The classic website will no longer be available as of June 25, 2024. Please use the modernized ClinicalTrials.gov.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    NCT04851873
Previous Study | Return to List | Next Study

Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04851873
Recruitment Status : Completed
First Posted : April 21, 2021
Results First Posted : January 5, 2024
Last Update Posted : January 5, 2024
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Spinal Muscular Atrophy
Intervention Genetic: OAV101
Enrollment 24
Recruitment Details Participants took part in 13 investigative sites across 9 countries.
Pre-assignment Details The screening period began after signature of the study informed consent. The study included a screening period of up to 45 days. On Day -1, participants were admitted to the hospital for pre-treatment baseline procedures. On Day 1, participants received the study treatment.
Arm/Group Title OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17 kg OAV101 1.1e14 vg/kg >17-21 kg
Hide Arm/Group Description 8.5-13 kg >13-17 kg >17-21 kg
Period Title: Overall Study
Started 7 8 9
Completed 7 8 9
Not Completed 0 0 0
Arm/Group Title OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17 kg OAV101 1.1e14 vg/kg >17-21 kg Total
Hide Arm/Group Description 8.5-13 kg >13-17 kg >17-21 kg Total of all reporting groups
Overall Number of Baseline Participants 7 8 9 24
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 7 participants 8 participants 9 participants 24 participants
3.027  (1.1458) 4.519  (1.1769) 6.137  (1.6018) 4.690  (1.8240)
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
 Number Analyzed 7 participants 8 participants 9 participants 24 participants
0 < 28 days
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
28 days - < 2 years
1
  14.3%
0
   0.0%
0
   0.0%
1
   4.2%
2 years - < 12 years
6
  85.7%
8
 100.0%
9
 100.0%
23
  95.8%
12 years - <18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
>= 18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 8 participants 9 participants 24 participants
Female
3
  42.9%
5
  62.5%
4
  44.4%
12
  50.0%
Male
4
  57.1%
3
  37.5%
5
  55.6%
12
  50.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 8 participants 9 participants 24 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
1
  14.3%
5
  62.5%
3
  33.3%
9
  37.5%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
6
  85.7%
1
  12.5%
6
  66.7%
13
  54.2%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
2
  25.0%
0
   0.0%
2
   8.3%
1.Primary Outcome
Title Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) by Weight Bracket
Hide Description An AE is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study.
Time Frame Up to Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set (The Safety Set comprised all participants who were administered investigational drug.)
Arm/Group Title OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17 kg OAV101 1.1e14 vg/kg >17-21 kg OAV101 1.1e14 vg/kg Overall
Hide Arm/Group Description:
8.5-13 kg
>13-17 kg
>17-21 kg
Overall
Overall Number of Participants Analyzed 7 8 9 24
Measure Type: Count of Participants
Unit of Measure: Participants
Any treatment-emergent adverse events
7
 100.0%
8
 100.0%
9
 100.0%
24
 100.0%
Any treatment-emergent adverse events related to OAV101
7
 100.0%
8
 100.0%
9
 100.0%
24
 100.0%
Any severe treatment-emergent adverse events
1
  14.3%
4
  50.0%
3
  33.3%
8
  33.3%
Any serious treatment-emergent adverse events
3
  42.9%
7
  87.5%
5
  55.6%
15
  62.5%
Serious treatment-emergent adverse events related to OAV101
1
  14.3%
4
  50.0%
2
  22.2%
7
  29.2%
Treatment-emergent adverse events leading to study discontinuation
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Treatment-emergent adverse events leading to death
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Treatment-emergent adverse events of special interest
7
 100.0%
7
  87.5%
9
 100.0%
23
  95.8%
2.Primary Outcome
Title Number of Participants With Important Identified and Important Potential Risks (Adverse Events of Special Interest (AESI)) by Risk Name and Weight Bracket
Hide Description

Important identified and important potential risks included the following AESIs: Hepatotoxicity, Thrombocytopenia, Cardiac adverse events, Dorsal root ganglia toxicity and Thrombotic microangiopathy.

These were assessed by the investigator.
Time Frame Up to Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set
Arm/Group Title OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17 kg OAV101 1.1e14 vg/kg >17-21 kg OAV101 1.1e14 vg/kg Overall
Hide Arm/Group Description:
8.5-13 kg
>13-17 kg
>17-21 kg
Overall
Overall Number of Participants Analyzed 7 8 9 24
Measure Type: Count of Participants
Unit of Measure: Participants
Hepatotoxicity
6
  85.7%
5
  62.5%
9
 100.0%
20
  83.3%
Transient thrombocytopenia
4
  57.1%
6
  75.0%
7
  77.8%
17
  70.8%
Cardiac adverse events
0
   0.0%
2
  25.0%
1
  11.1%
3
  12.5%
Thrombotic microangiopathy
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Dorsal root ganglia cell inflammation
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
3.Primary Outcome
Title Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Systolic and Diastolic Blood Pressure
Hide Description

Change from baseline in vital signs measurements - systolic and diastolic blood pressure (mmHg).

Systolic Blood Pressure-Low:<=5th percentile of the age(Any Age), High:>=90th percentile of the age, gender, and height group (<18 yrs).

Diastolic Blood Pressure-High:>=90th percentile of the age, gender, and height group(<18 yrs).
