An Investigator/Subject Blind, Randomized, Placebo-Controlled Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Doses of RO7034067 in Healthy Japanese Subjects

Hoffmann-La Roche1 个研究点分布在 1 个国家目标入组 24 人开始时间: 2017年3月22日最近更新: 2018年10月4日

适应症

干预措施

试验速览

阶段: 1 期
状态: 已完成
发起方: Hoffmann-La Roche
入组人数: 24
试验地点: 1
主要终点: Maximum Observed Plasma Concentration (Cmax) of Risdiplam

研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

This is a randomized, placebo-controlled study to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of single oral doses of Risdiplam in healthy Japanese participants.

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: Double (Participant, Investigator)

入排标准

年龄范围: 18 Years 至 60 Years（Adult）
性别: All
接受健康志愿者: 是

入选标准

•Participants of Japanese origin, with Japanese parents (for the provisional Caucasian cohort, participants must be male or female Caucasians with 4 Caucasian grand-parents)
•Healthy participants. Healthy status is defined by the absence of evidence of any active or chronic disease following a detailed medical and surgical history, a complete physical examination including ophthalmological examination, vital signs, 12-lead electrocardiogram (ECG), hematology, blood chemistry, serology and urinalysis
•A body mass index (BMI) between 18 to 30 kilograms per square meter (kg/m^2) inclusive
•Male participants must agree to use a barrier method of contraception from dosing until completion of the study and for 4 months thereafter
•Female participants must be either surgically sterile or post-menopausal

排除标准

•Medical history of cardiovascular disease, renal disease, liver disease, digestive system disease, blood dyscrasia, immunologic disease, diseases of the nervous system, endocrine disease, metabolic disease, lung disease, or with anamnesis and obstacles in kidney, liver, or cardiopulmonary function
•Participants with any clinically significant eye pathology
•Laboratory test (hematology, biochemistry, physical examination or vital signs) values outside the institutional normal range and rated as clinically significant abnormal at screening
•Any clinically significant abnormalities in ECG at screening
•Inherited long QT syndrome or known family history of arrhythmia
•Systolic blood pressure (SBP) higher than 140 millimeters of mercury (mmHg) or below 90 mmHg, and/or diastolic blood pressure (DBP) higher than 90 mmHg or below 50 mmHg in the supine position, as assessed at screening
•Positive result for human immunodeficiency virus (HIV) antigen and antibody, hepatitis B surface antigen (HBsAg) or hepatitis C virus (HCV) antibody at screening
•Donation or loss of blood over 500 milliliters (mL) within three months prior to screening
•History of smoking more than 10 cigarettes a day. Participants who have quit smoking or who have reduced their daily cigarette smoking to 10 or less for more than one month prior to screening are allowed
•Daily consumption of food or drink containing a large amount of methylxanthine (caffeine, theophylline, theobromine)
•Present or past history of substance addiction, dependence or abuse, such as abuse of drugs or alcohol
•Concomitant or previous participation in any clinical trial either within 90 days or 5 half-lives of the investigational drug, whichever is longer, before administration of study drug in this study
•Use of prescribed medications which have systemic exposure within one week before enrolment
•Use of any concomitant drug during the study (including over-the-counter medication and medications used in dentistry)
•Inability to meet the study requirements in the opinion of the Investigator

研究组 & 干预措施

Risdiplam '2' Milligrams (mg)

Experimental

A single dose of 2 mg Risdiplam will be administered to all participants randomized to this arm under fasted conditions as an oral drinking solution on Day 1.

干预措施: Risdiplam (Drug)

Risdiplam '6' mg

Experimental

A single dose of 6 mg Risdiplam will be administered to all participants randomized to this arm under fasted conditions as an oral drinking solution on Day 1.

干预措施: Risdiplam (Drug)

Risdiplam '12' mg

Experimental

A single dose of 12 mg Risdiplam will be administered to all participants randomized to this arm under fasted conditions as an oral drinking solution on Day 1.

干预措施: Risdiplam (Drug)

Placebo

Placebo Comparator

A single dose of placebo will be administered to all participants randomized to this arm under fasted conditions as an oral drinking solution on Day 1.

干预措施: Placebo (Drug)

结局指标

主要结局

Maximum Observed Plasma Concentration (Cmax) of Risdiplam

时间窗: Pre-dose (0 hour [hr]), 0.5, 1, 2, 3, 4, 4.5, 5, 6, 8, 10, 12, 24, 36, 48, 72, 120, 168, 264 hr postdose from Day 1

Time to Maximum Plasma Concentration (Tmax) of Risdiplam