Auditory Restoration and Outcomes After Gene Therapy and Cochlear Implantation: a PRospective Cohort Analysis (AURORA)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 180
- 试验地点
- 12
- 主要终点
- Auditory speech perception
研究概览
简要总结
This cohort study aims to explore the trends and differences in multidimensional perceptual levels of patients after cochlear implants or gene therapy, as well as to comprehensively assess the efficacy of gene therapy for congenital deafness, thus providing a reference for making a well-rounded postoperative rehabilitation protocol for gene therapy patients.
详细描述
Cochlear implant patients exhibit improved speech perception in quiet environments after surgery, but their music perception and speech perception under noise are still unsatisfactory. Gene therapy is a novel and promising treatment for congenital hearing loss to recover natural hearing sensations. To date, for patients with congenital deafness, there has been no study on their ability to perceive and understand sounds, such as speech perception in noise, music and directional perception, after hearing recovery owing to gene therapy. In addition, the difference between the two treatments in those perceptual levels postoperatively remains unknown.
Therefore, the investigators designed a single-center cohort study. Based on the intervention method, congenital deafness patients are divided into two groups: the cochlear implant group and the gene therapy group. In the present study, a full-scale evaluation of the two groups will be conducted. The battery encompasses auditory speech perception, cognition, psychological status, and auditory cortex development. A comparative analysis will be conducted to examine multidimensional differences between the two groups, shedding light on the divergent outcomes of gene therapy and cochlear implants for patients with congenital deafness.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 6 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Patients with congenital hearing loss with hearing thresholds ≥65 dB receive either gene therapy (previously received gene therapy and standardized postoperative rehabilitation and follow-up or plan to receive gene therapy), or cochlear implant surgery. Healthy participants with bilateral hearing thresholds within the normal range (≤20 dB), generally matched to the gene therapy group and the cochlear implant group by age and sex.
- •Age ≥ 6 months, regardless of gender. Mandarin Chinese as the native language. Participants and their guardians must provide informed consent before the trial, voluntarily sign a written consent form, and commit to follow-up at specified time points.
- •Capable of effective communication with researchers with the assistance of their guardians and willing to cooperate and comply with the researchers' requirements.
- •The participant's guardians should have a correct understanding of the trial and appropriate expectations regarding potential benefits.
排除标准
- •Presence of other otological disorders that may interfere with the surgical outcome or interpretation of study endpoints, such as middle/inner ear dysplasia or malformations revealed by CT/MRI scans performed within the past 3 months that may affect the therapeutic effect, vestibular-cochlear nerve abnormalities, acute/chronic otitis media, Meniere's disease, etc.
- •Presence of other severe congenital diseases. Presence of severe systemic diseases or diseases in the acute phase, such as pulmonary tuberculosis, active hepatitis B or C infection, active herpes zoster infection, pancreatitis, renal insufficiency, etc.
- •Individuals with low immunity, a history of immune deficiency or organ transplantation.
- •Individuals with a history of neurological or mental disorders, such as epilepsy or dementia.
- •Patients with contraindications to surgery or anesthesia, as assessed by a surgeon, anesthetist, or designated personnel, such as cardiovascular or cerebrovascular events in the past 6 months, allergies to the planned medications, etc.
- •Gene therapy group: gene therapy did not restore hearing; Cochlear implant group: presence of hereditary syndromic deafness or other conditions that seriously affect the efficacy evaluation.
- •Any other conditions for which the investigators consider the subject unsuitable for participation in this clinical study.
研究组 & 干预措施
Gene Therapy Group
The study will recruit gene therapy patients who are definitely diagnosed with autosomal recessive deafness 9 (DFNB9).
Cochlear Implant Group
The study will recruit cochlear implant patients with congenital deafness.
结局指标
主要结局
Auditory speech perception
时间窗: Preoperation, week 13, week 26 and week 52
The Angel Test: the Angel Test is a speech test software included the perception tests of environmental sound test, final recognition test, initial recognition test and lexical tone test in quiet environment. The higher the score the better the auditory speech ability.
Auditory speech perception
时间窗: Preoperatively and at 13, 26, and 52 weeks postoperatively, with subsequent assessments every 6 or 12 months up to 5 years after treatment.
Meaningful Auditory Integration Scale (MAIS)/Infant-toddler Meaningful Auditory Integration Scale (IT-MAIS): the MAIS (≥3 years old)/IT-MAIS (0\~3 years old) is an auditory perception test with a total score of 40 points. The higher the score the better the hearing ability.
次要结局
- Auditory cortex development(Preoperation, week 13, week 26 and week 52)
- Psychological status(Preoperation, week 26 and week 52)
- Cognitive function(Preoperation, week 13, week 26 and week 52)
- Growth and development assessment(Preoperatively and at 13, 26, and 52 weeks postoperatively, with subsequent assessments every 6 or 12 months up to 5 years after treatment.)
- Psychological status(Preoperatively and at 13, 26, and 52 weeks postoperatively, with subsequent assessments every 6 or 12 months up to 5 years after treatment.)
- Cortical auditory processing(Preoperatively and at 13, 26, and 52 weeks postoperatively, with subsequent assessments every 6 or 12 months up to 5 years after treatment.)