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

NCT04093349 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2024-11-27

No results posted yet for this study

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.

Conditions

  • Pompe Disease
  • Pompe Disease (Late-onset)
  • Glycogen Storage Disease Type 2
  • Glycogen Storage Disease Type II
  • LOPD
  • Lysosomal Storage Diseases
  • Acid Maltase Deficiency

Interventions

GENETIC

SPK-3006

adeno-associated viral (AAV) vector

Sponsors & Collaborators

  • Spark Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Tahseen Mozaffar, MD · University of California Irvine Health

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-10-01
Primary Completion
2032-04-30
Completion
2032-04-30
FDA Drug
Yes

Countries

  • United States
  • Canada
  • Denmark
  • France
  • Germany
  • Italy
  • Netherlands
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04093349 on ClinicalTrials.gov