Status:

COMPLETED

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

Lead Sponsor:

Novartis

Conditions:

Cryopyrin-Associated Periodic Syndromes

Familial Cold Autoinflammatory Syndrome

Eligibility:

All Genders

3+ years

Phase:

PHASE3

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 participate...

Eligibility Criteria

Inclusion

  • Male and female patients at least 3 years of age
  • 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
  • For patients under anakinra therapy or any other investigational IL-1 blocking therapy, these treatments should be discontinued prior to the baseline visit.
  • 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.
  • Patients who completed the CACZ885D2304 study may enter this study
  • Patients who completed the CACZ885D2201 study may enter this study
  • 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

  • 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.
  • History of being immunocompromised, including a positive HIV at screening (ELISA and Western blot) test result.
  • A positive Hepatitis B surface antigen (HBsAg) or Hepatitis C antibody test result is not allowed.
  • 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.
  • History of recurrent and/or evidence of active bacterial, fungal, or viral infections.
  • 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

Key Trial Info

Start Date :

May 1 2008

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

April 1 2010

Estimated Enrollment :

166 Patients enrolled

Trial Details

Trial ID

NCT00685373

Start Date

May 1 2008

End Date

April 1 2010

Last Update

November 4 2016

Active Locations (28)

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Page 1 of 7 (28 locations)

1

Little Rock Allergy and Asthma Clinic

Little Rock, Arkansas, United States, 72205

2

UCSF School of Medicine

San Francisco, California, United States, 94118

3

Allergy Center at Brookstone

Columbus, Georgia, United States, 31904

4

Rush-Presbyterian St. Lukes Medical Center

Chicago, Illinois, United States, 60612