Clinical trial NCT01801917: Efficacy and Tolerability of BAF312 in Patients With Polymyositis
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Polymyositis
Keywords: Polymyositis
Myositis
PM
chronic muscle inflammation
inflammatory myopathy
inclusion body myositis
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2013-03-01
Enrollment: 14
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 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).

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

Eligibility criteria: 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

Primary outcomes:
measure: 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)
timeFrame: Baseline, at 12 weeks
measure: Percent Change From Baseline at Week 12 for BAF312 2 mg, 10 mg or Placebo (Once Daily) Serum Creatine Kinase (CK) Levels
timeFrame: Baseline, at 12 weeks
Secondary outcomes:
measure: Six-minute Walking Distance (6MWD) at Week 12
timeFrame: Baseline, 12 weeks
measure: Six-minute Walking Distance (6MWD) at Week 24
timeFrame: Baseline, 24 weeks
measure: BAF312 Trough Plasma Concentrations (PK Set)
timeFrame: -7 Baseline, day 28, 56, 84
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Novartis Investigative Site
state: Arizona
city: Torono
country: Canada
facility: Novartis Investigative Site
state: Ontario
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: Bydgoszcz
country: Poland
facility: Novartis Investigative Site
city: Taichung
country: Taiwan
facility: Novartis Investigative Site
city: Taichung
country: Taiwan
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: