Trial Outcomes & Findings for Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 (NCT NCT02122952)
NCT ID: NCT02122952
Last Updated: 2022-09-15
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
15 participants
Primary outcome timeframe
2 years
Results posted on
2022-09-15
Participant Flow
Recruitment period May 2014 - Dec 2015
30 day screening period prior to enrollment and dosing.
Participant milestones
| Measure |
Cohort 1
6.7 X 10\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3)
AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
|
Cohort 2
2.0 X 10\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12)
AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
|
|---|---|---|
|
Overall Study
STARTED
|
3
|
12
|
|
Overall Study
COMPLETED
|
3
|
12
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
Baseline characteristics by cohort
| Measure |
Cohort 1
n=3 Participants
6.7 X 10\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3)
AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
|
Cohort 2
n=12 Participants
2.0 X 10\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12)
AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
|
Total
n=15 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
3 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
15 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
|
6.33 months
STANDARD_DEVIATION 0.751 • n=5 Participants
|
3.42 months
STANDARD_DEVIATION 2.063 • n=7 Participants
|
4.00 months
STANDARD_DEVIATION 2.209 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Hispanic or Latino
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
12 participants
n=7 Participants
|
15 participants
n=5 Participants
|
|
SMN2 copy number = 2
|
3 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
bi-allelic deletions of SMN1
|
3 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Exon 7 gene modifier mutation
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 yearsOutcome measures
| Measure |
Cohort 1
n=3 Participants
6.7 X 10\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3)
AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
|
Cohort 2
n=12 Participants
2.0 X 10\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12)
AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
|
|---|---|---|
|
Number of Participants That Experienced One Grade III or Higher Unanticipated, Treatment-related Toxicity That Presents With Clinical Symptoms and Requires Medical Treatment
|
1 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: Up to 13.6 months of agePermanent ventilation was defined as the requirement of ≥ 16-hour respiratory assistance, including non-invasive ventilatory support, per day continuously for ≥ 2 weeks in the absence of an acute reversible illness, excluding perioperative ventilation.
Outcome measures
| Measure |
Cohort 1
n=3 Participants
6.7 X 10\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3)
AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
|
Cohort 2
n=12 Participants
2.0 X 10\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12)
AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
|
|---|---|---|
|
Number of Participants Who Experienced Permanent Ventilation or Death
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline to 24 months post-dosePopulation: Includes all treated participants with non-missing data. For a major, systemic, and externally imposed limitation of movement that prevented accurate assessment of multiple items, the total score was regarded as missing. Also, CHOP-INTEND assessments were discontinued once participants achieved higher functioning status (2 consecutive scores ≥62).
Score ranges from 0 to 64, where 64 is the maximum possible score. A higher score is indicative of higher/better motor function. CHOP-INTEND assessments were discontinued once patients achieved higher functioning status, so the number of available data points decreased over time.
Outcome measures
| Measure |
Cohort 1
6.7 X 10\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3)
AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
|
Cohort 2
n=6 Participants
2.0 X 10\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12)
AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
|
|---|---|---|
|
Percent Change From Baseline in Mean Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score
|
—
|
30.7 Percentage Change
Standard Deviation 145.61
|
SECONDARY outcome
Timeframe: 24 months post-dosePopulation: full analysis set- all treated patients
Improvement in motor function was determined by achievement of developmental milestones, specifically achievement of ability to sit unassisted for at least 30 seconds, determined by physical therapist and confirmed by an independent central video reviewer. Achievement of functional independent sitting was defined as the ability to maintain a sitting position independently for at least 30 seconds as confirmed per video evaluation by an expert central reviewer based on videos taken either at scheduled visits or provided by the parent/legal guardian.
