A Multi-Centre, Open-Label, Non-Controlled Trial on Safety and Efficacy of N8 in Previously Treated Paediatric Patients with Haemophilia A

• Conditions: Haemophilia A; MedDRA version: 12.1Level: LLTClassification code 10018937Term: Haemophilia A

• Registration Number: EUCTR2009-016383-36-LT
• Lead Sponsor: ovo Nordisk A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-02-21
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 60

Inclusion Criteria

• Male patients with severe (baseline FVIII =1%) haemophilia A
• Age <12 years and weight = 11 kg
• Documented history of a minimum 50 EDs to FVIII products
(prophylaxis/prevention/surgery/on-demand)
• No factor VIII inhibitors ( =0.6 BU/mL) at screening
• Documented negative FVIII inhibitor test(s) ( < 0.6 BU/mL ) or documented FVIII
recovery tests (within expected normal ranges) within first 50 EDs
• Immunocompetent, defined as either HIV negative or if HIV positive, CD4 >200
cells/µL according to medical records
Are the trial subjects under 18? yes
Number of subjects for this age range:
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

• Any history of FVIII inhibitors (=0.6 BU/mL)
• Surgery planned to occur during trial participation (exceptions are port placement,
dental extractions, and minor, uncomplicated emergent procedures)
• Platelet count < 50,000 platelets/µL
• Congenital or acquired coagulation disorders other than haemophilia A
• Ongoing treatment or planned treatment during the trial with chemotherapy,
immunomodulatory agents (e.g. intravenous immunoglobulin (IVIG), routine
systemic corticosteroids)
• Any disease or condition which, judged by the Investigator, could imply a potential
hazard to the patient, interfere with the trial participation or trial outcome

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 N8 in paediatric previously treated patients (PTPs) <12 years<br> of age with haemophilia A;Secondary Objective: • To evaluate pharmacokinetics of N8 in paediatric PTPs <12 years of age with <br> haemophilia A<br>• To evaluate efficacy of N8 in paediatric PTPs <12 years of age with haemophilia A<br>• To assess and compare Patient Reported Outcomes from baseline to end of trial in <br> paediatric PTPs <12 years of age with haemophilia A<br>;Primary end point(s): • The incidence rate of FVIII inhibitors (=0.6 BU/mL)