Clinical Trials Information | Amicus Therapeutics

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 Renal Impairment Safety and Pharmacokinetic Study (AT1001-025)

More Information:
www.clinicaltrials.gov: NCT04020055

Migalastat Monotherapy: Phase 3 Long-term Safety Study 042 (At1001-042 Study)

More Information:
www.clinicaltrials.gov: NCT02194985

Migalastat Monotherapy: Expanded Access Physician Initiated Request (MGM116188)

More Information:
www.clinicaltrials.gov: NCT01476163

Migalastat Monotherapy: Currently Enrolling Study 020 (AT1001-020 Study)

More Information:
www.clinicaltrials.gov: NCT03500094

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 Fabry 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

Batten Disease Clinical Trials

Batten CLN6 Gene Therapy: Phase 1/2 Study

More Information:
www.clinicaltrials.gov: NCT02725580

Batten CLN3 Gene Therapy: Phase 1/2 Study

More Information:
www.clinicaltrials.gov: NCT03770572