Trial Outcomes & Findings for Efficacy, Safety and Tolerability of ACZ885 in Pediatric Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease (NCT NCT01302860)
NCT ID: NCT01302860
Last Updated: 2017-03-29
Results Overview
Complete response was defined as clinical remission and serological remission. Clinical remission was defined as Physician global assessment of auto-inflammatory disease activity as absent or minimal (using a 5-point scale ranging from absent to severe) and assessment of skin disease as absent or minimal (using a 5-point scale ranging from absent to severe). Serological remission was defined as C reactive protein (CRP) or Serum amyloid A protein (SAA) to be less than (\<) 15 milligram per liter (mg/L) and \<10 mg/L respectively.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
17 participants
Primary outcome timeframe
Week 56
Results posted on
2017-03-29
Participant Flow
The study was conducted at 14 centers in 7 countries.
A total of 17 participants were enrolled into the study.
Participant milestones
| Measure |
Canakinumab
Participants received body weight stratified dose of canakinumab 2 milligrams/kilogram (mg/kg) subcutaneous (s.c.) injection every 8 weeks.
|
|---|---|
|
Overall Study
STARTED
|
17
|
|
Overall Study
COMPLETED
|
17
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Efficacy, Safety and Tolerability of ACZ885 in Pediatric Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
Baseline characteristics by cohort
| Measure |
Canakinumab
n=17 Participants
Participants received body weight stratified dose of canakinumab 2 mg/kg s.c. injection every 8 weeks.
|
|---|---|
|
Age, Continuous
|
1.9 years
STANDARD_DEVIATION 1.39 • n=5 Participants
|
|
Age, Customized
Infants and toddlers (28 days-23 months)
|
6 participants
n=5 Participants
|
|
Age, Customized
Children (2-5 years)
|
11 participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Week 56Population: The analysis was performed in Full analysis set (FAS), defined as all participants who received at least one dose of study drug under this study protocol.
Complete response was defined as clinical remission and serological remission. Clinical remission was defined as Physician global assessment of auto-inflammatory disease activity as absent or minimal (using a 5-point scale ranging from absent to severe) and assessment of skin disease as absent or minimal (using a 5-point scale ranging from absent to severe). Serological remission was defined as C reactive protein (CRP) or Serum amyloid A protein (SAA) to be less than (\<) 15 milligram per liter (mg/L) and \<10 mg/L respectively.
Outcome measures
| Measure |
Canakinumab
n=17 Participants
Participants received body weight stratified dose of canakinumab 2 mg/kg s.c. injection every 8 weeks.
|
|---|---|
|
Percentage of Participants Aged 4 Years or Younger With at Least One Complete Response at Week 56
|
94.1 Percentage of participants
|
SECONDARY outcome
Timeframe: Week 56Population: The analysis was performed in FAS population. Here, "Number of participants analysed" signifies participants aged 2 years or younger.
Complete response was defined as clinical remission and serological remission. Clinical remission was defined as Physician global assessment of auto-inflammatory disease activity as absent or minimal (using a 5-point scale ranging from absent to severe) and assessment of skin disease as absent or minimal (using a 5-point scale ranging from absent to severe). Serological remission was defined as CRP or SAA to be \<15 mg/L and \<10 mg/L respectively.
Outcome measures
| Measure |
Canakinumab
n=10 Participants
Participants received body weight stratified dose of canakinumab 2 mg/kg s.c. injection every 8 weeks.
|
|---|---|
|
Percentage of Participants Aged 2 Years or Younger With at Least One Complete Response at Week 56
|
90 Percentage of participants
|
SECONDARY outcome
Timeframe: Week 56Population: The analysis was performed in FAS population.
Participants were assessed based by physician on Physician's Global Assessment measured on a 5--point scale for auto inflammatory disease activity as: 0 = None/absent; 1 = Minimal; 2 = Mild; 3 = Moderate; 4 = Severe.
Outcome measures
| Measure |
Canakinumab
n=17 Participants
Participants received body weight stratified dose of canakinumab 2 mg/kg s.c. injection every 8 weeks.
|
|---|---|
|
Percentage of Participants With Defined Grades in Physician's Global Assessment Score at Week 56
Absent
|
70.6 Percentage of participants
|
|
Percentage of Participants With Defined Grades in Physician's Global Assessment Score at Week 56
Minimal
|
23.5 Percentage of participants
|
|
Percentage of Participants With Defined Grades in Physician's Global Assessment Score at Week 56
Mild
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades in Physician's Global Assessment Score at Week 56
Moderate
|
5.9 Percentage of participants
|
|
Percentage of Participants With Defined Grades in Physician's Global Assessment Score at Week 56
Severe
|
0 Percentage of participants
|
SECONDARY outcome
Timeframe: Week 56Population: The analysis was performed in FAS population.
