PolymyositisPhase 2NCT01801917
Efficacy and Tolerability of BAF312 in Patients With Polymyositis
A Phase 2 study testing BAF312 in people with Polymyositis, run by Novartis Pharmaceuticals at 8 sites in 6 countries.
Trial summary
This study assessed the efficacy, safety and tolerability of BAF312 administered orally in patients with clinically active polymyositis and also in patients with polymyositis who had shown inadequate response to corticosteroids and or DMARDs (disease modifying antirheumatic drugs). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Polymyositis
- What is being tested?
- BAF312
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 14 participants
- Where
- 8 sitesin 6 countries
- Study dates
- 24 Apr 2013to 5 Aug 2016
- 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
- "definite" or "probable" for polymyositis at least three months before Baseline
- active disease as defined by elevated CK levels, or other enzymes, or MRI/biopsy if enzymes are normal, and persisting muscle weakness
- stable dose of corticosteroid for at least 2 weeks prior to Baseline and should not have received a medium or high dose in the last 8 weeks prior to study entry.
- patients treated with methotrexate must have been on a stable dose for at least 6 weeks prior to Baseline.
Who cannot join
- Patients with overlap polymyositis, late-stage polymyositis, or other types of myositis.
- Preexisting severe cardiac or pulmonary involvement, malignancy of any organ system or significant eye diseases.
- Uncontrolled diabetes mellitus or diabetes complicated with organ involvement.
- Pregnant or nursing (lactating) women
Trial locations and contacts
8 sites in 6 countries.
8 sites in 6 countries
Hungary2 sites
Novartis Investigative Site
Budapest, 1083
Novartis Investigative Site
Debrecen, 4032
Taiwan2 sites
Novartis Investigative Site
Taichung, 40447
Novartis Investigative Site
Taichung, 40705
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
This study assessed the efficacy, safety and tolerability of BAF312 administered orally in patients with clinically active polymyositis and also in patients with polymyositis who had shown inadequate response to corticosteroids and or DMARDs (disease modifying antirheumatic drugs).
Read the detailed description
This study was stopped prematurely due to overall slow recruitment and no evidence for efficacy in a parallel study in dermatomyositis with an assumed similar pathophysiology. With very small sample sizes per group the overall results for this study including primary and all other efficacy and PD data are inconclusive
- Conditions
- Polymyositis
- Being tested
- Placebo, BAF312
- Keywords
- Polymyositis, Myositis, PM, chronic muscle inflammation, inflammatory myopathy, inclusion body myositis
- Medical subject headings
- Polymyositis, Myositis, Myositis, Inclusion Body
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
Matching placebo tablet for oral administration
BAF312Drug
BAF312 in 4 dosage strengths in tablet form: 0.25 mg, 0.5 mg, 1 mg, 2 mg for oral administration
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | 5 placebo tablets daily during non-titration phase Receives: Drug: Placebo |
| BAF312 2mg | Experimental | 1 tablet of BAF312 2 mg + 4 tablets of Placebo daily during non-titration phase Receives: Drug: BAF312 |
| BAF312 10 mg | Experimental | 5 tablets of BAF312 2 mg daily during non-titration phase Receives: Drug: BAF312 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline at Week 12 for BAF312 2 mg, 10 mg or Placebo (Once Daily) for Combined Efficacy Endpoint: Manual Muscle Testing in 24 Muscles (MMT24)
Manual Muscle Testing Scoring Sheet: Neck flexors, neck extensors and other designated muscles bilaterally (Biceps brachii, Deltoid middle, Quadriceps, Gluteus maximus, Gluteus medius, Trapezius, Iliopsoas, Hamstrings, Wrist extensors, Wrist Flexors, Ankle plantar flexors and Ankle dorsiflexors) were tested on a 0-10 scale by the Investigator. Posterior credibility interval from Bayesian analysis displayed as confidence interval. The scores range was 0 to 260. Higher scores indicate better outcome.
Measured: Baseline, at 12 weeks
Percent Change From Baseline at Week 12 for BAF312 2 mg, 10 mg or Placebo (Once Daily) Serum Creatine Kinase (CK) Levels
Serum creatine kinase (CK) were analyzed as part of the blood chemistry panel. Posterior credibility interval from Bayesian analysis displayed as confidence interval. The variable CK was log-transformed for statistical analysis and after estimation was converted to percent change from baseline divided by the mean baseline
Measured: Baseline, at 12 weeks
Secondary outcomes (3)
Six-minute Walking Distance (6MWD) at Week 12
This test assessed the distance a patient could walk in 6 minutes (Rutkove et al 2002). If the patient was not able to walk for 6 minutes then a 2 minute walking test was conducted
Measured: Baseline, 12 weeks
Six-minute Walking Distance (6MWD) at Week 24
This test assessed the distance a patient could walk in 6 minutes (Rutkove et al 2002). If the patient was not able to walk for 6 minutes then a 2 minute walking test was conducted
Measured: Baseline, 24 weeks
BAF312 Trough Plasma Concentrations (PK Set)
All blood samples were taken by either direct venipuncture or an indwelling cannula inserted in a forearm vein. For each sample, approximately 2 mL of blood was drawn. BAF312 was determined in ethylenediaminetetraacetic acid (EDTA) plasma using a validated liquid chromatography-tandem mass spectrometry (LC-MS/MS) bioanalytical method for the quantification. The anticipated lower limit of quantification (LLOQ) was 0.02 ng/mL using 0.1 mL of plasma
Measured: -7 Baseline, day 28, 56, 84
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Mar 2013 (estimated)
- Study start24 Apr 2013
- Primary completion5 Aug 2016
- Study completion5 Aug 2016
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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 14 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
- NCT01801917
- Sponsor's protocol ID
- CBAF312X2205
- Status last verified
- Jan 2018
- First submitted
- 1 Feb 2013
- Last updated
- 5 Jan 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Jan 2021. 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
- ClinicalTrials.gov ID
- NCT01801917
- Acronym / sponsor ID
- CBAF312X2205
- Conditions
- Polymyositis
- Keywords
- Polymyositis, Myositis, PM, chronic muscle inflammation, inflammatory myopathy, inclusion body myositis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 24 Apr 2013 (actual)
- Primary completion
- 5 Aug 2016 (actual)
- Study completion
- 5 Aug 2016 (actual)
- First posted
- 1 Mar 2013 (estimated)
- Results
- Posted 4 Jan 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study assessed the efficacy, safety and tolerability of BAF312 administered orally in patients with clinically active polymyositis and also in patients with polymyositis who had shown inadequate response to corticosteroids and or DMARDs (disease modifying antirheumatic drugs).
