Trial Outcomes & Findings for Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 (NCT NCT03461289)
NCT ID: NCT03461289
Last Updated: 2026-01-26
Results Overview
Independent sitting is defined by the World Health Organization Multicentre Growth Reference Study, confirmed by video recording, as a participant who sits up straight with head erect for at least 10 seconds; participant does not use arms or hands to balance body or support position.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
33 participants
Primary outcome timeframe
From Day 1 up to 18 Months of Age Visit (Up to a Maximum of Approximately 17 Months)
Results posted on
2026-01-26
Participant Flow
Participant milestones
| Measure |
Onasemnogene Abeparvovec-xioi
Participants received a single dose of onasemnogene abeparvovec-xioi administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 × 10\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study.
|
|---|---|
|
Overall Study
STARTED
|
33
|
|
Overall Study
COMPLETED
|
32
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Onasemnogene Abeparvovec-xioi
Participants received a single dose of onasemnogene abeparvovec-xioi administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 × 10\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study.
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|---|---|
|
Overall Study
Death
|
1
|
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Onasemnogene Abeparvovec-xioi
n=33 Participants
Participants received a single dose of onasemnogene abeparvovec-xioi administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 × 10\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study.
|
|---|---|
|
Age, Categorical
<=18 years
|
33 Participants
n=33 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=33 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=33 Participants
|
|
Age, Continuous
|
4.055 months
STANDARD_DEVIATION 1.2799 • n=33 Participants
|
|
Sex: Female, Male
Female
|
19 Participants
n=33 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=33 Participants
|
|
Region of Enrollment
Belgium
|
4 participants
n=33 Participants
|
|
Region of Enrollment
Italy
|
23 participants
n=33 Participants
|
|
Region of Enrollment
United Kingdom
|
4 participants
n=33 Participants
|
|
Region of Enrollment
France
|
2 participants
n=33 Participants
|
PRIMARY outcome
Timeframe: From Day 1 up to 18 Months of Age Visit (Up to a Maximum of Approximately 17 Months)Population: The analysis population was the Intent-to-Treat (ITT) population which consisted of symptomatic participants with bi-allelic deletion of SMN1 (exon 7/8 common homozygous deletions) and 2 copies of SMN2 without the known gene modifier mutation (c.859G\>C) who received an IV infusion of onasemnogene abeparvovec-xioi at less than 180 days of age.
Independent sitting is defined by the World Health Organization Multicentre Growth Reference Study, confirmed by video recording, as a participant who sits up straight with head erect for at least 10 seconds; participant does not use arms or hands to balance body or support position.
Outcome measures
| Measure |
Onasemnogene Abeparvovec-xioi
n=32 Participants
Participants received a single dose of onasemnogene abeparvovec-xioi administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 × 10\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study.
|
|---|---|
|
Number of Participants Who Achieve Independent Sitting for at Least 10 Seconds
|
14 Participants
|
SECONDARY outcome
Timeframe: Up to 14 months of agePopulation: The analysis population was the ITT population which consisted of symptomatic participants with bi-allelic deletion of SMN1 (exon 7/8 common homozygous deletions) and 2 copies of SMN2 without the known gene modifier mutation (c.859G\>C) who received an IV infusion of onasemnogene abeparvovec-xioi at less than 180 days of age.
Event-free survival at 14 months of age was defined as the number of participants who did not die, did not require permanent ventilation and did not withdraw from the study by 14 months of age.
Outcome measures
| Measure |
Onasemnogene Abeparvovec-xioi
n=32 Participants
Participants received a single dose of onasemnogene abeparvovec-xioi administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 × 10\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study.
|
|---|---|
|
Event-free Survival at 14 Months of Age
|
31 Participants
|
Adverse Events
Onasemnogene Abeparvovec-xioi
Serious events: 19 serious events
Other events: 32 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Onasemnogene Abeparvovec-xioi
n=33 participants at risk
Participants received a single dose of onasemnogene abeparvovec-xioi administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 × 10\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study.
