NN7999-3774. NNC0156-0009 in paediatric, previously treated subjects.
Research type
Research Study
Full title
Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children with Haemophilia B
IRAS ID
110919
Contact name
Michael Williams
Sponsor organisation
Novo Nordisk A/S
Eudract number
2011-000826-31
Clinicaltrials.gov Identifier
Duration of Study in the UK
5 years, 11 months, 0 days
Research summary
This trial investigates the safety, efficacy and pharmacokinetics (PK) of NNC-0156-0009 (also known as N9-GP or NN7999) in previously treated children with moderate to severe haemophilia B. This rare population of patients are predisposed to bleeding due to congenital absence of coagulation factor IX activity. Patients usually receive lifelong treatment with factor IX products from the age of approximately 2 years to prevent and/or treat bleeds.
N9-GP is a new factor IX product which is modified to prolong its duration of action.
To prevent bleeding, current marketed factor IX products typically need to be given approximately twice weekly by intravenous injection, causing significant inconvenience and discomfort and conveying risk of infection. Longer-acting factor IX products could reduce the frequency of dosing required for effective prophylaxis
Development of new products is conducted according to guidance published by the European Medicines Agency (EMA/CHMP/BPWP/144552/2009,). Trials involving paediatric subjects are required by this guideline for good reasons: Published data has shown altered pharmacokinetics of coagulation factors in children due to faster clearance (compared to adults). Effective prevention of bleeds during childhood can avoid complications such as irreversible joint damage and reduced mobility later in life.
Previous studies have evaluated NNC-0156-0009 in adult and adolescent subjects. This study is necessary to confirm safety and efficacy and explore pharmacokinetics of N9-GP in younger patients.
Following a successful screening visit, subjects will receive their first two doses of N9-GP in clinic. Thereafter, clinic visits will be scheduled every 4-8 weeks for one year. If patients wish to continue their participation, they may then enter the extension phase of the trial during which the visit frequency is gradually reduced to once every six months.
The study is sponsored by the pharmaceutical company Novo Nordisk A/S and will be conducted in specialist haemophilia centres.REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
12/NE/0314
Date of REC Opinion
5 Oct 2012
REC opinion
Further Information Favourable Opinion