A Phase 1b/2a, Double-blinded, Placebo-controlled, Multiple Doses, 2 Step-up Study Evaluating the Safety, Tolerability, PK/PD and Efficacy of Systemic NS101 in Healthy Volunteers and SSNHL Patients
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 138
- 试验地点
- 15
- 主要终点
- Phase 1b part: Change from Baseline in Safety Profiles
研究概览
简要总结
The goal of this 2 step-up, exploratory study is to test safey, tolerability and PK/PD profiles in healthy volunteers and safety, tolerability and efficacy in sudden sensorineural hearing loss patients as an early salvage therapy.
The main questions it aims to answer are:
- whether is it safe and tolerable when healthy volunteers and sudden sensorineural hearing loss patients take multiple doses of NS101 against FAM19A5
- whether is it effective in reversing hearing capability in sudden sensorineural hearing loss patients who fails to show sufficient recovery despite of oral standard steroid therapy.
Patients and heathly volunteers will be given NS101 15mg/kg or 30mg/kg systemically less than 3 months per protocol.
This is placebo controlled, double blinded study, which means there will be a group who receives placebo (i.e. fake drug) for study purpose.
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
- Triple (Participant, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 19 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •unilateral ideopathic SSNHL greater than 45dB at the average of 4 frequencies in PTA (contralateral hearing is less than 30dB)
- •Subject who has completed investigator-recognized standard treatment within 30 days of symptom onset and is able to undergo randomization visit within 35 days.
- •Subjects who, in the opinion of the investigator, have received sufficient systemic steroid therapy for approximately 2 weeks recovery
- •A combination regimen consisting of approximately 2 weeks of systemic steroid therapy and intratympanic steroid administration
排除标准
- •other otologic or systemic diseases
- •retrocochlear lesion
研究组 & 干预措施
Group A
NS101 15mg/kg IV infusion Biweekly for 6weeks in Healthy Volunteers (active:placebo=6:3)
干预措施: NS101 (Biological)
Group A
NS101 15mg/kg IV infusion Biweekly for 6weeks in Healthy Volunteers (active:placebo=6:3)
干预措施: Placebo (Biological)
Group B
NS101 30mg/kg IV infusion Biweekly for 6weeks in Healthy Volunteers (active:placebo=6:3)
干预措施: NS101 (Biological)
Group B
NS101 30mg/kg IV infusion Biweekly for 6weeks in Healthy Volunteers (active:placebo=6:3)
干预措施: Placebo (Biological)
Cohort A
NS101 15mg/kg IV infusion Biweekly for 12weeks in Sudden Sensorineural Hearing Loss patients
干预措施: NS101 (Biological)
Cohort B
Placebo 15mg/kg IV infusion Biweekly for 12weeks in Sudden Sensorineural Hearing Loss patients
干预措施: Placebo (Biological)
结局指标
主要结局
Phase 1b part: Change from Baseline in Safety Profiles
时间窗: Baseline, Day1, Day2, Day3, Day14, Day15, Day16, Day17, Day28, Day29, Day30, Day32, Day43
Adverse Event, Serious Adverse Event, ratio of Treatment related Serisous Adverse Event with causality \& severity will be descriptively measured and collected in the following time frames
Phase 2a part: Changes from Baseline in Safety Profiles
时间窗: Baseline, Week1, Week2, Week4, Week6, Week8, Week10, Week12, Week16, Week20
Adverse Event, Serious Adverse Event, ratio of Treatment related Serisous Adverse Event with causality \& severity will be descriptively measured and collected in the following time frames
次要结局
- Phase 1b part: Change from Baseline in PK/PD profiles & Immunological Assay(Baseline, Day1, Day2, Day3, Day14, Day15, Day16, Day17, Day28, Day29, Day30, Day32, Day43)
- Phase 2a part : Change from Baseline in PK/PD profiles & immunological Assay(Baseline, Week1, Week2, Week4, Week6, Week8, Week10, Week12, Week16, Week20)