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NCT03285126 · ClinicalTrials.gov registry record
Pompe Gene Therapy- Screening for Eligibility
A clinical trial, sponsored by Duke University.
- Completed
- Registry status
- 19
- Enrollment target
NCT03285126: Completed study, sponsored by Duke University.
NCT03285126 is a clinical trial that has completed, run by Duke University. The registered enrollment target is 19 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03285126 has completed, sponsored by Duke University.
- COMPLETED
- Registry status
- 19 participants
- Enrollment target
Study 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.
Primary Outcome
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 Details
| Field | Value |
|---|---|
| Enrollment Target | 19 participants |
| Start Date | 2017-11-01 |
| Est. Completion | 2019-06-29 |
What the finished NCT03285126 record still lists
NCT03285126 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 19 participants, a relatively small participant target.
The record links to 0 conditions, and to 0 interventions.
NCT03285126 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03285126 about?
NCT03285126 is a clinical study titled "Pompe Gene Therapy- Screening for Eligibility". 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, vi...
What is the current status of trial NCT03285126?
This trial is currently completed. The enrollment target is 19 participants. The study started on 2017-11-01. Estimated completion is 2019-06-29.
Who is sponsoring clinical trial NCT03285126?
This trial is sponsored by Duke University, which has 1,725 total clinical trials registered on ClinicalTrials.gov.
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