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| ID | Type | Description | Link |
|---|---|---|---|
| 2011-005154-57 | EudraCT Number |
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This trial will provide long-term safety, efficacy and tolerability of ACZ885 in CAPS patients that completed the CACZ885D2307 study
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| canakinumab | Experimental | Patients will receive a standard dose at an equivalent of 2 mg/kg s.c. of canakinumab (ACZ885) every 8 weeks. Possible dose and/or dosing regimen adjustments that can be administered include: 4 mg/kg s.c. (every 4 to 8 weeks) 6 mg/kg s.c. (every 4 to 8 weeks) 8 mg/kg s.c. (every 4 to 8 weeks) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ACZ885 | Biological |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| The Percentage of Participants Without Disease Relapse as Determined by the Physician's Global Assessment of Autoinflammatory Disease Activity, Assessment of Skin Disease and Serological Inflammation Markers. | Disease relapse following complete response is defined as inflammation markers: C-Reactive Protein (CRP) and/or Serum Amyloid A (SAA) result > 30 mg/L AND Physician's Global Assessment of Autoinflammatory Disease Activity > minimal or Physician's Global Assessment >= minimal AND Skin Disease Assessment > minimal. Physician's Global Assessment of Autoinflammatory Disease Activity and Skin Disease Assessment (urticarial skin rash) are completed by the investigator using a 5 point rating scale: absent, minimal, mild, moderate and severe. | Week /80, 104, 128, and 152 (A minimum of 6 months and maximum of 24 months) |
| Measure | Description | Time Frame |
|---|---|---|
| Immunogenicity of Canakinumab (ACZ885). Number of Participants With Anti-canakinumab Antibodies | Immunogenicity assessment included determination of anti-canakinumab (ACZ885) antibodies in serum samples using BIAcore system, with detection based on surface plasmon resonance technique. | minimum of 6 months and maximum of 24 months |
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Inclusion criteria:
Exclusion criteria:
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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| ID | Title | Description |
|---|---|---|
| FG000 | Canakinumab | Patients will receive a standard dose at an equivalent of 2 mg/kg s.c. of canakinumab (ACZ885) every 8 weeks. Possible dose and/or dosing regimen adjustments that can be administered include: 4 mg/kg s.c. (every 4 to 8 weeks) 6 mg/kg s.c. (every 4 to 8 weeks) 8 mg/kg s.c. (every 4 to 8 weeks) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations |
CRP and SAA were used as serologic 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. |
| Week 0, 80, 104, 128 and 152, last assessment |
| Frequency Counts of Physician's Global Assessment of Autoinflammatory Disease and Skin Disease | 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 | minimum of 6 months and maximum of 24 months |
| 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. | pre-vaccine dose, Day 28 post-vaccine |
| 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 | Saint 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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|
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| ID | Title | Description |
|---|---|---|
| BG000 | Canakinumab | Patients will receive a standard dose at an equivalent of 2 mg/kg s.c. of canakinumab (ACZ885) every 8 weeks. Possible dose and/or dosing regimen adjustments that can be administered include: 4 mg/kg s.c. (every 4 to 8 weeks) 6 mg/kg s.c. (every 4 to 8 weeks) 8 mg/kg s.c. (every 4 to 8 weeks) |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
|
| 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 | The Percentage of Participants Without Disease Relapse as Determined by the Physician's Global Assessment of Autoinflammatory Disease Activity, Assessment of Skin Disease and Serological Inflammation Markers. | Disease relapse following complete response is defined as inflammation markers: C-Reactive Protein (CRP) and/or Serum Amyloid A (SAA) result > 30 mg/L AND Physician's Global Assessment of Autoinflammatory Disease Activity > minimal or Physician's Global Assessment >= minimal AND Skin Disease Assessment > minimal. Physician's Global Assessment of Autoinflammatory Disease Activity and Skin Disease Assessment (urticarial skin rash) are completed by the investigator using a 5 point rating scale: absent, minimal, mild, moderate and severe. | Extension Full analysis set (FAS) consisted of all patients who received at least one dose of study drug in the extension study | Posted | Number | Percentage of participants | Week /80, 104, 128, and 152 (A minimum of 6 months and maximum of 24 months) |
|
|
| ||||||||||||||||||||||||||
| Secondary | Immunogenicity of Canakinumab (ACZ885). Number of Participants With Anti-canakinumab Antibodies | Immunogenicity assessment included determination of anti-canakinumab (ACZ885) antibodies in serum samples using BIAcore system, with detection based on surface plasmon resonance technique. | Extension Safety set consisted of all patients from the core study who received at least one dose of study drug in the extension study and had at least one post-treatment safety assessment. Of note, the statement that a patient had no AE also constituted a safety assessment. | Posted | Number | Participants | minimum of 6 months and maximum of 24 months |
|
| |||||||||||||||||||||||||||
| Secondary | Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations | CRP and SAA were used as serologic 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. | Extension Full analysis set (FAS) consisted of all patients who received at least one dose of study drug in the extension study | Posted | Mean | Standard Deviation | (mg/L) | Week 0, 80, 104, 128 and 152, last assessment |
|
| ||||||||||||||||||||||||||
| Secondary | Frequency Counts of Physician's Global Assessment of Autoinflammatory Disease and Skin Disease | 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 | Extension Full analysis set (FAS) consisted of all patients who received at least one dose of study drug in the extension study | Posted | Number | Percentage of participants | minimum of 6 months and maximum of 24 months |
|
| |||||||||||||||||||||||||||
| 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. | Extension Full analysis set (FAS) consisted of all patients who received at least one dose of study drug in the extension study. Out of 20 unique patient-vaccination cases, 17 cases were assessable for a vaccination response due to availability of pre dose antibody titer. | Posted | Number | vaccination cases | pre-vaccine dose, Day 28 post-vaccine | patient-vaccination cases | patient-vaccination cases |
|
