Phase I Study of Oral Guanabenz for Multiple Sclerosis
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 2
- 试验地点
- 1
- 主要终点
- Maximum tolerated dose (MTD)
研究概览
简要总结
Background:
- People with multiple sclerosis (MS) get lesions in their brain and spinal cord. These cause neurological symptoms and sometimes disability. Researchers want to see if a blood pressure drug called guanabenz can repair lesions and help people with MS.
Objective:
- To see if guanabenz is safe and well tolerated in people with MS.
Eligibility:
- People 18 55 years old with MS who have taken glatiramer acetate for the past year.
Design:
- Participants will be screened in a separate protocol. For 2 months, they will be examined and have magnetic resonance imaging (MRI) scans. This will decide if they are in the Stable or Active MS study group.
- The study will last 5 months. There will be up to 11 visits, 5 overnight.
- Visit 1: overnight stay at the clinic:
- Medical history and physical exam.
- Health questionnaire
- Bladder ultrasound scan
- Brain MRI
- Electrocardiogram (EKG) to measure heart electrical activity
- Blood will be drawn through an intravenous (IV) line.
- Participants may have tests of strength, muscle tone, and movement.
- They will get their first dose of the study drug, a tablet taken once a day.
- Participants will take the study drug at home and keep a medicine diary.
- The dose will slowly increase. Each time, participants will stay overnight at the clinic. They will have a physical exam, EKG, MRI, and IV blood draw.
- Visit 6: Participants will have a physical exam, MRI, and blood drawn. They will get a schedule to slowly lower their drug dose and stop taking guanabenz.
- Participants will have 2 final visits. They will have a physical exam, EKG, MRI, and IV blood draw.
详细描述
OBJECTIVE:
This Phase 1 clinical study will aim to determine whether therapeutically adequate dosages of guanabenz are safe and well tolerated in patients with multiple sclerosis (MS). It will further provide pharmacokinetic data important for determination of optimal dosing schedule for possible future Phase 2 study.
STUDY POPULATION:
Six patients, ages between 18-55 inclusive, and diagnosis of definite multiple sclerosis by 2010 Revised McDonald Diagnostic Criteria (Polman et al, 2010) will be enrolled. All patients will have been on treatment with glatiramer acetate, a Food and Drug Administration (FDA) approved disease-modifying therapy, for a minimum of year. Four of the patients will be clinically stable with no clinical relapse in the preceding year and no evidence of active inflammation by MRI during the 2-month screening period; 2 patients will be selected based on evidence of on-going, active inflammation seen by MRI during the screening period.
DESIGN:
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 55 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Treatment arm
干预措施: Guanabenz (Drug)
结局指标
主要结局
Maximum tolerated dose (MTD)
时间窗: 3.5 months
次要结局
- Pharmacokinetics of guanabenz in MS patients(3.5 months)