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set
Arm/Group Title OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17kg OAV101 1.1e14 vg/kg >17-21kg OAV101 1.1e14 vg/kg Overall
Hide Arm/Group Description:
8.5-13 kg
>13-17kg
>17-21kg
Overall
Overall Number of Participants Analyzed 7 8 9 24
Measure Type: Count of Participants
Unit of Measure: Participants
Systolic Blood Pressure (mmHg) Low
1
  14.3%
0
   0.0%
1
  11.1%
2
   8.3%
Systolic Blood Pressure (mmHg) High
7
 100.0%
8
 100.0%
8
  88.9%
23
  95.8%
Diastolic Blood Pressure (mmHg) High
7
 100.0%
7
  87.5%
8
  88.9%
22
  91.7%
4.Primary Outcome
Title Change From Baseline in Vital Signs Measurements - Systolic Blood Pressure (mmHg)
Hide Description [Not Specified]
Time Frame Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set
Arm/Group Title OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17kg OAV101 1.1e14 vg/kg >17-21kg OAV101 1.1e14 vg/kg Overall
Hide Arm/Group Description:
8.5-13 kg
>13-17kg
>17-21kg
Overall
Overall Number of Participants Analyzed 7 8 9 24
Mean (Standard Deviation)
Unit of Measure: mmHg
Change from baseline at Day 2 Number Analyzed 7 participants 8 participants 9 participants 24 participants
5.3  (12.65) -1.3  (13.01) 0.4  (10.36) 1.3  (11.75)
Change from baseline at Day 3 Number Analyzed 7 participants 8 participants 9 participants 24 participants
-2.3  (15.01) -5.6  (11.16) 3.0  (8.38) -1.4  (11.62)
Change from baseline at Week 1 Number Analyzed 7 participants 8 participants 9 participants 24 participants
13.4  (10.53) -7.4  (15.59) 2.0  (12.84) 2.2  (15.18)
Change from baseline at Week 2 Number Analyzed 7 participants 8 participants 9 participants 24 participants
11.7  (20.33) -3.8  (17.60) 1.8  (9.15) 2.8  (16.45)
Change from baseline at Week 3 (n=6,8,9,23) Number Analyzed 6 participants 8 participants 9 participants 23 participants
15.8  (8.04) -3.5  (12.87) -0.7  (7.25) 2.7  (12.33)
Change from baseline at Week 4 (n=6,8,8,22) Number Analyzed 6 participants 8 participants 8 participants 22 participants
12.3  (16.50) -3.5  (15.89) 6.8  (10.02) 4.5  (15.05)
Change from baseline at Week 6 (n=7,8,8,23) Number Analyzed 7 participants 8 participants 8 participants 23 participants
8.1  (12.92) -2.8  (24.15) 5.4  (15.39) 3.4  (18.13)
Change from baseline at Week 8 (n=7,7,9,23) Number Analyzed 7 participants 7 participants 9 participants 23 participants
3.9  (15.49) -0.7  (14.57) 3.6  (12.83) 2.3  (13.69)
Change from baseline at Week 10 Number Analyzed 7 participants 8 participants 9 participants 24 participants
10.0  (16.99) -5.6  (13.19) 4.6  (10.06) 2.8  (14.32)
Change from baseline at Week 13 Number Analyzed 7 participants 8 participants 9 participants 24 participants
6.1  (10.78) -4.5  (17.50) 1.4  (9.93) 0.8  (13.28)
Change from baseline at Week 26 Number Analyzed 7 participants 8 participants 9 participants 24 participants
-5.7  (9.05) -12.9  (15.34) -6.9  (14.99) -8.5  (13.46)
Change from baseline at Week 39 (n=7,7,9,23) Number Analyzed 7 participants 7 participants 9 participants 23 participants
16.4  (29.70) -10.3  (13.59) -3.9  (3.56) 0.3  (21.98)
Change from baseline at Week 52 Number Analyzed 7 participants 8 participants 9 participants 24 participants
5.0  (21.60) -7.8  (13.54) 0.1  (10.81) -1.1  (15.67)
5.Primary Outcome
Title Change From Baseline in Vital Signs Measurements - Diastolic Blood Pressure (mmHg)
Hide Description [Not Specified]
Time Frame Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set
Arm/Group Title OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17kg OAV101 1.1e14 vg/kg >17-21kg OAV101 1.1e14 vg/kg Overall
Hide Arm/Group Description:
8.5-13 kg
>13-17kg
>17-21kg
Overall
Overall Number of Participants Analyzed 7 8 9 24
Mean (Standard Deviation)
Unit of Measure: mmHg
Change from baseline at Day 2 (n=6,8,9,23) Number Analyzed 6 participants 8 participants 9 participants 23 participants
1.7  (12.42) 4.8  (13.22) -2.1  (13.04) 1.3  (12.71)
Change from baseline at Day 3 (n=6,8,9,23) Number Analyzed 6 participants 8 participants 9 participants 23 participants
-3.7  (12.29) -5.1  (13.91) 4.2  (5.45) -1.1  (11.22)
Change from baseline at Week 1 Number Analyzed 7 participants 8 participants 9 participants 24 participants
7.4  (20.07) 1.8  (13.56) 3.8  (13.41) 4.2  (15.13)
Change from baseline at Week 2 Number Analyzed 7 participants 8 participants 9 participants 24 participants
7.1  (16.07) 7.3  (11.30) 4.4  (10.33) 6.2  (12.05)
Change from baseline at Week 3 (n=6,8,9,23) Number Analyzed 6 participants 8 participants 9 participants 23 participants
12.0  (15.09) -1.5  (14.01) 5.0  (9.60) 4.6  (13.28)
Change from baseline at Week 4 (n=6,8,8,22) Number Analyzed 6 participants 8 participants 8 participants 22 participants
3.8  (19.27) 1.8  (9.97) 1.3  (8.53) 2.1  (12.12)
Change from baseline at Week 6 (n=6,8,8,22) Number Analyzed 6 participants 8 participants 8 participants 22 participants
10.3  (10.91) -0.5  (17.87) 6.5  (10.14) 5.0  (13.77)
Change from baseline at Week 8 (n=6,7,9,22) Number Analyzed 6 participants 7 participants 9 participants 22 participants
