Efficacy and Tolerability of BAF312 in Patients With Polymyositis

NCT01801917 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: CBAF312X2205
• Study Type: interventional
• Target: 14
• Locations: 6 countries
• Sites: 8

Brief 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).

Conditions

Polymyositis

Keywords

Polymyositis; Myositis; PM; chronic muscle inflammation; inflammatory myopathy; inclusion body myositis

Outcome Measures

Primary Outcomes (2)

• 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.

  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

  Baseline, at 12 weeks

Secondary Outcomes (3)

• Six-minute Walking Distance (6MWD) at Week 12

  Baseline, 12 weeks

• Six-minute Walking Distance (6MWD) at Week 24

  Baseline, 24 weeks

• BAF312 Trough Plasma Concentrations (PK Set)

  -7 Baseline, day 28, 56, 84

Study Arms (3)

Placebo

PLACEBO COMPARATOR

5 placebo tablets daily during non-titration phase

Drug: Placebo

BAF312 2mg

EXPERIMENTAL

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

Drug: BAF312

BAF312 10 mg

EXPERIMENTAL

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

Drug: BAF312

Interventions

Placebo DRUG

Matching placebo tablet for oral administration

Placebo

BAF312 DRUG

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

BAF312 10 mg; BAF312 2mg

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• "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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (8)

Novartis Investigative Site

Phoenix, Arizona, 85013, United States

Location

Novartis Investigative Site

Torono, Ontario, M5G 2C4, Canada

Location

Novartis Investigative Site

Prague, 128 50, Czechia

Location

Novartis Investigative Site

Budapest, 1083, Hungary

Location

Novartis Investigative Site

Debrecen, 4032, Hungary

Location

Novartis Investigative Site

Bydgoszcz, 85-168, Poland

Location

Novartis Investigative Site

Taichung, 40447, Taiwan

Location

Novartis Investigative Site

Taichung, 40705, Taiwan

Location

Related Links

• A Plain Language Trial Summary is available on novartisclinicaltrials.com

MeSH Terms

Conditions

Polymyositis; Myositis; Myositis, Inclusion Body

Interventions

siponimod

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@novartis.com

862-778-8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 1, 2013
• First Posted: March 1, 2013
• Study Start: April 24, 2013
• Primary Completion: August 5, 2016
• Study Completion: August 5, 2016
• Last Updated: January 5, 2021
• Results First Posted: January 4, 2018

Record last verified: 2018-01

Locations

CZ (1); US (1); HU (2); TW (2); PL (1); CA (1)