Efficacy and Tolerability of BAF312 in Patients With Polymyositis and Dermatomyositis

April 3, 2019 updated by: Novartis Pharmaceuticals

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

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 Overview

Status

Terminated

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

18

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Prague 2, Czechia, 128 50
        • Novartis Investigative Site
      • Budapest, Hungary, 1083
        • Novartis Investigative Site
      • Debrecen, Hungary, 4032
        • Novartis Investigative Site
      • Stockholm, Sweden, 17176
        • Novartis Investigative Site
      • Manchester, United Kingdom, M6 8HD
        • Novartis Investigative Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Novartis Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Experimental: BAF312

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants Who Responded to BAF312
Time Frame: 12 weeks
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2
Time Frame: 12 weeks
12 weeks
Mean Plasma Concentrations of BAF312
Time Frame: baseline to end of trial (day 196)
baseline to end of trial (day 196)
Summary of CRP Levels
Time Frame: 12 weeks
Biomarkers reflecting efficacy in reducing systemic inflammatory components of the disease using serum markers such as C-reactive protein (CRP)
12 weeks
Efficacy in Modifying Health-related Quality of Life Measured by SF-36
Time Frame: 12 weeks
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
Myositis Disease (MD) Activity Scores
Time Frame: Week 12
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
Physician Global Activity Assessment
Time Frame: Baseline, Week 12
Physician's overall assessment on a single 0-10 cm scale, where the higher score indicates higher disease activity.
Baseline, Week 12
Patient Global Activity Assessment
Time Frame: Baseline, Week 12
Patient's overall assessment on a single 0-10 cm scale, where the higher score indicates higher disease activity.
Baseline, Week 12
Manual Muscle Testing (MMT) - 8 Score
Time Frame: Baseline, Week 12
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
Health Assessment Questionnaire
Time Frame: Baseline, Week 12
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
Serum Levels of Muscle Enzymes
Time Frame: Baseline, Week 12
Baseline, Week 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 15, 2010

Primary Completion (Actual)

June 13, 2012

Study Completion (Actual)

June 13, 2012

Study Registration Dates

First Submitted

June 21, 2010

First Submitted That Met QC Criteria

June 21, 2010

First Posted (Estimate)

June 22, 2010

Study Record Updates

Last Update Posted (Actual)

April 25, 2019

Last Update Submitted That Met QC Criteria

April 3, 2019

Last Verified

April 1, 2019

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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