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Home >> Clinical Studies
Amicus Therapeutics is conducting several clinical studies in patients with Fabry
and Pompe disease at participating sites in more than 20 countries.
Fabry Disease
Migalastat HCl Monotherapy: Phase 3 Study 011 (The FACETS, or FAB-AT1001-011 Study)
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients with
Fabry Disease
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Status: Ongoing, recruitment closed. In December 2011, the study completed
enrollment of 67 patients, who were identified as having amenable α-Gal A mutations
and met additional entry criteria.
- Design: Placebo-controlled, double-blind Phase 3 study of migalastat HCl
- More information: www.clinicaltrials.gov: NCT00925301
Migalastat HCl Monotherapy: Phase 3 Study 012 (The ATTRACT, or FAB-AT1001-012 Study)
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy
in Patients with Fabry Disease
- Status: Ongoing, recruitment closed. Enrolled more than 50 patients, who were identified as having amenable α-Gal A mutations and had been receiving enzyme replacement therapy (ERT) for at least 12 months.
- Design: Randomized, open-label, 18-month Phase 3 study investigating the safety
and efficacy of migalastat HCI compared to current standard-of-care ERTs Fabrazyme®
(agalsidase beta) or Replagal® (agalsidase alfa) for Fabry disease
- More information: www.clinicaltrials.gov: NCT01218659 or www.fabrystudy.com
Migalastat HCl Monotherapy: Phase 2 Extension Study 205 (FAB-CL-205 Study)
Open Label Long-term Safety Study
of AT1001 in Patients with Fabry Disease Who Have Completed a Previous AT1001 Study
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Status: Ongoing, recruitment closed
- Design: Open-label Phase 2 extension study to evaluate the long-term safety
and tolerability and to explore the efficacy of migalastat HCl in patients who have
previously completed a Phase 2 clinical study of migalastat HCl for Fabry disease.
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More information: www.clinicaltrials.gov: NCT00526071
Migalastat HCl Co-Administered with ERT: Phase 2 Study 013 (AT1001-013 Study)
Drug-Drug Interaction Study Between AT1001 and Agalsidase in Subjects With Fabry
Disease
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Status: Ongoing, recruitment closed. Enrolled males diagnosed with Fabry disease who are currently on ERT, independent of α-Gal A mutation type
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Design: Open-label Phase 2 study to compare a single administration of oral
migalastat HCl co-administered with infused ERT (Fabrazyme or Replagal) versus ERT
alone. Each patient receives their current dose and regimen of ERT alone at one
infusion and oral migalastat HCl (150 mg or 450 mg) administered prior to ERT at
their next infusion
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More information: www.clinicaltrials.gov: NCT01196871 or www.fabrystudy.com
Pompe Disease
Duvoglustat HCl (AT2220) Co-Administered with ERT: Phase 2 Study 010 (AT2220-010 Study)
An Open-Label,
Multi-Center, Study to Investigate Drug-Drug Interactions Between Duvoglustat
HCl (AT2220) and Alglucosidase Alfa in Patients With Pompe Disease
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Status: Ongoing, recruitment closed. Enrolled males and females diagnosed with Pompe disease who are currently on ERT
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Design: Open-label Phase 2 study to evaluate the safety and pharmacokinetic
effects of four increasing oral doses of the pharmacological chaperone duvoglustat HCl co-administered
with ERT (alglucosidase alfa – Myozyme®/Lumizyme®) versus ERT alone for Pompe disease.
Each patient receives their current dose and regimen of ERT alone at one infusion
and one of four ascending oral doses of AT2220 administered prior to ERT at their
next infusion.
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More information: www.clinicaltrials.gov: NCT1380743 or www.pompestudy.com
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Amicus Therapeutics is committed to improving the lives of patients and families affected by rare and orphan diseases.
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