Efficacy, Safety and Tolerability of ACZ885 in Pediatric Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01302860
First received: February 22, 2011
Last updated: May 20, 2014
Last verified: May 2014
  Purpose

This trial will assess the safety, efficacy and tolerability of ACZ885 in patients aged 4 years and younger with cryopyrin associated periodic syndromes (CAPS)


Condition Intervention Phase
Cryopyrin-associated Periodic Syndromes
Familial Cold Autoinflammatory Syndrome
Muckle-Wells Syndrome
Neonatal Onset Multisystem Inflammatory Disease
Drug: ACZ885
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A One-year Open-label, Multicenter Trial to Assess Efficacy, Safety and Tolerability of Canakinumab (ACZ885) and the Efficacy and Safety of Childhood Vaccinations in Patients Aged 4 Years or Younger With Cryopyrin Associated Periodic Syndromes (CAPS)

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • To assess the efficacy of canakinumab with respect to the treatment response in CAPS patients 4 years and younger [ Time Frame: A maximum of 56 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • To assess the efficacy of canakinumab with respect to the treatment response in CAPS patients 2 years and younger [ Time Frame: A maximum of 56 weeks ] [ Designated as safety issue: No ]
  • Safety and tolerability as assessed by overall frequency of adverse events and number of patients completing the study in patients 2 years and younger and the overall population [ Time Frame: A maximum of 56 weeks ] [ Designated as safety issue: Yes ]
  • To assess the presence of protective antibody levels following immunization with inactivated (killed) vaccines [ Time Frame: A maximum of 56 weeks ] [ Designated as safety issue: Yes ]
  • To evaluate the safety of canakinumab treatment in pediatric patients receiving a concomitant vaccination [ Time Frame: A maximum of 56 weeks ] [ Designated as safety issue: Yes ]
  • To evaluate the proportion of patients with vaccinated-associated reactions [ Time Frame: A maximum of 56 weeks ] [ Designated as safety issue: No ]
  • To assess the reduction of inflammation marker (C-reactive protein (CRP) or serum amyloid A (SAA)) after treatment initiation [ Time Frame: A maximum of 56 weeks ] [ Designated as safety issue: No ]

Enrollment: 17
Study Start Date: November 2010
Estimated Study Completion Date: November 2014
Estimated Primary Completion Date: November 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Canakinumab Drug: ACZ885

  Eligibility

Ages Eligible for Study:   1 Month to 60 Months
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Male and female patients that are 28 days up to 60 months of age at the time of the screening visit.
  2. Body weight > or = 2.5 kg.
  3. Parent or legal guardian's written informed consent is required before any assessment is performed for patients.
  4. At study entry, patients should have a clinical diagnosis of FCAS, MWS, or NOMID and symptoms requiring pharmacological intervention. 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.
  5. For patients treated with an IL-1 blocking agent (i.e. anakinra, rilonacept), these treatments should be discontinued prior to the baseline visit and patients must demonstrate active disease prior to treatment.
  6. Patients who are scheduled to receive an immunization, according to their local vaccination guidelines, with an inactivated vaccine must be willing to participate in the assessment schedule for vaccinated patients.

Exclusion Criteria:

  1. Preterm neonates for whom, in the Investigator's judgment, participation in the study is not deemed appropriate.
  2. History of recurrent and/or evidence of active bacterial, fungal, or viral infections (including HIV).
  3. Patients with immunodeficiency or treatment with immunosuppressive drugs.
  4. Live vaccinations within < or = 3 months prior to screening. No live vaccinations will be allowed throughout the course of this study and up to 3 months following the last dose.
  5. Patients with an increased risk of tuberculosis (TB) infection according to following risk factors:

    • Patients with recent close contact with persons known to have active pulmonary TB disease
    • Foreign-born patients from countries with a high prevalence of tuberculosis
    • Patients with recent tuberculosis infection (including children > 6 months with a positive PPD test [defined as an induration of at least 10mm])
    • Patients with end-stage renal disease
    • Patients with diabetes mellitus
    • Patients receiving immunosuppressive therapy
    • Patients with hematologic cancers.
  6. Participation in another trial within the last 30 days or 5 half-lives of the investigational compound (whichever is longer).
  7. Familial and social conditions rendering regular medical assessment not possible.
  8. Pediatric patients with neutropenia (absolute neutrophil count [ANC] < 1.5 x 10 to the 9th/l)

Other protocol defined inclusion/exclusion criteria may apply

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01302860

Locations
Belgium
Novartis Investigative Site
Bruxelles, Belgium, 1200
Novartis Investigative Site
Laeken, Belgium, 1020
Canada, Ontario
Novartis Investigative Site
Toronto, Ontario, Canada, M5G 1X8
France
Novartis Investigative Site
Le Kremlin Bicetre, France, 94275
Germany
Novartis Investigative Site
Berlin, Germany, 13353
Novartis Investigative Site
Dresden, Germany, 01307
Novartis Investigative Site
St. Augustin, Germany, 53757
Novartis Investigative Site
Tübingen, Germany, 72076
Spain
Novartis Investigative Site
Granada, Andalucia, Spain, 18012
Novartis Investigative Site
Valencia, Comunidad Valenciana, Spain, 46026
Switzerland
Novartis Investigative Site
Lausanne, Switzerland, 1011
United Kingdom
Novartis Investigative Site
London, United Kingdom, WC1N 1EH
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01302860     History of Changes
Other Study ID Numbers: CACZ885D2307, 2009-016859-22
Study First Received: February 22, 2011
Last Updated: May 20, 2014
Health Authority: Germany: Paul-Ehrlich-Institut
Spain: Spanish Agency of Medicines
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Belgium: Belgian Health Care Knowledge Center
Canada: Health Canada
Switzerland: Swissmedic
United States: Food and Drug Administration

Keywords provided by Novartis:
Muckle-Wells Syndrome (MWS) or Neonatal Onset Multisystem Inflammatory Disease (NOMID)
Cryopyrin-associated periodic syndromes (CAPS)
Familial Cold Autoinflammatory Syndrome (FCAS)
children
systemic autoinflammatory disease
CIAS-1 gene
NALP-3
NLRP3
ACZ885
Ilaris
human monoclonal anti-human interleukin-1 beta (IL-beta)
antibody
autosomal dominant
familial autoinflammatory syndrome

Additional relevant MeSH terms:
Syndrome
Cellulitis
Eosinophilia
Cryopyrin-Associated Periodic Syndromes
Disease
Pathologic Processes
Skin Diseases, Infectious
Infection
Suppuration
Connective Tissue Diseases
Inflammation
Leukocyte Disorders
Hematologic Diseases
Hereditary Autoinflammatory Diseases
Genetic Diseases, Inborn
Skin Diseases, Genetic
Skin Diseases

ClinicalTrials.gov processed this record on October 01, 2014