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Safety and Efficacy of BAF312 in Dermatomyositis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT02029274
First received: December 2, 2013
Last updated: September 7, 2016
Last verified: September 2016
  Purpose
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 Intervention 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
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, 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.

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]
    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:
  • Adverse Events. All information obtained on adverse events will be displayed by treatment (dose group) and subject. [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    Changes from baseline in 6 Minute walking distance at 6 months of treatment will be assessed.


Enrollment: 17
Study Start Date: November 2013
Study Completion Date: February 2016
Primary Completion Date: February 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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
placebo
Drug: Placebo to BAF312
Placebo to BAF312 once daily

  Eligibility

Ages Eligible for Study:   18 Years to 70 Years   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02029274

Locations
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
Japan
Novartis Investigative Site
Chiba-city, Chiba, Japan, 260-0801
Novartis Investigative Site
Sendai city, Miyagi, Japan, 980-8574
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02029274     History of Changes
Other Study ID Numbers: CBAF312X2206  2013-001799-39 
Study First Received: December 2, 2013
Last Updated: September 7, 2016
Health Authority: United States: Food and Drug Administration

Keywords provided by Novartis:
Dermatomyositis

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

ClinicalTrials.gov processed this record on December 05, 2016