Clinical trial NCT01148810: Efficacy and Tolerability of BAF312 in Patients With Polymyositis and Dermatomyositis
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 and Dermatomyositis
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Polymyositis
Dermatomyositis
Keywords: Inflammatory muscle disease, Muscle weakness, Myositis, inflammatory myopathy, inclusion body myositis, skin rash, autoimmune, difficulty swallowing, dysphagia
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2010-06-22
Enrollment: 18
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

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

Primary outcomes:
measure: Number of Participants Who Responded to BAF312
timeFrame: 12 weeks
Secondary outcomes:
measure: Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2
timeFrame: 12 weeks
measure: Mean Plasma Concentrations of BAF312
timeFrame: baseline to end of trial (day 196)
measure: Summary of CRP Levels
timeFrame: 12 weeks
measure: Efficacy in Modifying Health-related Quality of Life Measured by SF-36
timeFrame: 12 weeks
measure: Myositis Disease (MD) Activity Scores
timeFrame: Week 12
measure: Physician Global Activity Assessment
timeFrame: Baseline, Week 12
measure: Patient Global Activity Assessment
timeFrame: Baseline, Week 12
measure: Manual Muscle Testing (MMT) - 8 Score
timeFrame: Baseline, Week 12
measure: Health Assessment Questionnaire
timeFrame: Baseline, Week 12
measure: Serum Levels of Muscle Enzymes
timeFrame: Baseline, Week 12
Other outcomes:
Locations:
city: Boston
country: United States
facility: Novartis Investigative Site
state: Massachusetts
city: Prague 2
country: Czechia
facility: Novartis Investigative Site
city: Budapest
country: Hungary
facility: Novartis Investigative Site
city: Debrecen
country: Hungary
facility: Novartis Investigative Site
city: Stockholm
country: Sweden
facility: Novartis Investigative Site
city: Manchester
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: