NCT03285126

Pompe Gene Therapy- Screening for Eligibility

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

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 19 (actual)
• Sponsor: Duke University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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)

Timeline

• Start date: 2017-11-01
• Primary completion: 2019-06-29
• Completion: 2019-06-29
• First posted: 2017-09-15
• Last updated: 2019-07-02

Locations

1 site across 1 country: United States