Detailed description
This study was stopped prematurely due to overall slow recruitment and no evidence for efficacy in a parallel study in dermatomyositis with an assumed similar pathophysiology. With very small sample sizes per group the overall results for this study including primary and all other efficacy and PD data are inconclusive
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: Placebo5 placebo tablets daily during non-titration phase |
| BAF312 2mg | Experimental | Drug: BAF3121 tablet of BAF312 2 mg + 4 tablets of Placebo daily during non-titration phase |
| BAF312 10 mg | Experimental | Drug: BAF3125 tablets of BAF312 2 mg daily during non-titration phase |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- "definite" or "probable" for polymyositis at least three months before Baseline
- active disease as defined by elevated CK levels, or other enzymes, or MRI/biopsy if enzymes are normal, and persisting muscle weakness
- stable dose of corticosteroid for at least 2 weeks prior to Baseline and should not have received a medium or high dose in the last 8 weeks prior to study entry.
- patients treated with methotrexate must have been on a stable dose for at least 6 weeks prior to Baseline.
Key exclusion criteria (4 listed)
- Patients with overlap polymyositis, late-stage polymyositis, or other types of myositis.
- Preexisting severe cardiac or pulmonary involvement, malignancy of any organ system or significant eye diseases.
- Uncontrolled diabetes mellitus or diabetes complicated with organ involvement.
- Pregnant or nursing (lactating) women
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 |
|---|---|---|
| PlaceboMatching placebo tablet for oral administration | Drug | Placebo |
| BAF312BAF312 in 4 dosage strengths in tablet form: 0.25 mg, 0.5 mg, 1 mg, 2 mg for oral administration | Drug | BAF312 10 mg, BAF312 2mg |
Primary outcome
Change From Baseline at Week 12 for BAF312 2 mg, 10 mg or Placebo (Once Daily) for Combined Efficacy Endpoint: Manual Muscle Testing in 24 Muscles (MMT24)
Time frame: Baseline, at 12 weeks
Manual Muscle Testing Scoring Sheet: Neck flexors, neck extensors and other designated muscles bilaterally (Biceps brachii, Deltoid middle, Quadriceps, Gluteus maximus, Gluteus medius, Trapezius, Iliopsoas, Hamstrings, Wrist extensors, Wrist Flexors, Ankle plantar flexors and Ankle dorsiflexors) were tested on a 0-10 scale by the Investigator. Posterior credibility interval from Bayesian analysis displayed as confidence interval. The scores range was 0 to 260. Higher scores indicate better outcome.
Percent Change From Baseline at Week 12 for BAF312 2 mg, 10 mg or Placebo (Once Daily) Serum Creatine Kinase (CK) Levels
Time frame: Baseline, at 12 weeks
Serum creatine kinase (CK) were analyzed as part of the blood chemistry panel. Posterior credibility interval from Bayesian analysis displayed as confidence interval. The variable CK was log-transformed for statistical analysis and after estimation was converted to percent change from baseline divided by the mean baseline
3 secondary outcomes
Six-minute Walking Distance (6MWD) at Week 12
Time frame: Baseline, 12 weeks
Six-minute Walking Distance (6MWD) at Week 24
Time frame: Baseline, 24 weeks
BAF312 Trough Plasma Concentrations (PK Set)
Time frame: -7 Baseline, day 28, 56, 84
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- A Plain Language Trial Summary is available on novartisclinicaltrials.comLink provided by the sponsor.
Related trials for Polymyositis
RecruitingObservational study
Natural History of Inflammatory Muscle Diseases
Inflammation In Skeletal Muscle
RecruitingPhase 1
A Study to Investigate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AZD5492 in Adult Participants With Systemic Lupus Erythematosus or Idiopathic Inflammatory Myopathies or Rheumatoid Arthritis.
Systemic Lupus Erythematosus, Idiopathic Inflammatory Myopathies
RecruitingObservational study
Environmental Risk Factors for the Anti-synthetase Syndrome
Myositis, Dermatomyositis
Enrolling by invitationPhase 3
A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Active Idiopathic Inflammatory Myopathy
Myositis, Active Idiopathic Inflammatory Myopathy
RecruitingPhase 2/3
RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy
Idiopathic Inflammatory Myopathy, Dermatomyositis
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
8 sites in 6 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, run by Novartis Pharmaceuticals at 8 sites in 6 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 4 inclusion and 4 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 5 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in Hungary, Taiwan, United States, Canada, Czechia and 1 more country. 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 4 Jan 2018). 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?
Change From Baseline at Week 12 for BAF312 2 mg, 10 mg or Placebo (Once Daily) for Combined Efficacy Endpoint: Manual Muscle Testing in 24 Muscles (MMT24) (time frame: Baseline, at 12 weeks). The study lists 2 primary outcomes in all. 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 5 Jan 2021.