Lysosomal Storage Diseases clinical trials at UC Irvine
1 in progress, 0 open to eligible people
Showing trials for
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
Sorry, in progress, not accepting new patients
The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts.
Orange, California and other locations
Our lead scientists for Lysosomal Storage Diseases research studies include Tahseen Mozaffar, MD.