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

Last updated: November 3, 2016
Sponsor: Novartis
Overall Status: Completed

Phase

3

Condition

White Cell Disorders

Soft Tissue Infections

Skin Wounds

Treatment

N/A

Clinical Study ID

NCT00685373
CACZ885D2306
  • Ages > 3
  • All Genders

Study 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

Inclusion Criteria:

  1. Male and female patients at least 3 years of age

  2. Diagnosis of Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome orNeonatal Onset Multisystem Inflammatory Disease. Prior agreement between theInvestigator and Novartis for study eligibility is required for patients who do nothave a molecular diagnosis of NALP3 mutations available (either testing not performed,or testing performed but negative)upon study entry. For those patients who have notbeen molecularly tested for NALP3 mutations, molecular testing should be performedduring the course of the study

  3. For patients under anakinra therapy or any other investigational IL-1 blockingtherapy, 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 flareor 2) at least two months have elapsed from their last injection even in the absenceof 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 studiesand for whom in the Investigator's judgment (with prior agreement from Novartis)continued treatment with ACZ885 in this study is considered appropriate.

Exclusion

Exclusion Criteria:

  1. Participation in any clinical investigation within 4 weeks prior to dosing or longerif required by local regulation with the exception of trials with anakinra, otherinvestigational IL-1 blocking therapies, and/or ACZ885.

  2. History of being immunocompromised, including a positive HIV at screening (ELISA andWestern blot) test result.

  3. A positive Hepatitis B surface antigen (HBsAg) or Hepatitis C antibody test result isnot allowed.

  4. No live vaccinations within 3 months prior to the start of the trial, during thetrial, 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 tolocal standard practice) (>= 5 mm induration) at 48 to 72 hours after administrationat the screening visit or within 2 months prior to the screening visit. Patients whohave a positive PPD skin test with a documentation of BCG vaccination, who are at lowenvironmental risk for tuberculosis (TB) infection or reactivation, and have anegative chest X-ray can be included. Other protocol-defined inclusion/exclusion criteria may apply

Study Design

Total Participants: 166
Study Start date:
May 01, 2008
Estimated Completion Date:
April 30, 2010

Connect with a study center

  • Novartis Investigative Site

    Laeken,
    Belgium

    Site Not Available

  • Novartis Investigative Site

    Le Kremlin Bicetre,
    France

    Site Not Available

  • Novartis Investigative Site

    Lille Cedex,
    France

    Site Not Available

  • Novartis Investigative Site

    Montpelier Cedex,
    France

    Site Not Available

  • Novartis Investigative site

    Nantes,
    France

    Site Not Available

  • Novartis Investigative site

    Berlin,
    Germany

    Site Not Available

  • Novartis Investigative Site

    Hamburg,
    Germany

    Site Not Available

  • Novartis Investigative site

    Heidelburg,
    Germany

    Site Not Available

  • Novartis Investigative Site

    Herne,
    Germany

    Site Not Available

  • Novartis Investigative Site

    Marburg,
    Germany

    Site Not Available

  • Novartis Investigative site

    Tubingen,
    Germany

    Site Not Available

  • Novartis Investigative site

    New Delhi,
    India

    Site Not Available

  • Novartis Investigative site

    Genova,
    Italy

    Site Not Available

  • Novartis Investigative Site

    Napoli,
    Italy

    Site Not Available

  • Novartis Investigative Site

    Padova,
    Italy

    Site Not Available

  • Novartis Investigative Site

    Rome,
    Italy

    Site Not Available

  • Novartis Investigative Site

    Trieste,
    Italy

    Site Not Available

  • Novartis Investigative Site

    Madrid,
    Spain

    Site Not Available

  • Novartis Investigative site

    Oviedo,
    Spain

    Site Not Available

  • Novartis Investigative Site

    Vigo,
    Spain

    Site Not Available

  • Novartis Investigative site

    Istanbul,
    Turkey

    Site Not Available

  • Novartis Investigative site

    London,
    United Kingdom

    Site Not Available

  • Little Rock Allergy and Asthma Clinic

    Little Rock, Arkansas 72205
    United States

    Site Not Available

  • UCSF School of Medicine

    San Francisco, California 94118
    United States

    Site Not Available

  • Allergy Center at Brookstone

    Columbus, Georgia 31904
    United States

    Site Not Available

  • Rush-Presbyterian St. Lukes Medical Center

    Chicago, Illinois 60612
    United States

    Site Not Available

  • Cleveland Clinic

    Cleveland, Ohio 44195
    United States

    Site Not Available

  • University of Wisconsin

    Madison, Wisconsin 53792
    United States

    Site Not Available

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