Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A

Summary

This trial is conducted globally. The aim of the trial is to evaluate the safety and efficacy, including pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in subjects with Haemophilia A.

Timeline

Start

2012-01-30

Primary completion

2018-12-10

Completion

2018-12-10

Outcome

Met primary endpoint

registry p <0.001; Poisson estimate 3.70 (95% CI 2.94 to 4.66) NCT01480180 ↗

registry p <0.001; Poisson estimate 3.27 (95% CI 2.59 to 4.11) NCT01480180 ↗

registry p <0.001; Poisson estimate 2.35 (95% CI 1.87 to 2.95) NCT01480180 ↗

registry p <0.001; Poisson estimate 4.39 (95% CI 3.09 to 6.24) NCT01480180 ↗

Publications

• Results Giangrande P, Chowdary P, Enhrenforth S, Hanabusa H, Leebeek FW, Lentz SR, Nemes L, Poulsen LH, Santagostino E, You CW, Clausen WHO, Oldenburg J and on behalf of for the pathfinder™2 Investigators. Clinical evaluation of novel recombinant glycopegylated FVIII (turoctocog alfa pegol, N8-GP): efficacy and safety in previously treated patients with severe hemophilia A - results of pathfinder™2 international trial. Journal of Thrombosis and Haemostasis (Abstracts) 2015; 13 (Supplement S2): 1-997 [OR212]
• Results Giangrande P, Andreeva T, Chowdary P, Ehrenforth S, Hanabusa H, Leebeek FW, Lentz SR, Nemes L, Poulsen LH, Santagostino E, You CW, Clausen WH, Jonsson PG, Oldenburg J; Pathfinder2 Investigators. Clinical evaluation of glycoPEGylated recombinant FVIII: Efficacy and safety in severe haemophilia A. Thromb Haemost. 2017 Jan 26;117(2):252-261. doi: 10.1160/TH16-06-0444. Epub 2016 Dec 1.
• Results Giangrande P, Abdul Karim F, Nemes L, You CW, Landorph A, Geybels MS, Curry N. Long-term safety and efficacy of N8-GP in previously treated adults and adolescents with hemophilia A: Final results from pathfinder2. J Thromb Haemost. 2020 Sep;18 Suppl 1(Suppl 1):5-14. doi: 10.1111/jth.14959.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	turoctocog alfa pegol	Protein / enzyme biologic	20 iu/kg	Intravenous
Subject	turoctocog alfa pegol	Protein / enzyme biologic	50 iu/kg	Intravenous
Subject	turoctocog alfa pegol	Protein / enzyme biologic	75 iu/kg	Intravenous

Indications