Pompe Gene Therapy- Screening for Eligibility
|ClinicalTrials.gov Identifier: NCT03285126|
Recruitment Status : Completed
First Posted : September 15, 2017
Last Update Posted : July 2, 2019
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.
|Condition or disease|
|Pompe Disease (Late-onset)|
|Study Type :||Observational|
|Actual Enrollment :||19 participants|
|Official Title:||Screening for Eligibility to Participate in a Clinical Trial of Gene Therapy for Late-onset Pompe Disease|
|Actual Study Start Date :||November 1, 2017|
|Actual Primary Completion Date :||June 29, 2019|
|Actual Study Completion Date :||June 29, 2019|
- 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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03285126
|United States, North Carolina|
|Duke University Medical Center|
|Durham, North Carolina, United States, 27701|
|Principal Investigator:||Dwight Koeberl||Duke University|