CTIS2024-511342-40-00招募中1 期
A Phase 1/2, Open-Label, Multicenter Trial with a Single Ascending Dose Cohort with Unilateral Intracochlear Injection Followed by a Bilateral Injection Expansion Cohort to Evaluate the Safety, Tolerability, and Efficacy of DB-OTO in Children and Infants with Biallelic hOTOF Mutations - DB-OTO-001
Regeneron Pharmaceuticals Inc.0 个研究点目标入组 22 人开始时间: 2024年7月1日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 22
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 至 17（—）
- 性别
- All
入选标准
- •Presence of pathogenic or likely pathogenic mutations in both alleles of the OTOF gene., Patient is <18 years of age and of the appropriate age to qualify for enrollment in the corresponding age cohort at the time the parent/legal guardian signs the informed consent form. Participant to provide assent, when applicable., Audiological Criteria: Infants =24 months of age: a. Absence of an ABR neural signal in response to a click stimulus =85 dB nHL in the ear(s) to be injected with DB-OTO. b. Presence of otoacoustic emissions (OAE) in the ear(s) to receive DB-OTO., Audiological Criteria: Children >24 months to <18 years of age: a. Absence of an ABR neural signal in response to a click stimulus =85 dB nHL in the ear(s) to be injected with DB-OTO. b. Presence of otoacoustic emissions (OAE) in the ear(s) to receive DB-OTO OR c. Presence of a cochlear microphonic in ears to receive DB-OTO., Willingness of at least 1 parent/legal guardian to consent to vaccinations for the patient in accordance with the country-specific pediatric immunization schedule, No clinically significant laboratory findings on clinical laboratory tests at time of Screening, No evidence that hearing loss is dependent on body temperature, From study start and for the duration of the short-term follow-up period (18 months): Female patients of childbearing potential and fertile males, must agree to use highly effective contraception. Female patients must agree not to become pregnant. Fertile male patients must agree not to father a child or donate sperm., Additional Protocol defined inclusion criteria may apply.
排除标准
- •History or presence of other permanent or untreatable hearing loss conditions., Prior or current cochlear implants in the ear(s) to be injected with DB-OTO, History of risk factor(s) for auditory neuropathy not caused by OTOF mutations including: prematurity, low birth weight, hyperbilirubinemia, neonatal intensive care unit (NICU) admission, and/or low Apgar scores., Prior or current history of malignancies., Prior or current history of meningitis., History of prior treatment with gene therapy., Surgical anatomy that would preclude the planned surgical approach as indicated by medical imaging (eg, computed tomography [CT] or magnetic resonance imaging [MRI]) in the ear(s) to be injected with DB-OTO., Additional Protocol defined exclusion criteria may apply.
研究者
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