Trial Outcomes & Findings for Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease (NCT NCT00976352)
NCT ID: NCT00976352
Last Updated: 2018-09-14
Results Overview
Change in Adeno-associated virus (AAV) antibody level; Change in Alglucosidase alpha (GAA) Antibody level
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
9 participants
Primary outcome timeframe
Change from baseline to 365 post study agent administration.
Results posted on
2018-09-14
Participant Flow
Subjects were recruited from the Pediatric Neuromuscular Disorders Clinic at the University of Florida. Subjects were also self-referred from the ClinicalTrials.gov listing.
All subjects underwent a screening process for eligibility determination as well as for safety evaluations.
Participant milestones
| Measure |
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.
|
|---|---|---|
|
Overall Study
STARTED
|
3
|
10
|
|
Overall Study
COMPLETED
|
3
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
4
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
Baseline characteristics by cohort
| Measure |
rAAV1-CMV-GAA Administration-cohort 1
n=3 Participants
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
n=6 Participants
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
n=9 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
3 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
90 months
n=5 Participants
|
67.3 months
n=7 Participants
|
74.8 months
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
3 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Change from baseline to 365 post study agent administration.Change in Adeno-associated virus (AAV) antibody level; Change in Alglucosidase alpha (GAA) Antibody level
Outcome measures
| Measure |
rAAV1-CMV-GAA Administration-cohort 1
n=3 Participants
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
n=6 Participants
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.
|
|---|---|---|
|
Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration.
AAV1antibody Level -Screening
|
40,031 mU/mL
Standard Deviation 65324.18978
|
29,638 mU/mL
Standard Deviation 12395.02473
|
|
Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration.
AAV1antibody Level - Day 365
|
5,509,882 mU/mL
Standard Deviation 3051265.999
|
1,907,161 mU/mL
Standard Deviation 3,189,557
|
|
Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration.
GAA Antibody level - Screening
|
415.6666667 mU/mL
Standard Deviation 61.71169527
|
330.4 mU/mL
Standard Deviation 271.1112318
|
|
Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration.
GAA Antibody level - Day 365
|
504.3333333 mU/mL
Standard Deviation 63.88531391
|
494.3333333 mU/mL
Standard Deviation 371.6481311
|
SECONDARY outcome
Timeframe: Baseline and 365 post study agent administrationMedian (range) Maximal Inspiratory Pressure (MIP), in cm H2O
Outcome measures
| Measure |
rAAV1-CMV-GAA Administration-cohort 1
n=3 Participants
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
n=6 Participants
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.
|
|---|---|---|
|
Maximal Inspiratory Pressure
Day 365
|
6 cm H2O
Interval 2.0 to 19.0
|
56 cm H2O
Interval 3.0 to 75.0
|
|
Maximal Inspiratory Pressure
Baseline
|
6 cm H2O
Interval 2.0 to 22.0
|
61 cm H2O
Interval 3.0 to 85.0
|
SECONDARY outcome
Timeframe: 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.
Outcome measures
| Measure |
rAAV1-CMV-GAA Administration-cohort 1
n=3 Participants
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
n=6 Participants
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.
|
|---|---|---|
|
Evaluation of Ventilatory Performance Benefit of rAAV1-CMV-GAA Gene Transfer and Respiratory Muscle Strength Training (RMST) Compared to RMST Alone.
Screening
|
6.85 cm H2O
Interval 1.6 to 26.1
|
60.8 cm H2O
Interval 1.9 to 82.6
|
|
Evaluation of Ventilatory Performance Benefit of rAAV1-CMV-GAA Gene Transfer and Respiratory Muscle Strength Training (RMST) Compared to RMST Alone.
Baseline
|
5.85 cm H2O
Interval 1.55 to 22.85
|
60.8 cm H2O
Interval 2.65 to 84.85
|
|
Evaluation of Ventilatory Performance Benefit of rAAV1-CMV-GAA Gene Transfer and Respiratory Muscle Strength Training (RMST) Compared to RMST Alone.
Day 365
|
5.7 cm H2O
Interval 1.65 to 19.1
|
55.6 cm H2O
Interval 2.9 to 75.1
|
SECONDARY outcome
Timeframe: Screening, Baseline, and Day 365 post study agent administrationBest 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.
Outcome measures
| Measure |
rAAV1-CMV-GAA Administration-cohort 1
n=3 Participants
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
n=6 Participants
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.
|
|---|---|---|
|
Evaluation of Tidal Volume Benefit of rAAV1-CMV-GAA Gene Transfer and Respiratory Muscle Strength Training, Compared to Respiratory Muscle Strength Training Alone.
Screening
|
2.8 mL/kg
Interval 0.7 to 4.0
|
7.9 mL/kg
Interval 0.14 to 8.7
|
|
Evaluation of Tidal Volume Benefit of rAAV1-CMV-GAA Gene Transfer and Respiratory Muscle Strength Training, Compared to Respiratory Muscle Strength Training Alone.
