PolymyositisPhase 2NCT01148810
Efficacy and Tolerability of BAF312 in Patients With Polymyositis and Dermatomyositis
A Phase 2 study testing BAF312 in people with Polymyositis and Dermatomyositis, run by Novartis Pharmaceuticals at 6 sites in 5 countries.
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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Polymyositis, Dermatomyositis
- What is being tested?
- BAF312
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 18 participants
- Where
- 6 sitesin 5 countries
- Study dates
- 15 Jun 2010to 13 Jun 2012
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Other idiopathic inflammatory myopathies
- Myopathy other than polymyositis and dermatomyositis
- Patients with late stages of disease
Trial locations and contacts
6 sites in 5 countries.
6 sites in 5 countries
Hungary2 sites
Novartis Investigative Site
Budapest, 1083
Novartis Investigative Site
Debrecen, 4032
United States1 site
Novartis Investigative Site
Boston, Massachusetts, 02115
About this study
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
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.
- Conditions
- Polymyositis, Dermatomyositis
- Being tested
- BAF312, Placebo
- Keywords
- Inflammatory muscle disease, Muscle weakness, Myositis, inflammatory myopathy, inclusion body myositis, skin rash, autoimmune, difficulty swallowing, dysphagia
- Medical subject headings
- Polymyositis, Dermatomyositis, Myositis, Muscle Weakness, Myositis, Inclusion Body, Exanthema, Deglutition Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BAF312Drug
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
| BAF312 | Experimental | Not described Receives: Drug: BAF312 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 12 weeks
Secondary outcomes (10)
Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2
Measured: 12 weeks
Mean Plasma Concentrations of BAF312
Measured: baseline to end of trial (day 196)
Summary of CRP Levels
Biomarkers reflecting efficacy in reducing systemic inflammatory components of the disease using serum markers such as C-reactive protein (CRP)
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline, Week 12
Serum Levels of Muscle Enzymes
Measured: Baseline, Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2010 (estimated)
- Study start15 Jun 2010
- Primary completion13 Jun 2012
- Study completion13 Jun 2012
Why it stopped: Trial was prematurely terminated due to enrollment challenges.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for PolymyositisStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01148810
- Sponsor's protocol ID
- CBAF312A2202
- Other IDs
- 2008-006311-21, 2008
- Status last verified
- Apr 2019
- First submitted
- 21 Jun 2010
- Last updated
- 25 Apr 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Study at a glance
From the registry record, last updated by the sponsor on 25 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01148810
- Acronym / sponsor ID
- CBAF312A2202
- Conditions
- Polymyositis, Dermatomyositis
- Keywords
- Inflammatory muscle disease, Muscle weakness, Myositis, inflammatory myopathy, inclusion body myositis, skin rash, autoimmune, difficulty swallowing, dysphagia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Trial was prematurely terminated due to enrollment challenges.
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 15 Jun 2010 (actual)
- Primary completion
- 13 Jun 2012 (actual)
- Study completion
- 13 Jun 2012 (actual)
- First posted
- 22 Jun 2010 (estimated)
- Results
- Posted 25 Apr 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: Placebo |
| BAF312 | Experimental | Drug: BAF312 |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (3 listed)
- Other idiopathic inflammatory myopathies
- Myopathy other than polymyositis and dermatomyositis
- Patients with late stages of disease
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BAF312 | Drug | BAF312 |
| Placebo | Drug | Placebo |
Primary outcome
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).
10 secondary outcomes
Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2
Time frame: 12 weeks
Mean Plasma Concentrations of BAF312
Time frame: baseline to end of trial (day 196)
Summary of CRP Levels
Time frame: 12 weeks
Efficacy in Modifying Health-related Quality of Life Measured by SF-36
Time frame: 12 weeks
Myositis Disease (MD) Activity Scores
Time frame: Week 12
Physician Global Activity Assessment
Time frame: Baseline, Week 12
Patient Global Activity Assessment
Time frame: Baseline, Week 12
Manual Muscle Testing (MMT) - 8 Score
Time frame: Baseline, Week 12
Health Assessment Questionnaire
Time frame: Baseline, Week 12
Serum Levels of Muscle Enzymes
Time frame: Baseline, Week 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Apr 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
6 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BAF312 in people with Polymyositis and Dermatomyositis, run by Novartis Pharmaceuticals at 6 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 25 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Hungary, United States, Czechia, Sweden, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Apr 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Who Responded to BAF312 (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Apr 2019.