Phase 3
N=17
Efficacy, Safety and Tolerability of ACZ885 in Pediatric Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
Cryopyrin-associated Periodic Syndromes · Familial Cold Autoinflammatory Syndrome · Muckle-Wells Syndrome · Neonatal Onset Multisystem Inflammatory Disease
Bottom Line
View on ClinicalTrials.gov: NCT01576367 ↗Enrolled (actual)
17
Serious AEs
47.1%
Results posted
Aug 2016
Primary outcome: Primary: The Percentage of Participants Without Disease Relapse as Determined by the Physician's Global Assessment of Autoinflammatory Disease Activity, Assessment of Skin Disease and Serological Inflammation Markers. — 94.1 Percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- ACZ885 (Biological)
- Age
- Pediatric · 1+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Oct 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Percentage of Participants Without Disease Relapse as Determined by the Physician's Global Assessment of Autoinflammatory Disease Activity, Assessment of Skin Disease and Serological Inflammation Markers. |
94.1 | — |
| SECONDARY Immunogenicity of Canakinumab (ACZ885). Number of Participants With Anti-canakinumab Antibodies |
— | — |
| SECONDARY Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations |
-5.4; -14.7; -3.8; -4.1; -4.3; -10.4 | — |
| SECONDARY Frequency Counts of Physician's Global Assessment of Autoinflammatory Disease and Skin Disease |
64.7; 29.4; 5.9; 0; 0; 94.1 | — |
| SECONDARY Number of Vaccination Cases With Protective Antibody Levels Following Immunization With Inactivated Vaccines |
16; 3 | — |
Summary
This trial will provide long-term safety, efficacy and tolerability of ACZ885 in CAPS patients that completed the CACZ885D2307 study
Eligibility Criteria
Inclusion criteria
- Patients who completed the core CACZ885D2307 study (a patient is defined as having completed the core study if they completed the study up to and including the EOS visit with no major protocol deviations in the core).
- Male and female patients that are ≥ 1 year of age at the time of the roll-over visit.
- Parent or legal guardian written informed consent must be obtained before any assessment in the extension CACZ885D2307E1 study is performed.
Exclusion criteria
- Patients for who continued treatment in the CACZ885D2307E1 extension study is not considered appropriate by the treating physician.
- Patients who discontinued from the core CACZ885D2307 study
Other protocol-defined inclusion/exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT01576367). 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.