Baseline
|
2.0 mL/kg
Interval 0.4 to 4.1
|
7.3 mL/kg
Interval 0.16 to 11.7
|
|
Evaluation of Tidal Volume Benefit of rAAV1-CMV-GAA Gene Transfer and Respiratory Muscle Strength Training, Compared to Respiratory Muscle Strength Training Alone.
Day 365
|
3.4 mL/kg
Interval 0.71 to 4.3
|
9.1 mL/kg
Interval 0.18 to 13.3
|
Adverse Events
rAAV1-CMV-GAA Administration-cohort 1
Serious events: 1 serious events
Other events: 3 other events
Deaths: 1 deaths
rAAV1-CMV-GAA Administration-cohort 2
Serious events: 3 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
rAAV1-CMV-GAA Administration-cohort 1
n=3 participants at risk
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
n=9 participants at risk
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.
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Right-sided spontaneous pneumothorax
|
33.3%
1/3 • Number of events 1 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
0.00%
0/9 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Respiratory, thoracic and mediastinal disorders
Bilateral pleural effusion
|
0.00%
0/3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
11.1%
1/9 • Number of events 1 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Cardiac disorders
Bilateral pericardial effusion
|
0.00%
0/3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
11.1%
1/9 • Number of events 1 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Cardiac disorders
Supraventricular tachycardia
|
33.3%
1/3 • Number of events 1 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
0.00%
0/9 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Respiratory, thoracic and mediastinal disorders
Right upper lobe collapse with respiratory distress and Clostridium difficile gastritis
|
0.00%
0/3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
11.1%
1/9 • Number of events 2 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Blood and lymphatic system disorders
Bacteremia accompanied by fever
|
0.00%
0/3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
11.1%
1/9 • Number of events 1 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Respiratory, thoracic and mediastinal disorders
Desaturation Episode
|
0.00%
0/3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
11.1%
1/9 • Number of events 1 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Respiratory, thoracic and mediastinal disorders
Hospitalization: dehydration, increased work of breathing
|
0.00%
0/3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
11.1%
1/9 • Number of events 1 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
General disorders
Hospitalization
|
0.00%
0/3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
33.3%
3/9 • Number of events 3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Respiratory, thoracic and mediastinal disorders
hospitalization, worsening CHF, pulmonary edema
|
0.00%
0/3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
11.1%
1/9 • Number of events 1 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
Other adverse events
| Measure |
rAAV1-CMV-GAA Administration-cohort 1
n=3 participants at risk
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
n=9 participants at risk
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.
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Tract Infection
|
66.7%
2/3 • Number of events 2 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
22.2%
2/9 • Number of events 3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax/Capnothorax
|
100.0%
3/3 • Number of events 5 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
33.3%
3/9 • Number of events 3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Respiratory, thoracic and mediastinal disorders
respiratory symthomps
|
100.0%
3/3 • Number of events 10 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
77.8%
7/9 • Number of events 18 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
0.00%
0/3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
11.1%
1/9 • Number of events 1 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
General disorders
fever, bacteremia, Dehydration
|
100.0%
3/3 • Number of events 4 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
55.6%
5/9 • Number of events 7 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary and chest problems
|
33.3%
1/3 • Number of events 2 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
22.2%
2/9 • Number of events 2 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Gastrointestinal disorders
Reflux and Diarrhea, G-tube problems
|
100.0%
3/3 • Number of events 3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
44.4%
4/9 • Number of events 8 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
General disorders
Pain
|
66.7%
2/3 • Number of events 3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
44.4%
4/9 • Number of events 9 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Skin and subcutaneous tissue disorders
Skin issue
|
66.7%
2/3 • Number of events 5 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
33.3%
3/9 • Number of events 6 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Respiratory, thoracic and mediastinal disorders
Trachestomy
|
0.00%
0/3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
11.1%
1/9 • Number of events 1 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Ear and labyrinth disorders
ear infection and fluids
|
33.3%
1/3 • Number of events 2 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
22.2%
2/9 • Number of events 2 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Eye disorders
eye infection
|
0.00%
0/3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
11.1%
1/9 • Number of events 2 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Musculoskeletal and connective tissue disorders
Contracturs and scoliosis
|
33.3%
1/3 • Number of events 2 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
11.1%
1/9 • Number of events 3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
General disorders
Hypokalemia and Hypoglycemia
|
33.3%
1/3 • Number of events 1 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
11.1%
1/9 • Number of events 2 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Cardiac disorders
Tachycardia
|
33.3%
1/3 • Number of events 2 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
0.00%
0/9 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Renal and urinary disorders
urinary issues
|
33.3%
1/3 • Number of events 1 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
22.2%
2/9 • Number of events 2 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
|
Blood and lymphatic system disorders
Nosebleed
|
0.00%
0/3 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
11.1%
1/9 • Number of events 1 • Data on adverse events were collected on all subjects for 1 year after dosing.
|
Additional Information
Dr. Barry Byrne, Professor and Associate Chair
University of Florida
Phone: 352-273-6563
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place