An observational study in Pompe Disease (Late-onset), sponsored by Duke University. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2019-07-02.
Sponsored by Duke University · Observational
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.
Adults with late on-set Pompe Disease.
Exclusion Criteria
Eligibility for gene therapy.
The purpose of this study is to determine eligibility for the proposed clinical trial of gene therapy in adults with late-onset Pompe disease. Eligibility is measure by a composite score of blood tests, Electrocardiogram, Pulmonary Function Tests, and Muscle Status Testing.
Time frame: Up to 2 days
This study is completed, as verified in Jun 2019. You cannot join it, but the record below documents what was studied.
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Glycogen Storage Disease Type II→
Duke University