A Double Blind, Randomized, Placebo-controlled Study to Evaluate, Safety, Tolerability, Efficacy and Preliminary Dose-response of BAF312 in Patients With Active Dermatomyositis (DM)
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 17
- 试验地点
- 1
- 主要终点
- Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score
研究概览
简要总结
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 .
详细描述
The study was prematurely terminated based on the results of an interim analysis where BAF312 did not demonstrate superior efficacy over placebo and a dose-response relationship was not observed. There were no safety concerns. Approximately 56 participants were planned to be randomized. A total of 17 participants were enrolled and randomized by the time the study was terminated.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- 未提供
研究组 & 干预措施
BAF312 0.5mg
During period 1, participants were uptitrated daily from BAF312 0.25 mg to 0.5 mg over a 10 day period. After, participants continued on 0.5 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.
干预措施: BAF312 (Drug)
BAF312 2mg
During period 1, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.
干预措施: BAF312 (Drug)
BAF312 10 mg
During period 1, participants were uptitrated daily from BAF312 0.25 mg to 10.0 mg over a 10 day period. After, participants continued on 10.0 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.
干预措施: BAF312 (Drug)
Placebo
During period 1, participants received matching placebo daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score
时间窗: Baseline, 6 months
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.
次要结局
- Peripheral Blood Lymphocyte Counts(baseline, 6 months)
- Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score(baseline, 3 months)
- Change From Baseline in 6 Minutes Walking Distance (6-MWD) Test(baseline, 6 months)
- BAF312 Plasma Concentration(6 months)