rhGAA

DRUG2 trials

Sponsors

Amicus Therapeutics, Valerion Therapeutics, LLC

Conditions

Pompe Disease

Phase 1

VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease

TerminatedNCT02898753

Valerion Therapeutics, LLCPompe Disease

Start: 2017-06-21End: 2020-03-25Updated: 2020-06-02

Phase 2

Drug-drug Interaction Study

CompletedNCT01380743

Amicus TherapeuticsPompe Disease

Start: 2011-10-31End: 2013-01-04Updated: 2025-06-26

Related Papers

VAL-1221 for the treatment of patients with Lafora disease: study protocol for a single-arm, open-label clinical trial

BMJ Open2024-10-018 citations