Participants were assessed by physician for skin disease (urticarial skin rash) measured on a 5--point scale as: 0 = None/absent; 1 = Minimal; 2 = Mild; 3 = Moderate; 4 = Severe.
Outcome measures
| Measure |
Canakinumab
n=17 Participants
Participants received body weight stratified dose of canakinumab 2 mg/kg s.c. injection every 8 weeks.
|
|---|---|
|
Percentage of Participants With Defined Grades in Physician Assessment of Skin Disease at Week 56
Absent
|
82.4 Percentage of participants
|
|
Percentage of Participants With Defined Grades in Physician Assessment of Skin Disease at Week 56
Minimal
|
5.9 Percentage of participants
|
|
Percentage of Participants With Defined Grades in Physician Assessment of Skin Disease at Week 56
Mild
|
11.8 Percentage of participants
|
|
Percentage of Participants With Defined Grades in Physician Assessment of Skin Disease at Week 56
Moderate
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades in Physician Assessment of Skin Disease at Week 56
Severe
|
0 Percentage of participants
|
SECONDARY outcome
Timeframe: Baseline, Week 56Population: The analysis was performed in FAS population. Here 'n' signifies those participants with evaluable measurements at both baseline and the post-baseline visit.
The CRP and SAA were used as inflammatory markers. The target level concentrations for CRP and SAA was ≤15 mg/L and ≤10 mg/L, respectively. Negative change in concentration of inflammatory markers indicated improvement.
Outcome measures
| Measure |
Canakinumab
n=17 Participants
Participants received body weight stratified dose of canakinumab 2 mg/kg s.c. injection every 8 weeks.
|
|---|---|
|
Change From Baseline in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations at Week 56
CRP (n=14)
|
-5.4 mg/L
Standard Deviation 6.28
|
|
Change From Baseline in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations at Week 56
SAA (n=16)
|
-54.4 mg/L
Standard Deviation 133.81
|
SECONDARY outcome
Timeframe: Day 1 (start of study treatment) up to Week 56 (end of study)Population: The analysis was performed in the safety set population defined as participants who received at least one dose of study drug. Here, 'n' signifies participants evaluable for this measure at specified time points for each group, respectively.
Adverse events (AEs) were defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events (SAEs) were defined as any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalization, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgement of investigators represent significant hazards.
Outcome measures
| Measure |
Canakinumab
n=17 Participants
Participants received body weight stratified dose of canakinumab 2 mg/kg s.c. injection every 8 weeks.
|
|---|---|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
AEs (Participants <=2 years, n=10)
|
10 participants
|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
AEs (Overall, n=17)
|
17 participants
|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
SAEs (Participants <=2 years, n=10)
|
3 participants
|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
SAEs (Overall, n=17)
|
4 participants
|
SECONDARY outcome
Timeframe: Day 1 (start of study treatment) to Week 56 (end of study)Population: The analysis was performed in the FAS population.
Participants received any one of the following inactivated vaccines as per the immunization program: Corynebacterium diphtheria, Bordetella pertussis, Neisseria meningitidis, Clostridium tetani, Influenza type A, Influenza type B, Haemophilus influenza B, Streptococcus pneumoniae, or Hepatitis B were determined.
Outcome measures
| Measure |
Canakinumab
n=17 Participants
Participants received body weight stratified dose of canakinumab 2 mg/kg s.c. injection every 8 weeks.
|
|---|---|
|
Percentage of Participants Receiving a Concomitant Vaccination During the Study
|
41.2 Percentage of participants
|
SECONDARY outcome
Timeframe: Day -14 (prior-vaccination), Day 0 (vaccination), Day 28, Day 57 (post-vaccination)Population: The analysis was performed in the FAS population. Here, "Number of participants analysed" signifies evaluable participants who received a total of 31 vaccinations during the study.
Participants who received any inactivated vaccines during the study were assessed for their ability to attain protective antibody levels against the vaccine (antigen) post immunization. Participants vaccinations were not assessed for a response if the antibody titre was already sufficient at pre-dose and maintained during the study.