Outcome measures
| Measure |
Cohort 1
n=3 Participants
6.7 X 10\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3)
AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
|
Cohort 2
n=12 Participants
2.0 X 10\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12)
AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
|
|---|---|---|
|
Number of Participants With Assessed Improvement in Motor Function
|
0 Participants
|
9 Participants
|
Adverse Events
Cohort 1
Serious events: 3 serious events
Other events: 3 other events
Deaths: 0 deaths
Cohort 2
Serious events: 10 serious events
Other events: 12 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1
n=3 participants at risk
6.7 X 10\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3)
AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
|
Cohort 2
n=12 participants at risk
2.0 X 10\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12)
AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
|
|---|---|---|
|
Infections and infestations
pneumonia
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
58.3%
7/12 • Number of events 10 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
rhinovirus infection
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 5 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
enterovirus infection
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
parainfluenzae virus infection
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
respiratory syncytial virus bronchiolitis
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
upper respiratory tract infection
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
25.0%
3/12 • Number of events 3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
adenovirus infection
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Investigations
human rhinovirus test positive
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
lower respiratory tract infection
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
penumonia aspiration
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
postoperative wound infection
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
respiratory distress
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Investigations
transaminases increased
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
atelectasis
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
bronchitis
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Metabolism and nutrition disorders
dehydration
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Investigations
enterovirus test positive
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Injury, poisoning and procedural complications
femur fracture
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
gastroenteritis
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
gastroenteritis viral
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
influenza
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Investigations
norovirus test positive
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Investigations
oxygen saturation decreased
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
pneumonia parainfluenzae viral
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
pneumonia viral
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Injury, poisoning and procedural complications
post procedural hemorrhage
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
respiratory failure
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Cardiac disorders
tachycardia
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
viral upper respiratory tract infection
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
pneumonia respiratory syncytial viral
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
Other adverse events
| Measure |
Cohort 1
n=3 participants at risk
6.7 X 10\^13 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=3)
AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
|
Cohort 2
n=12 participants at risk
2.0 X 10\^14 vg/kg of AVXS-101 delivered one-time through a venous catheter inserted into a peripheral vein (n=12)
AVXS-101: Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter
|
|---|---|---|
|
Gastrointestinal disorders
constipation
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
50.0%
6/12 • Number of events 8 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Gastrointestinal disorders
diarrhea
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
25.0%
3/12 • Number of events 3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Gastrointestinal disorders
gastroesophageal reflux disease
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
41.7%
5/12 • Number of events 6 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Gastrointestinal disorders
vomiting
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
66.7%
8/12 • Number of events 11 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
General disorders
pyrexia
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
58.3%
7/12 • Number of events 12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
bronchiolitis
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
25.0%
3/12 • Number of events 3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
conjunctivitis
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
ear infection
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
enterovirus infection
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
25.0%
3/12 • Number of events 4 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
gastroenteritis viral
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
41.7%
5/12 • Number of events 5 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
otitis media
|
66.7%
2/3 • Number of events 6 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
parainfluenzae virus infection
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
pharyngitis streptococcal
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
pneumonia
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
33.3%
4/12 • Number of events 4 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
rhinovirus infection
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
33.3%
4/12 • Number of events 5 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
upper respiratory tract infection
|
33.3%
1/3 • Number of events 3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
75.0%
9/12 • Number of events 25 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
urinary tract infection
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
viral upper respiratory tract infection
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Injury, poisoning and procedural complications
fall
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
25.0%
3/12 • Number of events 3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Investigations
human rhinovirus test positive
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
25.0%
3/12 • Number of events 4 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Investigations
transaminases increased
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
atelectasis
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
25.0%
3/12 • Number of events 3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
41.7%
5/12 • Number of events 11 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
nasal congestion
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
50.0%
6/12 • Number of events 9 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
respiratory failure
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
25.0%
3/12 • Number of events 5 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
rhinorrhoea
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
25.0%
3/12 • Number of events 4 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
wheezing
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Skin and subcutaneous tissue disorders
decubitus ulcer
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
16.7%
2/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Skin and subcutaneous tissue disorders
erythema
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Skin and subcutaneous tissue disorders
rash
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
41.7%
5/12 • Number of events 6 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Vascular disorders
