Clinical Studies

Fabry Disease

Migalastat Monotherapy: Phase 3 Long-Term Safety Study 041 (AT1001-041 Study, formerly MGM116041)

Long-Term, Open-Label Study of Migalastat (AT1001) in Subjects with Fabry Disease Who Completed Treatment in a Previous Monotherapy Trial with Migalastat

Completed. Patients are eligible to roll over into Study 041 upon completion of the primary treatment and open-label extension periods in the FACETS (Study 011), ATTRACT (Study 012), or Phase 2 Extension Study 205.

Open-label extension study to evaluate long-term safety and efficacy of migalastat.

More information: www.clinicaltrials.gov: NCT01458119


Migalastat Monotherapy: Phase 3 Long-Term Safety Study 042 (AT1001-042 Study)

Open-Label Extension Study of the Long-Term Effects of Migalastat in Patients with Fabry Disease

Recruiting. Intended to provide continued treatment with migalastat for subjects with Fabry disease who completed treatment of a previous migalastat monotherapy study.

Open-label extension study to assess the long-term safety and effectiveness of migalastat in subjects with Fabry disease who completed migalastat treatment in a previous study.

More information: www.clinicaltrials.gov: NCT02194985


Migalastat Monotherapy: Physician Initiated Request (MGM116188)

Program Allows Physicians to Request Permission from Amicus Therapeutics for Treatment Access to Migalastat for Specific Adult Patients with Fabry Disease

Ongoing, currently recruiting. Physicians may request permission to treat specific patients with migalastat. Patients have amenable GLA mutations, do not meet eligibility criteria for existing migalastat clinical studies, and are unsuitable for or unable to access ERT.

Open-label treatment in up to 20 patients for 6 months with renewal every 6 months.

More information: www.clinicaltrials.gov: NCT01476163


Fabry Disease
Completed Studies

Migalastat Monotherapy: Phase 3 Study 011 (The FACETS, or AT1001-011 Study)

Study of the Effects of Oral Migalastat (AT1001) in Patients with Fabry Disease

Completed. In December 2011, the study completed enrollment of 67 patients, who were identified as having amenable GLA mutations and met additional entry criteria.

Placebo-controlled, double-blind Phase 3 study of migalastat.

More information: www.clinicaltrials.gov: NCT00925301


Migalastat Monotherapy: Phase 3 Study 012 (The ATTRACT or AT1001-012 Study)

Study to Compare the Efficacy and Safety of Oral Migalastat (AT1001) and Enzyme Replacement Therapy in Patients with Fabry Disease

Completed. Enrolled more than 50 patients, who were identified as having amenable GLA mutations and had been receiving enzyme replacement therapy (ERT) for at least 12 months.

Randomized, open-label, 18-month Phase 3 study investigating the safety and efficacy of migalastat compared to current standard of care ERTs Fabrazyme® (agalsidase beta) or Replagal® (agalsidase alfa) for Fabry disease.

More information: www.clinicaltrials.gov: NCT01218659.