Trial · NCT00976352
Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
NCT00976352, Phase 1, Phase 2, Completed, University of Florida; results first posted 2017-11-17
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease. Trials, record NCT00976352. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00976352
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
- NCT number
- NCT00976352
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 9 participants, actual
- Lead sponsor
- University of Florida
- Conditions
- Pompe Disease
- Interventions
- rAAV1-CMV-GAA (study agent) Administration, RMST
- First posted
- 2009-09-14
- Last updated
- 2018-09-14
- Results first posted
- 2017-11-17
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration. | Change from baseline to 365 post study agent administration. | Change in Adeno-associated virus (AAV) antibody level; Change in Alglucosidase alpha (GAA) Antibody level |
| Secondary | Maximal Inspiratory Pressure | Baseline and 365 post study agent administration | Median (range) Maximal Inspiratory Pressure (MIP), in cm H2O |
| Secondary | Evaluation of Ventilatory Performance Benefit of rAAV1-CMV-GAA Gene Transfer and Respiratory Muscle Strength Training (RMST) Compared to RMST Alone. | Screening, Baseline, and 365 post study agent administration. | Median (range) maximal inspiratory pressure, in cm H2O. Timeframe for RMST training was 90 days prior to rAAV1-CMV-GAA gene transfer. Timeframe for following subjects after rAAV1-CMV-GAA gene transfer was 365 days. |
| Secondary | Evaluation of Tidal Volume Benefit of rAAV1-CMV-GAA Gene Transfer and Respiratory Muscle Strength Training, Compared to Respiratory Muscle Strength Training Alone. | Screening, Baseline, and Day 365 post study agent administration | Best effort tidal volume, referenced to body mass, without use of ventilator assistance. Timeframe for respiratory muscle strength training alone was 90 days prior to dosing, timeframe post adminstration of rAAV1-CMV-GAA was 365 days. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration. | Change from baseline to 365 post study agent administration. | mU/mL | MEAN | |
| SECONDARY | Maximal Inspiratory Pressure | Baseline and 365 post study agent administration | cm H2O | MEDIAN | |
| SECONDARY | Evaluation of Ventilatory Performance Benefit of rAAV1-CMV-GAA Gene Transfer and Respiratory Muscle Strength Training (RMST) Compared to RMST Alone. | Screening, Baseline, and 365 post study agent administration. | cm H2O | MEDIAN | |
| SECONDARY | Evaluation of Tidal Volume Benefit of rAAV1-CMV-GAA Gene Transfer and Respiratory Muscle Strength Training, Compared to Respiratory Muscle Strength Training Alone. | Screening, Baseline, and Day 365 post study agent administration | mL/kg | MEDIAN |
Source ClinicalTrials.gov
Results by group24
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration. | rAAV1-CMV-GAA Administration-cohort 1 | AAV1antibody Level -Screening | 40,031 | Standard Deviation 65324.18978 | mU/mL |
| Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration. | rAAV1-CMV-GAA Administration-cohort 2 | AAV1antibody Level -Screening | 29,638 | Standard Deviation 12395.02473 | mU/mL |
| Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration. | rAAV1-CMV-GAA Administration-cohort 1 | AAV1antibody Level - Day 365 | 5,509,882 | Standard Deviation 3051265.999 | mU/mL |
| Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration. | rAAV1-CMV-GAA Administration-cohort 2 | AAV1antibody Level - Day 365 | 1,907,161 | Standard Deviation 3,189,557 | mU/mL |
| Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration. | rAAV1-CMV-GAA Administration-cohort 1 | GAA Antibody level - Screening | 415.6666667 | Standard Deviation 61.71169527 | mU/mL |
| Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration. | rAAV1-CMV-GAA Administration-cohort 2 | GAA Antibody level - Screening | 330.4 | Standard Deviation 271.1112318 | mU/mL |
| Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration. | rAAV1-CMV-GAA Administration-cohort 1 | GAA Antibody level - Day 365 | 504.3333333 | Standard Deviation 63.88531391 | mU/mL |
| Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration. | rAAV1-CMV-GAA Administration-cohort 2 | GAA Antibody level - Day 365 | 494.3333333 | Standard Deviation 371.6481311 | mU/mL |
| Maximal Inspiratory Pressure | rAAV1-CMV-GAA Administration-cohort 1 | Baseline | 6 | Full Range 2 to 22 | cm H2O |
| Maximal Inspiratory Pressure | rAAV1-CMV-GAA Administration-cohort 2 | Baseline | 61 | Full Range 3 to 85 | cm H2O |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | rAAV1-CMV-GAA Administration-cohort 1 | 3 | |
| Overall Study | STARTED | rAAV1-CMV-GAA Administration-cohort 2 | 10 | |
| Overall Study | COMPLETED | rAAV1-CMV-GAA Administration-cohort 1 | 3 | |
| Overall Study | COMPLETED | rAAV1-CMV-GAA Administration-cohort 2 | 6 | |
| Overall Study | NOT COMPLETED | rAAV1-CMV-GAA Administration-cohort 1 | 0 | |
| Overall Study | NOT COMPLETED | rAAV1-CMV-GAA Administration-cohort 2 | 4 |
Source ClinicalTrials.gov
Adverse events54
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Respiratory Tract Infection | Respiratory, thoracic and mediastinal disorders | rAAV1-CMV-GAA Administration-cohort 1 | 2 | 3 | 2 |
| other | Respiratory Tract Infection | Respiratory, thoracic and mediastinal disorders | rAAV1-CMV-GAA Administration-cohort 2 | 2 | 9 | 3 |
| other | Pneumothorax/Capnothorax | Respiratory, thoracic and mediastinal disorders | rAAV1-CMV-GAA Administration-cohort 1 | 3 | 3 | 5 |
| other | Pneumothorax/Capnothorax | Respiratory, thoracic and mediastinal disorders | rAAV1-CMV-GAA Administration-cohort 2 | 3 | 9 | 3 |
| other | respiratory symthomps | Respiratory, thoracic and mediastinal disorders | rAAV1-CMV-GAA Administration-cohort 1 | 3 | 3 | 10 |
| other | respiratory symthomps | Respiratory, thoracic and mediastinal disorders | rAAV1-CMV-GAA Administration-cohort 2 | 7 | 9 | 18 |
| other | Pneumonia | Respiratory, thoracic and mediastinal disorders | rAAV1-CMV-GAA Administration-cohort 1 | 0 | 3 | 0 |
| other | Pneumonia | Respiratory, thoracic and mediastinal disorders | rAAV1-CMV-GAA Administration-cohort 2 | 1 | 9 | 1 |
| other | fever, bacteremia, Dehydration | General disorders | rAAV1-CMV-GAA Administration-cohort 1 | 3 | 3 | 4 |
| other | fever, bacteremia, Dehydration | General disorders | rAAV1-CMV-GAA Administration-cohort 2 | 5 | 9 | 7 |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | rAAV1-CMV-GAA Administration-cohort 1 | 1 | 3 |
| Total, all-cause mortality | rAAV1-CMV-GAA Administration-cohort 2 | 0 | 9 |
| Total, other adverse events | rAAV1-CMV-GAA Administration-cohort 1 | 3 | 3 |
| Total, other adverse events | rAAV1-CMV-GAA Administration-cohort 2 | 9 | 9 |
| Total, serious adverse events | rAAV1-CMV-GAA Administration-cohort 1 | 1 | 3 |
| Total, serious adverse events | rAAV1-CMV-GAA Administration-cohort 2 | 3 | 9 |
Source ClinicalTrials.gov
Papers9
The papers the trial lists on ClinicalTrials.gov.
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| P01HL059412 | Gene Therapy for Lung and Cardiovascular Disease | MUZYCZKA, NICHOLAS | UNIVERSITY OF FLORIDA | NATIONAL HEART, LUNG, AND BLOOD INSTITUTE | 1998 to 2012 |
Source NIH RePORTER
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| rAAV1-CMV-GAA Administration-cohort 1 vs rAAV1-CMV-GAA Administration-cohort 2: deaths from any cause | Trial comparisons |
| rAAV1-CMV-GAA Administration-cohort 1 vs rAAV1-CMV-GAA Administration-cohort 2: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00976352
GET https://conductscience.org/api/trials/v1/NCT00976352/results?page=2