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NCT02240407 · ClinicalTrials.gov registry record · Phase 1
Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease
A Phase 1 study, sponsored by University of Florida.
- Completed
- Registry status
- Phase 1
- Development phase
- 2
- Enrollment target
NCT02240407 is a Phase 1 study that has completed, run by University of Florida. The registered enrollment target is 2 participants.
The verdict
NCT02240407, a Phase 1 study, has completed, sponsored by University of Florida.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 2 participants
- Enrollment target
Study 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 within-participant, double-blind, randomized, phase I controlled study evaluating the toxicology, biodistribution and potential activity of re-administration of rAAV9-DES-hGAA injected intramuscularly into the TA. Nine participants (18 to 50-years old) who reside within the United States with Late-Onset Pompe Disease (LOPD) will be included. The goal of the immune modulation strategy is to ablate B-cells (Rituximab and Sirolimus) prior to the initial exposure to the study agent in one leg and the subsequent exposure of the same vector to the contralateral leg after four months. At each study agent dosing, the contralateral leg will receive excipient. Patients will act as their own controls. Repeated measures, at baseline and during the following 3 months after each injection, will assess the safety, biochemical and functional impact of the vector.
Interventions
- DRUG Diphenhydramine
- DRUG Rapamycin
- OTHER saline
- DRUG Rituxan
- GENETIC Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 2 participants |
| Start Date | 2017-10-17 |
| Est. Completion | 2021-08-26 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02240407
The ClinicalTrials.gov registry entry for NCT02240407 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 2 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is University of Florida, which has 1,106 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 5 interventions - of which Diphenhydramine is the first listed.
NCT02240407 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT02240407 about?
NCT02240407 is a clinical study titled "Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease". 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 within-participant, double-blind, randomized, phase I controlled study evaluating the toxicology, biodi...
What is the current status of trial NCT02240407?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 2 participants. The study started on 2017-10-17. Estimated completion is 2021-08-26.
What interventions are being tested in trial NCT02240407?
The interventions under investigation include: Diphenhydramine (DRUG), Rapamycin (DRUG), saline (OTHER), Rituxan (DRUG), Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (GENETIC).
Who is sponsoring clinical trial NCT02240407?
This trial is sponsored by University of Florida, which has 1,106 total clinical trials registered on ClinicalTrials.gov.
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