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 Long-term Safety Study 042 (At1001-042 Study)

More Information:
www.clinicaltrials.gov: NCT02194985

Migalastat Monotherapy: Active, Not Recruiting Study 020 (AT1001-020 Study)

More Information:
www.clinicaltrials.gov: NCT03500094

Migalastat Monotherapy: Currently Enrolling Study 036 (AT1001-036 Study)

More Information:
www.clinicaltrials.gov: NCT04049760

Migalastat Monotherapy: A Prospective, Observational Registry of Patients with Fabry Disease (AT1001-030)

More Information:
www.encepp.eu: EUPAS20599

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

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

NP-NN-ALL-00120320