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

• Conditions: Following EMEA guidelines NN7008-3545 trial is a prospective clinical phase 3a trial intended at demonstrating safety and efficacy of N8 in previously treated patients < 12 years of age with severe haemophilia A. The trial will continue until at least 50 patients have received at least 50 EDs to the product.; MedDRA version: 12.1Level: LLTClassification code 10053753Term: Hemophilia A without inhibitors

• Registration Number: EUCTR2009-016383-36-IT
• Lead Sponsor: OVO NORDISK

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-06-07
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

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

Inclusion Criteria

1. 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.) 2. Male patients with severe (baseline FVIII =1%) haemophilia A 3. Age <12 years and weight =11 kg 4. Documented history of a minimum 50 EDs to FVIII produc prophylaxis/prevention/surgery/on-demand)(Please refer to section 8.4.4.2) 5. No factor VIII inhibitors ( =0.6 BU/mL) at screening 6. Documented negative FVIII inhibitor test(s) ( < 0.6 BU/mL ) or documented FVIII recovery tests (within expected normal ranges) within first 50 EDs 7. 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

1. Known or suspected allergy to hamster protein or intolerance to trial product or related products 2. Previous participation in this trial defined as withdrawal after administration of trial product 3. Any history of FVIII inhibitors (=0.6 BU/mL)(Please refer to section 8.4.4.1) 4. Surgery planned to occur during the trial (exceptions are port placement, dental extractions, and minor, uncomplicated emergent procedures) 5. Platelet count <50,000 platelets/µL 6. Congenital or acquired coagulation disorders other than haemophilia A 7. Ongoing treatment or planned treatment during the trial with chemotherapy, immunomodulatory agents (e.g. intravenous immunoglobulin (IVIG), routine systemic corticosteroids) 8. The receipt of any investigational product within 30 days prior to inclusion in this trial 9. 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 10. Renal dysfunction defined as serum creatinine level = 2 times upper limit of normal or glomerular filtration rate decrease > 50% 11. Liver dysfunction defined as ALT or GGT = 2 times upper limit of normal, or jaundice. 12. Documented diagnosis of obesity (only for patients in the PK part) defined as weight equal to or greater than the 95th percentile for age (refer to Appendix E) 13. Unwillingness, language or other barriers precluding adequate understanding and/or cooperation from parents and child

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified