;Primary end point(s): Incidence rate of FVIII inhibitors (>0.6 BU/mL) will be evaluated from Visit 1 to End of Trial<br>Visit/Visit 7;Timepoint(s) of evaluation of this end point: from Visit 1 to End of Trial<br>Visit/Visit 7

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Haemostatic effect of NNC 0155-0000-0004 on treatment of bleeds assessed on a predefined<br>four point scale: Excellent, Good, Moderate and None will be evaluated from Visit 2 to End of<br>Trial Visit/Visit 7<br>- Annualized bleeding rate will be evaluated from Visit 2 to End of Trial Visit/Visit 7;Timepoint(s) of evaluation of this end point: from Visit 2 to End of Trial<br>Visit/Visit 7