NCT02191826已完成1 期
Study of SOM0226 in Familial Amyloid Polyneuropathy (FAP) Patients and Asymptomatic Carriers to Evaluate Protein Stabilization Activity
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 17
- 试验地点
- 1
- 主要终点
- TTR stabilization
研究概览
简要总结
Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis.
详细描述
This is an open label interventional Phase IIa proof of concept clinical trial designed in two phases separated by a washout period of 6 weeks (± 2 weeks) , to evaluate the TTR stabilization activity of SOM0226 in healthy volunteers (wild type), TTR-FAP patients and asymptomatic carriers.
- Phase A (24 hours): SOM0226 single dose
- Phase B (32 hours): SOM0226 multiple dose
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Participant is willing and able to give informed consent for participation in the study
- •Male or Female, aged 18 years or above at the time of consent
- •Two cohorts defined by TTR variant (wild type: healthy volunteers; TTR mutant: asymptomatic carriers with documented mutation in TTR or diagnosed TTR-FAP patients (stage 1 or 2) not undergoing pharmacological treatment with Vyndaqel
- •Body Mass Index (BMI) > 17.5 kg/m2
- •Female participants of child bearing potential and male participants whose partner is of child bearing potential must be willing to ensure that they or their partner use effective contraception during the study and for 2 month thereafter
- •Able (in the Investigators opinion) and willing to comply with all study requirements
排除标准
- •Female participant who is pregnant, lactating or planning pregnancy during the course of the study
- •Evidence of history of clinically significant hepatic disease
- •An ALT or AST measurement > 2 times the ULN (Upper Limit of Normal)
- •Scheduled elective surgery or other procedures requiring general anaesthesia during the study
- •Donation of blood during the study or within the past 4 weeks
- •Treatment (during the study or within the past 4 weeks) with a prescription or investigational drug for the treatment of TTR amyloidosis
- •Treatment with NSAIDs (nonsteroidal antiinflammatory drug) during the study or within the past 4 weeks. The following NSAID are allowed: acetylsalicylic acid, etodolac, ibuprofen, indomethacin, ketoprofen, nabumetone, naproxen, nimesulide, piroxicam and sulindac
研究组 & 干预措施
SOM0226 single dose
Experimental
干预措施: SOM0226 (Drug)
SOM0226 multiple doses
Experimental
干预措施: SOM0226 (Drug)
结局指标
主要结局
TTR stabilization
时间窗: 24 hours and 32 hours
TTR stabilization will be measured by determining the concentration of tetrameric TTR after denaturation. The ratio of TTR tetramer after denaturation / TTR tetramer before denaturation of plasma samples from treated subjects will be compared to their baseline ratio. TTR stabilization will be determined as percentage of the baseline ratio.
次要结局
- Pharmacodynamics assessment(24 hours and 32 hours)
- Safety(24 hours)
研究者
研究点 (1)
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