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
Condition: Cryopyrin-Associated Periodic Syndromes; Familial Cold Autoinflammatory Syndrome; Muckle Wells Syndrome; Neonatal Onset Multisystem Inflammatory Disease
MeSH terms: Cellulitis; Eosinophilia; Cryopyrin-Associated Periodic Syndromes; Syndrome
Intervention: Canakinumab (ACZ885)
Intervention description: 6 mL glass vial containing 150 mg lyophilized Canakinumab reconstituted with water for a subcutaneous injection every 8 weeks. Dosage based on body weight.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 166.0
Sex: All
Minimum age: 3 Years
Healthy volunteers: No
Keywords: Cryopyrin-associated periodic syndromes (CAPS):; Familial Cold Autoinflammatory Syndrome (FCAS),; Muckle-Wells Syndrome (MWS),; MWS with overlapping symptoms of Neonatal Onset Multisystem Inflammatory Disease (NOMID) or Neonatal Onset Multisystem Inflammatory Disease (NOMID),; children,; systemic autoinflammatory disease,; CIAS-1 gene,; NALP-3,; ACZ885,; human monoclonal anti-human interleukin-1beta (IL-1beta),; antibody,; autosomal dominant,; familial autoinflammatory syndrome

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
    

Eligibility criteria:

        Inclusion Criteria:

          1. Male and female patients at least 3 years of age

          2. Diagnosis of Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome or
             Neonatal Onset Multisystem Inflammatory Disease. Prior agreement between the
             Investigator and Novartis for study eligibility is required for patients who do not
             have a molecular diagnosis of NALP3 mutations available (either testing not performed,
             or testing performed but negative)upon study entry. For those patients who have not
             been molecularly tested for NALP3 mutations, molecular testing should be performed
             during the course of the study

          3. For patients under anakinra therapy or any other investigational IL-1 blocking
             therapy, these treatments should be discontinued prior to the baseline visit.

          4. Patients from the CACZ885A2102 study may enter this study. However, dosing at Visit 2
             (Baseline Visit) can only occur if either 1) the patient is experiencing disease flare
             or 2) at least two months have elapsed from their last injection even in the absence
             of flare, whichever is earlier.

          5. Patients who completed the CACZ885D2304 study may enter this study

          6. Patients who completed the CACZ885D2201 study may enter this study

          7. Patients who discontinued from the CACZ885A2102, CACZ885D2201 or CACZ885D2304 studies
             and for whom in the Investigator's judgment (with prior agreement from Novartis)
             continued treatment with ACZ885 in this study is considered appropriate.

        Exclusion Criteria:

          1. Participation in any clinical investigation within 4 weeks prior to dosing or longer
             if required by local regulation with the exception of trials with anakinra, other
             investigational IL-1 blocking therapies, and/or ACZ885.

          2. History of being immunocompromised, including a positive HIV at screening (ELISA and
             Western blot) test result.

          3. A positive Hepatitis B surface antigen (HBsAg) or Hepatitis C antibody test result is
             not allowed.

          4. No live vaccinations within 3 months prior to the start of the trial, during the
             trial, and up to 3 months following the last dose.

          5. History of recurrent and/or evidence of active bacterial, fungal, or viral infections.

          6. Positive tuberculin skin test reaction (PPD 5 tuberculin units or as according to
             local standard practice) (>= 5 mm induration) at 48 to 72 hours after administration
             at the screening visit or within 2 months prior to the screening visit. Patients who
             have a positive PPD skin test with a documentation of BCG vaccination, who are at low
             environmental risk for tuberculosis (TB) infection or reactivation, and have a
             negative chest X-ray can be included.

        Other protocol-defined inclusion/exclusion criteria may apply