Efficacy and Tolerability of BAF312 in Patients With Polymyositis and Dermatomyositis

Dermatomyositis Clinical Trial

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 and Dermatomyositis

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01148810
• Other study ID #: CBAF312A2202
• Secondary ID: 2008-006311-21
• Status: Terminated
• Phase: Phase 2
• Start date: June 15, 2010
• Est. completion date: June 13, 2012

Study information

• Verified date: April 2019
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 18
• Est. completion date: June 13, 2012
• Est. primary completion date: June 13, 2012
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

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

Related Conditions & MeSH terms

• Dermatomyositis
• Polymyositis

Intervention

Drug:
• BAF312

• Placebo

Locations

Country	Name	City	State
Czechia	Novartis Investigative Site	Prague 2
Hungary	Novartis Investigative Site	Budapest
Hungary	Novartis Investigative Site	Debrecen
Sweden	Novartis Investigative Site	Stockholm
United Kingdom	Novartis Investigative Site	Manchester
United States	Novartis Investigative Site	Boston	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

United States,  Czechia,  Hungary,  Sweden,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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).	12 weeks
Secondary	Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2		12 weeks
Secondary	Mean Plasma Concentrations of BAF312		baseline to end of trial (day 196)
Secondary	Summary of CRP Levels	Biomarkers reflecting efficacy in reducing systemic inflammatory components of the disease using serum markers such as C-reactive protein (CRP)	12 weeks
Secondary	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.	12 weeks
Secondary	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.	Week 12
Secondary	Physician Global Activity Assessment	Physician`s overall assessment on a single 0-10 cm scale, where the higher score indicates higher disease activity.	Baseline, Week 12
Secondary	Patient Global Activity Assessment	Patient`s overall assessment on a single 0-10 cm scale, where the higher score indicates higher disease activity.	Baseline, Week 12
Secondary	Manual Muscle Testing (MMT) - 8 Score	Manual Muscle Testing - 8 (MMT-8): Assessment of designated muscles manually by scoring each muscle from 0 to 10 where 0 is no strength and 10 is maximum strength. MMT- 8 includes 7 bilateral muscles (potential score 0-70 x 2) and one unilateral (axial) muscle (0-10 x1) so the total score ranges from 0 to 150 (maximum) where higher score indicates more strength.	Baseline, Week 12
Secondary	Health Assessment Questionnaire	Health Assessment Questionnaire (HAQ): This questionnaire is a patient reported outcome (PRO) which is self-administered by the patient. It is used to assess disability and comprises various categories related to usual daily activities. The patients report the amount of difficulty they have in performing some of these activities. Each question asks on a scale ranging from 0 to 3 if the categories can be performed without any difficulty (scale 0) up to cannot be done at all (scale 3). The total score is derived from these sub-scores and ranges from 0 to 3 where higher HAQ indicates more disability.	Baseline, Week 12
Secondary	Serum Levels of Muscle Enzymes		Baseline, Week 12