Active dermatomyositis MedDRA version: 18.0 Level: PT Classification code 10012503 Term: Dermatomyositis System Organ Class: 10040785 - Skin and subcutaneous tissue disorders
Conditions
Interventions
Product Code: BAF312 0.25 mg
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Siponimod
CAS Number: 1234627-85-0
Current Sponsor code: BAF312
Other descriptive name: BAF312 hemifumarate
Co
Sponsors
Novartis Pharma Services AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Written informed consent must be obtained before any assessment is performed. • Patients who have been defined as "definite" or "probable" based on the criteria of Bohan and Peter (Bohan and Peter 1975) for dermatomyositis at least 3 months before screening • Patients must have active disease as defined by muscle weakness • Patients may be on a stable dose of corticosteroid (up/equal to 20 mg once daily prednisone equivalent) • Patients currently treated with oral or subcutaneous MTX must have been a stable dose of no more/equal to than 25 mg per week • Patients currently treated with Azathioprine must have been a stable maintenance dose of no more/equal to 3 mg/kg/day • Negative cancer screening conducted in the 12 months prior to screening visit 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 50 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 6
Exclusion criteria
Exclusion criteria: • Dermatomyositis patients having overlap myositis or any other type of myositis including paraneoplastic myositis, drug-induced myopathy, necrotizing myositis • Preexisting severe cardiac or pulmonary conditions, 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 apply.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary aim of this study is to assess the efficacy of different doses of BAF312 on the MMT-24 after 6 months. The overall efficacy of BAF312 will be assessed by comparing the improvements of MMT-24 with every dose of BAF312 to that of placebo. Then the dose response curve of MMT-24 will be estimated with the aim to determine a target dose for the program.;Secondary Objective: To assess the effects of different doses of BAF312 on safety, pharmacokinetics and peripheral blood lymphocyte counts in active DM patients To assess the efficacy of different doses of BAF312 after 3 months of treatment in active DM patients as assessed by manual muscle testing using the MMT-8 scoring system;Primary end point(s): Manual Muscle Testing - 24 muscles (MMT-24). Efficacy of BAF312 will be assessed by comparing the improvements with every dose of BAF312 to that of placebo;Timepoint(s) of evaluation of this end point: 6 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: For Manual Muscle Testing : 3 months For all others: 6 months;Secondary end point(s): • Adverse Events. All information obtained on adverse events will be displayed by treatment (dose group) and subject. Secondary variables include the incidence of adverse events. • Pharmacokinetics. BAF312 plasma concentration data will be listed by treatment (dose group), subject and visit/sampling time point. Descriptive summary statistics will be provided by treatment and visit/sampling time point. Secondary variables include plasma BAF312 concentrations. • Peripheral blood lymphocyte counts. Absolute lymphocyte counts will be plotted against time by dose level. Secondary variables include peripheral blood lymphocyte counts. • Manual Muscle Testing - 24 muscles (MMT-24). Efficacy of BAF312 will be assessed by comparing the improvements with every dose of BAF312 to that of placebo. Changes from baseline in MMT-24 at 3 months will also be evaluated. The dose-response will be assessed in the same way as for the 6-month data. • 6 Minutes Walking Distance test. Efficacy of BAF312 will be assessed by comparing the changes in walking distance across all doses of BAF312 to that of placebo Changes from baseline in 6 Minute walking distance at 6 months of treatment will be assessed. | — |
Countries
Belgium, Canada, China, Czech Republic, Germany, Hungary, Japan, Netherlands, Poland, Switzerland, Taiwan, United States
Contacts
Public ContactPublic Information Desk
Novartis Hungary Kft. Pharma
Outcome results
None listed