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Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

NCT03893240 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 61

Last updated 2023-03-24

No results posted yet for this study

Summary

The purpose of this study is to obtain information pertaining to the occurrence of antibodies to investigational SPK-3006 capsid and GAA, GAA activity and GAA antigen levels in the usual care setting of Late-Onset Pompe Disease (LOPD) participants on an enzyme replacement regimen. Additionally, a careful evaluation of laboratory and functional testing in patients with LOPD may provide information to better understand the disease features and better drive the design of a future interventional investigational gene therapy trial. An understanding of the underlying status of liver and muscle health in individuals with LOPD may also inform best surveillance during the conduct of gene therapy trials.

Conditions

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

Interventions

DIAGNOSTIC_TEST

Neutralizing Antibody to SPK-3006 capsid

Collected during a single study visit to establish the occurrence of neutralizing antibodies to SPK-3006 capsid in participants with LOPD on an enzyme replacement regimen.

Sponsors & Collaborators

  • Spark Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Tahseen Mozaffar, MD · University of California Irvine Health

Study Design

Allocation
NA
Purpose
SCREENING
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-06-12
Primary Completion
2020-10-27
Completion
2020-10-27

Countries

  • United States
  • 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 NCT03893240 on ClinicalTrials.gov