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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

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Feldt-Rasmussen, U, Hughes, D, Sunder-Plassmann, G, Shankar, S, Nedd, K, Olivotto, I, Ortiz, D, Ohashi, T, Hamazaki, T, Skuban, N, Yu, J, Barth, JA & Nicholls, K 2020, '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', Molecular Genetics and Metabolism, vol. 131, no. 1-2, pp. 219-228. https://doi.org/10.1016/j.ymgme.2020.07.007

APA

Feldt-Rasmussen, U., Hughes, D., Sunder-Plassmann, G., Shankar, S., Nedd, K., Olivotto, I., Ortiz, D., Ohashi, T., Hamazaki, T., Skuban, N., Yu, J., Barth, J. A., & Nicholls, K. (2020). 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. Molecular Genetics and Metabolism, 131(1-2), 219-228. https://doi.org/10.1016/j.ymgme.2020.07.007

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Author

Feldt-Rasmussen, Ulla ; Hughes, Derralynn ; Sunder-Plassmann, Gere ; Shankar, Suma ; Nedd, Khan ; Olivotto, Iacopo ; Ortiz, Damara ; Ohashi, Toya ; Hamazaki, Takashi ; Skuban, Nina ; Yu, Julie ; Barth, Jay A ; Nicholls, Kathleen. / 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. In: Molecular Genetics and Metabolism. 2020 ; Vol. 131, No. 1-2. pp. 219-228.

Bibtex

@article{a01930a3608c4935b1f8d851d14d7bcd,
title = "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",
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 continued receiving migalastat (Group 1 [MM]); patients who received ERT every other week discontinued ERT and started migalastat treatment (Group 2 [EM]). Outcome measures included eGFR, left ventricular mass index (LVMi), composite clinical outcome (renal, cardiac or cerebrovascular events), and safety. Forty-six patients who completed the randomized treatment period continued into the OLE (Group 1 [MM], n = 31; Group 2 [EM], n = 15). eGFR remained stable in both treatment groups. LVMi decreased from baseline at month 30 in Group 1 (MM) in patients with left ventricular hypertrophy at baseline. Only 10% of patients experienced a new composite clinical event with migalastat treatment during the OLE. No new safety concerns were reported. In conclusion, in patients with Fabry disease and amenable GLA variants, migalastat 150 mg QOD was well tolerated and demonstrated durable, long-term stability of renal function and reduction in LVMi.",
author = "Ulla Feldt-Rasmussen and Derralynn Hughes and Gere Sunder-Plassmann and Suma Shankar and Khan Nedd and Iacopo Olivotto and Damara Ortiz and Toya Ohashi and Takashi Hamazaki and Nina Skuban and Julie Yu and Barth, {Jay A} and Kathleen Nicholls",
note = "Copyright {\textcopyright} 2020 The Authors. Published by Elsevier Inc. All rights reserved.",
year = "2020",
month = oct,
day = "6",
doi = "10.1016/j.ymgme.2020.07.007",
language = "English",
volume = "131",
pages = "219--228",
journal = "Molecular Genetics and Metabolism",
issn = "1096-7192",
publisher = "Academic Press",
number = "1-2",

}

RIS

TY - JOUR

T1 - Long-term efficacy and safety of migalastat treatment in Fabry disease

T2 - 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study

AU - Feldt-Rasmussen, Ulla

AU - Hughes, Derralynn

AU - Sunder-Plassmann, Gere

AU - Shankar, Suma

AU - Nedd, Khan

AU - Olivotto, Iacopo

AU - Ortiz, Damara

AU - Ohashi, Toya

AU - Hamazaki, Takashi

AU - Skuban, Nina

AU - Yu, Julie

AU - Barth, Jay A

AU - Nicholls, Kathleen

N1 - Copyright © 2020 The Authors. Published by Elsevier Inc. All rights reserved.

PY - 2020/10/6

Y1 - 2020/10/6

N2 - 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 continued receiving migalastat (Group 1 [MM]); patients who received ERT every other week discontinued ERT and started migalastat treatment (Group 2 [EM]). Outcome measures included eGFR, left ventricular mass index (LVMi), composite clinical outcome (renal, cardiac or cerebrovascular events), and safety. Forty-six patients who completed the randomized treatment period continued into the OLE (Group 1 [MM], n = 31; Group 2 [EM], n = 15). eGFR remained stable in both treatment groups. LVMi decreased from baseline at month 30 in Group 1 (MM) in patients with left ventricular hypertrophy at baseline. Only 10% of patients experienced a new composite clinical event with migalastat treatment during the OLE. No new safety concerns were reported. In conclusion, in patients with Fabry disease and amenable GLA variants, migalastat 150 mg QOD was well tolerated and demonstrated durable, long-term stability of renal function and reduction in LVMi.

AB - 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 continued receiving migalastat (Group 1 [MM]); patients who received ERT every other week discontinued ERT and started migalastat treatment (Group 2 [EM]). Outcome measures included eGFR, left ventricular mass index (LVMi), composite clinical outcome (renal, cardiac or cerebrovascular events), and safety. Forty-six patients who completed the randomized treatment period continued into the OLE (Group 1 [MM], n = 31; Group 2 [EM], n = 15). eGFR remained stable in both treatment groups. LVMi decreased from baseline at month 30 in Group 1 (MM) in patients with left ventricular hypertrophy at baseline. Only 10% of patients experienced a new composite clinical event with migalastat treatment during the OLE. No new safety concerns were reported. In conclusion, in patients with Fabry disease and amenable GLA variants, migalastat 150 mg QOD was well tolerated and demonstrated durable, long-term stability of renal function and reduction in LVMi.

U2 - 10.1016/j.ymgme.2020.07.007

DO - 10.1016/j.ymgme.2020.07.007

M3 - Journal article

C2 - 33012654

VL - 131

SP - 219

EP - 228

JO - Molecular Genetics and Metabolism

JF - Molecular Genetics and Metabolism

SN - 1096-7192

IS - 1-2

ER -

ID: 61808067