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Efficacy and Tolerability of BAF312 in Patients With Polymyositis

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ClinicalTrials.gov Identifier: NCT01801917
Recruitment Status : Terminated
First Posted : March 1, 2013
Results First Posted : January 4, 2018
Last Update Posted : January 4, 2018
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Description
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Brief Summary:
This study assessed the efficacy, safety and tolerability of BAF312 administered orally in patients with clinically active polymyositis and also in patients with polymyositis who had shown inadequate response to corticosteroids and or DMARDs (disease modifying antirheumatic drugs).

Condition or disease Intervention/treatment Phase
Polymyositis Drug: Placebo Drug: BAF312 Phase 2

Detailed Description:
This study was stopped prematurely due to overall slow recruitment and no evidence for efficacy in a parallel study in dermatomyositis with an assumed similar pathophysiology. With very small sample sizes per group the overall results for this study including primary and all other efficacy and PD data are inconclusive

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 14 participants
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
Actual Study Start Date : April 24, 2013
Actual Primary Completion Date : August 5, 2016
Actual Study Completion Date : August 5, 2016

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Placebo Comparator: Placebo
5 placebo tablets daily during non-titration phase
 Drug: Placebo
Matching placebo tablet for oral administration
Experimental: BAF312 2mg
1 tablet of BAF312 2 mg + 4 tablets of Placebo daily during non-titration phase
 Drug: BAF312
BAF312 in 4 dosage strengths in tablet form: 0.25 mg, 0.5 mg, 1 mg, 2 mg for oral administration
Experimental: BAF312 10 mg
5 tablets of BAF312 2 mg daily during non-titration phase
 Drug: BAF312
BAF312 in 4 dosage strengths in tablet form: 0.25 mg, 0.5 mg, 1 mg, 2 mg for oral administration


Outcome Measures
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Primary Outcome Measures :
  1. Change From Baseline at Week 12 for BAF312 2 mg, 10 mg or Placebo (Once Daily) for Combined Efficacy Endpoint: Manual Muscle Testing in 24 Muscles (MMT24) [ Time Frame: Baseline, at 12 weeks ]
    Manual Muscle Testing Scoring Sheet: Neck flexors, neck extensors and other designated muscles bilaterally (Biceps brachii, Deltoid middle, Quadriceps, Gluteus maximus, Gluteus medius, Trapezius, Iliopsoas, Hamstrings, Wrist extensors, Wrist Flexors, Ankle plantar flexors and Ankle dorsiflexors) were tested on a 0-10 scale by the Investigator. Posterior credibility interval from Bayesian analysis displayed as confidence interval. The scores range was 0 to 260. Higher scores indicate better outcome.

  2. Percent Change From Baseline at Week 12 for BAF312 2 mg, 10 mg or Placebo (Once Daily) Serum Creatine Kinase (CK) Levels [ Time Frame: Baseline, at 12 weeks ]
    Serum creatine kinase (CK) were analyzed as part of the blood chemistry panel. Posterior credibility interval from Bayesian analysis displayed as confidence interval. The variable CK was log-transformed for statistical analysis and after estimation was converted to percent change from baseline divided by the mean baseline


Secondary Outcome Measures :
  1. Six-minute Walking Distance (6MWD) at Week 12 [ Time Frame: Baseline, 12 weeks ]
    This test assessed the distance a patient could walk in 6 minutes (Rutkove et al 2002). If the patient was not able to walk for 6 minutes then a 2 minute walking test was conducted

  2. Six-minute Walking Distance (6MWD) at Week 24 [ Time Frame: Baseline, 24 weeks ]
    This test assessed the distance a patient could walk in 6 minutes (Rutkove et al 2002). If the patient was not able to walk for 6 minutes then a 2 minute walking test was conducted

  3. BAF312 Trough Plasma Concentrations (PK Set) [ Time Frame: -7 Baseline, day 28, 56, 84 ]
    All blood samples were taken by either direct venipuncture or an indwelling cannula inserted in a forearm vein. For each sample, approximately 2 mL of blood was drawn. BAF312 was determined in ethylenediaminetetraacetic acid (EDTA) plasma using a validated liquid chromatography-tandem mass spectrometry (LC-MS/MS) bioanalytical method for the quantification. The anticipated lower limit of quantification (LLOQ) was 0.02 ng/mL using 0.1 mL of plasma


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

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
Contacts and Locations
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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 ClinicalTrials.gov identifier (NCT number): NCT01801917


Locations
United States, Arizona
Novartis Investigative Site
Phoenix, Arizona, United States, 85013
Canada, Ontario
Novartis Investigative Site
Torono, Ontario, Canada, M5G 2C4
Czechia
Novartis Investigative Site
Prague 2, Czechia, 128 50
Hungary
Novartis Investigative Site
Budapest, Hungary, 1083
Novartis Investigative Site
Debrecen, Hungary, 4032
Poland
Novartis Investigative Site
Bydgoszcz, Poland, 85-168
Taiwan
Novartis Investigative Site
Taichung, Taiwan, 40447
Novartis Investigative Site
Taichung, Taiwan, 40705
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
More Information
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01801917     History of Changes
Other Study ID Numbers: CBAF312X2205
First Posted: March 1, 2013    Key Record Dates
Results First Posted: January 4, 2018
Last Update Posted: January 4, 2018
Last Verified: January 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Polymyositis
Myositis
PM
 chronic muscle inflammation
inflammatory myopathy
inclusion body myositis

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