Status:
COMPLETED
Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease
Lead Sponsor:
University of Florida
Collaborating Sponsors:
Lacerta Therapeutics
Conditions:
Pompe Disease
Eligibility:
All Genders
18-50 years
Phase:
PHASE1
Brief Summary
A recombinant AAV vector has been generated to carry the codon-optimized acid alpha-glucosidase (coGAA) gene expressed from a human desmin enhancer/promoter (DES). The proposed clinical trial is a wit...
Detailed Description
Enrolling in this study will entail participating in 18 months of study-related visits. Patients will be asked to come to the Clinical and Translational Research Building at the University of Florida ...
Eligibility Criteria
Inclusion
- Male or female subjects 18 to 50-years old
- Have a diagnosis of Pompe disease, as defined by protein assay AND/OR DNA sequence of the acid alpha-glucosidase gene, AND clinical symptoms of the disease
- Have residual ability to complete the 10 meter walk test
- Willing to discontinue aspirin, aspirin-containing products and other drugs that may alter platelet function, 7 days prior to dosing, resuming 24 hours after the dose has been administered
- Consistently taking enzyme replacement therapy (ERT) or remain off ERT from baseline until Day 520
- United States residents only.
Exclusion
- Be pregnant or nursing, and if the subject is of child bearing potential they should use contraception until the end of the study
- Have required oral or systemic corticosteroids within the last 15 days prior to baseline screening
- Have a platelet count less than 75,000/mm\^3
- Have an INR greater than 1.3
- Have seronegative to AAV9 capsid protein (neutralizing Ab titers \<1:5 and total binding Ab titer \<50 U/ml)
- Have transaminases and alkaline phosphatase more than ten times the upper limit of normal at screening or Day-1
- Have bilirubin and gamma-glutamyl transpeptidase greater than 2 times the upper limit of normal at screening or Day -1
- Have any chronic liver disease (aside from hepatic dysfunction related to Pompe disease) such as hepatitis B and C and cirrhosis
- Be currently, or within the past 30 days, participating in any other research protocol involving investigational agents or therapies
- Have history of platelet dysfunction, evidence of abnormal platelet function at screening, or history of recent use of drugs that may alter platelet function, which the subject is unable/unwilling to discontinue for study agent administration
- Have received gene transfer agents within the past 6 months
- Have any medical condition or circumstance for which an MRI evaluation is contraindicated
- Have any other concurrent condition that, in the opinion of the investigator, would make the subject unsuitable for the study
- Inconsistent with use of ERT.
Key Trial Info
Start Date :
October 17 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 26 2021
Estimated Enrollment :
2 Patients enrolled
Trial Details
Trial ID
NCT02240407
Start Date
October 17 2017
End Date
August 26 2021
Last Update
April 5 2022
Active Locations (1)
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1
Clinical and Translational Research Building (CTRB), University of Florida
Gainesville, Florida, United States, 32610