Efficacy and Safety of ACZ885 in Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
|ClinicalTrials.gov Identifier: NCT00685373|
Recruitment Status : Completed
First Posted : May 28, 2008
Results First Posted : May 27, 2011
Last Update Posted : November 4, 2016
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
|Condition or disease||Intervention/treatment||Phase|
|Cryopyrin-Associated Periodic Syndromes Familial Cold Autoinflammatory Syndrome Muckle Wells Syndrome Neonatal Onset Multisystem Inflammatory Disease||Drug: Canakinumab (ACZ885)||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||166 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-label, Long-term Safety and Efficacy Study of ACZ885 (Anti-interleukin-1β Monoclonal Antibody) Administered for at Least 6 Months in Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease|
|Study Start Date :||May 2008|
|Actual Primary Completion Date :||April 2010|
|Actual Study Completion Date :||April 2010|
Experimental: 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.
Drug: Canakinumab (ACZ885)
6 mL glass vial containing 150 mg lyophilized Canakinumab reconstituted with water for a subcutaneous injection every 8 weeks. Dosage based on body weight.
- 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 [ Time Frame: 2 years depending on when the participant enters the study ]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.
- 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. [ Time Frame: Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years ]
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.
- Immunogenicity of Canakinumab (ACZ885) [ Time Frame: Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years ]The number of participants who tested positive for anti-ACZ885 antibodies using the Biacore Assay at the end of the study.
- Pharmacokinetics [ Time Frame: Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years ]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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00685373
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