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 (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