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 Cryo

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 ]

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.


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