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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 | Patients will receive a standard dose at an equivalent of 2 mg/kg s.c. of canakinumab (ACZ885) every 8 weeks. Possible dose and/or dosing regimen adjustments that can be administered include: 4 mg/kg s.c. (every 4 to 8 weeks) 6 mg/kg s.c. (every 4 to 8 weeks) 8 mg/kg s.c. (every 4 to 8 weeks) | 8 | 17 | 16 | 17 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Conductive deafness | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Papillitis | General disorders | MedDRA | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Meningitis aseptic | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Eosinophilia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Angina pectoris | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Cryopyrin associated periodic syndrome | Congenital, familial and genetic disorders | MedDRA | Systematic Assessment |
| |
| Conductive deafness | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
| |
| Eye pruritus | Eye disorders | MedDRA | Systematic Assessment |
| |
| Iridocyclitis | Eye disorders | MedDRA | Systematic Assessment |
| |
| Papilloedema | Eye disorders | MedDRA | Systematic Assessment |
| |
| Uveitis | Eye disorders | MedDRA | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Aphthous stomatitis | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Gingival hypertrophy | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Mouth ulceration | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA | Systematic Assessment |
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| Milk allergy | Immune system disorders | MedDRA | Systematic Assessment |
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| Seasonal allergy | Immune system disorders | MedDRA | Systematic Assessment |
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| Abscess limb | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Cystitis | 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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| Gastroenteritis | Infections and infestations | MedDRA | Systematic Assessment |
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| Hand-foot-and-mouth disease | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Impetigo | Infections and infestations | MedDRA | Systematic Assessment |
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| Laryngitis | Infections and infestations | MedDRA | Systematic Assessment |
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| Lice infestation | Infections and infestations | MedDRA | Systematic Assessment |
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| Meningitis aseptic | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Molluscum contagiosum | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA | Systematic Assessment |
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| Paronychia | Infections and infestations | MedDRA | Systematic Assessment |
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| Pharyngitis streptococcal | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Vulval abscess | Infections and infestations | MedDRA | Systematic Assessment |
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| Arthropod bite | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Craniocerebral injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
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| Body temperature increased | Investigations | MedDRA | Systematic Assessment |
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| C-reactive protein increased | Investigations | MedDRA | Systematic Assessment |
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| CSF white blood cell count increased | Investigations | MedDRA | Systematic Assessment |
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| Serum amyloid A protein increased | Investigations | MedDRA | Systematic Assessment |
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| Transaminases increased | Investigations | MedDRA | Systematic Assessment |
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| Tympanometry abnormal | Investigations | MedDRA | Systematic Assessment |
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| Decreased appetite | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
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| Joint swelling | 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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| Burning sensation | Nervous system disorders | MedDRA | Systematic Assessment |
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| Cerebral ventricle dilatation | Nervous system disorders | MedDRA | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA | Systematic Assessment |
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| Hemiplegia | Nervous system disorders | MedDRA | Systematic Assessment |
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| Lethargy | Nervous system disorders | MedDRA | Systematic Assessment |
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| Loss of consciousness | Nervous system disorders | MedDRA | Systematic Assessment |
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| Motor developmental delay | Nervous system disorders | MedDRA | Systematic Assessment |
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| Pyramidal tract syndrome | Nervous system disorders | MedDRA | Systematic Assessment |
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| Seizure | Nervous system disorders | MedDRA | Systematic Assessment |
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| Speech disorder developmental | Nervous system disorders | MedDRA | Systematic Assessment |
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| Hypertonic bladder | Renal and urinary disorders | MedDRA | Systematic Assessment |
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| Breast pain | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
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| Vulvovaginal burning sensation | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
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| Epistaxis | 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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| Productive cough | 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 | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Dermatitis diaper | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Dry skin | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Eczema | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Hypersensitivity vasculitis | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Prurigo | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Pruritus | 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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| Rash maculo-papular | 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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| Urticaria | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
| 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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| patient-vaccination cases |
|
|