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

Phase 3

Completed

• Conditions: Muckle-Wells Syndrome; Familial Cold Autoinflammatory Syndrome; Cryopyrin-associated Periodic Syndromes; Neonatal Onset Multisystem Inflammatory Disease
• Interventions: Biological: ACZ885

• Registration Number: NCT01576367
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

This trial will provide long-term safety, efficacy and tolerability of ACZ885 in CAPS patients that completed the CACZ885D2307 study

Detailed Description

Not available

Study Timeline

• Study Start: 2012-01-16
• Study First Submitted: 2012-02-17
• Study First Submitted QC: 2012-04-10
• Study First Posted: 2012-04-12
• Primary Completion: 2015-10-13
• Study Completion: 2015-10-13
• Results First Submitted: 2016-06-22
• Results First Submitted QC: 2016-06-22
• Results First Posted: 2016-08-03
• Status Verified: 2018-08
• Last Update Submitted: 2018-08-13
• Last Update Posted: 2018-09-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 17

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
canakinumab	ACZ885	Patients will receive a standard dose at an equivalent of 2 mg/kg s.c. of canakinumab (ACZ885) every 8 weeks. Possible dose and/or dosing regimen adjustments that can be administered include: 4 mg/kg s.c. (every 4 to 8 weeks) 6 mg/kg s.c. (every 4 to 8 weeks) 8 mg/kg s.c. (every 4 to 8 weeks)

Primary Outcome Measures

Name	Time	Method
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.	Week /80, 104, 128, and 152 (A minimum of 6 months and maximum of 24 months)	Disease relapse following complete response is defined as inflammation markers: C-Reactive Protein (CRP) and/or Serum Amyloid A (SAA) result \> 30 mg/L AND Physician's Global Assessment of Autoinflammatory Disease Activity \> minimal or Physician's Global Assessment \>= minimal AND Skin Disease Assessment \> minimal. Physician's Global Assessment of Autoinflammatory Disease Activity and Skin Disease Assessment (urticarial skin rash) are completed by the investigator using a 5 point rating scale: absent, minimal, mild, moderate and severe.

Secondary Outcome Measures

Name	Time	Method
Immunogenicity of Canakinumab (ACZ885). Number of Participants With Anti-canakinumab Antibodies	minimum of 6 months and maximum of 24 months	Immunogenicity assessment included determination of anti-canakinumab (ACZ885) antibodies in serum samples using BIAcore system, with detection based on surface plasmon resonance technique.
Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations	Week 0, 80, 104, 128 and 152, last assessment	CRP and SAA were used as serologic inflammatory markers. The target level concentrations for CRP and SAA was ≤15 mg/L and ≤10 mg/L, respectively. Negative change in concentration of inflammatory markers indicated improvement.
Frequency Counts of Physician's Global Assessment of Autoinflammatory Disease and Skin Disease	minimum of 6 months and maximum of 24 months	Participants were assessed based by physician on Physician's Global Assessment measured on a 5--point scale for auto inflammatory disease activity as: 0 = None/absent; 1 = Minimal; 2 = Mild; 3 = Moderate; 4 = Severe
Number of Vaccination Cases With Protective Antibody Levels Following Immunization With Inactivated Vaccines	pre-vaccine dose, Day 28 post-vaccine	Participants who received any inactivated vaccines during the study were assessed for their ability to attain protective antibody levels against the vaccine (antigen) post immunization. Participants vaccinations were not assessed for a response if the antibody titre was already sufficient at pre-dose and maintained during the study.

Trial Locations

Locations (1)

Novartis Investigative Site; 🇬🇧 London, United Kingdom