NCT02015481已完成2 期
Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 25
- 试验地点
- 4
- 主要终点
- Safety Lab Evaluations
研究概览
简要总结
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
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
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
- •Males and females
- •18 - 80 years (inclusive) of age
- •Clinically and genetically diagnosed as OPMD
- •Moderate dysphagia (abnormal drinking test at screening and on the first dosing day, before drug administration)
- •Patients who provide written informed consent to participate in the study
- •Body Mass Index (BMI) <30 kg/m2
排除标准
- •Diabetes mellitus type 1 or 2
- •Other major diseases, e.g.: renal failure (creatinine clearance <60ml/min), liver failure and chronic liver diseases (e.g. hepatitis B or C) , HIV carriers, tuberculosis, SLE, rheumatoid polyarthritis, sarcoidosis, collagenosis
- •Uncontrolled heart disease , CHF,
- •Other neuromuscular diseases
- •Other disorders associated with esophageal dysphagia: e.g. severe gastroesophageal reflux (GERD), esophageal stricture due to mechanical or chemical trauma, infection (e.g. esophageal moniliasis), drug-induced dysphagia (e.g. bisphosphonates), esophageal rings and webs, spastic motility disorders of the esophagus.
- •History of malignancy (except non-invasive skin malignancy)
- •History of neck irradiation
- •Pregnant or currently lactating women
- •Obesity (BMI≥ 30) and associated morbidity
- •Prior pharyngeal myotomy
- •Weight loss of more than 10% in the last 12 months.
- •Known hypersensitivity to any ingredients in the injection
- •Patient receiving anticoagulant treatment (e.g. warfarin)
研究组 & 干预措施
Cabaletta 30gr.
Experimental
weekly IV of Cabaletta 30gr.
干预措施: Cabaletta (Drug)
结局指标
主要结局
Safety Lab Evaluations
时间窗: 24 weeks
Change from baseline in safety labs including hematology, coagulation, chemistry, renal function, and liver function tests at week 24 .
次要结局
- Drinking Test Score(24 weeks)
- Videofluoroscopy (VFS) Score(24 Weeks)
- SWAL-QOL, Swallowing Quality of Life Questionnaire(28 weeks)
研究者
研究点 (4)
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