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

Novartis

Status and phase: Completed; Phase 3

Conditions: Muckle Wells Syndrome

Treatments: Drug: ACZ885; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT00465985

CACZ885D2304

Details and patient eligibility

About

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.

Enrollment: 35 patients

Sex: All

Ages: 4 to 75 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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35 participants in 2 patient groups, including a placebo group

Part I, Part II-arm1, & Part III

Experimental group

Treatment:

Drug: ACZ885

Part II - arm 2

Placebo Comparator group

Treatment:

Drug: Placebo