Screening for Eligibility to Participate in a Clinical Trial of Gene Therapy for Late-onset Pompe Disease
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Pompe Disease (Late-onset)
- Sponsor
- Duke University
- Enrollment
- 19
- Locations
- 1
- Primary Endpoint
- Eligibility for gene therapy.
- Status
- Completed
- Last Updated
- 6 years ago
Overview
Brief 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.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Outcomes
Primary Outcomes
Eligibility for gene therapy.
Time Frame: Up to 2 days
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.