Long-Term Safety and Efficacy of Nonacog Beta Pegol (N9-GP) Administered for at Least 5 Years in Previously Treated Children with Hemophilia B.
Adolescent
Age Factors
Asia
Child
Child, Preschool
Drug Administration Schedule
Europe
Factor IX
/ administration & dosage
Hemophilia B
/ blood
Hemorrhage
/ blood
Hemostatics
/ administration & dosage
Humans
Infant
North America
Patient Safety
Polyethylene Glycols
/ administration & dosage
Recombinant Proteins
/ administration & dosage
Risk Assessment
Risk Factors
Time Factors
Treatment Outcome
Journal
Thrombosis and haemostasis
ISSN: 2567-689X
Titre abrégé: Thromb Haemost
Pays: Germany
ID NLM: 7608063
Informations de publication
Date de publication:
May 2020
May 2020
Historique:
entrez:
6
5
2020
pubmed:
6
5
2020
medline:
25
8
2020
Statut:
ppublish
Résumé
Long-term safety and efficacy data of extended half-life factor IX (FIX) prophylaxis in children with hemophilia B (HB) are sparse. paradigm 5 is a multinational, open-label, single-arm, phase III trial assessing once-weekly (40 IU/kg) prophylactic nonacog beta pegol (N9-GP) in previously treated patients (PTPs) aged ≤ 12 years with HB (FIX activity ≤ 2%). Primary endpoint: incidence of anti-FIX inhibitory antibodies (≥ 0.6 Bethesda Units). We present a 5-year analysis (
Identifiants
pubmed: 32369845
doi: 10.1055/s-0040-1709521
doi:
Substances chimiques
Hemostatics
0
Recombinant Proteins
0
nonacog beta pegol
27Y83O992Q
Polyethylene Glycols
3WJQ0SDW1A
Factor IX
9001-28-9
Types de publication
Clinical Trial, Phase III
Journal Article
Multicenter Study
Langues
eng
Sous-ensembles de citation
IM
Pagination
737-746Informations de copyright
Georg Thieme Verlag KG Stuttgart · New York.
Déclaration de conflit d'intérêts
M.C. has received grant/research support from: Baxalta (Shire), Bayer, Biogen, Novo Nordisk and Pfizer, has been a consultant for Baxalta/Shire/Takeda, Bayer, Biogen/Bioverativ/Sanofi, CSL Behring, Grifols, Novo Nordisk, Octapharma, Pfizer, and Roche, and is a member of speaker bureaus of Baxalta (Shire), Bayer, Biogen, CSL Behring, Grifols, Novo Nordisk, Octapharma, Pfizer, and Roche.S.K. has received grant/research support from: local principal investigator (PI) research funding for Bayer, Bioverativ, Daiichi Sankyo, Grifols, and Novo Nordisk, and is a member of speaker bureaus/advisory boards for Bayer, Bioverativ, and Novo Nordisk.M.Y.L. has no conflict of interest.M.T. has served on advisory boards for Bayer, Bioverativ, Chugai, and Novo Nordisk; has received research support in clinical trials as a local PI for Bioverativ, Chugai, CSL Behring, and Novo Nordisk, and has received speaker's fees from Bayer, Bioverativ, Chugai, CSL Behring, Novo Nordisk, and Shire.D.R. and C.S. are employees of Novo Nordisk A/S, Søborg, Denmark.E.S. has served on advisory boards for Bayer, Bioverativ, CSL Behring, Grifols, Kedrion, Novo Nordisk, Octapharma, Pfizer, Roche, Shire/Takeda, SOBI, Spark, and Uniqure and has been a member of speaker bureaus for Bayer, CSL Behring, Grifols, Kedrion, Novo Nordisk, Pfizer, Roche, Shire/Takeda, and SOBI.