|
|---|---|
|
Surgical and medical procedures
Gastrostomy
|
6.1%
2/33 • Number of events 3 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Surgical and medical procedures
Hospitalisation
|
3.0%
1/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Alanine aminotransferase increased
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Aspartate aminotransferase increased
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Coagulation test abnormal
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Pulmonary function test
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Cardiac disorders
Bradycardia
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Respiratory, thoracic and mediastinal disorders
Increased bronchial secretion
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
3.0%
1/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Nervous system disorders
Hypoxic-ischaemic encephalopathy
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Nervous system disorders
Loss of consciousness
|
3.0%
1/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
General disorders
Pyrexia
|
12.1%
4/33 • Number of events 4 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Gastrointestinal disorders
Dysphagia
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Gastrointestinal disorders
Vomiting
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Hepatobiliary disorders
Hypertransaminasaemia
|
3.0%
1/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Metabolism and nutrition disorders
Feeding disorder
|
6.1%
2/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Metabolism and nutrition disorders
Hypernatraemia
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Pneumonia
|
15.2%
5/33 • Number of events 9 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Gastroenteritis
|
9.1%
3/33 • Number of events 3 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Respiratory tract infection
|
9.1%
3/33 • Number of events 3 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Upper respiratory tract infection
|
9.1%
3/33 • Number of events 3 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Bronchiolitis
|
6.1%
2/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Lower respiratory tract infection
|
6.1%
2/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Respiratory syncytial virus infection
|
6.1%
2/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Exanthema subitum
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Nasopharyngitis
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Rhinitis
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Rhinovirus infection
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Urinary tract infection
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Viral infection
|
3.0%
1/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
Other adverse events
| Measure |
Onasemnogene Abeparvovec-xioi
n=33 participants at risk
Participants received a single dose of onasemnogene abeparvovec-xioi administered as an intravenous (IV) infusion over 60 minutes at a dose of 1.1 × 10\^14 vg/kg (vector genome per kilogram) on Day 1 of the overall study.
|
|---|---|
|
Injury, poisoning and procedural complications
Postoperative ileus
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Injury, poisoning and procedural complications
Stoma site haemorrhage
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Injury, poisoning and procedural complications
Stoma site inflammation
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Candida infection
|
6.1%
2/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Injury, poisoning and procedural complications
Vaccination complication
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Alanine aminotransferase increased
|
27.3%
9/33 • Number of events 11 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Aspartate aminotransferase increased
|
24.2%
8/33 • Number of events 9 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Oxygen saturation decreased
|
6.1%
2/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Troponin T increased
|
6.1%
2/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Blood alkaline phosphatase increased
|
3.0%
1/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Vascular disorders
Hypertension
|
12.1%
4/33 • Number of events 4 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Vascular disorders
Hypotension
|
3.0%
1/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Immune system disorders
Drug hypersensitivity
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
General disorders
Pyrexia
|
60.6%
20/33 • Number of events 29 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
General disorders
Crying
|
3.0%
1/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
General disorders
Extravasation
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
General disorders
Generalised oedema
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
General disorders
Hypothermia
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
General disorders
Necrosis
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Psychiatric disorders
Irritability
|
6.1%
2/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Psychiatric disorders
Nervousness
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Psychiatric disorders
Psychomotor retardation
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Injury, poisoning and procedural complications
Joint dislocation
|
9.1%
3/33 • Number of events 3 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Injury, poisoning and procedural complications
Contusion
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Injury, poisoning and procedural complications
Femur fracture
|
3.0%
1/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Injury, poisoning and procedural complications
Hand fracture
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Blood creatine phosphokinase MB increased
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Blood phosphorus decreased
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Blood urine present
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Body temperature increased
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Breath sounds
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Gamma-glutamyltransferase increased
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Haemoglobin decreased
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Haemophilus test positive
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Platelet count increased
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Respiratory syncytial virus test positive
|
3.0%
1/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Respirovirus test positive
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Investigations
Staphylococcus test positive
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Cardiac disorders
Tachycardia
|
6.1%
2/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Cardiac disorders
Bradycardia
|
3.0%
1/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Cardiac disorders
Cyanosis
|
3.0%
1/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Congenital, familial and genetic disorders
Cryptorchism
