Study of efficacy and tolerability for BAF312 compared to placebo in patients with polymyositis

Phase 1

Conditions: Polymyositis
MedDRA version: 18.0Level: PTClassification code 10036102Term: PolymyositisSystem Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Registration Number: EUCTR2012-002859-42-BE

Lead Sponsor: ovartis Pharma Services AG

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 45

Inclusion Criteria

-definite or probable for polymyositis at least three months before
Baseline
- active active myositis 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 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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 37
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 8

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
Other protocol-defined inclusion/exclusion criteria may apply.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Secondary Objective: - To assess the safety and tolerability of BAF312 in patients with PM<br>- To characterize the steady state pharmacokinetics of BAF312 in<br>patients with PM<br>- To assess the effect of BAF312 on muscle function-dependent physical<br>performance using the 6 minutes walking distance test (6MWD);Primary end point(s): Manual Muscle Testing (MMT) and serum creatine kinase (CK) levels --<br>Assessment of preliminary clinical efficacy of 2mg and 10mg BAF312<br>once daily using<br>MMT of 24 muscles (MMT-24) and serum CK levels or other enzymes, or<br>MRI/biopsy if enzymes are normal.;Timepoint(s) of evaluation of this end point: Baseline, over 12 weeks;Main Objective: This study will assess the efficacy, safety and tolerability of BAF312<br>administered orally in patients with clinically active polymyositis.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Adverse Events: Number of adverse events will be tabulated by body<br>systems and treatment.<br>- Steady state pharmacokinetics of BAF312<br>- Effect of BAF312 on muscle function-dependent physical performance<br>using the 6 minutes walking distance test (6MWD).;Timepoint(s) of evaluation of this end point: 24 weeks for AEs and 12 weeks for 6MWD