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This will provided long-term safety and efficacy data for ACZ885 (a fully human anti-interleukin-1β [anti-IL-1β] monoclonal antibody) given as an injection subcutaneously in patients who participated in the CACZ885A2102 (NCT00487708), CACZ885D2201 (NCT00685373) or CACZ885D2304(NCT00465985) studies or newly identified patients with the following cryopyrin-associated periodic syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome or Neonatal Onset Multisystem Inflammatory Disease.
The duration of this study was 6 months with a maximum duration of 2 years
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Canakinumab (ACZ885) | Experimental | Subcutaneous injection every 8 weeks based on participant's body weight. Body weight >40 kilogram (kg): 150 milligrams (mg) per injection and body weight <= 40 kg: 2 mg/kg per injection. For participants who did not experience sufficient symptomatic relief, an up-titration to the dose and/or more frequent doses were permitted as per protocol. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Canakinumab (ACZ885) | Drug | 6 mL glass vial containing 150 mg lyophilized Canakinumab reconstituted with water for a subcutaneous injection every 8 weeks. Dosage based on body weight. |
| Measure | Description | Time Frame |
|---|---|---|
| The Number of Participants With Adverse Events (AEs), Death, Serious Adverse Events (SAEs), Discontinuation of Study Drug Due to an AE, Infections and Infestations and Injection Site Reactions | The number of participants with Adverse Events and Infections & Infestations are regardless of study drug relationship by primary system organ class preferred term equal and/or greater than 2% in any group. The number of participants with mild injection site reactions= mild reactions observed on at least one occasion but no moderate or severe reactions. The number of participants with moderate injection site reactions= moderate reactions observed on at least one occasion but no severe reactions. | 2 years depending on when the participant enters the study |
| 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 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. |
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Inclusion Criteria:
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Little Rock Allergy and Asthma Clinic | Little Rock | Arkansas | 72205 | United States | ||
| UCSF School of Medicine |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 21859692 | Derived | Kuemmerle-Deschner JB, Hachulla E, Cartwright R, Hawkins PN, Tran TA, Bader-Meunier B, Hoyer J, Gattorno M, Gul A, Smith J, Leslie KS, Jimenez S, Morell-Dubois S, Davis N, Patel N, Widmer A, Preiss R, Lachmann HJ. Two-year results from an open-label, multicentre, phase III study evaluating the safety and efficacy of canakinumab in patients with cryopyrin-associated periodic syndrome across different severity phenotypes. Ann Rheum Dis. 2011 Dec;70(12):2095-102. doi: 10.1136/ard.2011.152728. Epub 2011 Aug 21. |
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This study included participants previously enrolled in CACZ885A2102 (NCT00487708), CACZ885D2304 (NCT00465985) and ACZ885 naive patients.
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| ID | Title | Description |
|---|---|---|
| FG000 | Canakinumab (ACZ885) | Subcutaneous injection every 8 weeks based on participant's body weight. Body weight >40 kilogram (kg): 150 milligrams (mg) per injection and body weight <= 40 kg: 2 mg/kg per injection. For participants who did not experience sufficient symptomatic relief, an up-titration to the dose and/or more frequent doses were permitted as per protocol. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years |
| Immunogenicity of Canakinumab (ACZ885) | The number of participants who tested positive for anti-ACZ885 antibodies using the Biacore Assay at the end of the study. | Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years |
| Pharmacokinetics | Mean Clearance from serum in Liter per Day (CLD) in adult participants >=18, pediatric participants <18 with body weight >40 kg and pediatric participants <18 with body weight <=40 kg. | Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years |
| San Francisco |
| California |
| 94118 |
| United States |
| Allergy Center at Brookstone | Columbus | Georgia | 31904 | United States |
| Rush-Presbyterian St. Lukes Medical Center | Chicago | Illinois | 60612 | United States |
| Cleveland Clinic | Cleveland | Ohio | 44195 | United States |
| University of Wisconsin | Madison | Wisconsin | 53792 | United States |
| Novartis Investigative Site | Laken | Belgium |
| Novartis Investigative Site | Le Kremlin-Bicêtre | France |
| Novartis Investigative Site | Lille | France |
| Novartis Investigative Site | Montpelier Cedex | France |
| Novartis Investigative site | Nantes | France |
| Novartis Investigative site | Berlin | Germany |
| Novartis Investigative Site | Hamburg | Germany |
| Novartis Investigative site | Heidelberg | Germany |
| Novartis Investigative Site | Herne | Germany |
| Novartis Investigative Site | Marburg | Germany |
| Novartis Investigative site | Tübingen | Germany |
| Novartis Investigative site | New Delhi | India |
| Novartis Investigative site | Genova | Italy |
| Novartis Investigative Site | Naples | Italy |
| Novartis Investigative Site | Padova | Italy |
| Novartis Investigative Site | Rome | Italy |
| Novartis Investigative Site | Trieste | Italy |
| Novartis Investigative Site | Madrid | Spain |
