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 ]

The primary aim of this study is to assess the efficacy of different doses of BAF312 on the MMT-24 after 6 months. The overall efficacy of BAF312 will be assessed by comparing the improvements of MMT-24 with every dose of BAF312 to that of placebo. Then the doseresponse curve of MMT-24 will be estimated with the aim to determine a target dose for the program.


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 ]

The primary aim of this study is to assess the efficacy of different doses of BAF312 on the MMT-8 after 6 months. The overall efficacy of BAF312 will be assessed by comparing the improvements of MMT-8 with every dose of BAF312 to that of placebo. Then the doseresponse curve of MMT-8 will be estimated with the aim to determine a target dose for the program.


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