Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at Orange, California and other locations
Dates
study started
estimated completion
Principal Investigator
Tahseen Mozaffar, MD
Photo 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 SPK-3006

Eligibility

You can join if…

Open to people ages 18 years and up

  • Provide written informed consent;
  • Males and Females ≥18 years of age with late-onset Pompe disease;
  • Received ERT for at least the previous 24 months
  • Have clinically moderate, late-onset Pompe disease characteristics;
  • Agree to use reliable contraception.

You CAN'T join if...

  • Active hepatitis B and/or C;
  • Significant underlying liver disease;
  • Human immunodeficiency virus (HIV) infection;
  • Prior hypersensitivity to rhGAA;
  • Pre-existing anti-AAV neutralizing antibody titers;
  • High titer antibody responses to rhGAA;
  • Requires any invasive ventilation or requires noninvasive ventilation while awake and upright;
  • Received any prior vector or gene transfer agent;
  • Active malignancy (except non-melanoma skin cancer);
  • History of liver cancer;
  • Pregnant or nursing women;
  • Any evidence of active infection at the time of SPK-3006 infusion.

Locations

  • University of California Irvine Health accepting new patients
    Orange California 92868 United States
  • Barrow Neurological Institute not yet accepting patients
    Phoenix Arizona 85013 United States

Lead Scientist at UC Irvine

  • Tahseen Mozaffar, MD
    Clinical Professor, Neurology. Authored (or co-authored) 109 research publications.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Spark Therapeutics
Links
Related Info
ID
NCT04093349
Phase
Phase 1/2
Study Type
Interventional
Last Updated