Trial reportJournal of thrombosis and haemostasis : JTH2020
Long-term safety and efficacy of N8-GP in previously treated adults and adolescents with hemophilia A: Final results from pathfinder2.
Trial report in Journal of thrombosis and haemostasis : JTH, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01480180 (A Multi-national Trial Evaluating Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Patients With Haemophilia A), which is not on this map. Cited by 13 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Multi-national Trial Evaluating Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Patients With Haemophilia A
Who cites it
13 citing papers in PubMed, 28 citations in OpenAlex.
- The efficacy, safety, and pharmacokinetics of N8-GP in previously treated Chinese patients with haemophilia A: results from the phase 3b pathfinder10 study.Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis · 2025Trial
- Long-Term Efficacy and Safety of Damoctocog Alfa Pegol Prophylaxis in Patients with Haemophilia A Aged 12-<18 Years at Enrolment into PROTECT VIII.Acta haematologica · 2025Trial
- The safety and efficacy of N8-GP (turoctocog alfa pegol) in previously untreated pediatric patients with hemophilia A.Blood advances · 2023Trial
- Confirmed long-term safety and efficacy of prophylactic treatment with BAY 94-9027 in severe haemophilia A: final results of the PROTECT VIII extension study.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Trial
- Long-term safety and efficacy of N8-GP in previously treated adults and adolescents with hemophilia A: Final results from pathfinder2.Journal of thrombosis and haemostasis : JTH · 2020Trial
- Long-term efmoroctocog alfa prophylaxis improves perceived pain, mental, and physical health in patients with hemophilia A:Therapeutic advances in hematology · 2024Article
- Immunogenicity assessment strategy for a chemically modified therapeutic protein in clinical development.Frontiers in immunology · 2024Article
- Reduced FVIII recovery associated with anti-FVIII PEG antibodies after BNT162b2 SARS-CoV-2 vaccination.Blood advances · 2023Article
- Predictive Modeling Identifies Total Bleeds at 12-Weeks Postswitch to N8-GP Prophylaxis as a Predictor of Treatment Response.Thrombosis and haemostasis · 2022Article
- Turoctocog alfa pegol (N8-GP) in severe hemophilia A: Long-term safety and efficacy in previously treated patients of all ages in the pathfinder8 study.Research and practice in thrombosis and haemostasis · 2022Article
- Current therapeutic approaches in the management of hemophilia-a consensus view by the Romanian Society of Hematology.Annals of translational medicine · 2021Review
- Illustrative Cases from the Pathfinder Clinical Trials of Patients with Hemophilia A Treated with Turoctocog Alfa Pegol (N8-GP).Patient preference and adherence · 2021Article
- An overview of the pathfinder clinical trials program: Long-term efficacy and safety of N8-GP in patients with hemophilia A.Journal of thrombosis and haemostasis : JTH · 2020Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 4 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundN8-GP (turoctocog alfa pegol; Esperoct
objectiveWe report end-of-trial efficacy and safety of N8-GP from pathfinder2.
methodspathfinder2 main phase and extension phase part 1 results have been previously reported. During extension phase part 2, patients could switch from N8-GP prophylaxis 50 IU/kg every fourth day (Q4D) or 75 IU/kg once weekly (Q7D), depending on bleeding status. Extension phase part 2 collected long-term safety and efficacy data for all regimens until trial end (first patient in main phase, 30 January 2012; trial end, 10 December 2018).
resultsOverall, 186 patients were exposed to N8-GP for up to 6.6 years (median 5.4 years). The estimated annualized bleeding rate (ABR) was 2.14 (median 0.84) for the Q4D prophylaxis arm and 1.31 (median 1.67) for the Q7D prophylaxis arm. Nearly 30% of patients experienced zero bleeds throughout the entire duration of the trial, the hemostatic response was 83.2% across all treatment arms, and patient-reported outcomes were maintained or slightly improved. No safety concerns were detected.
conclusionData from the completed pathfinder2 trial, one of the largest and longest-running clinical trials to investigate treatment of severe hemophilia A, demonstrate the efficacy and safety of N8-GP in previously treated adolescent and adult patients.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.