Dataset Information

Efficacy and safety of canakinumab in adolescents and adults with colchicine-resistant familial Mediterranean fever.

ABSTRACT: This open-label pilot study aimed to investigate the efficacy of canakinumab in colchicine-resistant familial Mediterranean fever (FMF) patients.Patients with one or more attacks in a month in the preceding 3 months despite colchicine were eligible to enter a 30-day run-in period. Patients who had an attack during the first run-in period advanced to a second 30-day period. At the first attack, patients started to receive three canakinumab 150 mg subcutaneous injections at 4-week intervals, and were then followed for an additional 2 months. Primary efficacy outcome measure was the proportion of patients with 50 % or more reduction in attack frequency. Secondary outcome measures included time to next attack following last canakinumab dose and changes in quality of life assessed by SF-36.Thirteen patients were enrolled in the run-in period and 9 advanced to the treatment period. All 9 patients achieved a 50 % or more reduction in attack frequency, and only one patient had an attack during the treatment period. C-reactive protein and serum amyloid A protein levels remained low throughout the treatment period. Significant improvement was observed in both physical and mental component scores of the Short Form-36 at Day 8. Five patients had an attack during the 2-month follow-up, occurring median 71 (range, 31 to 78) days after the last dose. Adverse events were similar to those observed in the previous canakinumab trials.Canakinumab was effective at controlling the attack recurrence in patients with FMF resistant to colchicine. Further investigations are warranted to explore canakinumab's potential in the treatment of patients with colchicine resistant FMF.ClinicalTrials.gov NCT01088880 . Registered 16 March 2010.

SUBMITTER: Gul A

PROVIDER: S-EPMC4559892 | biostudies-literature | 2015 Sep

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Efficacy and safety of canakinumab in adolescents and adults with colchicine-resistant familial Mediterranean fever.

Gül Ahmet A Ozdogan Huri H Erer Burak B Ugurlu Serdal S Kasapcopur Ozgur O Davis Nicole N Sevgi Serhan S

Arthritis research & therapy 20150904

<h4>Introduction</h4>This open-label pilot study aimed to investigate the efficacy of canakinumab in colchicine-resistant familial Mediterranean fever (FMF) patients.<h4>Method</h4>Patients with one or more attacks in a month in the preceding 3 months despite colchicine were eligible to enter a 30-day run-in period. Patients who had an attack during the first run-in period advanced to a second 30-day period. At the first attack, patients started to receive three canakinumab 150 mg subcutaneous i ...[more]

PMID: 26337145

Similar Datasets

Project description:BACKGROUND:Familial Mediterranean fever (FMF) is an inherited disorder caused by a number of mutations of the Mediterranean fever (MEFV) gene, coding a protein named pyrin that acts as a major regulatory component of the inflammasome. The first-line drug for FMF treatment is colchicine, but 10% of patients with FMF do not respond well to colchicine. Although the efficacy of tocilizumab (TCZ), which is a recombinant, humanized, antihuman interleukin 6 (IL-6) receptor monoclonal antibody, has been reported to prevent FMF attacks, the effects of TCZ on individuals with colchicine-resistant or colchicine-intolerant FMF have not been evaluated in a randomized clinical trial. METHODS/DESIGN:In this phase III, investigator-initiated, multicenter, double-blind, randomized, parallel-group trial, the efficacy and safety of TCZ will be compared with placebo in patients with colchicine-resistant or colchicine-intolerant FMF. The study will be conducted in nine centers in Japan. Participants (n =?24) will be randomly assigned to receive 162?mg of TCZ (n =?12) or placebo (n =?12) administered subcutaneously once weekly for 24?weeks. Rescue treatment will be allowed if rescue criteria are met. A primary endpoint is the number of fever attacks until 24?weeks. Secondary endpoints include the number of occurrences of accompanying symptoms during attacks; the time until a fever attack occurs; the duration of fever attacks; serum C-reactive protein and serum amyloid A; 36-item Short Form Health Survey; general evaluation by a physician (100-mm visual analogue scale); body temperature; the percentage of subjects who achieve FMF 50 at 12?weeks and 24?weeks; and pharmacodynamic assessment, including the measurement of serum TCZ level and soluble IL-6 receptor. DISCUSSION:The study is expected to produce evidence regarding the efficacy of a potential new therapeutic agent, TCZ, in improving the clinical course and outcome for patients with colchicine-resistant or colchicine-intolerant FMF. TRIAL REGISTRATION:University Hospital Medical Information Network Clinical Trials Registry, UMIN000028010 . Registered on 7 July 2017.

Dataset Information

Efficacy and safety of canakinumab in adolescents and adults with colchicine-resistant familial Mediterranean fever.

Publications

Efficacy and safety of canakinumab in adolescents and adults with colchicine-resistant familial Mediterranean fever.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets