Efficacy, Safety, and Tolerability of ACZ885 in Patients With Muckle-Wells Syndrome (REMITTER)
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ClinicalTrials.gov Identifier: NCT00465985 |
Recruitment Status :
Completed
First Posted : April 27, 2007
Results First Posted : February 11, 2011
Last Update Posted : August 28, 2017
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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.
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Muckle Wells Syndrome | Drug: ACZ885 Drug: Placebo | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 35 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official 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 |
Study Start Date : | April 2007 |
Actual Primary Completion Date : | October 2008 |

Arm | Intervention/treatment |
---|---|
Experimental: Part I, Part II-arm1, & Part III |
Drug: ACZ885 |
Placebo Comparator: Part II - arm 2 |
Drug: Placebo |
- 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.
- 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.
- 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.
- 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
- 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 ]
- Pharmacokinetics (CLD (L/d)) [ Time Frame: 48 weeks after study start ]Assessed serum clearance of ACZ885.
- Pharmacodynamics Measured by Interleukin-1β (IL-1β) Concentrations at End of Part I. [ Time Frame: until Week 8 ]
- Pharmacodynamics Measured by Interleukin-1β (IL-1β) Concentrations at End of Part II. [ Time Frame: 32 weeks after study start ]
- Pharmacodynamics Measured by Interleukin-1β (IL-1β) Concentrations at End of Part III. [ Time Frame: 48 weeks after study start ]

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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:
- 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

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 ClinicalTrials.gov identifier (NCT number): NCT00465985
United States, California | |
Novartis Investigative Site | |
San Francisco, California, United States, 94115 | |
United States, Illinois | |
Novartis Investigative Site | |
Chicago, Illinois, United States, 60612 | |
United States, Wisconsin | |
Novartis Investigative Site | |
Madison, Wisconsin, United States, 53792 | |
France | |
Novartis Investigative Site | |
Le Kremlin Bicetre, France | |
Novartis Investigational Site | |
Lille Cedex, France | |
Novartis Investigative Site | |
Montpellier Cedex, France | |
Novartis Investigative Site | |
Nantes, France | |
Novartis Investigative Site | |
Paris, France | |
Germany | |
Novartis Investigative Site | |
Tubingen, Germany | |
India | |
Novartis Investigative Site | |
New Delhi, India | |
Spain | |
Novartis Investigative Site | |
Barcelona, Spain | |
United Kingdom | |
Novartis Investigative Site | |
London, United Kingdom |
Responsible Party: | Novartis |
ClinicalTrials.gov Identifier: | NCT00465985 |
Other Study ID Numbers: |
CACZ885D2304 |
First Posted: | April 27, 2007 Key Record Dates |
Results First Posted: | February 11, 2011 |
Last Update Posted: | August 28, 2017 |
Last Verified: | July 2017 |
Muckle-Wells Syndrome children systemic autoinflammatory disease CIAS-1 gene NALP-3 |
ACZ885 human monoclonal anti-human interleukin-1beta (IL-1beta) antibody autosomal dominant familial autoinflammatory syndrome |
Cellulitis Eosinophilia Cryopyrin-Associated Periodic Syndromes Syndrome Disease Pathologic Processes Hereditary Autoinflammatory Diseases Genetic Diseases, Inborn Skin Diseases, Genetic |
Skin Diseases Skin Diseases, Infectious Infections Suppuration Connective Tissue Diseases Inflammation Leukocyte Disorders Hematologic Diseases |