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| ID | Type | Description | Link |
|---|---|---|---|
| 2009-016859-22 |
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This trial will assess the safety, efficacy and tolerability of ACZ885 in patients aged 4 years and younger with cryopyrin associated periodic syndromes (CAPS)
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Canakinumab | Experimental | Canakinumab s.c. injection (2 mg/kg) was administered every 8 weeks. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ACZ885 | Drug |
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| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Aged 4 Years or Younger With at Least One Complete Response at Week 56 | 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. | Week 56 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Aged 2 Years or Younger With at Least One Complete Response at Week 56 | 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. |
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Inclusion Criteria:
Exclusion Criteria:
Preterm neonates for whom, in the Investigator's judgment, participation in the study is not deemed appropriate.
History of recurrent and/or evidence of active bacterial, fungal, or viral infections (including HIV).
Patients with immunodeficiency or treatment with immunosuppressive drugs.
Live vaccinations within < or = 3 months prior to screening. No live vaccinations will be allowed throughout the course of this study and up to 3 months following the last dose.
Patients with an increased risk of tuberculosis (TB) infection according to following risk factors:
Participation in another trial within the last 30 days or 5 half-lives of the investigational compound (whichever is longer).
Familial and social conditions rendering regular medical assessment not possible.
Pediatric patients with neutropenia (absolute neutrophil count [ANC] < 1.5 x 10 to the 9th/l)
Other protocol defined inclusion/exclusion criteria may apply
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Brussels | 1200 | Belgium | |||
| Novartis Investigative Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31161734 | Derived | Brogan PA, Hofer M, Kuemmerle-Deschner JB, Kone-Paut I, Roesler J, Kallinich T, Horneff G, Calvo Penades I, Sevilla-Perez B, Goffin L, Lauwerys BR, Lachmann HJ, Uziel Y, Wei X, Laxer RM. Rapid and Sustained Long-Term Efficacy and Safety of Canakinumab in Patients With Cryopyrin-Associated Periodic Syndrome Ages Five Years and Younger. Arthritis Rheumatol. 2019 Nov;71(11):1955-1963. doi: 10.1002/art.41004. Epub 2019 Sep 9. |
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A total of 17 participants were enrolled into the study.
The study was conducted at 14 centers in 7 countries.
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| ID | Title | Description |
|---|---|---|
| FG000 | Canakinumab | Participants received body weight stratified dose of canakinumab 2 milligrams/kilogram (mg/kg) subcutaneous (s.c.) injection every 8 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Week 56 |
| Percentage of Participants With Defined Grades in Physician's Global Assessment Score at Week 56 | 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. | Week 56 |
| Percentage of Participants With Defined Grades in Physician Assessment of Skin Disease at Week 56 | 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. | Week 56 |
| Change From Baseline in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations at Week 56 | 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. | Baseline, Week 56 |
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | 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. | Day 1 (start of study treatment) up to Week 56 (end of study) |
| Percentage of Participants Receiving a Concomitant Vaccination During the Study | 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. | Day 1 (start of study treatment) to Week 56 (end of study) |
| Number of Vaccination Cases With Protective Antibody Levels Following Immunization With Inactivated Vaccines | 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. | Day -14 (prior-vaccination), Day 0 (vaccination), Day 28, Day 57 (post-vaccination) |
| Number of Participants With Anti-canakinumab Antibodies at Week 56 | Immunogenicity assessment included determination of anti-canakinumab (ACZ885) antibodies in serum samples using BIAcore system, with detection based on surface plasmon resonance technique. | Week 56 (End of study) |
| Laken |
| 1020 |
| Belgium |
| Novartis Investigative Site | Toronto | Ontario | M5G 1X8 | Canada |
| Novartis Investigative Site | Le Kremlin-Bicêtre | 94275 | France |
| Novartis Investigative Site | Berlin | 13353 | Germany |
| Novartis Investigative Site | Dresden | 01307 | Germany |
| Novartis Investigative Site | Sankt Augustin | 53757 | Germany |
| Novartis Investigative Site | Tübingen | 72076 | Germany |
| Novartis Investigative Site | Granada | Andalusia | 18012 | Spain |
| Novartis Investigative Site | Valencia | Valencia | 46026 | Spain |
| Novartis Investigative Site | Lausanne | 1011 | Switzerland |
| Novartis Investigative Site | London | WC1N 1EH | United Kingdom |
| COMPLETED |
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| NOT COMPLETED |
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The analysis was performed in Full analysis set population, defined as all participants who received at least one dose of study drug under this study protocol.
