Clinical Trial: Safety,Tolerability,Pharmacokinetics and Efficacy of CFZ533 in Moderate to Severe Myasthenia Gravis
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Preliminarily Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of CFZ533 in Patients With Moderate
Brief Summary: The purpose of this study is to evaluate safety, tolerability, pharmacokinetics/pharmacodynamics and efficacy of CFZ533 as an add-on therapy to standard of care in patients with moderate to severe myasthenia gravis (MG).
Detailed Summary:
Sponsor: Novartis Pharmaceuticals
Current Primary Outcome: Mean change from baseline in the Quantitative Myastenia Gravis (QMG) score [ Time Frame: At week 25 ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Mean changes from baseline in the MGC score [ Time Frame: From baseline to week 49 ]
- Proportion of patients with improvement or worsening by ≥ 3 points in the QMG score [ Time Frame: From baseline to week 49 ]
- Proportion of patients intolerant to steroid taper [ Time Frame: from week 13 to week 49 ]
- Proportion of patients who discontinued due to inefficacy or worsening [ Time Frame: from baseline to week 49 ]
- Mean change from baseline in the Myasthenia Gravis-specific Activities of Daily Living scale (MG-ADL) [ Time Frame: From baseline to week 24 ]
- Mean changes from baseline in the QMG score [ Time Frame: From baseline to week 49 ]
- Mean change from baseline in the Myasthenia Gravis Quality of Life (MG QOL-15) [ Time Frame: From baseline to week 24 ]
Original Secondary Outcome: Same as current
Information By: Novartis
Dates:
Date Received: June 23, 2015
Date Started: September 29, 2015
Date Completion: December 27, 2017
Last Updated: April 10, 2017
Last Verified: April 2017
