Journal article

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

Ulla Feldt-Rasmussen, Derralynn Hughes, Gere Sunder-Plassmann, Suma Shankar, Khan Nedd, Iacopo Olivotto, Damara Ortiz, Toya Ohashi, Takashi Hamazaki, Nina Skuban, Julie Yu, Jay A Barth, Kathleen Nicholls

Molecular Genetics and Metabolism | ACADEMIC PRESS INC ELSEVIER SCIENCE | Published : 2020

Abstract

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 m2. During the OLE, patients who received migalastat 150 mg every other day (QOD) during the randomized period conti..

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Grants

Funding Acknowledgements

This study was funded by Amicus Therapeutics, Inc. This study was designed, managed, and analyzed jointly by authors employed by Amicus Therapeutics, Inc. and external authors who were not paid for their work. Third-party medical writing assistance was provided by ApotheCom and supported by Amicus Therapeutics, Inc.