Efficacy and safety of asfotase alfa in infants and young children with hypophosphatasia: A phase 2 open-label study
EntityUAM. Departamento de Pediatría; Instituto de Investigación del Hospital de La Princesa (IP)
PublisherOxford University Press
10.1210/jc.2018-02335Journal of Clinical Endocrinology and Metabolism 104.7 (2019): 2735-2747
Funded byThis study was sponsored by Alexion Pharmaceuticals, Inc. (Boston, MA, USA). The study was also supported, in part, by the National Center for Advancing Translational Sciences, National Institutes of Health (NIH), through University of California, San Francisco–Clinical and Translational Science Institute (UCSF–CTSI) Grant Number UL1 TR000004 (to P.H.)
SubjectsHypophosphatasia; Efficacy and safety; Asfotase alfa; Infants/young children; Medicina
Rights© 2019 Endocrine Society.
Esta obra está bajo una Licencia Creative Commons Atribución 4.0 Internacional.
Long-term data on enzyme replacement treatment of hypophosphatasia (HPP) are limited. Objective: To evaluate efficacy and safety of asfotase alfa in patients aged #5 years with HPP followed for up to 6 years. Design: Phase 2 open-label study (July 2010 to September 2016). Setting: Twenty-two sites; 12 countries. Participants: Sixty-nine patients [median (range) age: 16.0 (0.02 to 72) months] with severe HPP and sign/symptom onset before age 6 months. Intervention: Asfotase alfa 2 mg/kg three times/week or 1 mg/kg six times/week subcutaneously. Main Outcome Measures: Primary efficacy measure: Radiographic Global Impression of Change (RGI-C) score [23 (severe worsening) to 13 (complete/near-complete healing)]. Additional outcome measures: respiratory status, growth, and safety. Post hoc analysis: characteristics of radiographic responders vs nonresponders at Year 1 (RGI-C: 12 vs ,12).
Google Scholar:Hofmann, Christine E. - Harmatz, Paul - Vockley, Jerry - Högler, Wolfgang - Nakayama, Hideki - Bishop, Nick - Martos Moreno, Gabriel Ángel - Moseley, Scott - Fujita, Kenji P. - Liese, Johannes - Rockman-Greenberg, Cheryl
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