臨床研究等提出・公開システム

Study of Efficacy and Safety of Canakinumab in Patients With Hereditary Periodic Fevers

Novartis Pharma K. K.

0120-003-293

Interventional

A randomized, double-blind, placebo controlled study

3

*Patient's written informed consent (or parent's written informed consent in case of pediatric patient) at screening
*Male and female patients at least 2 years of age at the time of the screening visit
*Confirmed diagnosis and active flare at randomization
*CRP >10mg/L at randomization

*Use of the following therapies (within varying protocol defined timeframes): Corticosteroids, anakinra, canakinumab, rilonacept, tocilizumab, TNF inhibitors, abatacept, tofacitinib, rituximab, leflunomide, thalidomide, cyclosporine, intravenous immunoglobulin, 6-Merceptopurine, azathioprine, cyclophosphamide, or chlorambucil, any other investigational biologics
*History of malignancy of any organ system (other than localized basal cell carcinoma of the skin or in - situ cervical cancer), treated or untreated
*Significant medical diseases, including but not limited to the following:
a. History of organ transplantation
b. Elevated liver enzymes >=2x ULN
c. Increase in total bilirubin
d. Serious hepatic disorder (Child-Pugh scores B or C)
e. Chronic Kidney Disease
f. Thyroid disease
g. Diagnosis of active peptic ulcer disease
h. Coagulopathy
i. Significant CNS effects including vertigo and dizziness
*Any conditions or significant medical problems which immunecompromise the patient and/or places the patient at unacceptable risk for immunomodulatory therapy
*Live vaccinations within 3 months prior to the start of the trial, during the trial, and up to 3 months following the last dose

Other protocol defined inclusion/exclusion may apply

Both

Hereditary Periodic Fevers

investigational material(s)
Generic name etc : ACZ885
INN of investigational material : canakinumab
Therapeutic category code : 399 Agents affecting metabolism, n.e.c.
Dosage and Administration for Investigational material : subcutaneous injection

control material(s)
Generic name etc : Placebo
INN of investigational material :
Therapeutic category code : --- Other
Dosage and Administration for Investigational material : subcutaneous injection

Proportion of participants with resolution of initial flare at time of the randomization and absence of new flares [ Time Frame: 16 weeks ]
To demonstrate significant reduction of disease activity with canakinumab versus placebo

Percentage of participants who achieve Physician's global assessment < 2 [ Time Frame: 16 weeks ]
Assessment of Physician's global assessment
Percentage of participantswith the serologic remission [ Time Frame: 16 weeks ]
Normalization of C-reactive protein, Serum Amyloid A