Trial Outcomes & Findings for Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency (NCT NCT02168686)
NCT ID: NCT02168686
Last Updated: 2023-10-05
Results Overview
Number and proportion of subjects experiencing treatment-related adverse events related to ADVM-043
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
6 participants
Primary outcome timeframe
From ADVM-043 infusion through End-of-Study visit at 52 weeks
Results posted on
2023-10-05
Participant Flow
Participant milestones
| Measure |
Part A: Dose 1
Single IV infusion of ADVM-043 at 8E13 total vg
|
Part A: Dose 2
Single IV infusion of ADVM-043 at 4E14 total vg
|
Part A: Dose 3
Single IV infusion of ADVM-043 at 1.2E15 total vg
|
|---|---|---|---|
|
Overall Study
STARTED
|
2
|
2
|
2
|
|
Overall Study
COMPLETED
|
2
|
2
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency
Baseline characteristics by cohort
| Measure |
Part A: Dose 1
n=2 Participants
Single IV infusion of ADVM-043 at 8E13 total vg
|
Part A: Dose 2
n=2 Participants
Single IV infusion of ADVM-043 at 4E14 total vg
|
Part A: Dose 3
n=2 Participants
Single IV infusion of ADVM-043 at 1.2E15 total vg
|
Total
n=6 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=93 Participants
|
2 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
5 Participants
n=483 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
1 Participants
n=483 Participants
|
|
Age, Continuous
|
40.5 years
n=93 Participants
|
53.5 years
n=4 Participants
|
61.0 years
n=27 Participants
|
51.7 years
n=483 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
3 Participants
n=483 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
3 Participants
n=483 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=93 Participants
|
2 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
6 Participants
n=483 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Region of Enrollment
United States
|
2 participants
n=93 Participants
|
2 participants
n=4 Participants
|
2 participants
n=27 Participants
|
6 participants
n=483 Participants
|
PRIMARY outcome
Timeframe: From ADVM-043 infusion through End-of-Study visit at 52 weeksNumber and proportion of subjects experiencing treatment-related adverse events related to ADVM-043
Outcome measures
| Measure |
Part A: Dose 1
n=2 Participants
Single IV infusion of ADVM-043 at 8E13 total vg
|
Part A: Dose 2
n=2 Participants
Single IV infusion of ADVM-043 at 4E14 total vg
|
Part A: Dose 3
n=2 Participants
Single IV infusion of ADVM-043 at 1.2E15 total vg
|
Total
All Participants
|
|---|---|---|---|---|
|
Treatment-emergent Adverse Events Related to ADVM-043
|
0 Participants
|
1 Participants
|
2 Participants
|
—
|
PRIMARY outcome
Timeframe: From ADVM-043 infusion through End-of-Study visit at 52 weeksNumber of participants with ≥1 abnormal shift from Baseline in neutrophil count, hemoglobin, important serum chemistry parameters
Outcome measures
| Measure |
Part A: Dose 1
n=2 Participants
Single IV infusion of ADVM-043 at 8E13 total vg
|
Part A: Dose 2
n=2 Participants
Single IV infusion of ADVM-043 at 4E14 total vg
|
Part A: Dose 3
n=2 Participants
Single IV infusion of ADVM-043 at 1.2E15 total vg
|
Total
n=6 Participants
All Participants
|
|---|---|---|---|---|
|
Abnormal Changes in Clinical Laboratory Parameters
Alanine Transaminase Shifts to High (max >2.0 to ≤3.0×upper limit of normal
|
0 participants
|
1 participants
|
1 participants
|
2 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Alanine Transaminase Shifts to High (max >3.0×upper limit of normal)
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Serum Glucose Shifts to High
|
2 participants
|
1 participants
|
1 participants
|
4 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Neutrophil Count Shifts to High
|
2 participants
|
1 participants
|
2 participants
|
5 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Neutrophil Count Shifts to Low
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Hemoglobin Shifts to High
|
0 participants
|
1 participants
|
1 participants
|
2 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Hemoglobin Shifts to Low
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Alanine Transaminase Shifts to High (max >1.0 to ≤2.0×upper limit of normal)
|
1 participants
|
1 participants
|
1 participants
|
3 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Aspartate Transaminase Shifts to High (max >1.0 to ≤2.0×upper limit of normal)
|
0 participants
|
1 participants
|
1 participants
|
2 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Aspartate Transaminase Shifts to High (max >2.0 to ≤3.0×upper limit of normal)
|
0 participants
|
0 participants
|
1 participants
|
1 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Aspartate Transaminase Shifts to High (max >3.0×upper limit of normal)
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Alkaline Phosphatase Shifts to High
|
0 participants
|
0 participants
|
1 participants
|
1 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Creatinine Shifts to High
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Creatinine Shifts to Low
|
0 participants
|
0 participants
|
1 participants
|
1 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Creatine Kinase Shifts to High
|
1 participants
|
0 participants
|
1 participants
|
2 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Creatine Kinase Shifts to Low
|
1 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Albumin Shifts to High
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Albumin Shifts to Low
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Direct Bilirubin Shifts to High
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Direct Bilirubin Shifts to Low
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Changes in Clinical Laboratory Parameters
Serum Glucose Shifts to Low
|
1 participants
|
0 participants
|
2 participants
|
3 participants
|
SECONDARY outcome
Timeframe: At Week 52Population: Not analyzed per the statistical analysis plan because participants initiated PAT therapy
Change from baseline at Week 52 of plasma concentration of M-specific A1AT for subjects who did not receive PAT post-dose Note: 1. Two subjects in Dose 1 Arm/Group, had results available at Week 52; the remaining 4 subjects in Dose 2 Arm/Group and Dose 3 Arm/Group had resumed PAT therapy after Week 24, and their results were censored from the Week 52 timepoint. 2. While data on the Total Plasma Concentrations of A1AT up to 52 Weeks were collected for 1 study participant in Part A: Dose 3, no data were collected on the Change in Plasma Concentrations of M-specific A1AT due to the initiation of PAT after 24 weeks in Part A: Dose 3 subjects.
