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

Novartis

Status and phase: Completed; Phase 3

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

Treatments: Biological: ACZ885

Study type: Interventional

Funder types: Industry

Identifiers

NCT01576367

2011-005154-57 (EudraCT Number)

CACZ885D2307E1

Details and patient eligibility

About

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

Enrollment: 17 patients

Sex: All

Ages: 1 to 4 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. 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).
2. Male and female patients that are ≥ 1 year of age at the time of the roll-over visit.
3. Parent or legal guardian written informed consent must be obtained before any assessment in the extension CACZ885D2307E1 study is performed.

Exclusion criteria

1. Patients for who continued treatment in the CACZ885D2307E1 extension study is not considered appropriate by the treating physician.
2. Patients who discontinued from the core CACZ885D2307 study

Other protocol-defined inclusion/exclusion criteria may apply.

Trial design

17 participants in 1 patient group

canakinumab

Experimental group

Description:

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)

Treatment:

Biological: ACZ885