A 24-month, Open-label, Prospective, Multicenter Interventional, Single-arm Study Assessing the Efficacy and Safety of Fingolimod (Gilenya) 0.5 mg in Relapsing Multiple Sclerosis (RMS) Patients in China
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 98
- 试验地点
- 14
- 主要终点
- Adjusted Annualized Relapse Rate (ARR) in Adult Group
研究概览
简要总结
The main purpose of this study was to assess the efficacy and safety of 0.5mg Fingolimod (Gilenya) in Chinese patients with relapsing multiple sclerosis (RMS)
详细描述
This was a 24-month, open-label, multicenter, interventional, single-arm study to collect efficacy and, safety of oral fingolimod 0.5 mg/day in approximately 100 relapsing multiple sclerosis (RMS) participants in China.
The study consisted of three Phases:
- Screening (up to 1 month): After signing informed consent, participants entered a Screening Phase to determine eligibility according to inclusion and exclusion criteria.
- Treatment Period (24 months): On visit Day 1, all eligibility criteria were confirmed, including a pre-dose ECG and vital signs. The first dose of study drug was taken in the clinic on Day 1 and the participant was monitored for 6 hours after the first dose administration before discharge. Participants returned to site for evaluation at month 1 and then every three months until the end of treatment up to 24 months.
- Follow Up (2 months): Subjects who completed Treatment Period or discontinued from treatment returned for the Follow-up visit 2 months after the last dose of study drug.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 10 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant 10 to 17 years old inclusive with weight > 40kg.
- •Participant 18 to 65 years old inclusive;
- •Participants with relapsing multiple sclerosis
- •Participants never used fingolimod before enrollment
- •Subjects with Expanded Disability Status Scale (EDSS) score of 0 - 6.0 (inclusive) at Screening
排除标准
- •Participants with certain cardiovascular conditions and/or findings in the screening ECG.
- •Diagnosis of macular edema during screening visit.
- •Increased risk for opportunistic infections
- •Participants with known active malignancies.
- •Participants who have been treated with teriflunomide within 3.5 months prior to baseline, except if active washout.
- •Participants with severe active infections, active chronic infection.
- •Participants with severe liver impairment.
- •Pregnant confirmed by a positive pregnancy test or nursing (lactating) women.
研究组 & 干预措施
Fingolimod
Fingolimod 0.5 mg capsule taken orally once daily
干预措施: Fingolimod 0.5mg (Drug)
结局指标
主要结局
Adjusted Annualized Relapse Rate (ARR) in Adult Group
时间窗: Baseline to Month 24
A confirmed relapse is any relapse that is accompanied by an increase of at least 0.5 on the EDSS or an increase of 1 point on two different Functional Systems (FS) of the EDSS or 2 points on one of the FS (excluding Bowel/Bladder or Cerebral FS) as confirmed by the treating physician. The adjusted annualized relapse rate (ARR) was estimated by a negative binomial regression model with log-link function, the cumulative number of confirmed MS relapses per subject as the response variable, number of relapses in the previous two years before enrollment and baseline EDSS as continuous covariates. Natural log of time on study in years was used as the offset variable to account for the varying lengths of subjects' time in the study. The adjusted ARR (i.e.model-based estimate adjusted for covariates) and the corresponding 95% confidence interval were obtained. As per SAP this analysis was only performed for the Adult group. Descriptive data is presented in subsequent OMs.
次要结局
- Number of Participants With Treatment Emergent Adverse Events (AE) and Serious Adverse Events (SAE)(From first dose of study treatment to 45 days after last study dose up to aproximately 25.5 months)
- Annualized Rate of the Number of New or Newly Enlarged T2 Lesions(Baseline to end of treatment (up to Month 24))
- Change From Baseline in Number of New or Newly Enlarged T2 Lesions(Baseline up to Month 24)
- Change From Baseline in T2 Lesion Volume(Baseline up to Month 24)
- Number of Gd-enhancing T1 Lesions Per Scan in Adult Group(Baseline up to Month 24)
- Number of Gd-enhancing T1 Lesions(Baseline up to Month 24)
- Change From Baseline in Gd-enhancing T1 Lesion Volume(Baseline up to Month 24)
- Number of T1 Hypo-intense Lesions(Baseline up to Month 24)
- Change From Baseline in T1 Hypo-intense Lesion Volume(Baseline up to Month 24)