Efficacy and Tolerability of BAF312 in Patients With Polymyositis

Novartis

Status and phase: Terminated; Phase 2

Conditions: Polymyositis

Treatments: Drug: Placebo; Drug: BAF312

Study type: Interventional

Funder types: Industry

Identifiers

NCT01801917

CBAF312X2205

Details and patient eligibility

About

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).

Full 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

Enrollment: 14 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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14 participants in 3 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

5 placebo tablets daily during non-titration phase

Treatment:

Drug: Placebo

BAF312 2mg

Experimental group

Description:

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

Treatment:

Drug: BAF312

BAF312 10 mg

Experimental group

Description:

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

Treatment:

Drug: BAF312