- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01148810
Efficacy and Tolerability of BAF312 in Patients With Polymyositis and Dermatomyositis
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
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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Prague 2, Czechia, 128 50
- Novartis Investigative Site
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Budapest, Hungary, 1083
- Novartis Investigative Site
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Debrecen, Hungary, 4032
- Novartis Investigative Site
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Stockholm, Sweden, 17176
- Novartis Investigative Site
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Manchester, United Kingdom, M6 8HD
- Novartis Investigative Site
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Massachusetts
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Boston, Massachusetts, United States, 02115
- Novartis Investigative Site
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
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
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 |
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Placebo Comparator: Placebo
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Experimental: BAF312
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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
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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).
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12 weeks
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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
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12 weeks
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Mean Plasma Concentrations of BAF312
Time Frame: baseline to end of trial (day 196)
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baseline to end of trial (day 196)
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Summary of CRP Levels
Time Frame: 12 weeks
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Biomarkers reflecting efficacy in reducing systemic inflammatory components of the disease using serum markers such as C-reactive protein (CRP)
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12 weeks
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Efficacy in Modifying Health-related Quality of Life Measured by SF-36
Time Frame: 12 weeks
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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.
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12 weeks
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Myositis Disease (MD) Activity Scores
Time Frame: Week 12
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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.
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Week 12
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Physician Global Activity Assessment
Time Frame: Baseline, Week 12
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Physician's overall assessment on a single 0-10 cm scale, where the higher score indicates higher disease activity.
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Baseline, Week 12
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Patient Global Activity Assessment
Time Frame: Baseline, Week 12
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Patient's overall assessment on a single 0-10 cm scale, where the higher score indicates higher disease activity.
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Baseline, Week 12
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Manual Muscle Testing (MMT) - 8 Score
Time Frame: Baseline, Week 12
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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.
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Baseline, Week 12
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Health Assessment Questionnaire
Time Frame: Baseline, Week 12
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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.
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Baseline, Week 12
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Serum Levels of Muscle Enzymes
Time Frame: Baseline, Week 12
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Baseline, Week 12
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Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Nervous System Diseases
- Skin Diseases
- Musculoskeletal Diseases
- Connective Tissue Diseases
- Muscular Diseases
- Neuromuscular Diseases
- Myositis
- Dermatomyositis
- Polymyositis
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Immunosuppressive Agents
- Immunologic Factors
- Sphingosine 1 Phosphate Receptor Modulators
- Siponimod
Other Study ID Numbers
- CBAF312A2202
- 2008-006311-21 (EudraCT Number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
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
Studies a U.S. FDA-regulated device product
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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