A Single-Center, Randomized, Double-Blind, Parallel-Group, Dose-Controlled Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Patients With Machado-Joseph Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Safety laboratory tests
研究概览
简要总结
- This is an exploratory, randomized, parallel-group, dose escalation and dose-controlled study without a placebo arm.
- Eligible patients will be randomized in a 1:1 ratio (double-blind) to receive Cabaletta in 2 doses, once weekly for 22 weeks (total of 24 weeks of treatment).
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
- Triple (Participant, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Men and women, 18 - 75 years
- •Clinically diagnosed as Machado-Joseph disease/Spinocerebellar ataxia 3 confirmed by genetic testing
- •With disease stage 2 or less
- •Stable doses of all medications for 30 days prior to study entry and for the duration of the study.
- •Body Mass Index (BMI) ≤32 kg/m
- •Ability to ambulate with or without assistance
排除标准
- •Diabetes mellitus type 1 or 2
- •Other major diseases
- •Uncontrolled heart disease, chronic heart failure (CHF).
- •Other neurological diseases.
- •Ataxia derived from any other cause than genetically-confirmed spinocerebellar ataxia
- •Presence of psychosis, bipolar disorder, untreated depression
- •History of malignancy (except non-invasive skin malignancy).
研究组 & 干预措施
Cabaletta 15gr
Cabaletta 15gr
干预措施: Cabaletta for IV infusion once weekly during 24 weeks (Drug)
Cabaletta 30gr
Cabaletta 30gr
干预措施: Cabaletta for IV infusion once weekly during 24 weeks (Drug)
结局指标
主要结局
Safety laboratory tests
时间窗: 28weeks
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
12-lead ECG
时间窗: 28weeks
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Vital signs
时间窗: 28weeks
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Physical examination
时间窗: 28 weeks
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Adverse events
时间窗: 28 weeks
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
次要结局
- Disease markers(27 weeks)