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Clinical Trial Summary

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.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT04093349
Study type Interventional
Source Spark Therapeutics
Contact
Status Active, not recruiting
Phase Phase 1/Phase 2
Start date October 1, 2020
Completion date April 2032

See also
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