Pompe Gene Therapy- Screening for Eligibility

Completed

• Conditions: Pompe Disease (Late-onset)

• Registration Number: NCT03285126
• Lead Sponsor: Duke University

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2017-09-13
• Study First Submitted QC: 2017-09-13
• Study First Posted: 2017-09-15
• Study Start: 2017-11-01
• Status Verified: 2019-06
• Primary Completion: 2019-06-29
• Study Completion: 2019-06-29
• Last Update Submitted: 2019-07-01
• Last Update Posted: 2019-07-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 19

Inclusion Criteria

Not provided

Read More

Exclusion Criteria

Not provided

Read More

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Eligibility for gene therapy.	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.

Trial Locations

Locations (1)

Duke University Medical Center; 🇺🇸 Durham, North Carolina, United States