Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease

ClinicalTrials.gov ID: NCT00976352

Public ClinicalTrials.gov record NCT00976352. 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.

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ClinicalTrials.gov public records Last synced Jul 21, 2026, 1:42 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients With Pompe Disease

Study identification

NCT ID: NCT00976352
Recruitment status: Completed
Study type: Interventional
Phase: Phase 1, Phase 2
Lead sponsor: University of Florida; Other
Enrollment: 9 participants

Conditions and interventions

Conditions

Pompe Disease

Interventions

rAAV1-CMV-GAA (study agent) Administration Drug
RMST Other

Drug · Other

Eligibility (public fields only)

Age range: 2 Years to 18 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: Aug 31, 2010
Primary completion: Nov 30, 2015
Completion: Nov 30, 2015
Last update posted: Sep 13, 2018

2010 – 2015

United States locations

U.S. sites: 1
U.S. states: 1
U.S. cities: 1

Facility	City	State	ZIP	Site status
Shands at the 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 NCT00976352, 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 Sep 13, 2018 · Synced Jul 21, 2026

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The complete protocol, eligibility criteria, and contact information for NCT00976352 live on ClinicalTrials.gov.

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