A Pivotal, Multicenter, Open-label, Randomized Withdrawal, Non-Treatment Concurrent Control Study to Assess the Safety, Tolerability, and Efficacy of Cabaletta® in OPMD Patients Who Participated in Study BBCO-001
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- Change in swallowing quality of life
研究概览
简要总结
This will be a multi-center, randomized withdrawal, open-label, non-treatment concurrent control, parallel group study. Patients completing protocol BBCO-001 will be offered the opportunity to enter into this 12-month randomized withdrawal protocol.
详细描述
This will be a multi-center, randomized withdrawal, open-label, non-treatment concurrent control, parallel group study. Patients completing protocol BBCO-001 will be offered the opportunity to enter into this 12-month randomized withdrawal protocol.
Eligible patients will be randomized to one of the following treatment arms:
- Treatment Arm 1: active treatment; continuation of 30 g IV Cabaletta once a week over an additional 52 weeks.
- Treatment Arm 2: no-treatment concurrent control; discontinuation of IV Cabaletta and follow-up over 52 weeks.
IV Cabaletta will be administered once a week to patients in Treatment Arm 1. All patients, regardless of treatment arm allocation, will undergo the same safety and efficacy assessments during the monthly site visits.
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
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult men and women who participated and completed study BBCO-001
- •Clinical and genetic diagnosis of OPMD
- •Able to provide written informed consent to participate in this study
- •Able to understand the requirements of the study and willing to comply with the requirements of the study
排除标准
- •Pregnant or lactating
- •Currently receiving anticoagulant treatment (e.g., warfarin)
- •Any life-threatening illness, medical condition or organ system dysfunction which, in the investigator's opinion, could compromise the subject's safety
- •Known hypersensitivity to any ingredient in the Cabaletta IV infusion
- •Currently participating in another clinical trial (other than BBCO-001) or have completed an interventional trial less than 30 days prior to the planned treatment start date
研究组 & 干预措施
Arm 1
Trehalose 30 g for IV infusion administered every week over an additional 52 weeks
干预措施: Tehalose 30gr (Drug)
结局指标
主要结局
Change in swallowing quality of life
时间窗: 52 weeks
long term effect of Cabaletta on disease progression as measured by the changes in the patient's swallowing quality of life
Change in disease markers
时间窗: 52 weeks
long term effect of Cabaletta on disease progression as measured by the changes in the disease markers
次要结局
- Long-term safety and tolerability of repeated intravenous (IV) of Cabaletta 30 g(52 weeks)