Trial
NCT00688597
Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease
Phase IITERMINATEDSAFETYConfidence: HIGH
Sponsor
Amicus Therapeutics
Collaborators
—
Condition
Pompe Disease
Intervention
Duvoglustat
Why stopped
Per protocol, 3 sequential dose cohorts were planned. Study discontinued by Sponsor based upon serious adverse events in first 2 of 3 participants in Cohort 1.
Provenance
Dataset: 2026-07-02 • Source: ClinicalTrials.gov API v2 • Last update: 2025-09-11