10.5  (14.57) 3.0  (17.68) 5.9  (11.72) 6.2  (14.18)
Change from baseline at Week 10 (n=6,8,9,23) Number Analyzed 6 participants 8 participants 9 participants 23 participants
6.7  (15.55) -2.0  (8.88) 4.8  (11.74) 2.9  (12.01)
Change from baseline at Week 13 (n=6,8,9,23) Number Analyzed 6 participants 8 participants 9 participants 23 participants
9.7  (15.37) 0.4  (13.54) 0.1  (11.03) 2.7  (13.20)
Change from baseline at Week 26 Number Analyzed 7 participants 8 participants 9 participants 24 participants
-0.7  (9.05) -9.1  (10.26) -1.2  (7.55) -3.7  (9.41)
Change from baseline at Week 39 (n=7,7,9,23) Number Analyzed 7 participants 7 participants 9 participants 23 participants
2.4  (15.73) -4.4  (13.01) -0.4  (9.62) -0.8  (12.44)
Change from baseline at Week 52 Number Analyzed 7 participants 8 participants 9 participants 24 participants
0.3  (11.74) -2.0  (11.89) 1.7  (5.52) 0.0  (9.60)
6.Primary Outcome
Title Change From Baseline in Vital Signs Measurements - Respiratory Rate (Breaths/Min)
Hide Description Change from baseline in vital signs measurements - Respiratory Rate (breaths/min)
Time Frame Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set
Arm/Group Title OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17kg OAV101 1.1e14 vg/kg >17-21kg OAV101 1.1e14 vg/kg Overall
Hide Arm/Group Description:
8.5-13 kg
>13-17kg
>17-21kg
Overall
Overall Number of Participants Analyzed 7 8 9 24
Mean (Standard Deviation)
Unit of Measure: breaths/min)
Change from baseline at Day 2 Number Analyzed 7 participants 8 participants 9 participants 24 participants
-1.1  (3.08) -1.6  (3.74) 0.8  (3.96) -0.6  (3.66)
Change from baseline at Day 3 Number Analyzed 7 participants 8 participants 9 participants 24 participants
-0.1  (5.87) -0.1  (4.52) 0.4  (4.30) 0.1  (4.66)
Change from baseline at Week 1 Number Analyzed 7 participants 8 participants 9 participants 24 participants
2.7  (6.78) -0.1  (4.22) 1.6  (5.61) 1.3  (5.45)
Change from baseline at Week 2 Number Analyzed 7 participants 8 participants 9 participants 24 participants
2.4  (5.44) -0.8  (5.01) 1.4  (4.16) 1.0  (4.81)
Change from baseline at Week 3 (n=6,8,9,23) Number Analyzed 6 participants 8 participants 9 participants 23 participants
5.5  (8.96) -1.8  (5.09) 1.8  (5.02) 1.5  (6.63)
Change from baseline at Week 4 (n=7,8,8,23) Number Analyzed 7 participants 8 participants 8 participants 23 participants
2.6  (4.54) 1.4  (7.35) 1.4  (4.00) 1.7  (5.31)
Change from baseline at Week 6 (n=7,8,8,23) Number Analyzed 7 participants 8 participants 8 participants 23 participants
5.1  (9.56) -0.6  (4.75) 0.3  (6.43) 1.4  (7.19)
Change from baseline at Week 8 (n=7,7,9,23) Number Analyzed 7 participants 7 participants 9 participants 23 participants
3.3  (7.45) -0.6  (4.47) 0.0  (4.42) 0.8  (5.52)
Change from baseline at Week 10 Number Analyzed 7 participants 8 participants 9 participants 24 participants
3.3  (6.58) -1.0  (4.31) -0.3  (3.61) 0.5  (4.99)
Change from baseline at Week 13 Number Analyzed 7 participants 8 participants 9 participants 24 participants
1.7  (6.75) -0.5  (3.51) -0.6  (2.92) 0.1  (4.44)
Change from baseline at Week 26 Number Analyzed 7 participants 8 participants 9 participants 24 participants
4.1  (8.69) 0.8  (3.69) 0.7  (4.24) 1.7  (5.71)
Change from baseline at Week 39 (n=7,7,9,23) Number Analyzed 7 participants 7 participants 9 participants 23 participants
1.4  (5.03) -1.7  (4.35) -0.6  (4.56) -0.3  (4.61)
Change from baseline at Week 52 Number Analyzed 7 participants 8 participants 9 participants 24 participants
1.7  (7.34) -0.5  (4.66) -2.2  (3.19) -0.5  (5.18)
7.Primary Outcome
Title Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min)
Hide Description Change from baseline in vital signs measurements - Pulse Rate (beats/min
Time Frame Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set
Arm/Group Title OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17kg OAV101 1.1e14 vg/kg >17-21kg OAV101 1.1e14 vg/kg Overall
Hide Arm/Group Description:
8.5-13 kg
>13-17kg
>17-21kg
Overall
Overall Number of Participants Analyzed 7 8 9 24
Mean (Standard Deviation)
Unit of Measure: beats/min
Change from baseline at Day 2 Number Analyzed 7 participants 8 participants 9 participants 24 participants
3.3  (18.54) 6.3  (4.43) 16.3  (26.54) 9.2  (19.35)
Change from baseline at Day 3 Number Analyzed 7 participants 8 participants 9 participants 24 participants
-1.4  (19.29) -3.1  (14.21) 10.2  (14.19) 2.4  (16.36)
Change from baseline at Week 1 Number Analyzed 7 participants 8 participants 9 participants 24 participants
1.4  (14.27) -9.6  (18.04) -3.9  (18.84) -4.3  (17.19)
Change from baseline at Week 2 Number Analyzed 7 participants 8 participants 9 participants 24 participants
-13.4  (21.25) -8.8  (15.21) 4.7  (12.12) -5.1  (17.39)
Change from baseline at Week 3 (n=6,8,9,23) Number Analyzed 6 participants 8 participants 9 participants 23 participants
21.0  (21.83) -3.8  (12.46) 13.1  (15.12) 9.3  (18.60)
Change from baseline at Week 4 (n=7,8,8,23) Number Analyzed 7 participants 8 participants 8 participants 23 participants
-4.7  (17.27) -3.5  (22.82) 12.8  (16.23) 1.8  (19.95)