hypertension
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Immune system disorders
food allergy
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Immune system disorders
hypersensitivity
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
General disorders
catheter site inflammation
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
General disorders
catheter site pain
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
General disorders
pain
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
General disorders
secretion discharge
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Injury, poisoning and procedural complications
femur fracture
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Injury, poisoning and procedural complications
humerus fracture
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Injury, poisoning and procedural complications
lower limb fracture
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Injury, poisoning and procedural complications
mouth injury
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Injury, poisoning and procedural complications
procedural pain
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Injury, poisoning and procedural complications
tibia fracture
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Injury, poisoning and procedural complications
traumatic haematoma
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Injury, poisoning and procedural complications
wound
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Investigations
aspartate aminotransferase increased
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Investigations
enterovirus test positive
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Investigations
eosinophil count increased
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Investigations
haemoglobin decreased
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Cardiac disorders
bradycardia
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Cardiac disorders
tachycardia
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Cardiac disorders
ventricular hypertrophy
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Blood and lymphatic system disorders
anaemia
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
acute respiratory failure
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
aspiration
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
dyspnoea
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
epistaxis
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
hypoxia
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
nasal oedema
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
pleural effusion
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
respiratory tract congestion
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
rhinitis allergic
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
snoring
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
tachypnoea
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Respiratory, thoracic and mediastinal disorders
upper respiratory tract congestion
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Eye disorders
blepharitis
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Eye disorders
chalazion
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Eye disorders
dry eye
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Gastrointestinal disorders
abdominal distension
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Gastrointestinal disorders
abdominal pain
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Gastrointestinal disorders
dysphagia
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Gastrointestinal disorders
gastric hypomotility
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Gastrointestinal disorders
haematemesis
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Gastrointestinal disorders
haematochezia
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Gastrointestinal disorders
hiatus hernia
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Gastrointestinal disorders
nausea
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Gastrointestinal disorders
teething
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Skin and subcutaneous tissue disorders
acne infantile
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Skin and subcutaneous tissue disorders
alopecia
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Skin and subcutaneous tissue disorders
dermatitis allergic
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Skin and subcutaneous tissue disorders
eczema
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Skin and subcutaneous tissue disorders
excessive granulation tissue
|
33.3%
1/3 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Skin and subcutaneous tissue disorders
rash generalised
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Skin and subcutaneous tissue disorders
skin discolouration
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Skin and subcutaneous tissue disorders
urticaria
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Musculoskeletal and connective tissue disorders
mastication disorder
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Musculoskeletal and connective tissue disorders
muscular weakness
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Musculoskeletal and connective tissue disorders
osteopenia
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Musculoskeletal and connective tissue disorders
scoliosis
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Metabolism and nutrition disorders
dehydration
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Metabolism and nutrition disorders
fluid overload
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Metabolism and nutrition disorders
hyperglycaemia
|
33.3%
1/3 • Number of events 3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Metabolism and nutrition disorders
hypoglycaemia
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
alpha haemolytic streptococcal infection
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
catheter site cellulitis
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
clostridium difficile colitis
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
influenza
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
lower respiratory tract infection
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 2 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
metapneumovirus infection
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
oral candidiasis
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
otitis externa
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
otitis media acute
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
pharyngitis
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
pneumonia viral
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
pseudomonas infection
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
respiratory tract infection
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
staphylococcal bacteraemia
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
staphylococcal infection
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
tonsillitis
|
33.3%
1/3 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
0.00%
0/12 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
viral infection
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
|
Infections and infestations
wound infection
|
0.00%
0/3 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
8.3%
1/12 • Number of events 1 • Adverse events were collected from the single dose of study treatment until 24 months post dose
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee PI will provide proposed publication or presentation for review 60 days prior; Sponsor informs PI in writing of revisions required to protect Sponsor's confidential and proprietary technical information and to address inaccurate data or inappropriate interpretations. At expiration of such 60 days, PI may proceed unless Sponsor has notified in writing that such proposed publication or presentation discloses the Sponsor's confidential and proprietary technical information.
- Publication restrictions are in place
Restriction type: OTHER