Safety and Efficacy of BAF312 in Dermatomyositis



Status:Terminated
Healthy:No
Age Range:18 - 75
Updated:1/17/2019
Start Date:August 25, 2013
End Date:February 17, 2016

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A Double Blind, Randomized, Placebo-controlled Study to Evaluate, Safety, Tolerability, Efficacy and Preliminary Dose-response of BAF312 in Patients With Active Dermatomyositis (DM)

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.

Key Inclusion Criteria:

Written informed consent must be obtained before any assessment is performed.

- Patients who have been defined as "definite" or "probable" based on the criteria of
Bohan and Peter (Bohan and Peter 1975) for dermatomyositis at least 3 months before
screening

- Patients must have active disease as defined by muscle weakness

- Patients may be on a stable dose of corticosteroid (up/equal to 20 mg once daily
prednisone equivalent)

- Patients currently treated with oral or subcutaneous MTX must have been a stable dose
of no more/equal to than 25 mg per week

- Patients currently treated with Azathioprine must have been a stable maintenance dose
of no more/equal to 3 mg/kg/day

- Negative cancer screening conducted in the 12 months prior to screening visit

Key Exclusion Criteria

- Dermatomyositis patients having overlap myositis or any other type of myositis
including paraneoplastic myositis, drug-induced myopathy, necrotizing myositis

- Preexisting severe cardiac or pulmonary conditions, malignancy of any organ system or
significant eye diseases.

- Uncontrolled diabetes mellitus or diabetes complicated with organ involvement.

- Pregnant or nursing (lactating) women
We found this trial at
7
sites
Boston, Massachusetts 02118
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Kansas City, Kansas 66160
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Kansas City, KS
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Los Angeles, California 90095
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Los Angeles, CA
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Miami, Florida 33136
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Miami, FL
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Orange, California 92868
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Orange, CA
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Phoenix, Arizona 85012
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Phoenix, AZ
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Prague 2, Czech Republic 12850
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Prague 2,
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