Efficacy and Tolerability of BAF312 in Patients With Polymyositis
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ClinicalTrials.gov Identifier: NCT01801917 |
Recruitment Status :
Terminated
First Posted : March 1, 2013
Results First Posted : January 4, 2018
Last Update Posted : January 5, 2021
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Polymyositis | Drug: Placebo Drug: BAF312 | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 14 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
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 |
Actual Study Start Date : | April 24, 2013 |
Actual Primary Completion Date : | August 5, 2016 |
Actual Study Completion Date : | August 5, 2016 |

Arm | Intervention/treatment |
---|---|
Placebo Comparator: Placebo
5 placebo tablets daily during non-titration phase
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Drug: Placebo
Matching placebo tablet for oral administration |
Experimental: BAF312 2mg
1 tablet of BAF312 2 mg + 4 tablets of Placebo daily during non-titration phase
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Drug: BAF312
BAF312 in 4 dosage strengths in tablet form: 0.25 mg, 0.5 mg, 1 mg, 2 mg for oral administration |
Experimental: BAF312 10 mg
5 tablets of BAF312 2 mg daily during non-titration phase
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Drug: BAF312
BAF312 in 4 dosage strengths in tablet form: 0.25 mg, 0.5 mg, 1 mg, 2 mg for oral administration |
- 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
- Six-minute Walking Distance (6MWD) at Week 12 [ Time Frame: Baseline, 12 weeks ]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
- Six-minute Walking Distance (6MWD) at Week 24 [ Time Frame: Baseline, 24 weeks ]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
- BAF312 Trough Plasma Concentrations (PK Set) [ Time Frame: -7 Baseline, day 28, 56, 84 ]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

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Ages Eligible for Study: | 18 Years to 75 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- "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.
Exclusion Criteria:
- 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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01801917
United States, Arizona | |
Novartis Investigative Site | |
Phoenix, Arizona, United States, 85013 | |
Canada, Ontario | |
Novartis Investigative Site | |
Torono, Ontario, Canada, M5G 2C4 | |
Czechia | |
Novartis Investigative Site | |
Prague 2, Czechia, 128 50 | |
Hungary | |
Novartis Investigative Site | |
Budapest, Hungary, 1083 | |
Novartis Investigative Site | |
Debrecen, Hungary, 4032 | |
Poland | |
Novartis Investigative Site | |
Bydgoszcz, Poland, 85-168 | |
Taiwan | |
Novartis Investigative Site | |
Taichung, Taiwan, 40447 | |
Novartis Investigative Site | |
Taichung, Taiwan, 40705 |
Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
Responsible Party: | Novartis Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT01801917 |
Other Study ID Numbers: |
CBAF312X2205 |
First Posted: | March 1, 2013 Key Record Dates |
Results First Posted: | January 4, 2018 |
Last Update Posted: | January 5, 2021 |
Last Verified: | January 2018 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Polymyositis Myositis PM |
chronic muscle inflammation inflammatory myopathy inclusion body myositis |
Polymyositis Myositis Muscular Diseases |
Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases |