Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study.
1-Deoxynojirimycin
/ administration & dosage
Adolescent
Adult
Aged
Biomarkers, Pharmacological
/ metabolism
Enzyme Replacement Therapy
Fabry Disease
/ drug therapy
Female
Humans
Hypertrophy, Left Ventricular
/ chemically induced
Kidney
/ drug effects
Male
Middle Aged
Mutation
/ genetics
Young Adult
alpha-Galactosidase
/ genetics
Chaperone
Clinical trial
Fabry disease
Lysosomal disorders
Migalastat
Journal
Molecular genetics and metabolism
ISSN: 1096-7206
Titre abrégé: Mol Genet Metab
Pays: United States
ID NLM: 9805456
Informations de publication
Date de publication:
Historique:
received:
27
03
2020
revised:
25
06
2020
accepted:
19
07
2020
pubmed:
6
10
2020
medline:
1
7
2021
entrez:
5
10
2020
Statut:
ppublish
Résumé
Results from the 18-month randomized treatment period of the phase 3 ATTRACT study demonstrated the efficacy and safety of oral migalastat compared with enzyme replacement therapy (ERT) in patients with Fabry disease who previously received ERT. Here, we report data from the subsequent 12-month, migalastat-only, open-label extension (OLE) period. ATTRACT (Study AT1001-012; NCT01218659) was a randomized, open-label, active-controlled study in patients aged 16-74 years with Fabry disease, an amenable GLA variant, and an estimated glomerular filtration rate (eGFR) ≥30 mL/min/1.73 m
Identifiants
pubmed: 33012654
pii: S1096-7192(20)30176-1
doi: 10.1016/j.ymgme.2020.07.007
pii:
doi:
Substances chimiques
Biomarkers, Pharmacological
0
1-Deoxynojirimycin
19130-96-2
migalastat
C4XNY919FW
alpha-Galactosidase
EC 3.2.1.22
Banques de données
ClinicalTrials.gov
['NCT01218659']
Types de publication
Clinical Trial, Phase III
Journal Article
Randomized Controlled Trial
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
219-228Informations de copyright
Copyright © 2020 The Authors. Published by Elsevier Inc. All rights reserved.
Déclaration de conflit d'intérêts
Declaration of competing interest UFR has served on advisory boards for Amicus Therapeutics, Shire, Freeline, and Sanofi Genzyme, as a speaker for Amicus Therapeutics and Sanofi Genzyme, and has received research funding from Shire, Amicus Therapeutics, and Sanofi Genzyme. DH has received honoraria and research funding from Amicus Therapeutics, Shire, Sanofi Genzyme, Protalix, and Actelion. GSP has served on advisory boards for Amicus Therapeutics and Greenovation, as a speaker for Sanofi Genzyme, and has received research funding from Amicus Therapeutics, Idorsia, and Shire. SS has served as an investigator for Amicus Therapeutics. K Nedd received fees from Sanofi Genzyme and Shire-Takeda. IO has received research funding from and served as a speaker for Shire, Sanofi Genzyme, and MyoKardia. DO has served on advisory boards for Sanofi Genzyme. TH has nothing to disclose. TO has received research funding from DSP, Sanofi Genzyme, and Avrobio. NS, JY, and JAB are employees of and hold stock in Amicus Therapeutics. K Nicholls has served on advisory boards for Amicus Therapeutics, Sanofi Genzyme, and Shire, as a speaker for Amicus Therapeutics, and has received research funding from Sanofi Genzyme and Shire.