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
18.2%
6/33 • Number of events 7 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
6.1%
2/33 • Number of events 3 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Respiratory, thoracic and mediastinal disorders
Lung consolidation
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
3.0%
1/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory disorder
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Respiratory, thoracic and mediastinal disorders
Tachypnoea
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Respiratory, thoracic and mediastinal disorders
Use of accessory respiratory muscles
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Nervous system disorders
Dizziness
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Nervous system disorders
Hypersomnia
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Nervous system disorders
Seizure
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Eye disorders
Strabismus
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Ear and labyrinth disorders
Tympanic membrane hyperaemia
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Gastrointestinal disorders
Vomiting
|
24.2%
8/33 • Number of events 15 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Gastrointestinal disorders
Constipation
|
21.2%
7/33 • Number of events 7 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Gastrointestinal disorders
Diarrhoea
|
15.2%
5/33 • Number of events 8 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
12.1%
4/33 • Number of events 4 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Gastrointestinal disorders
Teething
|
9.1%
3/33 • Number of events 3 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Gastrointestinal disorders
Salivary hypersecretion
|
6.1%
2/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Gastrointestinal disorders
Abdominal pain
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Gastrointestinal disorders
Duodenal ulcer
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Gastrointestinal disorders
Gastritis
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Gastrointestinal disorders
Oral pain
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Renal and urinary disorders
Leukocyturia
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Hepatobiliary disorders
Hypertransaminasaemia
|
24.2%
8/33 • Number of events 25 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Hepatobiliary disorders
Hepatic steatosis
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Product Issues
Device occlusion
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
9.1%
3/33 • Number of events 3 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Skin and subcutaneous tissue disorders
Rash
|
9.1%
3/33 • Number of events 3 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Skin and subcutaneous tissue disorders
Acne infantile
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Skin and subcutaneous tissue disorders
Dermatitis allergic
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Skin and subcutaneous tissue disorders
Dermatitis atopic
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Skin and subcutaneous tissue disorders
Excessive granulation tissue
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Skin and subcutaneous tissue disorders
Ingrowing nail
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Skin and subcutaneous tissue disorders
Skin reaction
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
6.1%
2/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Musculoskeletal and connective tissue disorders
Muscle contracture
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Musculoskeletal and connective tissue disorders
Torticollis
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Endocrine disorders
Adrenal insufficiency
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Metabolism and nutrition disorders
Failure to thrive
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Metabolism and nutrition disorders
Feeding disorder
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Metabolism and nutrition disorders
Hyperphosphatasaemia
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Upper respiratory tract infection
|
30.3%
10/33 • Number of events 16 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Nasopharyngitis
|
12.1%
4/33 • Number of events 8 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Respiratory tract infection
|
12.1%
4/33 • Number of events 4 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Ear infection
|
9.1%
3/33 • Number of events 3 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Gastroenteritis
|
9.1%
3/33 • Number of events 3 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Impetigo
|
6.1%
2/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Pneumonia
|
6.1%
2/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Rhinitis
|
6.1%
2/33 • Number of events 3 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Urinary tract infection
|
6.1%
2/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Bacteriuria
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Bronchitis
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Conjunctivitis
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Cystitis
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Dermatitis infected
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Fungal infection
|
3.0%
1/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Gastroenteritis viral
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Hordeolum
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Influenza
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Laryngitis
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Lower respiratory tract infection
|
3.0%
1/33 • Number of events 2 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Otitis media
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Pharyngitis bacterial
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Respiratory tract infection viral
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Roseola
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Urinary tract infection bacterial
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
|
Infections and infestations
Varicella
|
3.0%
1/33 • Number of events 1 • From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months)
Non-serious treatment emergent AEs were collected from Day 1 until 18 months of age visit.
|
Additional Information
EMEA Medical Information
Novartis Gene Therapies EU Limited
Phone: +353 (1) 566-2364
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Sponsor can review results communications prior to public release \& has the right to request changes to communications regarding trial results for a period of 30 to 60 days with an additional 6 months, if needed, from the time submitted to the Sponsor for review in order to either remove references to Sponsor's Confidential Information or delay such results communications to permit Sponsor to obtain appropriate Intellectual Property protection as set forth in each of the agreements.
- Publication restrictions are in place
Restriction type: OTHER