A Three-part, Multicenter, Open Label, Single Dose Study to Assess the Safety, Tolerability, and Efficacy of Intra Labyrinthine (IL) CGF166 in Patients With Severe-to-profound Hearing Loss
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 22
- 试验地点
- 1
- 主要终点
- Summary of Change From Baseline in Treated Ear's Pure Tone Audiometry Air Conduction Threshold by Frequency for Last Study Visit
研究概览
简要总结
The goal of the study was to evaluate the safety, tolerability, and the potential ability of CGF166 delivered through IL-infusion to improve hearing. CGF166 is a recombinant adenovirus 5 (Ad5) vector containing a cDNA encoding the human Atonal transcription factor (Hath1).
详细描述
This study evaluated the safety, tolerability, and potential efficacy of CGF166 and the associated delivery procedures in patients with severe-to-profound unilateral or bilateral hearing loss. Eligible patients were required to have documented, non-fluctuating hearing loss.
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 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- •Patients with hearing loss caused by genetic/developmental disorders, e.g., cochlea aplasia
- •Patients with existing conductive hearing loss or mixed hearing loss as judged by the Principal Investigator following a thorough review of all of the trial hearing assessments;
- •Patients with a history of cochlear implant in the study ear
- •Hearing loss due to any other cause that would not be expected to respond to hair cell regeneration, for example mechanical trauma or central auditory lesions or lack of an auditory nerve
- •Patients who will require ototoxic drugs as routine therapy over the course of the study, for example cystic fibrosis patients
- •Any contraindication to the planned surgery or anesthesia as determined by the surgeon or anesthesiologist
- •Previous surgery in the study ear
- •Any otological history, such as chronic otitis, cholesteatoma, tympanic membrane perforation, that suggests poor candidacy for cochlear implant or inner ear surgery or suggests potential interference with study auditory or vestibular function tests
- •Pregnant women
- •Abnormal vital signs and/or ECG that suggest potential contraindication for planned study anesthesia
- •Past serious adverse reaction to anesthesia
- •Meniere's Disease
- •History of radiation therapy to the head and neck
- •Participation in a clinical trial within the last 30 days
- •Immunocompromised patients, as judged by the investigators based on patient history, physical exam and CBC
研究组 & 干预措施
CGF166 dose 20 uL
single dose volume #1
干预措施: CGF166 (Drug)
CGF166 dose 30 and 40 uL
single dose volume #2
干预措施: CGF166 (Drug)
CGF166 dose 40 uL
single dose volume #3
干预措施: CGF166 (Drug)
CGF166 dose 60 uL
single dose volume #4
干预措施: CGF166 (Drug)
CFG166 dose 30 uL
Single dose volume #5
干预措施: CGF166 (Drug)
结局指标
主要结局
Summary of Change From Baseline in Treated Ear's Pure Tone Audiometry Air Conduction Threshold by Frequency for Last Study Visit
时间窗: Week 52
Summary of change from baseline in pure tone audiometry air conduction threshold by frequency for last study visit is presented in table below.
Summary of Change From Baseline in Non-treated Ear's Pure Tone Audiometry Air Conduction Threshold by Frequency for Last Study Visit
时间窗: Week 52
Summary of change from baseline in Non-treated ear's pure tone audiometry air conduction threshold by frequency for last study visit is presented in table below.
Number of Participants With Adverse Events
时间窗: week 52
AE tables are below in the Adverse Events section of this report.
Number of Adverse Events
时间窗: week 52
AE tables are below in the Adverse Events section of this report.
Summary of Pure Tone Audiometry in Treated Ear Compared to Pretreatment Values
时间窗: Days 29, 57, 85, 113, 141, 169, 358, 537, 600
Summary of pure tone audiometry air conduction thresholds at frequency 0.125 KHz
Summary Pure Tone Audiometry Bone Conduction Thresholds in Treated Ear by Time and Frequency
时间窗: Days 29, 57, 85, 113, 141, 169, 358,537, EoS
Summary of pure tone audiometry bone conduction thresholds by time and frequency 0.250 KHz
次要结局
- Number of Participants With Response in Vestibular Function in Treated Ear Compared to Pretreatment Values(24 months)
- Number of Participants With Changes in Auditory Functions (Speech Recognition) and Vestibular Functions Before and After IL Infusion of CGF166 Between the Study Ear and the Contralateral Ear(24 months)
- Number of Participants With Change in Brainstem Auditory Evoked Responses (BAER) Compared to Pretreatment Values(24 months)