A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Status Active, not recruiting

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

Clinical Trial Details

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

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See also
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