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 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 CLN3 Gene Therapy: Phase 1/2 Study
More Information:
www.clinicaltrials.gov: NCT03770572
NP-NN-ALL-00120320