| Novartis Investigative site | Oviedo | Spain |
| Novartis Investigative Site | Vigo | Spain |
| Novartis Investigative site | Istanbul | Turkey (Türkiye) |
| Novartis Investigative site | London | United Kingdom |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Canakinumab (ACZ885) | Subcutaneous injection every 8 weeks based on participant's body weight. Body weight >40 kilogram (kg): 150 milligrams (mg) per injection and body weight <= 40 kg: 2 mg/kg per injection. For participants who did not experience sufficient symptomatic relief, an up-titration to the dose and/or more frequent doses were permitted as per protocol. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Number | participants |
| ||||||||||||||||||||||||||
| Gender | Count of Participants | Participants |
| ||||||||||||||||||||||||||
| Disease Characteristics | Number | 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 Number of Participants With Adverse Events (AEs), Death, Serious Adverse Events (SAEs), Discontinuation of Study Drug Due to an AE, Infections and Infestations and Injection Site Reactions | The number of participants with Adverse Events and Infections & Infestations are regardless of study drug relationship by primary system organ class preferred term equal and/or greater than 2% in any group. The number of participants with mild injection site reactions= mild reactions observed on at least one occasion but no moderate or severe reactions. The number of participants with moderate injection site reactions= moderate reactions observed on at least one occasion but no severe reactions. | Safety Population defined as all participants who received at least one dose of study drug. | Posted | Number | participants | 2 years depending on when the participant enters the study |
|
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | The Percentage of Participants Without Disease Relapse as Determined by the Physician's Global Assessment of Autoinflammatory Disease Activity, Assessment of Skin Disease and 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. | Safety Population defined as all participants who had at least one dose of study drug and were included in the Relapse Assessment. | Posted | Number | Percentage of participants | Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Immunogenicity of Canakinumab (ACZ885) | The number of participants who tested positive for anti-ACZ885 antibodies using the Biacore Assay at the end of the study. | Safety Population defined as all participants who received at least one dose of study drug and were tested for anti-ACZ885 antibodies at the end of the study. | Posted | Number | participants | Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Pharmacokinetics | Mean Clearance from serum in Liter per Day (CLD) in adult participants >=18, pediatric participants <18 with body weight >40 kg and pediatric participants <18 with body weight <=40 kg. | Safety population defined as all participants who received at least 1 dose of study drug. 3 participants were excluded because dosing information was not available at the time of analysis. | Posted | Mean | Standard Deviation | L/day | Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 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 (ACZ885) | Subcutaneous injection every 8 weeks based on participant's body weight. Body weight >40 kilogram (kg): 150 milligrams (mg) per injection and body weight <= 40 kg: 2 mg/kg per injection. For participants who did not experience sufficient symptomatic relief, an up-titration to the dose and/or more frequent doses were permitted as per protocol. | 18 | 166 | 124 | 166 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Muckle-Wells syndrome | Congenital, familial and genetic disorders | MedDRA | Systematic Assessment |
| |
| Radicular cyst | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Abdominal abscess | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Chronic tonsillitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| H1N1 influenza | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Drug exposure during pregnancy | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Forearm fracture | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Hepatitis C antibody positive | Investigations | MedDRA | Systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA | Systematic Assessment |
| |
| Carpal tunnel syndrome | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Nerve root compression | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Pregnancy | Pregnancy, puerperium and perinatal conditions | MedDRA | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Nephrotic syndrome | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Miscarriage of partner | Social circumstances | MedDRA | Systematic Assessment |
| |
| Abortion induced | Surgical and medical procedures | MedDRA | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal 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.
| 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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| Title | Measurements |
|---|---|
|
| >= 75 years |
|
| Neonatal Onset Multi-System Inflammatory Syndrome |
|
| Information Missing |
|
| Title | Measurements |
|---|---|
|
| Discontinuation of study drug due to an AE |
|
| Infections and Infestations |
|
| Any Injection Site Reactions |
|
| Mild Injection Site Reactions |
|
| Moderate Injection Site Reactions |
|
| Participants |
|
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|
|