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Title: Safety and Efficacy of Elosulfase Alfa in Australian Patients with Morquio a Syndrome: A Phase 3b Study
Authors: Owens, Penny ;Bhattacharya, K.;Balasubramaniam, S.;Murray, K.;Peters, H.;Ketteridge, D.;Inwood, A.;Lee, J.;Ellaway, C.;Wong, M.
Affliation: Central Coast Local Health District
Issue Date: 2020
Source: 8:e20200001
Journal title: Journal of Inborn Errors of Metabolism and Screening
Department: Paediatrics
Abstract: Abstract The safety and efficacy of elosulfase alfa were evaluated in a multicenter, open-label, phase 3b study in Australian Morquio A patients, consisting of a 49-week initial phase and an extension phase until elosulfase alfa was government funded. Thirteen patients (1-27 years) were enrolled. No new safety concerns were identified over 138 weeks. Most drug-related adverse events were mild or moderate in severity; none led to study discontinuation. After 49 weeks of treatment, median improvements from baseline were seen in the 6-minute walk test (+41.0 m), 3-minute stair climb test (+14.0 stairs/min), forced vital capacity (+16.4%), forced expiratory volume in 1 second (+14.1%), urine keratan sulfate (-7.1 µg/mg creatinine), and pain intensity. Growth, cardiac function, sleep, and quality of life results were mixed or stable. These results provide further evidence of the acceptable safety/tolerability profile of elosulfase alfa. The improvements in endurance, pulmonary function, and pain support findings from previous studies.
DOI: 10.1590/2326-4594-JIEMS-2020-0001
ISSN: 2326-4594
Publicaton type: Journal Article
Study or Trial: Multicentre Studies
Appears in Collections:Health Service Research

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