Clinical Trial: 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
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
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 Perio
Brief Summary:
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
Detailed Summary:
Sponsor: Novartis
Current Primary Outcome: 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 ]
Original Primary Outcome: Adverse events (including infections); the number and frequency of Injection site reactions
Current Secondary Outcome:
- 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.
Original Secondary Outcome:
- The proportion of patients with disease relapse as determined by the Physician's global assessment of autoinflammatory disease activity, assessment of skin disease and inflammation markers
- Immunogenicity
- Pharmacokinetics
Information By: Novartis
Dates:
Date Received: May 27, 2008
Date Started: May 2008
Date Completion:
Last Updated: November 3, 2016
Last Verified: September 2016