A Prospective, Observational Study Evaluating Persistence on Treatment, Safety, Tolerability, and Effectiveness of Diroximel Fumarate in the Real-World Setting (EXPERIENCE-US Study)
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- Biogen
- 入组人数
- 75
- 试验地点
- 18
- 主要终点
- Percentage of Participants on Treatment with DRF at 1 Year
研究概览
简要总结
The primary objective of the study is to characterize the persistence to therapy in participants with relapsing forms of multiple sclerosis (RMS) treated with diroximel fumarate (DRF) in routine clinical practice. The secondary objectives of the study are to assess short-term persistence to treatment; to assess long-term persistence on treatment; to assess the effect of DRF on relapses; to assess the impact of DRF on cognition; to assess the impact of DRF on participant reported outcomes (PROs) and to explore the real-world safety profile of DRF (ie, gastrointestinal [GI] tolerability, lymphocyte dynamics, adverse events [AEs] leading to discontinuation, and serious adverse events [SAEs]).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Have a diagnosis of MS and satisfy the approved therapeutic indication for DRF per the USPI.
- •DRF prescribed and planned to be initiated within 60 days after enrollment.
排除标准
- •History of gastric bypass or required use of feeding tubes.
- •Have received prior treatment with DRF.
- •Note: Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Diroximel Fumarate
Participants with RMS who are receiving diroximel fumarate orally in routine clinical practice will be enrolled.
干预措施: Diroximel Fumarate (Drug)
结局指标
主要结局
Percentage of Participants on Treatment with DRF at 1 Year
时间窗: 1 year
次要结局
- Percentage of Participants on Treatment with DRF at 3 Months(3 months)
- Change in Processing Speed Test (PST) Score from Baseline(Baseline up to 2 years)
- Change in Disability, as Measured by Patient Determined Disease Steps (PDDS)(Baseline up to 2 years)
- Percentage of Participants on Treatment with DRF at 2 Years(2 years)
- Change in Score for Each Domain of Quality of Life in Neurological Disorders (Neuro- QoL™) Questionnaire(Baseline up to 2 years)
- Annualized Relapse Rate (ARR) with DRF(At 1 and 2 years)
- Percentage of Participants Relapsed(At 1 and 2 years)
- Number of Participants categorized by the types of actions taken to mitigate GI AEs(Up to 32 months)
- Change in Work Productivity and Activity Impairment Due to Multiple Sclerosis (WPAI- MS) Scores from Baseline(Baseline up to 2 years)
- Number of Participants with Serious Adverse Events (SAEs)(Up to 32 months)
- Percent Change in Median ALC from Baseline(Baseline up to 2 years)
- Number of Participants with Lymphopenia According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI/CTCAE)Severity Grading(Up to 32 months)
- Number of Participants with Gastrointestinal (GI) Adverse Events (AEs)(Up to 32 months)
- Number of Participants with AEs Leading to Treatment Discontinuation(Up to 32 months)
- Median Absolute Lymphocyte Count (ALC) Over Time(Baseline up to 2 years)