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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00976352
First Posted: September 14, 2009
Last Update Posted: November 17, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
University of Florida
Results First Submitted: June 1, 2017  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Sequential Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Pompe Disease
Interventions: Drug: rAAV1-CMV-GAA (study agent) Administration
Other: RMST

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Subjects were recruited from the Pediatric Neuromuscular Disorders Clinic at the University of Florida. Subjects were also self-referred from the ClinicalTrials.gov listing.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
All subjects underwent a screening process for eligibility determination as well as for safety evaluations.

Reporting Groups
  Description
rAAV1-CMV-GAA Administration-cohort 1

rAAV1-CMV-GAA (study agent) Administration: 1.0 x 10e12 vector genomes. The following study assessments/interventions will be completed: Respiratory Muscle Strength Training (RMST), Safety labs, pulmonary function testing.

rAAV1-CMV-GAA (study agent) Administration: rAAV1-CMV-GAA via intramuscular injection into the diaphragm. Dose selection for cohort 1: 1.0 x 10e12 vector genomes Cohort 1 will have a total of 3 participants enrolled. Dose selection for cohort 2 and 3: 5.0 x 10e12 vector genomes rAAV1-CMV-GAA. Cohort 2 = 6 subjects.

RMST: After enrollment and screening visit, the subject will be given a RMST prescription and will complete RMST for a minimum of 4 weeks prior to study agent administration. RMST prescription will be adjusted as needed at Day 14, 90, 180, and 270.

rAAV1-CMV-GAA Administration-cohort 2

rAAV1-CMV-GAA (study agent) Administration: 5.0 x 10e12 vector genomes Cohort 2 = 6 subjects. The following study assessments/interventions will be completed: Respiratory Muscle Strength Training (RMST), Safety labs, pulmonary function testing.

rAAV1-CMV-GAA (study agent) Administration: rAAV1-CMV-GAA via intramuscular injection into the diaphragm. Dose selection for cohort 1: 1.0 x 10e12 vector genomes Cohort 1 will have a total of 3 participants enrolled. Dose selection for cohort 2 and 3: 5.0 x 10e12 vector genomes rAAV1-CMV-GAA. Cohort 2 = 6 subjects.

RMST: After enrollment and screening visit, the subject will be given a RMST prescription and will complete RMST for a minimum of 4 weeks prior to study agent administration. RMST prescription will be adjusted as needed at Day 14, 90, 180, and 270.


Participant Flow:   Overall Study
    rAAV1-CMV-GAA Administration-cohort 1   rAAV1-CMV-GAA Administration-cohort 2
STARTED   3   10 
COMPLETED   3   6 [1] 
NOT COMPLETED   0   4 
[1] Last evaluation of last subject in cohort.



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
rAAV1-CMV-GAA Administration-cohort 1

rAAV1-CMV-GAA (study agent) Administration: 1.0 x 10e12 vector genomes. The following study assessments/interventions will be completed: Respiratory Muscle Strength Training (RMST), Safety labs, pulmonary function testing.

rAAV1-CMV-GAA (study agent) Administration: rAAV1-CMV-GAA via intramuscular injection into the diaphragm. Dose selection for cohort 1: 1.0 x 10e12 vector genomes Cohort 1 will have a total of 3 participants enrolled. Dose selection for cohort 2 and 3: 5.0 x 10e12 vector genomes rAAV1-CMV-GAA. Cohort 2 = 6 subjects.

RMST: After enrollment and screening visit, the subject will be given a RMST prescription and will complete RMST for a minimum of 4 weeks prior to study agent administration. RMST prescription will be adjusted as needed at Day 14, 90, 180, and 270.

rAAV1-CMV-GAA Administration-cohort 2

rAAV1-CMV-GAA (study agent) Administration: 5.0 x 10e12 vector genomes Cohort 2 = 6 subjects. The following study assessments/interventions will be completed: Respiratory Muscle Strength Training (RMST), Safety labs, pulmonary function testing.

rAAV1-CMV-GAA (study agent) Administration: rAAV1-CMV-GAA via intramuscular injection into the diaphragm. Dose selection for cohort 1: 1.0 x 10e12 vector genomes Cohort 1 will have a total of 3 participants enrolled. Dose selection for cohort 2 and 3: 5.0 x 10e12 vector genomes rAAV1-CMV-GAA. Cohort 2 = 6 subjects.

RMST: After enrollment and screening visit, the subject will be given a RMST prescription and will complete RMST for a minimum of 4 weeks prior to study agent administration. RMST prescription will be adjusted as needed at Day 14, 90, 180, and 270.

Total Total of all reporting groups

Baseline Measures
   rAAV1-CMV-GAA Administration-cohort 1   rAAV1-CMV-GAA Administration-cohort 2   Total 
Overall Participants Analyzed 
[Units: Participants]
 3   6   9 
Age 
[Units: Participants]
Count of Participants
     
<=18 years      3 100.0%      6 100.0%      9 100.0% 
Between 18 and 65 years      0   0.0%      0   0.0%      0   0.0% 
>=65 years      0   0.0%      0   0.0%      0   0.0% 
Age 
[Units: Months]
Mean (Full Range)
 90 
 (66 to 108) 
 67.3 
 (30 to 180) 
 74.8 
 (30 to 180) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      0   0.0%      3  50.0%      3  33.3% 
Male      3 100.0%      3  50.0%      6  66.7% 
Region of Enrollment 
[Units: Participants]
Count of Participants
     
United States   3   6   9 


  Outcome Measures
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1.  Primary:   Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration.   [ Time Frame: Change from baseline to 365 post study agent administration. ]

2.  Secondary:   Maximal Inspiratory Pressure   [ Time Frame: Baseline and 365 post study agent administration ]

3.  Secondary:   Evaluation of Ventilatory Performance Benefit of rAAV1-CMV-GAA Gene Transfer and Respiratory Muscle Strength Training (RMST) Compared to RMST Alone.   [ Time Frame: Screening, Baseline, and 365 post study agent administration. ]

4.  Secondary:   Evaluation of Tidal Volume Benefit of rAAV1-CMV-GAA Gene Transfer and Respiratory Muscle Strength Training, Compared to Respiratory Muscle Strength Training Alone.   [ Time Frame: Screening, Baseline, and Day 365 post study agent administration ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Dr. Barry Byrne, Professor and Associate Chair
Organization: University of Florida
phone: 352-273-6563
e-mail: bbyrne@ufl.edu


Publications of Results:
Other Publications:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: University of Florida
ClinicalTrials.gov Identifier: NCT00976352     History of Changes
Other Study ID Numbers: PGTC PD-AAV004
P01HL059412-06 ( U.S. NIH Grant/Contract )
First Submitted: July 13, 2009
First Posted: September 14, 2009
Results First Submitted: June 1, 2017
Results First Posted: November 17, 2017
Last Update Posted: November 17, 2017