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TerminatedNCT01801917Updated Jan 5, 2021Results posted

Efficacy and Tolerability of BAF312 in Patients With Polymyositis

A Phase 2 interventional study of Placebo and BAF312 in Polymyositis, sponsored by Novartis Pharmaceuticals. Terminated at 8 sites in 6 countries. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2021-01-05.

Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
14
Allocation
Randomized
Ages
18 Years to 75 Years
Sex
All
01

Study 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 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

02

Conditions studied

  • Polymyositis

Keywords

  • Polymyositis
  • Myositis
  • PM
  • chronic muscle inflammation
  • inflammatory myopathy
  • inclusion body myositis
03

In context

Polymyositis

73 studies on the registry are indexed under Polymyositis; 19 are open to participants now.

This study's enrollment of 14 is below the median of 33 across 48 interventional studies indexed under Polymyositis.

Browse Polymyositis studies →

Lead sponsor

Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.

Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.

Browse Novartis Pharmaceuticals studies →All Novartis studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 75 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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/ if enzymes are normal, and persisting muscle weakness
  • stable dose of 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

Exclusion Criteria:

  • Patients with overlap polymyositis, late-stage polymyositis, or other types of myositis.
  • Preexisting severe cardiac or pulmonary involvement, of any organ system or significant eye diseases.
  • Uncontrolled diabetes mellitus or diabetes complicated with organ involvement.
  • Pregnant or nursing (lactating) women
Terms in these criteria, in plain language(3)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Biopsy and archival tissue

A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.

Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
14 participants (actual)

Study arms

  • Placebo comparator
    Placebo

    5 placebo tablets daily during non-titration phase

    Drug: Placebo

  • Experimental
    BAF312 2mg

    1 tablet of BAF312 2 mg + 4 tablets of Placebo daily during non-titration phase

    Drug: BAF312

  • Experimental
    BAF312 10 mg

    5 tablets of BAF312 2 mg daily during non-titration phase

    Drug: BAF312

Interventions

  • DrugPlacebo

    Matching placebo tablet for oral administration

  • DrugBAF312

    BAF312 in 4 dosage strengths in tablet form: 0.25 mg, 0.5 mg, 1 mg, 2 mg for oral administration

06

What researchers measure

Primary outcomes

  1. 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.

    Time frame: Baseline, at 12 weeks

  2. 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

    Time frame: Baseline, at 12 weeks

Secondary outcomes

  1. 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

    Time frame: Baseline, 12 weeks

  2. 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

    Time frame: Baseline, 24 weeks

  3. 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

    Time frame: -7 Baseline, day 28, 56, 84

07

Results

Posted Jan 4, 2018

Participant flow

Period 1 - Randomized
Participant flow — Period 1 - Randomized
MilestoneBAF312 2mg/BAF312 2mgBAF312 10 mg/BAF312 10 mgPlacebo/BAF312 2 mgPlacebo/BAF312 10 mg
Started7241
Completed6231
Not completed1010
Withdrew: Adverse event1010
Extension - All Active
Participant flow — Extension - All Active
MilestoneBAF312 2mg/BAF312 2mgBAF312 10 mg/BAF312 10 mgPlacebo/BAF312 2 mgPlacebo/BAF312 10 mg
Started6030
Completed6030
Not completed0000

Outcome measures

PrimaryChange 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.

Time frame:
Baseline, at 12 weeks
Reported as:
Mean · scores on a scale
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)
scores on a scaleBAF312 2mgBAF312 10 mgPlacebo
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)11.2 (3.5 to 19.2)39.0 (10.7 to 67.2)9.1 (-1.6 to 20.0)
Statistical analysis
  • BAF312 2mg vs Placebo · Bayesian: 0.586
  • BAF312 2mg vs Placebo · Bayesian: 0.022
  • BAF312 10 mg vs Placebo · Bayesian: 0.963
  • BAF312 10 mg vs Placebo · Bayesian: 0.837
PrimaryPercent 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

Time frame:
Baseline, at 12 weeks
Reported as:
Mean · U/L
Percent Change From Baseline at Week 12 for BAF312 2 mg, 10 mg or Placebo (Once Daily) Serum Creatine Kinase (CK) Levels
U/LBAF312 2mgBAF312 10 mgPlacebo
Percent Change From Baseline at Week 12 for BAF312 2 mg, 10 mg or Placebo (Once Daily) Serum Creatine Kinase (CK) Levels-19.7 (-32.3 to -4.7)-55.6 (-77.1 to -13.5)-0.5 (-21.8 to 25.9)
SecondarySix-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

Time frame:
Baseline, 12 weeks
Reported as:
Mean · meters
Six-minute Walking Distance (6MWD) at Week 12
metersBAF312 2mgBAF312 10 mgPlacebo
Period 1, Week 12362.47 ± 52.02393.00 ± 0.0303.10 ± 112.480
Distance walked,change from BL at Wk 1246.82 ± 65.6423.00 ± 0.0-6.40 ± 21.981
SecondarySix-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

Time frame:
Baseline, 24 weeks
Reported as:
Mean · meters
Six-minute Walking Distance (6MWD) at Week 24
metersBAF312 2mgPlacebo/BAF312 2 mg
Period 2, Week 24364.60 ± 73.803329.33 ± 186.551
Distance walked,change from baseline at Wk 2448.95 ± 91.9224.33 ± 51.637
SecondaryBAF312 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

