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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 )

February 1, 2013
March 1, 2013
August 1, 2017
January 4, 2018
January 4, 2018
April 24, 2013
August 5, 2016   (Final data collection date for primary outcome measure)
  • 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.
  • 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
Combined efficacy endpoint: Manual Muscle Testing (MMT) and serum creatine kinase (CK) levels [ Time Frame: 12 weeks ]
Assessment of preliminary clinical efficacy of 2mg BAF312 once daily using MMT of 8 muscles (MMT-8) and serum CK levels
Complete list of historical versions of study NCT01801917 on ClinicalTrials.gov Archive Site
  • 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
  • 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
  • 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
Adverse Events [ Time Frame: 24 weeks ]
Number and percentage of adverse events will be tabulated by body systems and treatment.
Not Provided
Not Provided
 
Efficacy and Tolerability of BAF312 in Patients With Polymyositis
A Multi-centre Double-blind, Placebo Controlled, Proof of Concept Study to Evaluate the Efficacy and Tolerability of BAF312 in Patients With Polymyositis
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).
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
Interventional
Phase 2
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Polymyositis
  • Drug: Placebo
    Matching placebo tablet for oral administration
  • Drug: BAF312
    BAF312 in 4 dosage strengths in tablet form: 0.25 mg, 0.5 mg, 1 mg, 2 mg for oral administration
  • Placebo Comparator: Placebo
    5 placebo tablets daily during non-titration phase
    Intervention: Drug: Placebo
  • Experimental: BAF312 2mg
    1 tablet of BAF312 2 mg + 4 tablets of Placebo daily during non-titration phase
    Intervention: Drug: BAF312
  • Experimental: BAF312 10 mg
    5 tablets of BAF312 2 mg daily during non-titration phase
    Intervention: Drug: BAF312
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Terminated
14
August 5, 2016
August 5, 2016   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
18 Years to 75 Years   (Adult, Senior)
No
Contact information is only displayed when the study is recruiting subjects
Canada,   Czechia,   Hungary,   Poland,   Taiwan,   United States
Belgium,   Czech Republic,   Netherlands,   Switzerland
 
NCT01801917
CBAF312X2205
No
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Not Provided
Novartis ( Novartis Pharmaceuticals )
Novartis Pharmaceuticals
Not Provided
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Novartis
January 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP