Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at Orange, California and other locations
Dates
study started
completion around
Principal Investigator
by Tahseen Mozaffar, MD
Headshot of Tahseen Mozaffar
Tahseen Mozaffar

Description

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.

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

Keywords

Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency, Glycogen Storage Disease, Metabolic Diseases, Disease, SPK-3006

Eligibility

Locations

  • University of California Irvine Health
    Orange California 92868 United States
  • Barrow Neurological Institute
    Phoenix Arizona 85013 United States

Lead Scientist at UC Irvine

  • Tahseen Mozaffar, MD
    Clinical Professor, Neurology, School of Medicine. Authored (or co-authored) 160 research publications

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Spark Therapeutics, Inc.
ID
NCT04093349
Phase
Phase 1/2 research study
Study Type
Interventional
Participants
About 4 people participating
Last Updated