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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: Completed Phase 1 study, sponsored by University of Florida.
NCT02240407 is a Phase 1 study that has completed, run by University of Florida. The registered enrollment target is 2 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (97% lower). According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
Safety will be tested by clinical pathology tests, blood assay for vector genomes, antibodies against GAA and T-cell ELISPOT against GAA and AAV.
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 |
What the finished NCT02240407 record still lists
NCT02240407 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 2 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (97% lower).
The record links to 0 conditions, and to 5 interventions - of which Diphenhydramine is the first listed.
NCT02240407 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02240407's enrollment target sits among peer trials
2 2000th of 2000 the lowest of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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