This study is in progress, not accepting new patients

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

a study on Pompe Disease Lysosomal Storage Diseases Metabolic Disease

Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at UC Irvine
Dates
study started
completion around
Principal Investigator
by Tahseen Mozaffar, MD (uci)

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

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
    Orange California 92868 United States
  • Barrow Neurological Institute
    Phoenix Arizona 85013 United States

Lead Scientist at University of California Health

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