A 96-Week, Prospective, Multicenter, Randomised, Double-Blind, Placebo-Controlled, Phase 3 Study to Compare Efficacy and Safety of Masitinib Dose Titration to 4.5 mg/kg/Day Versus Placebo in the Treatment of Patients With Primary Progressive or Secondary Progressive Multiple Sclerosis Without Relapse
试验速览
- 阶段
- 3 期
- 状态
- 暂停
- 发起方
- 入组人数
- 800
- 试验地点
- 37
- 主要终点
- Time to confirmed progression
研究概览
简要总结
To evaluate the efficacy and safety of oral masitinib versus placebo in the treatment of patients with primary progressive or secondary progressive multiple sclerosis without relapse.
详细描述
Masitinib is a selective tyrosine kinase inhibitor, targeting innate immune cells (mast cells and microglia) that are involved in the pathophysiology of progressive multiple sclerosis (MS). This is a multicenter, double-blind, randomized, placebo-controlled, comparative study of oral masitinib in the treatment of patients with progressive MS who were progressing but not clinically active.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Main inclusion criteria include:
- •Patients with either primary progressive or secondary progressive multiple sclerosis with onset of symptoms at least five years before baseline and with no relapse diagnosed according to the 2017 revised McDonald's criteria at least two years before screening
- •Patients with Expanded Disability Status Scale (EDSS) score between 3.0 to 6.0 (both inclusive) at screening and baseline
- •Patients with an EDSS score progression ≥1 point with no improvement during 2 years
- •Absence of T1 Gadolinium-enhancing brain lesions as measured by MRI at screening
排除标准
- •Patients suffering from a disease other than MS that would better explain the patient's neurological clinical signs and symptoms and/or MRI lesions observed at screening
- •Inability to complete screening MRI (contraindications for MRI) and/or any known allergy or hypersensitivity or any contra-indication to gadolinium macrocyclic
- •Patients treated with other disease modifying treatments in the time frames and conditions mentioned under previous treatment wash out period, assessed at baseline
- •Patients with lymphocytes <1.0 × 10^9/L at screening and at baseline
研究组 & 干预措施
Masitinib (4.5)
Participants receive masitinib (3.0 mg/kg/day), given orally twice daily, with a dose escalation to 4.5 mg/kg/day after 4 weeks of treatment. Each ascending dose titration is subjected to a safety control.
干预措施: Masitinib (4.5) (Drug)
Placebo
Participants receive a matched dose placebo, given orally twice daily.
干预措施: Placebo (Drug)
结局指标
主要结局
Time to confirmed progression
时间窗: 96 weeks
Time to disability progression, confirmed by two consecutive visits, wherein progression of disability is measured by the Expanded Disability Status Scale (EDSS) with progression defined as a 1-point worsening for baseline EDSS score ≤5.5, or 0.5-point worsening for baseline EDSS score \>5.5. The Expanded Disability Status Scale (EDSS) is a method of quantifying disability in multiple sclerosis and monitoring changes in the level of disability over time. The EDSS provides a total score on a scale that ranges from 0 to 10, in 0.5-point increments, with higher scores indicating greater disability. The first levels 1.0 to 4.5 refer to people with a high degree of ambulatory ability and the subsequent levels 5.0 to 9.5 refer to the loss of ambulatory ability.
次要结局
- Time to Expanded Disability Status Scale (EDSS) score of 7.0(96 weeks)
- Multiple Sclerosis Quality of Life (MSQOL)-54(96 weeks)
- Overall Change in Expanded Disability Status Scale (EDSS) Score(96 weeks)
- Brain Magnetic Resonance Imaging Assessments(96 weeks)