Clinical Trials

Clinical trials are integral to bringing forward breakthroughs. At Amicus, we believe that all study participants are equal clinical research partners in the drug development process. For all of our clinical trials, we actively seek and incorporate input from health care providers, patients and patient advocates – who are why we create medicines in the first place.  

We are immensely grateful to our collaborators for their crucial role in bringing forward our medicines, and in shaping the medicines we are developing for the future. 

Here’s information about our clinical trials open to patients today: 

  • A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
    More information: www.clinicaltrials.gov (ID NCT06904261)
  • Migalastat Monotherapy: Phase 3 Renal Study (AT1001-025 Study)
    More information: www.clinicaltrials.gov (ID NCT04020055)
  • A Prospective Observational Study of Women with Fabry Disease (AT1001-037 Study)
    More information: www.clinicaltrials.gov (ID NCT04252066)
  • A Prospective Observational Registry of Patients with Fabry Disease (AT1001-030)
    More information: www.encepp.eu (ID EUPAS20599)
  • AT-GAA: Phase 3 ROSSELLA Study (ATB200-08 Study; patients 0 to <18 years, IOPD)
    More information: www.clinicaltrials.gov  (ID NCT04808505) 
  • A Global Prospective Observational Registry of Patients With Pompe Disease (POM-005)
    More information:www.clinicaltrials.gov  (ID NCT06121011)
  • AT-GAA: Phase 3 ZIP Study (ATB200-04 Study; patients 0 to <18 years, LOPD)
    More Information: www.clinicaltrials.gov: NCT03911505 
  • Focal Segmental Glomerulosclerosis (FSGS) Clinical TrialsDMX-200: Phase 3 ACTION3 Study*
    More information: www.clinicaltrials.gov (ID NCT05183646)
    *Dimerix is the sponsor of this study, Amicus has U.S. commercial rights and will be responsible for U.S. regulatory submissions
  • Migalastat Monotherapy: Phase 3 Renal Study (AT1001-025 Study)
    More information: www.clinicaltrials.gov (ID NCT04020055)