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Pompe Gene Therapy- Screening for Eligibility

NCT03285126 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 19

Last updated 2019-07-02

No results posted yet for this study

Summary

The purpose of this study is to determine eligibility for the future clinical trial of gene therapy in adults with late-onset Pompe disease.

This screening protocol will enroll up to 20 adults patients (\>=18 yo) with late-onset Pompe disease. Study assessments include review of medical history, vital signs, physical examination, muscle function testing, lung function testing, blood and urine collection, serum pregnancy test and ECG.

The study results will be collected to perform descriptive statistical analysis and used to determine eligibility for the proposed clinical trial of gene therapy in the future.

Conditions

  • Pompe Disease (Late-onset)

Sponsors & Collaborators

Principal Investigators

  • Dwight Koeberl · Duke University

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-11-01
Primary Completion
2019-06-29
Completion
2019-06-29

Countries

  • United States

Study Locations

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Entities

Companies

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 NCT03285126 on ClinicalTrials.gov