Outcome measures
| Measure |
Part A: Dose 1
n=2 Participants
Single IV infusion of ADVM-043 at 8E13 total vg
|
Part A: Dose 2
Single IV infusion of ADVM-043 at 4E14 total vg
|
Part A: Dose 3
Single IV infusion of ADVM-043 at 1.2E15 total vg
|
Total
n=2 Participants
All Participants
|
|---|---|---|---|---|
|
Change in Plasma Concentrations of M-specific A1AT up to 52 Weeks
|
88.050 uM
Standard Deviation 19.955
|
—
|
—
|
88.050 uM
Standard Deviation 19.955
|
SECONDARY outcome
Timeframe: At Week 52Population: Not analyzed per the statistical analysis plan because participant initiated PAT therapy
Change from baseline at Week 24 and Week 52 of total A1At plasma concentration for subjects who did not receive PAT post -dose
Outcome measures
| Measure |
Part A: Dose 1
n=2 Participants
Single IV infusion of ADVM-043 at 8E13 total vg
|
Part A: Dose 2
n=2 Participants
Single IV infusion of ADVM-043 at 4E14 total vg
|
Part A: Dose 3
n=2 Participants
Single IV infusion of ADVM-043 at 1.2E15 total vg
|
Total
n=6 Participants
All Participants
|
|---|---|---|---|---|
|
Changes in Total Plasma Concentrations of A1AT up to 52 Weeks
Mean change from Baseline at Week 24 serum Total Protein
|
1.230 uM
Interval -0.08 to 2.54
|
1.870 uM
Interval 0.14 to 3.6
|
1.145 uM
Interval 0.2 to 2.09
|
1.415 uM
Interval -0.08 to 3.6
|
|
Changes in Total Plasma Concentrations of A1AT up to 52 Weeks
Mean change from Baseline at Week 52 serum Total Protein
|
1.220 uM
Interval -0.44 to 2.88
|
—
|
0.640 uM
Interval 0.64 to 0.64
|
1.027 uM
Interval -0.44 to 2.88
|
Adverse Events
Part A: Dose 1
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Part A: Dose 2
Serious events: 2 serious events
Other events: 2 other events
Deaths: 0 deaths
Part A: Dose 3
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Part A: Dose 1
n=2 participants at risk
Single IV infusion of ADVM-043 at 8E13 total vg
|
Part A: Dose 2
n=2 participants at risk
Single IV infusion of ADVM-043 at 4E14 total vg
|
Part A: Dose 3
n=2 participants at risk
Single IV infusion of ADVM-043 at 1.2E15 total vg
|
|---|---|---|---|
|
Infections and infestations
Clostridium difficile colitis
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Infections and infestations
Influenza
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Respiratory, thoracic and mediastinal disorders
Chronic obstructive pulmonary disease
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Meningioma
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
Other adverse events
| Measure |
Part A: Dose 1
n=2 participants at risk
Single IV infusion of ADVM-043 at 8E13 total vg
|
Part A: Dose 2
n=2 participants at risk
Single IV infusion of ADVM-043 at 4E14 total vg
|
Part A: Dose 3
n=2 participants at risk
Single IV infusion of ADVM-043 at 1.2E15 total vg
|
|---|---|---|---|
|
Infections and infestations
Bronchitis
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Infections and infestations
Pneumonia
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Infections and infestations
Chronic sinusitis
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Infections and infestations
Tooth abscess
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Infections and infestations
Viral upper respiratory tract infection
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Gastrointestinal disorders
Dysphagia
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Gastrointestinal disorders
Toothache
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Investigations
Transaminases increased
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
100.0%
2/2 • Number of events 3 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Investigations
Cortisol decreased
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Investigations
Crystal urine present
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Respiratory, thoracic and mediastinal disorders
Chronic obstructive pulmonary disease
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
General disorders
Face oedema
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
General disorders
Oedema peripheral
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Musculoskeletal and connective tissue disorders
Bursitis
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Psychiatric disorders
Anxiety disorder
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Psychiatric disorders
Insomnia
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Reproductive system and breast disorders
Benign prostatic hyperplasia
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
|
Vascular disorders
Hypertension
|
50.0%
1/2 • Number of events 1 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
0.00%
0/2 • From ADVM-043 infusion through End-of-Study visit at 52 weeks
Subject incidence of treatment-emergent serious adverse events
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Disclosure restrictions on the PIs are that Sponsor can review communications by the PI prior to public release and can embargo communications regarding trial results for varying time periods from the time submitted to the sponsor for review. The Sponsor may require changes to the communication after review and may delay publication upon request by the Sponsor to allow the Sponsor to seek/obtain patent protection.
- Publication restrictions are in place
Restriction type: OTHER