Change from baseline at Week 6 (n=7,8,8,23) Number Analyzed 7 participants 8 participants 8 participants 23 participants
12.9  (20.70) 0.0  (9.59) 8.8  (17.64) 7.0  (16.58)
Change from baseline at Week 8 (n=7,7,9,23) Number Analyzed 7 participants 7 participants 9 participants 23 participants
-3.6  (15.75) -6.3  (22.69) 6.4  (17.63) -0.5  (18.83)
Change from baseline at Week 10 Number Analyzed 7 participants 8 participants 9 participants 24 participants
-3.9  (25.14) -4.3  (16.57) 9.7  (14.13) 1.1  (19.08)
Change from baseline at Week 13 Number Analyzed 7 participants 8 participants 9 participants 24 participants
-6.4  (12.23) 2.6  (15.65) 8.4  (18.06) 2.2  (16.28)
Change from baseline at Week 26 Number Analyzed 7 participants 8 participants 9 participants 24 participants
-6.9  (12.85) -6.3  (17.00) 3.8  (14.34) -2.7  (15.12)
Change from baseline at Week 39 (n=7,7,9,23) Number Analyzed 7 participants 7 participants 9 participants 23 participants
-4.7  (21.85) -7.7  (13.23) 3.1  (17.12) -2.6  (17.54)
Change from baseline at Week 52 Number Analyzed 7 participants 8 participants 9 participants 24 participants
-8.9  (10.32) -16.4  (14.38) 4.4  (16.46) -6.4  (16.35)
8.Primary Outcome
Title Summary of Participants Meeting Criteria for Potentially Clinically Significant Vital Sign Values by Weight Bracket - Temperature
Hide Description

Change from baseline in vital signs measurements - temperature (degrees Celsius)

Temperature-Low:<=35ºC(Any Age),High:>=38.4ºC(<18 yrs).
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set
Arm/Group Title OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17kg OAV101 1.1e14 vg/kg >17-21kg OAV101 1.1e14 vg/kg Overall
Hide Arm/Group Description:
8.5-13 kg
>13-17kg
>17-21kg
Overall
Overall Number of Participants Analyzed 7 8 9 24
Measure Type: Count of Participants
Unit of Measure: Participants
Temperature (ºC) Low
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Temperature (ºC) High
1
  14.3%
0
   0.0%
1
  11.1%
2
   8.3%
9.Primary Outcome
Title Change From Baseline in Vital Signs Measurements - Temperature (Degrees Celsius)
Hide Description [Not Specified]
Time Frame Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set
Arm/Group Title OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17kg OAV101 1.1e14 vg/kg >17-21kg OAV101 1.1e14 vg/kg Overall
Hide Arm/Group Description:
8.5-13 kg
>13-17kg
>17-21kg
Overall
Overall Number of Participants Analyzed 7 8 9 24
Mean (Standard Deviation)
Unit of Measure: degrees Celsius
Change from baseline at Day 2 Number Analyzed 7 participants 8 participants 9 participants 24 participants
0.07  (0.502) -0.11  (0.714) 0.07  (0.918) 0.01  (0.722)
Change from baseline at Day 3 Number Analyzed 7 participants 8 participants 9 participants 24 participants
0.11  (0.261) -0.48  (0.719) 0.12  (0.319) -0.08  (0.541)
Change from baseline at Week 1 Number Analyzed 7 participants 8 participants 9 participants 24 participants
-0.07  (0.386) -0.44  (0.563) 0.00  (0.548) -0.17  (0.528)
Change from baseline at Week 2 Number Analyzed 7 participants 8 participants 9 participants 24 participants
0.30  (0.592) -0.41  (0.649) -0.09  (0.478) -0.08  (0.618)
Change from baseline at Week 3 (n=6,8,9,23) Number Analyzed 6 participants 8 participants 9 participants 23 participants
0.32  (0.556) -0.24  (0.403) 0.02  (0.387) 0.01  (0.474)
Change from baseline at Week 4 (n=7,8,8,23) Number Analyzed 7 participants 8 participants 8 participants 23 participants
0.16  (0.673) -0.31  (0.775) 0.05  (0.431) -0.04  (0.645)
Change from baseline at Week 6 (n=7,8,8,23) Number Analyzed 7 participants 8 participants 8 participants 23 participants
0.26  (0.458) -0.18  (0.599) -0.09  (0.500) -0.01  (0.535)
Change from baseline at Week 8 (n=7,7,9,23) Number Analyzed 7 participants 7 participants 9 participants 23 participants
0.13  (0.547) -0.21  (0.654) 0.01  (0.386) -0.02  (0.521)
Change from baseline at Week 10 Number Analyzed 7 participants 8 participants 9 participants 24 participants
0.34  (0.351) -0.45  (0.727) 0.01  (0.553) -0.05  (0.635)
Change from baseline at Week 13 Number Analyzed 7 participants 8 participants 9 participants 24 participants
0.13  (0.399) -0.06  (0.701) 0.09  (0.473) 0.05  (0.525)
Change from baseline at Week 26 Number Analyzed 7 participants 8 participants 9 participants 24 participants
0.31  (0.453) -0.15  (0.532) 0.03  (0.324) 0.05  (0.460)
Change from baseline at Week 39 (n=7,7,9,23) Number Analyzed 7 participants 7 participants 9 participants 23 participants
0.33  (0.350) -0.10  (0.622) -0.11  (0.491) 0.03  (0.518)
Change from baseline at Week 52 Number Analyzed 7 participants 8 participants 9 participants 24 participants
0.63  (0.836) -0.53  (0.819) -0.11  (0.401) -0.03  (0.814)
10.Primary Outcome
Title Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level
Hide Description

Change from baseline in vital signs measurements - oxygen saturation level (%).

Oxygen saturation is the fraction of oxygen-saturated hemoglobin relative to total hemoglobin (unsaturated+saturated) in the blood and then multiplied by 100.
