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Efficacy and Tolerability of BAF312 in Patients With Polymyositis

This study has been terminated.
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals ) Identifier:
First received: February 1, 2013
Last updated: February 19, 2017
Last verified: February 2017
This study will assess the efficacy, safety and tolerability of BAF312 administered orally in patients with clinically active polymyositis and also in patients with polymyositis who have shown inadequate response to corticosteroids and or DMARDs (disease modifying antirheumatic drugs).

Condition Intervention Phase
Polymyositis Drug: Placebo Drug: BAF312 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Multi-centre Double-blind, Placebo Controlled, Proof of Concept Study to Evaluate the Efficacy and Tolerability of BAF312 in Patients With Polymyositis

Resource links provided by NLM:

Further study details as provided by Novartis ( Novartis Pharmaceuticals ):

Primary Outcome Measures:
  • Combined efficacy endpoint: Manual Muscle Testing (MMT) and serum creatine kinase (CK) levels, or other enzymes, or MRI/biopsy if enzymes are normal. [ Time Frame: 12 weeks ]
    Assessment of preliminary clinical efficacy of 2mg and 10mg BAF312 once daily using MMT of 24 muscles (MMT-24) and serum CK levels, or other enzymes, or MRI/biopsy if enzymes are normal.

Secondary Outcome Measures:
  • Adverse Events (AEs), 6 minute walking distance test (MWD) and pharmacokinetics [ Time Frame: 24 weeks for AEs and 12 weeks for 6MWD ]
    Number of adverse events will be tabulated by body systems and treatment. Distance traveled over a 6 minute time period will be measured. Steady state pharmacokinetics of BAF312 will be characterized.

Enrollment: 14
Study Start Date: April 2013
Study Completion Date: August 2016
Primary Completion Date: August 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: Placebo
Drug: Placebo
Experimental: BAF312 2mg
BAF312 2 mg
Drug: BAF312
Experimental: BAF312 10 mg
BAF312 10 mg
Drug: BAF312


Ages Eligible for Study:   18 Years to 75 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • "definite" or "probable" for polymyositis at least three months before Baseline
  • active disease as defined by elevated CK levels, or other enzymes, or MRI/biopsy if enzymes are normal, and persisting muscle weakness
  • stable dose of corticosteroid for at least 2 weeks prior to Baseline and should not have received a medium or high dose in the last 8 weeks prior to study entry.
  • patients treated with methotrexate must have been on a stable dose for at least 6 weeks prior to Baseline.

Exclusion Criteria:

  • Patients with overlap polymyositis, late-stage polymyositis, or other types of myositis.

    • Preexisting severe cardiac or pulmonary involvement, 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 may apply.

  Contacts and Locations
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Please refer to this study by its identifier: NCT01801917

United States, Arizona
Novartis Investigative Site
Phoenix, Arizona, United States, 85013
Canada, Ontario
Novartis Investigative Site
Torono, Ontario, Canada, M5G 2C4
Czech Republic
Novartis Investigative Site
Prague 2, Czech Republic, 128 50
Novartis Investigative Site
Budapest, Hungary, 1083
Novartis Investigative Site
Debrecen, Hungary, 4032
Novartis Investigative Site
Bydgoszcz, Poland, 85-168
Novartis Investigative Site
Taichung, Taiwan, 40447
Novartis Investigative Site
Taichung, Taiwan, 40705
Sponsors and Collaborators
Novartis Pharmaceuticals
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis Pharmaceuticals Identifier: NCT01801917     History of Changes
Other Study ID Numbers: CBAF312X2205
Study First Received: February 1, 2013
Last Updated: February 19, 2017

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Polymyositis, Myositis, PM

Additional relevant MeSH terms:
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases processed this record on September 21, 2017