Safety and Efficacy of BAF312 in Dermatomyositis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02029274
Recruitment Status : Completed
First Posted : January 7, 2014
Last Update Posted : September 8, 2016
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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 .

Condition or disease Intervention/treatment Phase
Active Dermatomyositis Drug: BAF312 0.5 mg Drug: BAF312 2 mg Drug: BAF312 10 mg Drug: Placebo to BAF312 Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 17 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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.
Study Start Date : November 2013
Actual Primary Completion Date : February 2016
Actual Study Completion Date : February 2016

Arm Intervention/treatment
Experimental: BAF312 0.5mg
BAF312 0.5 mg/day
Drug: BAF312 0.5 mg
BAF312 0.5 mg once daily

Experimental: BAF312 2mg
BAF312 2 mg/day
Drug: BAF312 2 mg
BAF312 2 mg once daily

Experimental: BAF312 10 mg
BAF312 10 mg/day
Drug: BAF312 10 mg
BAF312 10 mg once daily

Placebo Comparator: Placebo
Drug: Placebo to BAF312
Placebo to BAF312 once daily

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

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
  • Other protocol-defined inclusion/exclusion criteria apply.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02029274

United States, Arizona
Novartis Investigative Site
Phoenix, Arizona, United States, 85028
United States, California
Novartis Investigative Site
Los Angeles, California, United States, 90095
Novartis Investigative Site
Orange, California, United States, 92868
United States, Florida
Novartis Investigative Site
Miami, Florida, United States, 33136
Novartis Investigative Site
Tampa, Florida, United States, 33612
United States, Kansas
Novartis Investigative Site
Kansas City, Kansas, United States, 66160
United States, Massachusetts
Novartis Investigative Site
Boston, Massachusetts, United States, 02115
Czech Republic
Novartis Investigative Site
Prague 2, Czech Republic, 128 50
Novartis Investigative Site
Chiba-city, Chiba, Japan, 260-0801
Novartis Investigative Site
Sendai city, Miyagi, Japan, 980-8574
Sponsors and Collaborators
Novartis Pharmaceuticals
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals

Responsible Party: Novartis Pharmaceuticals Identifier: NCT02029274     History of Changes
Other Study ID Numbers: CBAF312X2206
2013-001799-39 ( EudraCT Number )
First Posted: January 7, 2014    Key Record Dates
Last Update Posted: September 8, 2016
Last Verified: September 2016

Keywords provided by Novartis ( Novartis Pharmaceuticals ):

Additional relevant MeSH terms:
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Connective Tissue Diseases
Skin Diseases