clinical trial · NCT04093349
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
Spark Therapeutics, Inc.·phase2·active not recruiting·n = 4
Pompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2Glycogen Storage Disease Type IILOPDLysosomal Storage DiseasesAcid Maltase DeficiencySPK-3006
brief 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.
started
Oct 1, 2020
primary completion
Apr 1, 2032
completion
Apr 1, 2032
last updated
Nov 27, 2024
official title
Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol