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Safety, Efficacy, Pharmacokinetics, Pharmacodynamics of ACZ885 in Patients With NALP3 Mutations and Clinical Symptoms

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00487708
Recruitment Status : Completed
First Posted : June 19, 2007
Last Update Posted : February 17, 2009
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Brief Summary:
This study will investigate the clinical efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of ACZ885, administered intravenously and subcutaneously to patients with NALP3 mutations whose clinical symptoms are either untreated or insufficiently treated and require medical intervention.

Condition or disease Intervention/treatment Phase
NALP3 Mutation Drug: canakinumab Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 34 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label, Phase II Dose Titration Study of ACZ885 (Human Anti-IL-1β Monoclonal Antibody) to Assess the Clinical Efficacy, Safety, Pharmacokinetics and Pharmacodynamics in Patients With NALP3 Mutations
Study Start Date : January 2005
Actual Primary Completion Date : July 2008
Actual Study Completion Date : July 2008

Resource links provided by the National Library of Medicine

Drug Information available for: Canakinumab

Arm Intervention/treatment
Experimental: 1
Drug: canakinumab

Primary Outcome Measures :
  1. Response to treatment and time to relapse after ACZ885 administration according to monthly investigator's clinical assessments, laboratory monitoring, and patient diaries. [ Time Frame: Every month ]

Secondary Outcome Measures :
  1. Assessment of safety,tolerability and immunogenicity of ACZ885 at each clinical visit. Evaluation of ACZ885 PK and PD at each clinical visit Evaluate efficacy towards hearing loss(every 4 months),kidney function (every 4 months),neurological symptoms [ Time Frame: Every month ]

Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 75 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients aged 4 to 75 years (inclusive)
  • Body weight ≥ 12 kg and < 100 kg.
  • Females of child-bearing potential must have a negative pregnancy test. Additional birth control details to be provided at screening.
  • Documented molecular diagnosis of NALP3 mutations and clinical symptoms that are either untreated or insufficiently treated and require medical intervention.
  • Patients under anakinra therapy or any other IL-1 blocking therapy, whose clinical symptoms improved under treatment and are willing to discontinue that therapy until a relapse becomes evident.
  • Patients with a very severe characteristics requiring oral prednisone are eligible if the dose is stable (≤ 0.4 mg/kg/day or ≤ 20 mg/day, whichever is lower) for at least 1 week prior to the screening visit. Steroid therapy may be tapered during treatment with ACZ885 at the discretion of the investigator.
  • Parents' or legal guardian's written informed consent (patient's informed consent for ≥ 18 years of age) and child's assent, if appropriate, are required prior to study participation.

Exclusion Criteria:

  • Participation in any clinical trial investigation (except trials with anakinra) within 4 weeks prior to dosing or longer per local regulation
  • Antiinflammatory therapy with colchicine, chlorambucil, dapsone, azathioprine, mycophenolate mofetil, within 3 weeks prior to dosing. Therapeutic antibodies (e.g. anti-TNF-alpha antibodies) must be discontinued at least 60 days before dosing.
  • Donation or loss of 400 mL or more of blood within 8 weeks prior to dosing.
  • A past personal or close family medical history of clinically significant ECG abnormalities or prolonged QT-interval syndrome.
  • History of

    • Immunocompromise, including a positive HIV result.
    • Positive Hepatitis B surface antigen or Hepatitis C test result.
    • Drug or alcohol abuse within the 12 months prior to dosing.
    • Tuberculosis.
    • Renal transplant.
    • Evidence of lymphoma.
  • Active medical condition preventing participation in the study such as infection, poorly controlled diabetes etc.
  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00487708

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Novartis Investigator Site
Lille, France
Novartis Investigator Site
Berlin, Germany
Novartis Investigator Site
Dresden, Germany
Novartis Investigator Site
Heidelberg, Germany
Novartis Investigator Site
Marburg, Germany
Novartis investigative site
Nuernberg, Germany
Novartis Investigator Site
Tuebingen, Germany
Novartis Investigator Site
New Dehli, India
Novartis Investigator Site
Oviedo, Spain
United Kingdom
Novartis Investigative site
London, United Kingdom
Sponsors and Collaborators
Novartis Pharmaceuticals
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Principal Investigator: Novartis Investigative site

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: External Affairs, Novartis Pharmaceuticals Identifier: NCT00487708     History of Changes
Other Study ID Numbers: CACZ885A2102
First Posted: June 19, 2007    Key Record Dates
Last Update Posted: February 17, 2009
Last Verified: February 2009
Keywords provided by Novartis:
NALP3 mutations
Muckle-Wells Syndrome
Additional relevant MeSH terms:
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Antibodies, Monoclonal
Immunologic Factors
Physiological Effects of Drugs