Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease
Public ClinicalTrials.gov record NCT02240407. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Evaluation of Re-administration of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV9-DES-hGAA) in Patients With Late-Onset Pompe Disease (LOPD)
Study identification
- NCT ID
- NCT02240407
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 1
- Lead sponsor
- University of Florida
- Other
- Enrollment
- 2 participants
Conditions and interventions
Conditions
Interventions
- Acetaminophen Drug
- Diphenhydramine Drug
- LMX 4 Topical Cream Drug
- Lidocaine Drug
- Rapamycin Drug
- Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase Genetic
- Rituxan Drug
- saline Other
Drug · Genetic · Other
Eligibility (public fields only)
- Age range
- 18 Years to 50 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 16, 2017
- Primary completion
- Aug 25, 2021
- Completion
- Aug 25, 2021
- Last update posted
- Apr 4, 2022
2017 – 2021
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Clinical and Translational Research Building (CTRB), University of Florida | Gainesville | Florida | 32610 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02240407, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 4, 2022 · Synced May 20, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT02240407 live on ClinicalTrials.gov.