This study investigated 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 was composed of 2 periods: a double-blind period 1 with BAF312 administered at different daily doses (0.5, 2, 10 mg and placebo) and a fixed-dose Period 2 in which BAF312 was administered at the dose of 2 mg daily .
The study was prematurely terminated based on the results of an interim analysis where BAF312 did not demonstrate superior efficacy over placebo and a dose-response relationship was not observed. There were no safety concerns. Approximately 56 participants were planned to be randomized. A total of 17 participants were enrolled and randomized by the time the study was terminated.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
17
Novartis Investigative Site
Phoenix, Arizona, United States
Novartis Investigative Site
Los Angeles, California, United States
Novartis Investigative Site
Orange, California, United States
Novartis Investigative Site
Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score
Each muscles tested was evaluated on a 0 - 10 scale where 0 indicated the weakest muscle score and 10 indicated the strongest muscle score. The total MMT24 score ranged from 0 - 240, where an increasing trend in the values indicates improvement. A positive change from baseline indicates improvement.
Time frame: Baseline, 6 months
BAF312 Plasma Concentration
Time frame: 6 months
Peripheral Blood Lymphocyte Counts
Absolute lymphocyte counts
Time frame: baseline, 6 months
Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score
Each muscles tested was evaluated on a 0-10 scale where 0 indicated the weakest muscle score and 10 indicated the strongest muscle score. the total MMT24 score ranged from 0-240, where an increasing trend in the values indicates improvement. A positive change from baseline indicates improvement.
Time frame: baseline, 3 months
Change From Baseline in 6 Minutes Walking Distance (6-MWD) Test
Time frame: baseline, 6 months
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Miami, Florida, United States
Novartis Investigative Site
Kansas City, Kansas, United States
Novartis Investigative Site
Boston, Massachusetts, United States
Novartis Investigative Site
Prague, Czech Republic, Czechia
Novartis Investigative Site
Chiba, Chiba, Japan
Novartis Investigative Site
Sendai, Miyagi, Japan