Efficacy and Tolerability of BAF312 in Patients With Polymyositis and Dermatomyositis

Phase 2TerminatedNCT01148810

Novartis Pharmaceuticals18 enrolled

Overview

This study determined the efficacy, safety, tolerability and the PK profile of BAF312, a novel immunomodulator, in polymyositis and dermatomyositis patients who were not responsive to traditional immunosuppressive and/or corticosteroid therapy. The study consisted of a 12 week, randomized, placebo controlled period, followed by another 12 weeks where all subjects received BAF312 treatment.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Polymyositis Dermatomyositis

Interventions

BAF312DRUG

PlaceboDRUG

Eligibility

Sex: ALLMin age: 18 YearsMax age: 75 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Patients with disease at least 3 months before study * Muscle weakness * Received corticosteroids with or with out disease modifying antirheumatic drugs at least 3 months before study however not responding to this therapy Exclusion Criteria: * Other idiopathic inflammatory myopathies * Myopathy other than polymyositis and dermatomyositis * Patients with late stages of disease

Locations (6)

Novartis Investigative Site

Boston, Massachusetts, United States

Novartis Investigative Site

Prague, Czechia

Novartis Investigative Site

Budapest, Hungary

Novartis Investigative Site

Debrecen, Hungary

Outcomes

Primary Outcomes

Number of Participants Who Responded to BAF312

Preliminary clinical efficacy of BAF312 in patients with Polymyositis and dermatomyositis (PM/DM) using the International Myositis Assessment and Clinical Studies Group (IMACS) core set measures (including manual muscle testing, Physician's Global Activity Assessment (on a horizontal 10 cm visual analogue scale), Patient Global Activity Assessment (on a horizontal 10 cm visual analogue scale), Physical Function (Health Assessment Questionnaire), Muscle-associated Enzymes (CK, LDH, AST, ALT, aldolase) and Extra-Muscular Activity Assessment (Extra-muscular portion of Myositis Disease Activity Assessment Tool).

Time frame: 12 weeks

Secondary Outcomes

Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2

Time frame: 12 weeks

Mean Plasma Concentrations of BAF312

Time frame: baseline to end of trial (day 196)

Summary of CRP Levels

Biomarkers reflecting efficacy in reducing systemic inflammatory components of the disease using serum markers such as C-reactive protein (CRP)

Time frame: 12 weeks

Efficacy in Modifying Health-related Quality of Life Measured by SF-36

Short Form (36) Health Survey. The SF-36 consists of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score the more disability. The higher the score the less disability i.e., a score of zero is equivalent to maximum disability.

Time frame: 12 weeks

Myositis Disease (MD) Activity Scores

Myositis Disease Activity Scores. This is a combined tool that captures the physician's assessment of disease activity of various organ systems via the MYOSITIS INTENTION TO TREAT ACTIVITY INDEX (MITAX) and via the MYOSITIS DISEASE ACTIVITY ASSESSMENT VISUAL ANALOGUE SCALES (MYOACT) It rates the physician's overall assessment of the ongoing current disease activity for various systems by drawing a vertical mark on the 10-cm line for each system according to the following scale: left end of line = no evidence of disease activity, midpoint of line = moderate disease activity, and right end of line = extreme or maximum disease activity.

Data from ClinicalTrials.gov

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