Active Dermatomyositis Clinical Trial
Official title:
A Double Blind, Randomized, Placebo-controlled Study to Evaluate, Safety, Tolerability, Efficacy and Preliminary Dose-response of BAF312 in Patients With Active Dermatomyositis (DM)
| Verified date | December 2018 |
| Source | Novartis |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
This study investigated the dose response relationship for the efficacy and safety of BAF312 compared to placebo in active DM patients over a treatment period of 6+6 months and to determine the minimum dose required for a maximal clinical effect. The study was composed of 2 periods: a double-blind period 1 with BAF312 administered at different daily doses (0.5, 2, 10 mg and placebo) and a fixed-dose Period 2 in which BAF312 was administered at the dose of 2 mg daily .
| Status | Terminated |
| Enrollment | 17 |
| Est. completion date | February 17, 2016 |
| Est. primary completion date | February 17, 2016 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years to 75 Years |
| Eligibility | Key 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 Key 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 |
| Country | Name | City | State |
|---|---|---|---|
| Czechia | Novartis Investigative Site | Prague 2 | Czech Republic |
| Japan | Novartis Investigative Site | Chiba-city | Chiba |
| Japan | Novartis Investigative Site | Sendai city | Miyagi |
| United States | Novartis Investigative Site | Boston | Massachusetts |
| United States | Novartis Investigative Site | Kansas City | Kansas |
| United States | Novartis Investigative Site | Los Angeles | California |
| United States | Novartis Investigative Site | Miami | Florida |
| United States | Novartis Investigative Site | Orange | California |
| United States | Novartis Investigative Site | Phoenix | Arizona |
| Lead Sponsor | Collaborator |
|---|---|
| Novartis Pharmaceuticals |
United States, Czechia, Japan,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score | Each muscles tested was evaluated on a 0 - 10 scale where 0 indicated the weakest muscle score and 10 indicated the strongest muscle score. The total MMT24 score ranged from 0 - 240, where an increasing trend in the values indicates improvement. A positive change from baseline indicates improvement. | Baseline, 6 months | |
| Secondary | BAF312 Plasma Concentration | 6 months | ||
| Secondary | Peripheral Blood Lymphocyte Counts | Absolute lymphocyte counts | baseline, 6 months | |
| Secondary | Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score | Each muscles tested was evaluated on a 0-10 scale where 0 indicated the weakest muscle score and 10 indicated the strongest muscle score. the total MMT24 score ranged from 0-240, where an increasing trend in the values indicates improvement. A positive change from baseline indicates improvement. | baseline, 3 months | |
| Secondary | Change From Baseline in 6 Minutes Walking Distance (6-MWD) Test | baseline, 6 months |