Open-Label Phase 3 Long-Term Safety Study of Migalastat

Summary

This was a long-term, open-label study of migalastat (123 milligrams \[mg\] of migalastat \[equivalent to 150 mg of migalastat hydrochloride\]) (migalastat) in participants with Fabry disease who completed treatment in a previous monotherapy trial with migalastat.

Timeline

Start

2011-10-14

Primary completion

2016-02-17

Completion

2016-02-17

Outcome

Missed primary endpoint

Stopped: “Amicus Therapeutics discontinued Study AT1001-041 for logistical reasons.”

paper 13 of 32 patients (41%) who received migalastat and 9 of 32 patients (28%) who received placebo had a response at 6 months (P=0.30). PMID 27509102 ↗

Publications

• Germain DP, Hughes DA, Nicholls K, Bichet DG, Giugliani R, Wilcox WR, Feliciani C, Shankar SP, Ezgu F, Amartino H, Bratkovic D, Feldt-Rasmussen U, Nedd K, Sharaf El Din U, Lourenco CM, Banikazemi M, Charrow J, Dasouki M, Finegold D, Giraldo P, Goker-Alpan O, Longo N, Scott CR, Torra R, Tuffaha A, Jovanovic A, Waldek S, Packman S, Ludington E, Viereck C, Kirk J, Yu J, Benjamin ER, Johnson F, Lockhart DJ, Skuban N, Castelli J, Barth J, Barlow C, Schiffmann R. Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat. N Engl J Med. 2016 Aug 11;375(6):545-55. doi: 10.1056/NEJMoa1510198.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	migalastat	Other / unclassified	150 mg	Oral

Indications