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Pompe Gene Therapy- Screening for Eligibility

Pompe Disease (Late-onset) Clinical Trial

Official title:
Screening for Eligibility to Participate in a Clinical Trial of Gene Therapy for Late-onset Pompe Disease

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03285126
Study type Observational
Source Duke University
Contact
Status Completed
Phase
Start date November 1, 2017
Completion date June 29, 2019
View Details
See also
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