Time Frame Baseline, Days 2 and 3, Weeks 1, 2, 3, 4, 6, 8, 10, 13, 26, 39 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set
Arm/Group Title OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17kg OAV101 1.1e14 vg/kg >17-21kg OAV101 1.1e14 vg/kg Overall
Hide Arm/Group Description:
8.5-13 kg
>13-17kg
>17-21kg
Overall
Overall Number of Participants Analyzed 7 8 9 24
Mean (Standard Deviation)
Unit of Measure: % Oxygen Saturated
Change from baseline at Day 2 Number Analyzed 7 participants 8 participants 9 participants 24 participants
0.0  (0.82) -0.3  (1.04) -0.4  (1.13) -0.3  (0.99)
Change from baseline at Day 3 Number Analyzed 7 participants 8 participants 9 participants 24 participants
0.3  (1.80) -0.1  (0.83) -1.3  (1.12) -0.5  (1.41)
Change from baseline at Week 1 Number Analyzed 7 participants 8 participants 9 participants 24 participants
0.1  (1.07) -0.4  (2.00) -0.8  (1.20) -0.4  (1.47)
Change from baseline at Week 2 Number Analyzed 7 participants 8 participants 9 participants 24 participants
-0.7  (1.89) -1.0  (1.51) -0.8  (2.11) -0.8  (1.79)
Change from baseline at Week 3 (n=6,8,9,23) Number Analyzed 6 participants 8 participants 9 participants 23 participants
-1.0  (1.90) -0.3  (1.28) -1.1  (1.05) -0.8  (1.38)
Change from baseline at Week 4 (n=7,8,8,23) Number Analyzed 7 participants 8 participants 8 participants 23 participants
0.0  (1.91) -0.5  (1.31) -0.8  (0.89) -0.4  (1.38)
Change from baseline at Week 6 (n=7,8,8,23) Number Analyzed 7 participants 8 participants 8 participants 23 participants
-0.1  (1.21) -1.0  (1.51) -0.6  (1.19) -0.6  (1.31)
Change from baseline at Week 8 (n=7,7,9,23) Number Analyzed 7 participants 7 participants 9 participants 23 participants
-0.7  (1.89) 0.0  (1.15) -0.4  (0.88) -0.4  (1.31)
Change from baseline at Week 10 Number Analyzed 7 participants 8 participants 9 participants 24 participants
0.3  (1.89) -1.0  (1.93) -0.7  (1.32) -0.5  (1.72)
Change from baseline at Week 13 Number Analyzed 7 participants 8 participants 9 participants 24 participants
0.1  (1.57) -0.5  (1.07) -0.4  (1.67) -0.3  (1.43)
Change from baseline at Week 26 Number Analyzed 7 participants 8 participants 9 participants 24 participants
-0.6  (1.51) -0.1  (1.36) -0.2  (1.99) -0.3  (1.60)
Change from baseline at Week 39 (n=7,7,8,22) Number Analyzed 7 participants 7 participants 8 participants 22 participants
-1.0  (1.41) 0.1  (0.90) -0.4  (1.19) -0.4  (1.22)
Change from baseline at Week 52 Number Analyzed 7 participants 8 participants 9 participants 24 participants
-1.0  (2.00) -0.6  (1.51) -0.1  (1.54) -0.5  (1.64)
11.Secondary Outcome
Title Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development.
Hide Description The World Health Organization-Multicentre Growth Reference Study (WHO-MGRS) and Bayley scale of Infant and Toddler Development was modified and combined into a single scale expressly for this study, to measure developmental motor milestones. These were assessed via the milestone checklist, formed of 10 yes/no questions with optional video documentation. The developmental milestones are: head control, sitting with support, sitting without support, sitting without support for 30 seconds, hands-and-knees crawling, pulls to stand, standing with assistance, walking with assistance, standing alone and walking alone. A yes response indicates that the patient reached a particular development milestone.
Time Frame Baseline, Week 26 and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17 kg OAV101 1.1e14 vg/kg >17-21 kg OAV101 1.1e14 vg/kg Overall
Hide Arm/Group Description:
8.5-13 kg
>13-17 kg
>17-21 kg
Overall
Overall Number of Participants Analyzed 7 8 9 24
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline Head control (Bayley GM #4) Number Analyzed 7 participants 8 participants 9 participants 24 participants
7
 100.0%
8
 100.0%
9
 100.0%
24
 100.0%
Baseline Sits with support (Bayley GM #19) Number Analyzed 7 participants 8 participants 9 participants 24 participants
7
 100.0%
8
 100.0%
9
 100.0%
24
 100.0%
Baseline Sitting without support (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
6
  85.7%
7
  87.5%
8
  88.9%
21
  87.5%
Baseline Sits without support 30 s (Bayley GM #26) Number Analyzed 7 participants 8 participants 9 participants 24 participants
6
  85.7%
7
  87.5%
8
  88.9%
21
  87.5%
Baseline Hands-and-knees crawling (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
1
  14.3%
3
  37.5%
6
  66.7%
10
  41.7%
Baseline Pulls to stand (Bayley GM #35) Number Analyzed 7 participants 8 participants 9 participants 24 participants
1
  14.3%
3
  37.5%
5
  55.6%
9
  37.5%
Baseline Standing with assistance (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
0
   0.0%
2
  25.0%
5
  55.6%
7
  29.2%
Baseline Walking with assistance (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
0
   0.0%
2
  25.0%
5
  55.6%
7
  29.2%
Baseline Standing alone (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
0
   0.0%
2
  25.0%
4
  44.4%
6
  25.0%
Baseline Walking alone (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
0
   0.0%
2
  25.0%
4
  44.4%
6
  25.0%
Week 26 Head control (Bayley GM #4) Number Analyzed 7 participants 8 participants 9 participants 24 participants
7
 100.0%
8
 100.0%
9
 100.0%
24
 100.0%
Week 26 Sits with support (Bayley GM #19) Number Analyzed 7 participants 8 participants 9 participants 24 participants
7
 100.0%
8
 100.0%
9
 100.0%
24
 100.0%
Week 26 Sitting without support (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
7
 100.0%
5
  62.5%
9
 100.0%
21
  87.5%