Outcome measures
| Measure |
Canakinumab
n=7 Participants
Participants received body weight stratified dose of canakinumab 2 mg/kg s.c. injection every 8 weeks.
|
|---|---|
|
Number of Vaccination Cases With Protective Antibody Levels Following Immunization With Inactivated Vaccines
Positive response for antibody levels
|
18 vaccination cases
|
|
Number of Vaccination Cases With Protective Antibody Levels Following Immunization With Inactivated Vaccines
No pre-dose antibody levels
|
13 vaccination cases
|
SECONDARY outcome
Timeframe: Week 56 (End of study)Population: The analysis was performed in the safety set population. Here, 'Number of participants analysed' signifies participants who had immunogenicity samples taken and analyzed during the study.
Immunogenicity assessment included determination of anti-canakinumab (ACZ885) antibodies in serum samples using BIAcore system, with detection based on surface plasmon resonance technique.
Outcome measures
| Measure |
Canakinumab
n=15 Participants
Participants received body weight stratified dose of canakinumab 2 mg/kg s.c. injection every 8 weeks.
|
|---|---|
|
Number of Participants With Anti-canakinumab Antibodies at Week 56
|
0 participants
|
Adverse Events
Canakinumab
Serious events: 4 serious events
Other events: 17 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Canakinumab
n=17 participants at risk
Participants received body weight stratified dose of canakinumab 2 mg/kg s.c. injection every 8 weeks.
|
|---|---|
|
Congenital, familial and genetic disorders
Cryopyrin associated periodic syndrome
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Congenital, familial and genetic disorders
Cryptorchism
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Gastrointestinal disorders
Diarrhoea
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Gastrointestinal disorders
Vomiting
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Influenza
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Lung infection
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Wound infection staphylococcal
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Injury, poisoning and procedural complications
Femur fracture
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
Other adverse events
| Measure |
Canakinumab
n=17 participants at risk
Participants received body weight stratified dose of canakinumab 2 mg/kg s.c. injection every 8 weeks.
|
|---|---|
|
Blood and lymphatic system disorders
Eosinophilia
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Congenital, familial and genetic disorders
Cryopyrin associated periodic syndrome
|
11.8%
2/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Ear and labyrinth disorders
Conductive deafness
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Eye disorders
Iridocyclitis
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Eye disorders
Papilloedema
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Gastrointestinal disorders
Abdominal pain
|
17.6%
3/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Gastrointestinal disorders
Diarrhoea
|
23.5%
4/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Gastrointestinal disorders
Gastritis
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Gastrointestinal disorders
Stomatitis
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Gastrointestinal disorders
Toothache
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Gastrointestinal disorders
Vomiting
|
23.5%
4/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
General disorders
Fatigue
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
General disorders
Pyrexia
|
35.3%
6/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Immune system disorders
Drug hypersensitivity
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Bronchitis
|
23.5%
4/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Conjunctivitis
|
23.5%
4/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Ear infection
|
23.5%
4/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Eczema infected
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Enterobiasis
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Escherichia urinary tract infection
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Gastritis viral
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Gastroenteritis
|
17.6%
3/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Gastroenteritis viral
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Laryngitis
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Molluscum contagiosum
|
11.8%
2/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Nasopharyngitis
|
41.2%
7/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Oral fungal infection
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Oral herpes
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Otitis externa
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Pharyngitis
|
11.8%
2/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Rhinitis
|
35.3%
6/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Tinea pedis
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Tonsillitis
|
11.8%
2/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Tonsillitis bacterial
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Upper respiratory tract infection
|
41.2%
7/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Urinary tract infection
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Infections and infestations
Varicella
|
23.5%
4/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Investigations
CSF white blood cell count increased
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Investigations
Transaminases increased
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
11.8%
2/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Nervous system disorders
Headache
|
23.5%
4/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Nervous system disorders
Motor developmental delay
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Nervous system disorders
Speech disorder developmental
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
23.5%
4/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Skin and subcutaneous tissue disorders
Pruritus generalised
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Skin and subcutaneous tissue disorders
Rash
|
11.8%
2/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
|
Surgical and medical procedures
Hearing aid therapy
|
5.9%
1/17 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV), for up to 4 years. All other adverse events are monitored from First Participant First Treatment until LPLV, for up to 4 years.
|
Additional Information
Study Director
Novartis Pharmaceuticals
Phone: 862 -778 -8300
Results disclosure agreements
- Principal investigator is a sponsor employee 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 (ie, data from all sites) in the clinical trial.
- Publication restrictions are in place
Restriction type: OTHER