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| ID | Title | Description |
|---|---|---|
| BG000 | Canakinumab | Participants received body weight stratified dose of canakinumab 2 mg/kg s.c. injection every 8 weeks. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Age, Customized | Number | participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Participants Aged 4 Years or Younger With at Least One Complete Response at Week 56 | 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. | 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. | Posted | Number | Percentage of participants | Week 56 |
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| Secondary | Percentage of Participants Aged 2 Years or Younger With at Least One Complete Response at Week 56 | 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. | The analysis was performed in FAS population. Here, "Number of participants analysed" signifies participants aged 2 years or younger. | Posted | Number | Percentage of participants | Week 56 |
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| Secondary | Percentage of Participants With Defined Grades in Physician's Global Assessment Score at Week 56 | 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. | The analysis was performed in FAS population. | Posted | Number | Percentage of participants | Week 56 |
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| Secondary | Percentage of Participants With Defined Grades in Physician Assessment of Skin Disease at Week 56 | 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. | The analysis was performed in FAS population. | Posted | Number | Percentage of participants | Week 56 |
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| Secondary | Change From Baseline in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations at Week 56 | 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. | The analysis was performed in FAS population. Here 'n' signifies those participants with evaluable measurements at both baseline and the post-baseline visit. | Posted | Mean | Standard Deviation | mg/L | Baseline, Week 56 |
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| Secondary | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | 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. | 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. | Posted | Number | participants | Day 1 (start of study treatment) up to Week 56 (end of study) |
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| Secondary | Percentage of Participants Receiving a Concomitant Vaccination During the Study | 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. | The analysis was performed in the FAS population. | Posted | Number | Percentage of participants | Day 1 (start of study treatment) to Week 56 (end of study) |
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| Secondary | Number of Vaccination Cases With Protective Antibody Levels Following Immunization With Inactivated Vaccines | 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. | 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. | Posted | Number | vaccination cases | Day -14 (prior-vaccination), Day 0 (vaccination), Day 28, Day 57 (post-vaccination) |
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| Secondary | Number of Participants With Anti-canakinumab Antibodies at Week 56 | Immunogenicity assessment included determination of anti-canakinumab (ACZ885) antibodies in serum samples using BIAcore system, with detection based on surface plasmon resonance technique. | 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. | Posted | Number | participants | Week 56 (End of study) |
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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.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Canakinumab | Participants received body weight stratified dose of canakinumab 2 mg/kg s.c. injection every 8 weeks. | 4 | 17 | 17 | 17 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cryopyrin associated periodic syndrome | Congenital, familial and genetic disorders | MedDRA | Systematic Assessment |
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| Cryptorchism | Congenital, familial and genetic disorders | MedDRA | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Influenza | Infections and infestations | MedDRA | Systematic Assessment |
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| Lung infection | Infections and infestations | MedDRA | Systematic Assessment |
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| Wound infection staphylococcal | Infections and infestations | MedDRA | Systematic Assessment |
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| Femur fracture | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Eosinophilia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
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| Cryopyrin associated periodic syndrome | Congenital, familial and genetic disorders | MedDRA | Systematic Assessment |
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| Conductive deafness | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
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| Iridocyclitis | Eye disorders | MedDRA | Systematic Assessment |
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| Papilloedema | Eye disorders | MedDRA | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Gastritis | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Stomatitis | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Toothache | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Fatigue | General disorders | MedDRA | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA | Systematic Assessment |
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| Drug hypersensitivity | Immune system disorders | MedDRA | Systematic Assessment |
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| Bronchitis | Infections and infestations | MedDRA | Systematic Assessment |
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| Conjunctivitis | Infections and infestations | MedDRA | Systematic Assessment |
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| Ear infection | Infections and infestations | MedDRA | Systematic Assessment |
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| Eczema infected | Infections and infestations | MedDRA | Systematic Assessment |
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| Enterobiasis | Infections and infestations | MedDRA | Systematic Assessment |
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| Escherichia urinary tract infection | Infections and infestations | MedDRA | Systematic Assessment |
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| Gastritis viral | Infections and infestations | MedDRA | Systematic Assessment |
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| Gastroenteritis | Infections and infestations | MedDRA | Systematic Assessment |
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| Gastroenteritis viral | Infections and infestations | MedDRA | Systematic Assessment |
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| Laryngitis | Infections and infestations | MedDRA | Systematic Assessment |
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| Molluscum contagiosum | Infections and infestations | MedDRA | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA | Systematic Assessment |
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| Oral fungal infection | Infections and infestations | MedDRA | Systematic Assessment |
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| Oral herpes | Infections and infestations | MedDRA | Systematic Assessment |
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| Otitis externa | Infections and infestations | MedDRA | Systematic Assessment |
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| Pharyngitis | Infections and infestations | MedDRA | Systematic Assessment |
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| Rhinitis | Infections and infestations | MedDRA | Systematic Assessment |
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| Tinea pedis | Infections and infestations | MedDRA | Systematic Assessment |
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| Tonsillitis | Infections and infestations | MedDRA | Systematic Assessment |
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| Tonsillitis bacterial | Infections and infestations | MedDRA | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA | Systematic Assessment |
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| Varicella | Infections and infestations | MedDRA | Systematic Assessment |
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| Skin abrasion | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| CSF white blood cell count increased | Investigations | MedDRA | Systematic Assessment |
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| Transaminases increased | Investigations | MedDRA | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA | Systematic Assessment |
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| Motor developmental delay | Nervous system disorders | MedDRA | Systematic Assessment |
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| Speech disorder developmental | Nervous system disorders | MedDRA | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Dermatitis diaper | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Pruritus generalised | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Hearing aid therapy | Surgical and medical procedures | MedDRA | Systematic Assessment |
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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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | 862 -778 -8300 |
| ID | Term |
|---|---|
| D056587 | Cryopyrin-Associated Periodic Syndromes |
| C566739 | Multiple Pterygium Syndrome, Autosomal Dominant |
| ID | Term |
|---|---|
| D056660 | Hereditary Autoinflammatory Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012873 | Skin Diseases, Genetic |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D000094482 | Chronic Inducible Urticaria |
| D000080223 | Chronic Urticaria |
| D014581 | Urticaria |
| D017445 | Skin Diseases, Vascular |
| D000096703 | Cold Urticaria |
| D006969 | Hypersensitivity, Immediate |
| D006967 | Hypersensitivity |
| D007154 | Immune System Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| C541220 | canakinumab |
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