Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2008-006311-21 | EudraCT Number |
Not provided
Not provided
Not provided
Trial was prematurely terminated due to enrollment challenges.
Not provided
Not provided
Not provided
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator |
| |
| BAF312 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BAF312 | Drug |
| ||
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2 | 12 weeks | |
| Mean Plasma Concentrations of BAF312 | baseline to end of trial (day 196) | |
Not provided
Inclusion Criteria:
Exclusion Criteria:
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Not provided
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Boston | Massachusetts | 02115 | United States | ||
| Novartis Investigative Site |
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
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The study was terminated after 18 patients had been enrolled, of which 14 were eligible for the primary efficacy analysis.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | BAF312/BAF312 | 2 tablets each of BAF312 5mg for oral administration in period 1 and 2 tablets each of BAF312 5mg in period 2 |
| FG001 | Placebo/BAF312 | 2 tablets of Placebo for oral administration in period 1 and 2 tablets each of BAF312 5mg in period 2 |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Randomized set: all patients with Polymyositi/Dermatomyositis were enrolled and randomized in the study.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | BAF312/BAF312 | 2 tablets each of BAF312 5mg for oral administration in period 1 and 2 tablets each of BAF312 5mg in period 2 |
| BG001 | Placebo/BAF312 | 2 tablets of Placebo for oral administration in period 1 and 2 tablets each of BAF312 5mg in period 2 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Randomized set |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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). | All patients with evaluable (or complete) PD measurement and no major protocol deviations which impact on PD data were included in the PD analysis set. | Posted | Count of Participants | Participants | 12 weeks |
|
Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit) up to approximately 2 years.
This was a randomized, double-blind, placebo-controlled trial followed by an open label extension period where all patients received BAF312.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | BAF312/BAF312 | 2 tablets each of BAF312 5mg for oral administration in period 1 and 2 tablets each of BAF312 5mg in period 2 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cholelithiasis | Hepatobiliary disorders | MedDRA (15.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Palpitations | Cardiac disorders | MedDRA (15.0) | Systematic Assessment |
The study was terminated due to new clinical data from overall BAF312 program (<10 mg/day already have optimal pharmacodynamic effect & clinical efficacy). There was no safety concern testing 10 mg BAF312 that would have required study termination.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | 862-778-8300 | Novartis.email@novartis.com |
| ID | Term |
|---|---|
| D017285 | Polymyositis |
| D003882 | Dermatomyositis |
| D009220 | Myositis |
| D018908 | Muscle Weakness |
| D018979 | Myositis, Inclusion Body |
| D005076 | Exanthema |
| D003680 | Deglutition Disorders |
| ID | Term |
|---|---|
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C578989 | siponimod |
Not provided
Not provided
Not provided
Not provided
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Not provided
|
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| Serum Levels of Muscle Enzymes | Baseline, Week 12 |
| Prague |
| 128 50 |
| Czechia |
| Novartis Investigative Site | Budapest | 1083 | Hungary |
| Novartis Investigative Site | Debrecen | 4032 | Hungary |
| Novartis Investigative Site | Stockholm | 17176 | Sweden |
| Novartis Investigative Site | Manchester | M6 8HD | United Kingdom |
| Withdrawal by Subject |
|
| Protocol Violation |
|
| BG002 | Total | Total of all reporting groups |
| Standard Deviation |
| Years |
|
| Sex: Female, Male | Randomized set | Count of Participants | Participants |
|
2 tablets each of BAF312 5mg for oral administration in period 1 and 2 tablets each of BAF312 5mg in period 2
| OG001 | Placebo/BAF312 | 2 tablets of Placebo for oral administration in period 1 and 2 tablets each of BAF312 5mg in period 2 |
|
|
|
| Secondary | Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2 | The Safety set included all patients who received at least one dose of study medication. | Posted | Count of Participants | Participants | 12 weeks |
|
|
|