Week 26 Sits without support 30 s (Bayley GM #26) Number Analyzed 7 participants 8 participants 9 participants 24 participants
7
 100.0%
5
  62.5%
8
  88.9%
20
  83.3%
Week 26 Hands-and-knees crawling (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
2
  28.6%
3
  37.5%
6
  66.7%
11
  45.8%
Week 26 Pulls to stand (Bayley GM #35) Number Analyzed 7 participants 8 participants 9 participants 24 participants
2
  28.6%
3
  37.5%
5
  55.6%
10
  41.7%
Week 26 Standing with assistance (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
1
  14.3%
3
  37.5%
5
  55.6%
9
  37.5%
Week 26 Walking with assistance (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
1
  14.3%
3
  37.5%
4
  44.4%
8
  33.3%
Week 26 Standing alone (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
0
   0.0%
3
  37.5%
4
  44.4%
7
  29.2%
Week 26 Walking alone (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
0
   0.0%
2
  25.0%
4
  44.4%
6
  25.0%
Week 52 Head control (Bayley GM #4) Number Analyzed 7 participants 8 participants 9 participants 24 participants
7
 100.0%
8
 100.0%
9
 100.0%
24
 100.0%
Week 52 Sits with support (Bayley GM #19) Number Analyzed 7 participants 8 participants 9 participants 24 participants
7
 100.0%
7
  87.5%
9
 100.0%
23
  95.8%
Week 52 Sitting without support (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
7
 100.0%
7
  87.5%
8
  88.9%
22
  91.7%
Week 52 Sits without support 30 s (Bayley GM #26) Number Analyzed 7 participants 8 participants 9 participants 24 participants
7
 100.0%
6
  75.0%
8
  88.9%
21
  87.5%
Week 52 Hands-and-knees crawling (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
2
  28.6%
3
  37.5%
6
  66.7%
11
  45.8%
Week 52 Pulls to stand (Bayley GM #35) (n=7,8,8,23) Number Analyzed 7 participants 8 participants 8 participants 23 participants
2
  28.6%
3
  37.5%
5
  62.5%
10
  43.5%
Week 52 Standing with assistance (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
2
  28.6%
3
  37.5%
5
  55.6%
10
  41.7%
Week 52 Walking with assistance (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
1
  14.3%
3
  37.5%
4
  44.4%
8
  33.3%
Week 52 Standing alone (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
0
   0.0%
3
  37.5%
4
  44.4%
7
  29.2%
Week 52 Walking alone (WHO MGRS) Number Analyzed 7 participants 8 participants 9 participants 24 participants
0
   0.0%
2
  25.0%
4
  44.4%
6
  25.0%
12.Secondary Outcome
Title Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age
Hide Description The HFMSE was devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE is formed of 33 assessments. Each motor skill item is scored on a 3 point Likert scale from 0 (no response) to 2 (full response), with a total score range of 0 to 66. A higher score indicates a higher level of ability.
Time Frame Baseline, Week 4, Week 13, Week 26, Week 39 and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Full Analysis Set (FAS) who had an available value for the outcome measure at the specified timepoints. The FAS includes all participants who were administered investigational drug.
Arm/Group Title OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17 kg OAV101 1.1e14 vg/kg >17-21 kg OAV101 1.1e14 vg/kg Overall
Hide Arm/Group Description:
8.5-13 kg
>13-17 kg
>17-21 kg
Overall
Overall Number of Participants Analyzed 5 6 9 20
Mean (Standard Deviation)
Unit of Measure: HFMSE total score on a scale
Change from baseline at Week 4 (n=4,6,9,19) Number Analyzed 4 participants 6 participants 9 participants 19 participants
3.8  (2.99) 2.3  (2.66) 3.1  (3.10) 3.0  (2.83)
Change from baseline at Week 13 (n=4,6,9,19) Number Analyzed 4 participants 6 participants 9 participants 19 participants
1.3  (7.89) 4.5  (3.99) 3.9  (3.98) 3.5  (4.83)
Change from baseline at Week 26 (n=5,6,9,20) Number Analyzed 5 participants 6 participants 9 participants 20 participants
3.4  (5.18) 4.3  (5.05) 3.1  (4.14) 3.6  (4.45)
Change from baseline at Week 39 (n=5,5,9,19) Number Analyzed 5 participants 5 participants 9 participants 19 participants
2.6  (6.11) -0.6  (4.72) 4.3  (3.71) 2.6  (4.87)
Change from baseline at Week 52 (n=5,6,7,18) Number Analyzed 5 participants 6 participants 7 participants 18 participants
3.0  (5.24) 3.7  (5.75) 4.3  (4.07) 3.7  (4.73)
13.Secondary Outcome
Title Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age.
Hide Description The RULM assesses motor performance in the upper limbs from childhood through adulthood in ambulatory and non-ambulatory individuals with SMA. 'The scale consists of an entry item to establish functional levels and 19 items covering distal to proximal movements. The entry item is a modified version of the Brooke scale, including activities ranging from no functional use of hands (score 0) to full bilateral shoulder abduction (score 6). The entry item does not contribute to the total score but serves as a functional classification of overall upper limb functional ability. Of the remaining 19 items, 18 are scored on a 3 point scoring system and 1 item is scored on a 2 point scoring system. The test is performed unilaterally using the limb preferred by the participant. The total score ranges from 0, if all the items cannot be performed, to 37, if all the activities are achieved fully without any compensation. ' Higher scores indicate higher levels of motor ability.
Time Frame Baseline, Week 4, Week 13, Week 26, Week 39 and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Full Analysis Set (FAS) who had an available value for the outcome measure at the specified timepoints. The FAS includes all participants who were administered investigational drug.
