A Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Trial of Trehalose for the Treatment of
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 试验地点
- 3
- 主要终点
- Drinking Test Time
研究概览
简要总结
BB-OPMD-202 is a randomized, double-blind, placebo-controlled study of IV trehalose for treatment of OPMD. The study includes a 4-week screening period, a 24-week blinded treatment period during which patients will receive weekly infusions of trehalose or placebo, followed by a 24-week open-label extension period during which all patients will receive weekly infusions of trehalose. Patients will undergo a safety follow-up assessment 4 weeks after their last treatment.
详细描述
After signing informed consent, patients will undergo two rounds of ice-cold water and nectar drinking tests at least 1 week apart to confirm oropharyngeal dysfunction. Patients who have confirmed oropharyngeal dysfunction, i.e., an ice-cold water drinking test time of 8 seconds or greater at both rounds, in addition to an SSQ score of >235, will be enrolled. Baseline values for all safety and efficacy parameters will be established during the screening period. Patients will be randomized in a 1:1 ratio, to trehalose or placebo, at the time of enrollment. Randomization will be stratified according to the patient's score on the SSQ at screening (≤ 799 or ≥ 800).
Patients randomized to trehalose will receive a 1-hour IV infusion of trehalose at a dose of 0.75 g/kg weekly for 24 weeks. Patients randomized to placebo (normal saline) will receive a weight-based equal volume of placebo weekly for 24 weeks.
After Week 24, patients may transition to an open-label extension of the study (extension period). During the extension period, patients will be treated with weekly infusion of trehalose at a dose of 0.75 g/kg for 24 weeks, followed by a 4-week safety follow-up (total duration of study = 56 weeks).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
The Treatment Period of the study is double-blind. The Extension Period is open label.
入排标准
- 年龄范围
- 50 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically confirmed OPMD with a (GCN)13 size PABPN1 mutation
- •A score greater than 235 on the Sydney Swallow Questionnaire at screening
- •Confirmation of oropharyngeal dysfunction by abnormal ice-cold water drinking test result, defined as drinking 80 cc of ice-cold water in ≥ 8 seconds at both drinking tests (at least 1 week apart) during the screening period
排除标准
- •History of pharyngeal myotomy.
- •Esophageal dilatation within the last 12 months.
- •Treatment with botulinum toxin (any location) within 1 year prior to screening.
- •Diagnosis of any other muscle disorder.
- •Prior head and neck surgery or radiation.
- •Oropharyngeal injury or oropharyngeal cancer.
- •Other esophageal disease that may be the cause of the dysphagia.
- •Previously diagnosed with diabetes or a hemoglobin A1c (HgbA1c) result > 6.0% at screening.
- •Prior treatment with IV trehalose.
- •Known hypersensitivity to trehalose.
- •Non-ambulatory (Use of a cane or short leg braces are permitted).
- •Prior history of stroke (ischemic or hemorrhagic).
- •Pregnancy or breast feeding.
- •History of alcohol or drug abuse within the last 5 years.
- •Evidence of hepatitis B, hepatitis C, or HIV infection at screening.
- •Currently receiving anti-coagulant treatment (e.g., warfarin, enoxaparin) other than anti-platelet treatments, which are not a reason for exclusion.
- •Currently participating in another clinical trial or has completed an interventional trial less than 90 days prior to planned first dosing.
研究组 & 干预措施
Trehalose
Trehalose 9% solution: The dose is 0.75 g/kg administered IV over 60 ± 5 minutes once weekly.
干预措施: Trehalose (Drug)
0.9% Normal Saline
Normal saline: weight-based volume administered IV over 60 ± 5 minutes once weekly.
干预措施: Trehalose (Drug)
结局指标
主要结局
Drinking Test Time
时间窗: 24 weeks
Change from baseline in timed drinking tests with 80 cc of ice-cold water and nectar.
次要结局
- Muscle Strength Testing(24 weeks)
- Stair Climb Test(24 weeks)
- Timed Up and Go Test(24 weeks)
- 30-Second Lift Test(24 weeks)
- EuroQol-5D-5L(24 weeks)
- Swallowing Quality of Life(24 weeks)
- Sydney Swallow Questionnaire(24 weeks)