Safety and Efficacy of Elosulfase Alfa in Australian Patients with Morquio a Syndrome: A Phase 3b Study
J. inborn errors metab. screen
; 8: e20200001, 2020. tab, graf
Article
in English
|
LILACS-Express
| LILACS
| ID: biblio-1135005
Responsible library:
BR1.1
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.
Full text:
Available
Collection:
International databases
Database:
LILACS
Type of study:
Controlled clinical trial
/
Prognostic study
Aspects:
Patient-preference
Language:
English
Journal:
J. inborn errors metab. screen
Journal subject:
Medicina Cl¡nica
/
Patologia
Year:
2020
Document type:
Article
Affiliation country:
Australia
/
United States
Institution/Affiliation country:
BioMarin Pharmaceutical Inc/US
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Perth Childrens Hospital/AU
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Queensland Childrens Hospital/AU
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Sydney Childrens Hospitals Network/AU
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The Royal Childrens Hospital Melbourne/AU
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University of Sydney/AU
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Womens and Childrens Hospital Adelaide/AU