Efficacy and Tolerability of BAF312 in Patients With Polymyositis and Dermatomyositis
Primary Purpose
Polymyositis, Dermatomyositis
Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
BAF312
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Polymyositis focused on measuring Inflammatory muscle disease, Muscle weakness, Myositis, inflammatory myopathy, inclusion body myositis, skin rash, autoimmune, difficulty swallowing, dysphagia
Eligibility Criteria
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
Sites / Locations
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
Arms of the Study
Arm 1
Arm 2
Arm Type
Placebo Comparator
Experimental
Arm Label
Placebo
BAF312
Arm Description
Outcomes
Primary Outcome Measures
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).
Secondary Outcome Measures
Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2
Mean Plasma Concentrations of BAF312
Summary of CRP Levels
Biomarkers reflecting efficacy in reducing systemic inflammatory components of the disease using serum markers such as C-reactive protein (CRP)
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.
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.
Physician Global Activity Assessment
Physician's overall assessment on a single 0-10 cm scale, where the higher score indicates higher disease activity.
Patient Global Activity Assessment
Patient's overall assessment on a single 0-10 cm scale, where the higher score indicates higher disease activity.
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.
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.
Serum Levels of Muscle Enzymes
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01148810
Brief Title
Efficacy and Tolerability of BAF312 in Patients With Polymyositis and Dermatomyositis
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
Study Type
Interventional
2. Study Status
Record Verification Date
April 2019
Overall Recruitment Status
Terminated
Why Stopped
Trial was prematurely terminated due to enrollment challenges.
Study Start Date
June 15, 2010 (Actual)
Primary Completion Date
June 13, 2012 (Actual)
Study Completion Date
June 13, 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Polymyositis, Dermatomyositis
Keywords
Inflammatory muscle disease, Muscle weakness, Myositis, inflammatory myopathy, inclusion body myositis, skin rash, autoimmune, difficulty swallowing, dysphagia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
18 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Title
BAF312
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
BAF312
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Number of Participants Who Responded to BAF312
Description
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 Outcome Measure Information:
Title
Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2
Time Frame
12 weeks
Title
Mean Plasma Concentrations of BAF312
Time Frame
baseline to end of trial (day 196)
Title
Summary of CRP Levels
Description
Biomarkers reflecting efficacy in reducing systemic inflammatory components of the disease using serum markers such as C-reactive protein (CRP)
Time Frame
12 weeks
Title
Efficacy in Modifying Health-related Quality of Life Measured by SF-36
Description
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
Title
Myositis Disease (MD) Activity Scores
Description
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.
Time Frame
Week 12
Title
Physician Global Activity Assessment
Description
Physician's overall assessment on a single 0-10 cm scale, where the higher score indicates higher disease activity.
Time Frame
Baseline, Week 12
Title
Patient Global Activity Assessment
Description
Patient's overall assessment on a single 0-10 cm scale, where the higher score indicates higher disease activity.
Time Frame
Baseline, Week 12
Title
Manual Muscle Testing (MMT) - 8 Score
Description
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.
Time Frame
Baseline, Week 12
Title
Health Assessment Questionnaire
Description
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.
Time Frame
Baseline, Week 12
Title
Serum Levels of Muscle Enzymes
Time Frame
Baseline, Week 12
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigative Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Novartis Investigative Site
City
Prague 2
ZIP/Postal Code
128 50
Country
Czechia
Facility Name
Novartis Investigative Site
City
Budapest
ZIP/Postal Code
1083
Country
Hungary
Facility Name
Novartis Investigative Site
City
Debrecen
ZIP/Postal Code
4032
Country
Hungary
Facility Name
Novartis Investigative Site
City
Stockholm
ZIP/Postal Code
17176
Country
Sweden
Facility Name
Novartis Investigative Site
City
Manchester
ZIP/Postal Code
M6 8HD
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing 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
Learn more about this trial
Efficacy and Tolerability of BAF312 in Patients With Polymyositis and Dermatomyositis
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