Arm/Group Title OAV101 1.1e14 vg/kg 8.5-13 kg OAV101 1.1e14 vg/kg >13-17 kg OAV101 1.1e14 vg/kg >17-21 kg OAV101 1.1e14 vg/kg 8.5-13 kg Overall
Hide Arm/Group Description:
8.5-13 kg
>13-17 kg
>17-21 kg
Overall
Overall Number of Participants Analyzed 4 6 8 18
Mean (Standard Deviation)
Unit of Measure: RULM total score on a scale
Change from baseline at Week 4 Number Analyzed 4 participants 6 participants 8 participants 18 participants
2.8  (2.36) 1.2  (1.33) 1.0  (1.93) 1.4  (1.89)
Change from baseline at Week 13 Number Analyzed 4 participants 6 participants 8 participants 18 participants
4.3  (1.50) 0.8  (2.32) 2.0  (2.56) 2.1  (2.52)
Change from baseline at Week 26 Number Analyzed 4 participants 6 participants 8 participants 18 participants
5.3  (2.50) 1.3  (1.21) 1.6  (2.88) 2.3  (2.74)
Change from baseline at Week 39 (n=4,5,8,17) Number Analyzed 4 participants 5 participants 8 participants 17 participants
7.3  (2.22) -0.6  (1.34) 2.0  (4.34) 2.5  (4.29)
Change from baseline at Week 52 (n=3,6,8,17) Number Analyzed 3 participants 6 participants 8 participants 17 participants
6.0  (3.46) 0.3  (1.63) 1.8  (4.71) 2.0  (4.02)
Time Frame Adverse events are reported from the single dose of study treatment until end of study, up to maximum duration of 12 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title OAV101 1.1e14 vg/kg 8.5 to 13 kg OAV101 1.1e14 vg/kg Greater Than 13 to 17 kg OAV101 1.1e14 vg/kg Greater Than 17 to 21 kg OAV101 1.1e14 vg/kg Overall
Hide Arm/Group Description 8.5 to 13 kg Greater than 13 to 17 kg Greater than 17 to 21 kg Overall
All-Cause Mortality
OAV101 1.1e14 vg/kg 8.5 to 13 kg OAV101 1.1e14 vg/kg Greater Than 13 to 17 kg OAV101 1.1e14 vg/kg Greater Than 17 to 21 kg OAV101 1.1e14 vg/kg Overall
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/7 (0.00%)   0/8 (0.00%)   0/9 (0.00%)   0/24 (0.00%) 
Hide Serious Adverse Events
OAV101 1.1e14 vg/kg 8.5 to 13 kg OAV101 1.1e14 vg/kg Greater Than 13 to 17 kg OAV101 1.1e14 vg/kg Greater Than 17 to 21 kg OAV101 1.1e14 vg/kg Overall
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   3/7 (42.86%)   7/8 (87.50%)   5/9 (55.56%)   15/24 (62.50%) 
Blood and lymphatic system disorders         
Anaemia † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Bicytopenia † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Thrombocytopenia † 1  0/7 (0.00%)  2/8 (25.00%)  1/9 (11.11%)  3/24 (12.50%) 
Gastrointestinal disorders         
Constipation † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Vomiting † 1  0/7 (0.00%)  2/8 (25.00%)  1/9 (11.11%)  3/24 (12.50%) 
General disorders         
Asthenia † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Pyrexia † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Hepatobiliary disorders         
Hepatic cytolysis † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Hepatotoxicity † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Hypertransaminasaemia † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Infections and infestations         
COVID-19 † 1  0/7 (0.00%)  0/8 (0.00%)  2/9 (22.22%)  2/24 (8.33%) 
COVID-19 pneumonia † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Gastroenteritis viral † 1  1/7 (14.29%)  1/8 (12.50%)  0/9 (0.00%)  2/24 (8.33%) 
Pneumonia † 1  0/7 (0.00%)  0/8 (0.00%)  2/9 (22.22%)  2/24 (8.33%) 
Respiratory tract infection † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Subglottic laryngitis † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Varicella † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Injury, poisoning and procedural complications         
Femur fracture † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Radius fracture † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Investigations         
Liver function test abnormal † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Liver function test increased † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Metabolism and nutrition disorders         
Dehydration † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Hypoglycaemia † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Musculoskeletal and connective tissue disorders         
Hip deformity † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Renal and urinary disorders         
Urinary retention † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
1
Term from vocabulary, MedDRA (26.0)
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
OAV101 1.1e14 vg/kg 8.5 to 13 kg OAV101 1.1e14 vg/kg Greater Than 13 to 17 kg OAV101 1.1e14 vg/kg Greater Than 17 to 21 kg OAV101 1.1e14 vg/kg Overall
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   7/7 (100.00%)   8/8 (100.00%)   9/9 (100.00%)   24/24 (100.00%) 
Blood and lymphatic system disorders         
Anaemia † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Thrombocytopenia † 1  1/7 (14.29%)  2/8 (25.00%)  2/9 (22.22%)  5/24 (20.83%) 
Cardiac disorders         
Bradycardia † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Pericardial effusion † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Tachycardia † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Endocrine disorders         
Cushing's syndrome † 1  1/7 (14.29%)  0/8 (0.00%)  2/9 (22.22%)  3/24 (12.50%) 
Cushingoid † 1  0/7 (0.00%)  1/8 (12.50%)  1/9 (11.11%)  2/24 (8.33%) 
Eye disorders         
Blepharitis † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Eye irritation † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Periorbital swelling † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Gastrointestinal disorders         
Abdominal pain † 1  2/7 (28.57%)  0/8 (0.00%)  0/9 (0.00%)  2/24 (8.33%) 
Abdominal pain upper † 1  0/7 (0.00%)  2/8 (25.00%)  2/9 (22.22%)  4/24 (16.67%) 
Constipation † 1  1/7 (14.29%)  2/8 (25.00%)  0/9 (0.00%)  3/24 (12.50%) 
Diarrhoea † 1  2/7 (28.57%)  1/8 (12.50%)  0/9 (0.00%)  3/24 (12.50%) 
Faecaloma † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Gastritis † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Gastrooesophageal reflux disease † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Leukoplakia oral † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Nausea † 1  3/7 (42.86%)  2/8 (25.00%)  3/9 (33.33%)  8/24 (33.33%) 
