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
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|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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00685373