A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients with Severe Haemophilia A

Phase 1

• Conditions: Haemophilia A; MedDRA version: 20.0Level: LLTClassification code 10018938Term: Haemophilia A (Factor VIII)System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2012-001711-23-LT
• Lead Sponsor: ovo Nordisk A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-03-21
• Last Update Posted: 6 November 2018

Recruitment & Eligibility

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

Inclusion Criteria

- Male patients with severe congenital haemophilia A (FVIII activity level < 1%)
- Age below 12 years at screening (for Turkey only: Age above 3 and below 12 years at screening)
- Weight =10 kg
- Documented history of > 150 ED to FVIII products for patients aged 6-11 years and > 50 ED to FVIII products for patients aged 0-5 years (for Turkey only: Documented history of > 150 ED to FVIII products for patients aged 6-11 years and > 50 ED to FVIII products for patients aged 3-5 years)
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

Any history of FVIII inhibitors

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate immunogenicity of NNC 0129-0000-1003 (hereafter referred to as N8-GP);Secondary Objective: - To evaluate safety other than immunogenicity of N8-GP<br>- To evaluate efficacy of N8-GP in prophylaxis and treatment of bleeding episodes<br>- To evaluate pharmacokinetic properties of N8-GP and compare to previous FVIII product;Primary end point(s): Incidence of inhibitory antibodies against coagulation factor VIII (FVIII) =0.6 Bethesda units;Timepoint(s) of evaluation of this end point: During the main phase of the trial (from 0-26 weeks of treatment)