Efficacy and Tolerability of BAF312 in Patients With Polymyositis

This study is currently recruiting participants. (see Contacts and Locations)
Verified October 2015 by Novartis
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
First received: February 1, 2013
Last updated: October 15, 2015
Last verified: October 2015
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
Drug: Placebo
Drug: BAF312
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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:

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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: Yes ]
    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.

Estimated Enrollment: 45
Study Start Date: April 2013
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: December 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
Genders Eligible for Study:   Both
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
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: NCT01801917

Contact: Novartis Pharmaceuticals 1-888-669-6682
Contact: Novartis Pharmaceuticals

  Show 37 Study Locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01801917     History of Changes
Other Study ID Numbers: CBAF312X2205
Study First Received: February 1, 2013
Last Updated: October 15, 2015
Health Authority: Canada: Health Canada
United States: Food and Drug Administration
Czech Republic: State Institute for Drug Control
Hungary: National Institute of Pharmacy
Poland: Ministry of Health
Belgium: Federal Agency for Medicinal Products and Health Products
Taiwan: Center for Drug Evaluation
Switzerland: Swissmedic

Keywords provided by Novartis:
Polymyositis, Myositis, PM

Additional relevant MeSH terms:
Muscular Diseases
Musculoskeletal Diseases
Nervous System Diseases
Neuromuscular Diseases

ClinicalTrials.gov processed this record on November 24, 2015