Clinical Trial: Safety and Efficacy of BAF312 in Dermatomyositis
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Double Blind, Randomized, Placebo-controlled Study to Evaluate, Safety, Tolerability, Efficacy and Preliminary Dose-response of BAF312 in Patients With Active Dermatomyositis
Brief Summary: This study investigates the dose response relationship for the efficacy and safety of BAF312 compared to placebo in active DM patients over a treatment period of 6+6 months and to determine the minimum dose required for a maximal clinical effect. The study is composed of 2 periods: a double-blind period I with BAF312 administered at different daily doses (0.5, 2, 10 mg and placebo) and a fixed-dose Period II in which BAF312 will be administered at the dose of 2 mg daily .
Detailed Summary:
Sponsor: Novartis Pharmaceuticals
Current Primary Outcome: Manual Muscle Testing - 24 muscles (MMT-24). Efficacy of BAF312 will be assessed by comparing the improvements with every dose of BAF312 to that of placebo. [ Time Frame: 6 months ]
Original Primary Outcome: Manual Muscle Testing -8 muscles (MMT-8). Efficacy of BAF312 will be assessed by comparing the improvements with every dose of BAF312 to that of placebo. [ Time Frame: 6 months ]
Current Secondary Outcome:
- Adverse Events. All information obtained on adverse events will be displayed by treatment (dose group) and subject. [ Time Frame: 6 months ]Secondary variables include the incidence of adverse events.
- Pharmacokinetics. BAF312 plasma concentration data will be listed by treatment (dose group), subject and visit/sampling time point. Descriptive summary statistics will be provided by treatment and visit/sampling time point. [ Time Frame: 6 months ]Secondary variables include plasma BAF312 concentrations.
- Peripheral blood lymphocyte counts. Absolute lymphocyte counts will be plotted against time by dose level. [ Time Frame: 6 months ]Secondary variables include peripheral blood lymphocyte counts.
- Manual Muscle Testing - 24 muscles (MMT-24). Efficacy of BAF312 will be assessed by comparing the improvements with every dose of BAF312 to that of placebo. [ Time Frame: 3 months ]Changes from baseline in MMT-24 at 3 months will also be evaluated. The dose-response will be assessed in the same way as for the 6-month data.
- 6 Minutes Walking Distance test. Efficacy of BAF312 will be assessed by comparing the changes in walking distance across all doses of BAF312 to that of placebo [ Time Frame: 6 months ]Changes from baseline in 6 Minute walking distance at 6 months of treatment will be assessed.
Original Secondary Outcome:
- Adverse Events. All information obtained on adverse events will be displayed by treatment (dose group) and subject. [ Time Frame: 6 months ]Secondary variables include the incidence of adverse events.
- Pharmacokinetics. BAF312 plasma concentration data will be listed by treatment (dose group), subject and visit/sampling time point. Descriptive summary statistics will be provided by treatment and visit/sampling time point. [ Time Frame: 6 months ]Secondary variables include plasma BAF312 concentrations.
- Peripheral blood lymphocyte counts. Absolute lymphocyte counts will be plotted against time by dose level. [ Time Frame: 6 months ]Secondary variables include peripheral blood lymphocyte counts.
- Manual Muscle Testing-8 muscles (MMT-8). Efficacy of BAF312 will be assessed by comparing the improvements with every dose of BAF312 to that of placebo. [ Time Frame: 3 months ]Changes from baseline in MMT-8 at 3 months will also be evaluated. The dose-response will be assessed in the same way as for the 6-month data.
Information By: Novartis
Dates:
Date Received: December 2, 2013
Date Started: November 2013
Date Completion:
Last Updated: September 7, 2016
Last Verified: September 2016