A study to check how safe, beneficial and tolerable the drug canakinumab is for patients with Muckle-Wells Syndrome

Conditions: Muckle-Wells Syndrome (Autoinflammatory Disease)
MedDRA version: 14.1Level: PTClassification code 10064569Term: Muckle-Wells syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Registration Number: EUCTR2006-005455-15-Outside-EU/EEA

Lead Sponsor: ovartis Pharma Services AG

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: A

Sex: All

Target Recruitment: 35

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).

Other protocol-defined inclusion criteria may apply.
Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 29
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

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 exclusion criteria may apply

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: 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.<br>Part I is an 8-week open-label, active treatment period to identify ACZ885 responders.<br>Part II is a double-blind, placebo-controlled period to assess primarily the efficacy of ACZ885 compared to placebo.<br>Part III is an open-label, active treatment period where patients will receive ACZ885 every 8 weeks after withdrawal or completion of Part II.;Secondary Objective: ;Primary end point(s): 1. Percent of Participants With Disease Flare in Part II (After 24 Weeks of the Double-blind Part)<br>2. Number of Participants Who Experienced a Disease Flare in Part II ;Timepoint(s) of evaluation of this end point: 32 weeks after study start

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): 1. Number of Participants With Treatment Response in Part I (After 8 Weeks)<br>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)<br>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.<br>4. Pharmacokinetics (CLD (L/d))<br>5. Pharmacodynamics Measured by Interleukin-1ß (IL-1ß) Concentrations at End of Part I.<br>6. Pharmacodynamics Measured by Interleukin-1ß (IL-1ß) Concentrations at End of Part II.<br>7. Pharmacodynamics Measured by Interleukin-1ß (IL-1ß) Concentrations at End of Part III. ;Timepoint(s) of evaluation of this end point: 1. 8 weeks after study start<br>2. 32 weeks after study start<br>3. Week 8 and Week 32<br>4. 48 weeks after study start<br>5. until Week 8<br>6. 32 weeks after study start<br>7. 48 weeks after study start