Efficacy and Tolerability of BAF312 in Patients With Polymyositis

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2015 by Novartis
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
First received: February 1, 2013
Last updated: February 17, 2015
Last verified: February 2015

This study will assess the efficacy, safety and tolerability of BAF312 administered orally in patients with clinically active 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, Investigator)
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 [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
    Assessment of preliminary clinical efficacy of 2mg BAF312 once daily using MMT of 8 muscles (MMT-8) and serum CK levels

Secondary Outcome Measures:
  • Adverse Events [ Time Frame: 24 weeks ] [ Designated as safety issue: Yes ]
    Number of adverse events will be tabulated by body systems and treatment.

Estimated Enrollment: 30
Study Start Date: April 2013
Estimated Study Completion Date: January 2016
Estimated Primary Completion Date: January 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: Placebo
Drug: Placebo
Experimental: BAF312
Active drug
Drug: BAF312
Patients randomized to active treatment will undergo a titration period of 6 days with BAF312. From day 6 patients will receive BAF312 (2 mg) for the entire duration of Period 1. Both patients randomized on active and on placebo will be offered to enter in the all-active extension Period 2, after the completion of Period 1.

Detailed Description:

This non-confirmatory PoC study will employ a randomized, parallel, double-blind, placebo-controlled, multi-center design followed by an optional, all-active (BAF312) extension period. Approximately 30 patients with PM will be enrolled. The study will consist of

  • Screening period: 13 days
  • Baseline period: 6 days
  • Period 1 - randomized treatment period: 12 weeks
  • Period 2 - optional all active (BAF312) extension period: 12 weeks
  • Follow up: 4 weeks after the last drug administration. Patients who complete Period 1 may opt to enter Period 2 allowing all placebo patients of Period 1 to be switched to BAF312; patients already receiving BAF312 in Period 1 to continue to receive active treatment and patients demonstrating clinical improvements to (further) taper down the dose of corticosteroid if they have not done so already after week 8 of Period 1

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 and persisting muscle weakness
  • must have received and inadequately responding to standard therapies (corticosteroid alone or with disease modifying antirheumatic drugs
  • 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 36 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: February 17, 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

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 October 08, 2015