Upper gastrointestinal haemorrhage † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Vomiting † 1  5/7 (71.43%)  5/8 (62.50%)  7/9 (77.78%)  17/24 (70.83%) 
General disorders         
Malaise † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Pyrexia † 1  4/7 (57.14%)  1/8 (12.50%)  5/9 (55.56%)  10/24 (41.67%) 
Hepatobiliary disorders         
Hypertransaminasaemia † 1  1/7 (14.29%)  1/8 (12.50%)  5/9 (55.56%)  7/24 (29.17%) 
Immune system disorders         
Hypersensitivity † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Infections and infestations         
COVID-19 † 1  1/7 (14.29%)  4/8 (50.00%)  2/9 (22.22%)  7/24 (29.17%) 
Conjunctivitis † 1  1/7 (14.29%)  1/8 (12.50%)  0/9 (0.00%)  2/24 (8.33%) 
Gastroenteritis † 1  1/7 (14.29%)  0/8 (0.00%)  2/9 (22.22%)  3/24 (12.50%) 
Infectious mononucleosis † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Influenza † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Nasopharyngitis † 1  2/7 (28.57%)  2/8 (25.00%)  1/9 (11.11%)  5/24 (20.83%) 
Otitis media † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Pneumonia † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Respiratory tract infection † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Rhinitis † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Upper respiratory tract infection † 1  4/7 (57.14%)  2/8 (25.00%)  2/9 (22.22%)  8/24 (33.33%) 
Urinary tract infection † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Viral skin infection † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Injury, poisoning and procedural complications         
Fall † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Joint dislocation † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Procedural nausea † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Procedural pain † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Stoma site erythema † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Torus fracture † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Investigations         
Alanine aminotransferase increased † 1  1/7 (14.29%)  0/8 (0.00%)  1/9 (11.11%)  2/24 (8.33%) 
Aspartate aminotransferase increased † 1  1/7 (14.29%)  0/8 (0.00%)  1/9 (11.11%)  2/24 (8.33%) 
Blood lactate dehydrogenase increased † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Blood potassium abnormal † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Blood urea increased † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Electrocardiogram T wave inversion † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Haemoglobin decreased † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Hepatic enzyme increased † 1  0/7 (0.00%)  2/8 (25.00%)  0/9 (0.00%)  2/24 (8.33%) 
Liver function test abnormal † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Liver function test increased † 1  1/7 (14.29%)  0/8 (0.00%)  1/9 (11.11%)  2/24 (8.33%) 
Platelet count abnormal † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Platelet count decreased † 1  3/7 (42.86%)  2/8 (25.00%)  2/9 (22.22%)  7/24 (29.17%) 
Prothrombin time prolonged † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Transaminases increased † 1  3/7 (42.86%)  2/8 (25.00%)  1/9 (11.11%)  6/24 (25.00%) 
Weight increased † 1  0/7 (0.00%)  1/8 (12.50%)  1/9 (11.11%)  2/24 (8.33%) 
Metabolism and nutrition disorders         
Decreased appetite † 1  1/7 (14.29%)  0/8 (0.00%)  1/9 (11.11%)  2/24 (8.33%) 
Dehydration † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Vitamin D deficiency † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Musculoskeletal and connective tissue disorders         
Arthralgia † 1  0/7 (0.00%)  0/8 (0.00%)  3/9 (33.33%)  3/24 (12.50%) 
Back pain † 1  0/7 (0.00%)  1/8 (12.50%)  1/9 (11.11%)  2/24 (8.33%) 
Muscular weakness † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Osteopenia † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Pain in extremity † 1  0/7 (0.00%)  0/8 (0.00%)  3/9 (33.33%)  3/24 (12.50%) 
Nervous system disorders         
Dizziness † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Headache † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Lethargy † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Tremor † 1  0/7 (0.00%)  2/8 (25.00%)  0/9 (0.00%)  2/24 (8.33%) 
Psychiatric disorders         
Hallucination † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Insomnia † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Irritability † 1  0/7 (0.00%)  0/8 (0.00%)  2/9 (22.22%)  2/24 (8.33%) 
Renal and urinary disorders         
Pollakiuria † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Respiratory, thoracic and mediastinal disorders         
Cough † 1  3/7 (42.86%)  0/8 (0.00%)  0/9 (0.00%)  3/24 (12.50%) 
Epistaxis † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Nasal congestion † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Nocturnal dyspnoea † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Oropharyngeal pain † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Respiratory tract congestion † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Sneezing † 1  0/7 (0.00%)  2/8 (25.00%)  0/9 (0.00%)  2/24 (8.33%) 
Upper respiratory tract congestion † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Skin and subcutaneous tissue disorders         
Dermatitis † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
Eczema infantile † 1  1/7 (14.29%)  0/8 (0.00%)  0/9 (0.00%)  1/24 (4.17%) 
Hypertrichosis † 1  0/7 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/24 (4.17%) 
Rash † 1  1/7 (14.29%)  1/8 (12.50%)  0/9 (0.00%)  2/24 (8.33%) 
Rash erythematous † 1  0/7 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  1/24 (4.17%) 
1
Term from vocabulary, MedDRA (26.0)
†
Indicates events were collected by systematic assessment
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
Phone: + 1 862 778 8300
EMail: Novartis.email@Novartis.com
Layout table for additonal information
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT04851873    
Other Study ID Numbers: COAV101A12306
2020-005995-37 ( EudraCT Number )
First Submitted: April 6, 2021
First Posted: April 21, 2021
Results First Submitted: November 27, 2023
Results First Posted: January 5, 2024
Last Update Posted: January 5, 2024