Phase 3
N=35
Efficacy, Safety, and Tolerability of ACZ885 in Patients With Muckle-Wells Syndrome
Muckle Wells Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT00465985 ↗Enrolled (actual)
35
Serious AEs
3.0%
Results posted
Feb 2011
Primary outcome: Primary: Percent of Participants With Disease Flare in Part II (After 24 Weeks of the Double-blind Part) — 0; 81.3 percent of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- ACZ885 (Drug); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 4+ yrs
- Sex
- All
- Sponsor
- Novartis
- Primary completion
- Oct 2008
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent of Participants With Disease Flare in Part II (After 24 Weeks of the Double-blind Part) |
0; 81.3 | — |
| PRIMARY Number of Participants Who Experienced a Disease Flare in Part II |
0; 13 | — |
| SECONDARY Number of Participants With Treatment Response in Part I (After 8 Weeks) |
31; 2; 1; 1 | — |
| SECONDARY 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) |
8; 0; 7; 4; 0; 8 | — |
| SECONDARY 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. |
1.10; 19.93; 2.27; 71.09 | — |
| SECONDARY Pharmacokinetics (CLD (L/d)) |
0.177 | — |
| SECONDARY Pharmacodynamics Measured by Interleukin-1β (IL-1β) Concentrations at End of Part I. |
19.047 | — |
| SECONDARY Pharmacodynamics Measured by Interleukin-1β (IL-1β) Concentrations at End of Part II. |
21.943; 0.596 | — |
| SECONDARY Pharmacodynamics Measured by Interleukin-1β (IL-1β) Concentrations at End of Part III. |
23.018 | — |
Summary
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.
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT00465985). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.