| Secondary | Mean Plasma Concentrations of BAF312 | PK Analysis set in all disease types | Posted | Mean | Standard Deviation | ng/ml | 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) | The Safety set included all patients who received at least one dose of study medication. | Posted | Mean | Standard Deviation | mg/L | 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. | All patients with evaluable (or complete) PD measurement and no major protocol deviations which impact on PD data were included in the PD analysis set. | Posted | Mean | Standard Deviation | score on a scale | 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. | PD set in all disease types | Posted | Mean | Standard Deviation | cm | 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. | PD set in all disease types | Posted | Mean | Standard Deviation | cm | 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. | PD set in all disease types | Posted | Mean | Standard Deviation | cm | 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. | PD set in all disease types | Posted | Mean | Standard Deviation | scores on a scale | 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. | PD set in all disease types | Posted | Mean | Standard Deviation | Score on a scale | Baseline, Week 12 |
|
|
|
| Secondary | Serum Levels of Muscle Enzymes | PD set in all disease types | Posted | Mean | Standard Deviation | U/L | Baseline, Week 12 |
|
|
|
| 0 |
| 16 |
| 0 |
| 16 |
| 12 |
| 16 |
| EG001 | Placebo/BAF312 | 2 tablets of Placebo for oral administration in period 1 and 2 tablets each of BAF312 5mg in period 2 | 0 | 10 | 3 | 10 | 7 | 10 |
| Peritonitis | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Spinal compression fracture | Injury, poisoning and procedural complications | MedDRA (15.0) | Systematic Assessment |
|
| Cataract | Eye disorders | MedDRA (15.0) | Systematic Assessment |
|
| Conjunctivitis | Eye disorders | MedDRA (15.0) | Systematic Assessment |
|
| Macular oedema | Eye disorders | MedDRA (15.0) | Systematic Assessment |
|
| Periorbital oedema | Eye disorders | MedDRA (15.0) | Systematic Assessment |
|
| Vision blurred | Eye disorders | MedDRA (15.0) | Systematic Assessment |
|
| Visual acuity reduced | Eye disorders | MedDRA (15.0) | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Aphthous stomatitis | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Dental caries | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Dysphagia | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Frequent bowel movements | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Haemorrhoids | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Salivary hypersecretion | Gastrointestinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Oedema peripheral | General disorders | MedDRA (15.0) | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (15.0) | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Oral herpes | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Tinea versicolour | Infections and infestations | MedDRA (15.0) | Systematic Assessment |
|
| Spinal compression fracture | Injury, poisoning and procedural complications | MedDRA (15.0) | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Intervertebral disc compression | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Basal cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (15.0) | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA (15.0) | Systematic Assessment |
|
| Dysgeusia | Nervous system disorders | MedDRA (15.0) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (15.0) | Systematic Assessment |
|
| Sciatica | Nervous system disorders | MedDRA (15.0) | Systematic Assessment |
|
| Tremor | Nervous system disorders | MedDRA (15.0) | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (15.0) | Systematic Assessment |
|
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Mechanical urticaria | Skin and subcutaneous tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Rosacea | Skin and subcutaneous tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Skin mass | Skin and subcutaneous tissue disorders | MedDRA (15.0) | Systematic Assessment |
|
| Haematoma | Vascular disorders | MedDRA (15.0) | Systematic Assessment |
|
| Hypertension | Vascular disorders | MedDRA (15.0) | Systematic Assessment |
|
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012871 | Skin Diseases |
| D020879 | Neuromuscular Manifestations |
| D009461 | Neurologic Manifestations |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012816 | Signs and Symptoms |
| D004935 | Esophageal Diseases |
| D005767 | Gastrointestinal Diseases |
| D004066 | Digestive System Diseases |
| D010608 | Pharyngeal Diseases |
| D010038 | Otorhinolaryngologic Diseases |
| Steroid stable |
|
| Title | Measurements |
|---|---|
|
| Day 28 |
|
|
| Day 56 |
|
|
| Day 84 |
|
|
| Day 92 |
|
|
| Day 112 |
|
|
| Day 140 |
|
|
| Day 168 |
|
|
| Day 196 |
|
|
| Cutaneous disease activity |
|
|
| Week 12 |
|
|
| Week 12 |
|
|
| Week 12 |
|
|
| Week 12 |
|
|
| Creatine Kinase - Week 12 |
|
|