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 (pathfinder™5)

Novo Nordisk

Status and phase: Completed; Phase 3

Conditions: Haemophilia A; Congenital Bleeding Disorder

Treatments: Drug: turoctocog alfa pegol

Study type: Interventional

Funder types: Industry

Identifiers

NCT01731600

NN7088-3885

U1111-1129-6009 (Other Identifier)

2012-001711-23 (EudraCT Number)

JapicCTI-132214 (Registry Identifier)

Details and patient eligibility

About

This trial is conducted globally. The aim of the trial is to investigate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who have undergone treatment with previous factor VIII (FVIII) products.

Enrollment: 68 patients

Sex: Male

Ages: Under 11 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male patients with severe congenital haemophilia A (FVIII activity level below 1%)
• Weight above or equal to 10 kg - Documented history of 150 exposure days (ED) to FVIII products for patients aged 6-11 years and above 50 ED to FVIII products for patients aged 0-5 years

Exclusion criteria

• Any history of FVIII inhibitors

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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68 participants in 1 patient group

N8-GP

Experimental group

Treatment:

Drug: turoctocog alfa pegol