Bilateral Essential Tremor Treatment With Gamma Knife
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 3
- 主要终点
- Change in QUEST Score
研究概览
简要总结
Combined Phase II/III, multi-center, prospective, single-blinded trial.
Ten (10) patients with essential tremor who previously underwent successful and uncomplicated GK thalamotomy for essential tremor will undergo a contralateral treatment. The incidence of side effects will be determined at 3 months postoperatively, graded per the CTCAE v5 and analyzed by a data safety monitoring board. Upon successful review, this Phase II trial will be converted to a Phase III trial of utility that will enrol 40 additional patients. The primary outcome will be the change in QUEST score at 12 months postoperatively, as well as a patient-reported assessment of Health Utility. Secondary outcomes will include objective tremor, gait and speech assessments (filmed and scored by blinded evaluators), as well as quality of life questionnaires and adverse events questionnaires. Outcomes will be assessed at baseline, as well as 3, 6, 12, 24 and 36 months post-operatively.
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
盲法说明
All assessments filmed on video and scored by a blinded evaluator.
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
- •Patients must be ≥ 18 years
- •The patient has a primary diagnosis of ET or ET plus confirmed by a movement disorder neurologist;
- •The patient previously underwent a GK thalamotomy (> 12 months ago);
- •The tremor on the untreated side negatively impacts the patients' quality of life;
- •The patient wants treatment of the contralateral side.
排除标准
- •Clinically relevant gait and/or balance impairment (e.g. wide-based, more than 2 falls/months, wheelchair, walker, cane use);
- •Clinically relevant speech impairment (e.g. impairment of intelligibility);
- •Inability to comply with the follow-up schedule;
- •Refusal of the treating physician.
结局指标
主要结局
Change in QUEST Score
时间窗: 12 months
Difference in QUEST score between baseline and the 12-month follow-up, with statistical significance defined as p = 0.05 and a minimum clinically important difference (MCID) defined as 5
Patient-based Assessment of Utility
时间窗: 12 months
A patient-reported assessment of utility after 12 months using the question "Knowing what you know now, would you do this treatment again?".
次要结局
- Gait Assessment (missteps)(Baseline, post operative month 3, 6, 12, 24, 36)
- Falls(Baseline, post operative month 3, 6, 12, 24, 36)
- Dysgeusia(Baseline, post operative month 3, 6, 12, 24, 36)
- Tremor (CRST)(Baseline, post operative month 3, 6, 12, 24, 36)
- Quality of life (CRST part C)(Baseline, post operative month 3, 6, 12, 24, 36)
- Gait Assessment (NRS-11)(Baseline, post operative month 3, 6, 12, 24, 36)
- Speech Assessment (NRS-11)(Baseline, post operative month 3, 6, 12, 24, 36)
- Numbness(Baseline, post operative month 3, 6, 12, 24, 36)
- Other adverse events(Baseline, post operative month 3, 6, 12, 24, 36)
- Quality of life (QUEST)(Baseline, post operative month 3, 6, 12, 24, 36)
- Health Utility (EQ-5D-5L)(Baseline, post operative month 3, 6, 12, 24, 36)
研究者
Christian Iorio-Morin
Assistant professor of neurosurgery
Université de Sherbrooke