Time frame:
-7 Baseline, day 28, 56, 84
Reported as:
Mean · ng/mL
BAF312 Trough Plasma Concentrations (PK Set)
ng/mLBAF312 2mgBAF312 10 mg
Day - 70 ± 00 ± 0
Day 2825.3 ± 11.2182 ± 0
Day 5625.1 ± 12.6270 ± 0
Day 8421.4 ± 10.1240 ± 0

Adverse events

Collected over 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.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Period 1 BAF312 2mg—1/7 (14.3%)6/7 (85.7%)
Period 1 BAF312 10mg—0/2 (0%)2/2 (100%)
Period 1 Placebo—0/5 (0%)4/5 (80%)
Period 2 BAF312 2mg/ BAF312 2mg—0/6 (0%)4/6 (66.7%)
Period 2 Placebo/ BAF312 2mg—0/3 (0%)2/3 (66.7%)
Most frequent serious events
Most frequent serious events
EventPeriod 1 BAF312 2mgPeriod 1 BAF312 10mgPeriod 1 PlaceboPeriod 2 BAF312 2mg/ BAF312 2mgPeriod 2 Placebo/ BAF312 2mg
Haemolytic anaemiaBlood and lymphatic system disorders1/70/20/50/60/3
Haemolytic uraemic syndromeBlood and lymphatic system disorders1/70/20/50/60/3
Acute kidney injuryRenal and urinary disorders1/70/20/50/60/3
Most frequent other events
Showing 10 of 35
Most frequent other events
EventPeriod 1 BAF312 2mgPeriod 1 BAF312 10mgPeriod 1 PlaceboPeriod 2 BAF312 2mg/ BAF312 2mgPeriod 2 Placebo/ BAF312 2mg
Eye painEye disorders0/71/20/50/60/3
VomitingGastrointestinal disorders0/71/20/50/60/3
Chest discomfortGeneral disorders0/71/20/50/60/3
Urinary tract infectionInfections and infestations0/71/20/50/60/3
DizzinessNervous system disorders0/71/22/50/60/3
HeadacheNervous system disorders2/70/22/50/60/3
CataractEye disorders1/70/20/50/61/3
DiarrhoeaGastrointestinal disorders1/70/20/50/61/3
Upper respiratory tract infectionInfections and infestations0/70/20/51/61/3
Gamma-glutamyltransferase increasedInvestigations0/70/20/50/61/3

Baseline characteristics

Age, Continuous
Age, Continuous(years)BAF312 2mg/BAF312 2mgBAF312 10 mg/BAF312 10 mgPlacebo/BAF312 2 mgPlacebo/BAF312 10 mgTotal
Mean50.3 ± 14.7847.0 ± 21.2148.0 ± 8.8353.0 ± 0.049.4 ± 12.51
Sex: Female, Male
Sex: Female, Male(Participants)BAF312 2mg/BAF312 2mgBAF312 10 mg/BAF312 10 mgPlacebo/BAF312 2 mgPlacebo/BAF312 10 mgTotal
Female522110
Male20204
Race/Ethnicity, Customized
Race/Ethnicity, Customized(participants)BAF312 2mg/BAF312 2mgBAF312 10 mg/BAF312 10 mgPlacebo/BAF312 2 mgPlacebo/BAF312 10 mgTotal
Caucasian713011
Black00011
Asian01102
Disease duration
Disease duration(years)BAF312 2mg/BAF312 2mgBAF312 10 mg/BAF312 10 mgPlacebo/BAF312 2 mgPlacebo/BAF312 10 mgTotal
Mean5.6 ± 4.465.4 ± 2.742.7 ± 1.6716.9 ± 05.6 ± 4.77
Baseline MMT24 Score
Baseline MMT24 Score(scores on a scale)BAF312 2mg/BAF312 2mgBAF312 10 mg/BAF312 10 mgPlacebo/BAF312 2 mgPlacebo/BAF312 10 mgTotal
Mean202.6 ± 41.74184.0 ± 19.80189.5 ± 45.65166.0 ± 0193.6 ± 37.90
Taking DMARD at baseline
Taking DMARD at baseline(participants)BAF312 2mg/BAF312 2mgBAF312 10 mg/BAF312 10 mgPlacebo/BAF312 2 mgPlacebo/BAF312 10 mgTotal
Number724114
08

Study locations

8 sites
  • Novartis Investigative Site
    Phoenix, Arizona 85013, United States
  • Novartis Investigative Site
    Torono, Ontario M5G 2C4, Canada
  • Novartis Investigative Site
    Prague 2, 128 50, Czechia
  • Novartis Investigative Site
    Budapest, 1083, Hungary
  • Novartis Investigative Site
    Debrecen, 4032, Hungary
  • Novartis Investigative Site
    Bydgoszcz, 85-168, Poland
  • Novartis Investigative Site
    Taichung, 40447, Taiwan
  • Novartis Investigative Site
    Taichung, 40705, Taiwan
09

References and documents

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jan 5, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT01801917
Lead sponsor
Novartis Pharmaceuticals
Responsible party
Sponsor
First posted
Mar 1, 2013
Start date
Apr 24, 2013
Primary completion
Aug 5, 2016
Completion
Aug 5, 2016
Results posted
Jan 4, 2018
Last update
Jan 5, 2021

Study contacts

Novartis Pharmaceuticals
study director · Novartis Pharmaceuticals

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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