A Single Center, Single Ascending Dose, Double-Blind, Randomized, Placebo-Controlled Trial to Establish Safety and the Maximum Tolerated Dose of Intravenous Trehalose Solution in Healthy Subjects
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 24
- 试验地点
- 1
- 主要终点
- Safety and tolerability of escalating doses of intravenously administered trehalose (incidence of adverse events and serious adverse events, including clinically significant laboratory abnormalities)
研究概览
简要总结
This will be a double-blind, randomized, placebo-controlled, single ascending dose study performed in healthy subjects.
The study will include up to four escalating dose cohorts with eight (8) subjects in each cohort.
In each cohort, eligible subjects will be randomized in a 3:1 ratio to receive single IV administration of 9% trehalose (Treatment Arm 1) or placebo (Treatment Arm 2).
All subjects, regardless of their treatment arm assignment, will undergo the same evaluations and will receive the study drug at the clinic. Each subject will continue to be followed for one week post dosing.
详细描述
This is a double-blind, randomized, placebo-controlled, single ascending dose study performed in healthy subjects.
The study will include up to four escalating dose cohorts with eight (8) subjects in each cohort.
In each cohort, eligible subjects will be randomized in a 3:1 ratio to receive single IV administration of 9% trehalose (Treatment Arm 1) or placebo (Treatment Arm 2).
All subjects, regardless of their treatment arm assignment, will undergo the same evaluations and will receive the study drug at the clinic. Each subject will continue to be followed for one week post dosing.
Cohorts 1 to 3 After all subjects in a given cohort complete their 1-week follow-up visit (Visit 4), a Safety Review Committee (SRC) will review the safety and PK data of that cohort. If no safety concerns are identified, and the exposure data supports a higher dose is acceptable, the SRC will approve continuation into the next cohort (dose level).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 盲法
- Triple (Participant, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 55 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Healthy men and women between 18 and 55 years (inclusive) of age
- •Body Mass Index (BMI) 19 to 29.9 kg/m2 (inclusive) and weighing at least 55 kg.
- •Subjects in general good health in the opinion of the investigator
- •Blood pressure and heart rate within normal limits
- •Female subjects must have a negative serum pregnancy test during the Screening period (Day -28 to -1) and be willing and able to use a medically acceptable method of birth control or be postmenopausal.
排除标准
- •Diabetes mellitus type 1 or 2 or HbA1c > 5.6 % at Screening
- •History of significant medical disorder
- •Any clinically significant abnormality in safety laboratory tests during the Screening period (Day -28 to -1)
- •Known contraindication, hypersensitivity and/or allergy to trehalose
- •Any acute illness (e.g. acute infection) within 72 hours
- •Participation in another clinical trial with drugs received within 3 months prior to dosing
- •Positive serum pregnancy test determined during the Screening period or currently lactating women
- •ECG with clinically significant finding recorded during the Screening period
- •Positive HIV, Hepatitis B or Hepatitis C serology at Screening
- •Known history of alcohol or drug abuse in the past 5 years
- •Positive urinary drug screen determined during the Screening period
研究组 & 干预措施
Trehalose 9%
Single dose administration of Trehalose 9% for IV infusion.
干预措施: Trehalose for IV Infusion (Drug)
Saline 0.9%
Single dose administration of 0.9% saline in the same volume and duration as Treatment Arm 1 (9% trehalose)
干预措施: Saline 0.9% IV (Drug)
结局指标
主要结局
Safety and tolerability of escalating doses of intravenously administered trehalose (incidence of adverse events and serious adverse events, including clinically significant laboratory abnormalities)
时间窗: Will be assessed during the entire study. At screening, at day -1 before drug administration, and day 1 of drug administration before, during and after drug administration, and at day 8 the follow up visit
Safety will be assessed by the incidence of adverse events and serious adverse events, including clinically significant laboratory abnormalities.
次要结局
- Maximum-tolerated dose (MTD) of trehalose administered intravenously (Averse events, vitals signs)(Will be assessed during the entire study. At screening, at day -1 before drug administration, and day 1 of drug administration before, during and after drug administration, and at day 8 the follow up visit)
- Pharmacokinetics (PK) of plasma and urine trehalose(Will be assessed on the day of drug administration, before drug administration and up to 12hours following administration.)
- Pharmacokinetics (PK) of serum and urine glucose(Will be assessed on the day of drug administration, before drug administration and up to 12hours following administration.)