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

Hereditary Periodic Fevers Clinical Trial

Official title: A Randomized, Double-blind, Placebo Controlled Study of Canakinumab in Patients With Hereditary Periodic Fevers (TRAPS, HIDS, or crFMF), With Subsequent Randomized Withdrawal/Dosing Frequency Reduction and Open-label Long-term Treatment Epochs

Status Completed

Clinical Trial Summary

This study is to determine whether canakinumab is able to induce and maintain a clinically meaningful reduction of disease activity in participants with Hereditary Periodic Fevers (HPF) compared to placebo.

Clinical Trial Description

This study consists of 3 randomized cohorts (one per condition of colchicine resistant/intolerant Familial Mediterranean Fever (crFMF), Hyper Immunoglobulin D Syndrome (also known as mevalonate kinase deficiency (HIDS/MKD), and Tumor Necrosis Factor Receptor Associated Periodic Syndrome (TRAPS), and 4 study epochs:

1. Epoch 1: a screening epoch to assess participant's eligibility;

2. Epoch 2: a randomized treatment epoch of 16 weeks where participants are randomized to canakinumab 150 mg every 4 weeks (q4w) or to placebo to obtain efficacy and safety data in a double-blind placebo controlled parallel-arm setting. This epoch contained 2 possible escape options :

1. early blinded escape option for non responders from Day 8 to Day 28 with here an add-on dose of 150mg canakinumab followed by blinded uptitration at the next scheduled visit (Day 29)

2. late unblinded escape option for non responders from Day 29 to Day 112; with open-label uptitration

3. Epoch 3: a randomized withdrawal epoch of 24 weeks where canakinumab responders from the randomized treatment epoch were re-randomized to canakinumab 150mg q8w or placebo to assess the potential for canakinumab to maintain clinical efficacy at a reduced dosing frequency;

4. Epoch 4: an open-label treatment epoch of 72 weeks to collect long-term ;

Related Conditions & MeSH terms

• Amyloidosis
• Familial Mediterranean Fever
• Fever
• Hereditary Autoinflammatory Diseases
• Hereditary Periodic Fevers

• NCT number: NCT02059291
• Study type: Interventional
• Source: Novartis
• Status: Completed
• Phase: Phase 3
• Start date: June 27, 2014
• Completion date: July 4, 2017