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

Phase 1

• Conditions: Haemophilia A; MedDRA version: 14.1 Level: LLT Classification code 10018937 Term: Haemophilia A System 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-GR
• Lead Sponsor: ovo Nordisk A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2012-04-25
• Study Completion: 2018-12-05
• Last Update Posted: 28 February 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• 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 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: 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
- 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) with haemophilia A;<br> Secondary Objective: - To evaluate efficacy of NNC 0155-0000-0004 in treatment of bleeds in paediatric PUP with haemophilia A<br> - To evaluate preventive effect of NNC 0155-0000-0004 on bleeds in paediatric PUP with haemophilia A<br> ;Primary end point(s): Incidence rate of FVIII inhibitors (=0.6 BU/mL) ;Timepoint(s) of evaluation of this end point: Will be evaluated from Visit 2 to End of Trial Visit/Visit 5

Secondary Outcome Measures

Name	Time	Method
<br> Secondary end point(s): - Haemostatic effect of NNC 0155-0000-0004 on treatment of bleeds assessed on a predefined four point scale: Excellent, Good, Moderate and None<br> - Annualized bleeding rate<br> ;<br> Timepoint(s) of evaluation of this end point: Will be evaluated from Visit 2 to End of Trial Visit/Visit 5<br>