Efficacy and Tolerability of BAF312 in Patients With Polymyositis and Dermatomyositis

NCT01148810 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: CBAF312A22022008-006311-21
• Study Type: interventional
• Target: 18
• Locations: 5 countries
• Sites: 6

Brief 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. 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

Keywords

Inflammatory muscle disease, Muscle weakness, Myositis, inflammatory myopathy, inclusion body myositis, skin rash, autoimmune, difficulty swallowing, dysphagia

Outcome Measures

Primary Outcomes (1)

• 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).

  12 weeks

Secondary Outcomes (10)

• Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2

  12 weeks

• Mean Plasma Concentrations of BAF312

  baseline to end of trial (day 196)

• Summary of CRP Levels

  12 weeks

• Efficacy in Modifying Health-related Quality of Life Measured by SF-36

  12 weeks

• Myositis Disease (MD) Activity Scores

  Week 12

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Drug: Placebo

BAF312

EXPERIMENTAL

Drug: BAF312

Interventions

BAF312 DRUG

BAF312

Placebo DRUG

Placebo

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• Other idiopathic inflammatory myopathies
• Myopathy other than polymyositis and dermatomyositis
• Patients with late stages of disease

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (6)

Novartis Investigative Site

Boston, Massachusetts, 02115, United States

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

Stockholm, 17176, Sweden

Location

Novartis Investigative Site

Manchester, M6 8HD, United Kingdom

Location

MeSH Terms

Conditions

Polymyositis; Dermatomyositis; Myositis; Muscle Weakness; Myositis, Inclusion Body; Exanthema; Deglutition Disorders

Interventions

siponimod

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Neuromuscular Manifestations; Neurologic Manifestations; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Esophageal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Pharyngeal Diseases; Otorhinolaryngologic Diseases

Limitations and Caveats

The study was terminated due to new clinical data from overall BAF312 program (\<10 mg/day already have optimal pharmacodynamic effect \& clinical efficacy). There was no safety concern testing 10 mg BAF312 that would have required study termination.

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: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 21, 2010
• First Posted: June 22, 2010
• Study Start: June 15, 2010
• Primary Completion: June 13, 2012
• Study Completion: June 13, 2012
• Last Updated: April 25, 2019
• Results First Posted: April 25, 2019

Record last verified: 2019-04

Data Sharing

• IPD Sharing: Will share

Locations

HU (2); US (1); CZ (1); SE (1); GB (1)