Clinical Trial: Evaluate the Safety and Efficacy of Canakinumab in Pediatric Patients With Colchicine Intolerant or Colchicine Resistant Familial Mediterranean Fever (FMF)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A 6 Month Phase 2, Multi-Center, Open-label, Single Arm Study to Evaluate the Safety and Efficacy of Treatment With Canakinumab in Pediatric Patients With Colchicine Intolerant or Colchicine Resistant

Brief Summary:

A study designed to evaluate the role of treatment with a biological agent - Canakinumab in pediatric (age 4-20) Familial Mediterranean Fever (FMF) patients that are intolerant or resistant for colchicine treatment.

The study hypothesis is that Canakinumab will reduce attack frequency and severity.


Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: To measure the effect of canakinumab on the frequency of FMF attacks, defined as percentage of subjects with at least 50% reduction in the attack frequency during a 3 month treatment period [ Time Frame: 0-3 months ]

Original Primary Outcome: To measure the effect of canakinumab on the frequency of FMF attacks defined as percentage of subjects with at least 50% reduction in the attack frequency during 3 month treatment period [ Time Frame: 0-3 months ]

Current Secondary Outcome:

  • To assess the effect of canakinumab with regard to percentage of subjects with no attacks during the 3 months treatment period [ Time Frame: 0-3 months ]
  • To evaluate the safety and tolerability of canakinumab by monitoring adverse events (AEs) and subject discontinuations due to an AE [ Time Frame: 3 months ]
  • To assess the change in frequency of FMF attacks during the treatment period [ Time Frame: 3 months ]


Original Secondary Outcome:

  • To assess the effect of canakinumab with regard to percentage of subjects with no attacks during the 3 months treatment period [ Time Frame: 0-3 months ]
  • To evaluate the safety and tolerability of canakinumab by monitoring adverse events (AE) and subject discontinuations due to an AE [ Time Frame: 3 months ]
  • To assess the change in frequency of FMF attacks during the treatment period [ Time Frame: 3 months ]


Information By: Novartis

Dates:
Date Received: June 21, 2010
Date Started: December 2010
Date Completion:
Last Updated: November 16, 2016
Last Verified: November 2016