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Completed

NCT Number: NCT00465985

Efficacy, Safety, and Tolerability of ACZ885 in Patients With Muckle-Wells Syndrome

This study is designed to provide efficacy and safety data for ACZ885 (a fully human anti-interleukin-1beta (anti-IL-1beta) monoclonal antibody) administered as an injection subcutaneously (s.c.) in patients with Muckle-Wells Syndrome.

Part I is an 8-week open-label, active treatment period to identify ACZ885 responders.

Part II is a double-blind, placebo-controlled period to assess primarily the efficacy of ACZ885 compared to placebo.

Part III is an open-label, active treatment period where patients will receive ACZ885 every 8 weeks after withdrawal or completion of Part II.

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Key information

Conditions

Muckle Wells Syndrome Chronic Disease Chronic Inducible Urticaria Chronic Urticaria Cold Urticaria Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cryopyrin-Associated Periodic Syndromes Disease Attributes Genetic Diseases, Inborn Hereditary Autoinflammatory Diseases Hypersensitivity Hypersensitivity, Immediate Immune System Diseases Multiple Pterygium Syndrome, Autosomal Dominant Pathologic Processes Pathological Conditions, Signs and Symptoms Skin Diseases Skin Diseases, Genetic Skin Diseases, Vascular Skin and Connective Tissue Diseases Urticaria

Age range

4 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Novartis Investigative Site, Le Kremlin-Bicêtre, France

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Molecular diagnosis of NALP3 mutations and clinical picture resembling Muckle-Wells Syndrome.
  • Muckle-Wells Syndrome patients who participated in the CACZ885A2102 study, will have the option to participate in this study upon disease flare
  • Muckle-Wells Syndrome patients requiring medical intervention either untreated or treated (i.e. under ACZ885, anakinra, or any other investigational IL-1 blocking therapy).

Exclusion criteria

  • History of being immunocompromised, including a positive HIV at screening test result.
  • 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 significant medical conditions, which in the Investigator's opinion would exclude the patient from participating in this trial.
  • History of recurrent and/or evidence of active bacterial, fungal, or viral infections.
  • Positive tuberculin skin test at 48 to 72 hours after administration at the screening visit or within 2 months prior to the screening visit, according to national guidelines.

Other protocol-defined inclusion/exclusion criteria may apply

Treatment and study plan

ACZ885

Drug

Placebo

Drug

Primary outcomes

  1. Percent of Participants With Disease Flare in Part II (After 24 Weeks of the Double-blind Part)

    Time frame: 32 weeks after study start

    Determined by the Physician's global assessment of autoinflammatory disease activity, assessment of skin disease and inflammation markers. Data expressed as a percent of participants who had experienced a flare by the end of Part II.

  2. Number of Participants Who Experienced a Disease Flare in Part II

    Time frame: 32 weeks after study start

    Disease flare is determined by the Physician's global assessment of autoinflammatory disease activity, assessment of skin disease and inflammation markers. Disease Flare = the C-reactive protein and/or serum amyloid A (SAA) > 30 mg/L and either a PGA > minimal, or PGA equal to minimal and > minimal SD.

Secondary outcomes

  1. Number of Participants With Treatment Response in Part I (After 8 Weeks)

    Time frame: 8 weeks after study start

    Treatment response was based on Physician's global assessment(PGA) of autoinflammatory disease activity, assessment of skin disease(SD) and serum values of C-reactive protein(CRP) and/or serum amyloid A(SAA). Complete Response (CR):PGA and SD ≤ minimal and normal CRP and/or SAA. Partial Response (PR): a reduction of CRP and/or SAA from baseline (BL) by >30% but not reaching normal values and PGA improvement from BL by at least one category. Disease flare: a CRP and/or SAA > 30 mg/L and either PGA > minimal or PGA = minimal and SD > minimal. Non-responders = no PR by Day 8 or no CR by Day 15.

  2. Investigator's Clinical Assessment of Autoinflammatory Disease Activity & Participant's Assessment of Symptoms at End of Part II (After 24 Weeks of the Double-blind Part)

    Time frame: 32 weeks after study start

    A 5-point scale was used for the Physician's global assessment on autoinflammatory disease activity (absent, minimal, mild, moderate and severe) and for the assessment of the following items:

    • skin disease (urticarial skin rash)
    • arthralgia
    • myalgia
    • headache/migraine
    • conjunctivitis
    • fatigue/malaise
    • other symptoms related to autoinflammatory syndrome
    • other symptoms not related to autoinflammatory syndrome

  3. Change in Inflammation Markers at the End of Part II (C-reactive Protein and/or Serum Amyloid A) (After 24 Weeks of the Double-blind Part) From Week 8.

    Time frame: Week 8 and Week 32

  4. Pharmacokinetics (CLD (L/d))

    Time frame: 48 weeks after study start

    Assessed serum clearance of ACZ885.

  5. Pharmacodynamics Measured by Interleukin-1β (IL-1β) Concentrations at End of Part I.

    Time frame: until Week 8

  6. Pharmacodynamics Measured by Interleukin-1β (IL-1β) Concentrations at End of Part II.

    Time frame: 32 weeks after study start

  7. Pharmacodynamics Measured by Interleukin-1β (IL-1β) Concentrations at End of Part III.

    Time frame: 48 weeks after study start

Sponsors and collaborators

Lead sponsor

Novartis

Industry

Registry information

Official study title

A Three-part,Multicenter Study,With a Randomized,Double-blind,Placebo Controlled,Withdrawal Design in Part II to Assess Efficacy,Safety,and Tolerability of ACZ885(Anti-interleukin-1beta Monoclonal Antibody)in Patients With Muckle-Wells Syndrome

Acronym: REMITTER

Important dates

Study start
2007
Primary completion
2008
First posted
Apr 27, 2007
Registry last updated
Aug 28, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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