Trial record 11 of 15 for:    "Muckle-Wells syndrome"

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

This study has been completed.
Sponsor:
Information provided by:
Novartis
ClinicalTrials.gov Identifier:
NCT00487708
First received: June 18, 2007
Last updated: February 16, 2009
Last verified: February 2009
  Purpose

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 Intervention Phase
NALP3 Mutation
Drug: canakinumab
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]

Enrollment: 34
Study Start Date: January 2005
Study Completion Date: July 2008
Primary Completion Date: July 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
ACZ885
Drug: canakinumab

  Eligibility

Ages Eligible for Study:   4 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  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: NCT00487708

Locations
France
Novartis Investigator Site
Lille, France
Germany
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
India
Novartis Investigator Site
New Dehli, India
Spain
Novartis Investigator Site
Oviedo, Spain
United Kingdom
Novartis Investigative site
London, United Kingdom
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Principal Investigator: Novartis Investigative site
  More Information

No publications provided by Novartis

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: External Affairs, Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00487708     History of Changes
Other Study ID Numbers: CACZ885A2102
Study First Received: June 18, 2007
Last Updated: February 16, 2009
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency
Germany: Paul-Ehrlich-Institut
Spain: Comité Ético de Investigación Clínica

Keywords provided by Novartis:
ACZ885
NALP3 mutations
Interleukin-1beta
Muckle-Wells Syndrome

ClinicalTrials.gov processed this record on August 20, 2014