Status:
COMPLETED
Pompe Gene Therapy- Screening for Eligibility
Lead Sponsor:
Duke University
Conditions:
Pompe Disease (Late-onset)
Eligibility:
All Genders
18+ years
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 patien...
Eligibility Criteria
Inclusion
- Inclusion Criteria
- Diagnosis of Pompe disease by blood or skin fibroblast GAA assay and two pathogenic variants in the GAA gene.
- Age: at least 18 years at enrollment.
- Subjects are capable of giving written consent.
- Receiving ERT at a stable dose for at least 104 weeks.
- Exclusion Criteria
- Any condition that would interfere with participation in the study as determined by the principal investigator.
- Pregnancy or nursing mothers.
- History of active hepatitis B, hepatitis C, or cirrhosis
- Receiving any investigational agent.
Exclusion
Key Trial Info
Start Date :
November 1 2017
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
June 29 2019
Estimated Enrollment :
19 Patients enrolled
Trial Details
Trial ID
NCT03285126
Start Date
November 1 2017
End Date
June 29 2019
Last Update
July 2 2019
Active Locations (1)
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1
Duke University Medical Center
Durham, North Carolina, United States, 27701