Programs & Pipeline ❯ Clinical Trials
Clinical Trials
We support the disease communities – and their families
View ongoing and completed clinical studies for the following conditions:
Fabry Disease Clinical Trials
Note to healthcare providers and patients: Amicus Therapeutics will provide support to patients and healthcare providers to help provide continued access to treatment. For more information, contact EarlyAccess@amicusrx.com.
A Thank You to All Participants: At Amicus, we believe that all study participants are equal clinical research partners in the drug development process. Without the support of their families, friends, and healthcare teams, the Amicus clinical research program could not move forward. Amicus would like to express its gratitude to the Fabry community and all involved in the ongoing and completed clinical trials and all of our clinical research.
Migalastat Monotherapy: Phase 3 Long-term Safety Study (AT1001-042 Study)
More Information:
www.clinicaltrials.gov: NCT02194985
Migalastat Monotherapy: Phase 3 Safety Study (AT1001-020 Study; pediatric patients 12 to <18 years)
More Information:
www.clinicaltrials.gov: NCT03500094
Migalastat Monotherapy: Phase 3 Safety Study (AT1001-036 Study; pediatric patients >12 years)
More Information:
www.clinicaltrials.gov: NCT04049760
Migalastat Monotherapy: Phase 3 Renal Study (AT1001-025 Study)
More Information:
clinicaltrials.gov: NCT04020055
A Prospective Observational Registry of Patients with Fabry Disease (AT1001-030)
More Information:
www.encepp.eu: EUPAS20599
A Prospective Observational Study of Women with Fabry Disease (AT1001-037 Study)
More Information:
clinicaltrials.gov: NCT04252066
Pompe Disease Clinical Trials
A Thank You to All Participants: At Amicus, we believe that all study participants are equal clinical research partners in the drug development process. Without the support of their families, friends, and healthcare teams, the Amicus clinical research program could not move forward. Amicus would like to express its gratitude to the Pompe disease community and all involved in the ongoing and completed clinical trials and all of our clinical research.
AT-GAA: Phase 1/2 Safety Study (ATB200-02 Study)
More Information:
www.clinicaltrials.gov: NCT02675465
AT-GAA: Phase 3 PROPEL Study (ATB200-03 Study)
More Information:
www.clinicaltrials.gov: NCT03729362
Official PROPEL study website: www.pompestudy.com
AT-GAA: Phase 3 ZIP Study (ATB200-04 Study; patients 0 to <18 years, LOPD)
More Information:
www.clinicaltrials.gov: NCT03911505
AT-GAA: Phase 3 ROSSELLA Study (ATB200-08 Study; patients 0 to <18 years, IOPD)
More Information:
www.clinicaltrials.gov: NCT04808505
A Global Prospective Observational Registry of Patients With Pompe Disease (POM-005)
More Information:
www.clinicaltrials.gov: NCT06121011
Focal Segmental Glomerulosclerosis (FSGS) Clinical Trials
DMX-200: Phase 3 ACTION3 Study*
More Information:
www.clinicaltrials.gov: NCT05183646
*Dimerix is the sponsor of this study, Amicus has U.S. commercial rights and will be responsible for U.S. regulatory submissions