A Phase 1/2, Open-label, Single Dose Study of SKY-GJB2 Given as a Single Unilateral Intracochlear Administration in Pediatric Subjects With GJB2-mediated Hearing Loss
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 10
- 试验地点
- 4
研究概览
简要总结
Skylark Bio is conducting a study of an investigational new drug called SKY-GJB2 and its delivery device, the SKY-CAT. SKY-GJB2 is a gene therapy that is being developed to treat children who have hearing loss due to changes in the GJB2 gene (also referred to as Connexin 26). The SKY-CAT is a device that delivers the gene therapy.
The purpose of this study is to:
Learn about the safety and tolerability (does not cause ongoing discomfort) of SKY-GJB2 Evaluate the efficacy of SKY-GJB2 (how well is works)
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 9 Months 至 7 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female aged 9 months to 7 years at the time of gene therapy administration
- •Subjects must have at least two (biallelic) pathogenic or likely pathogenic variants in the GJB2 gene
- •Bilateral sensorineural hearing loss as assessed by ABR. Hearing loss at ≥85 dB HL for at least one of the frequencies (500-4000 Hz) in the study treatment ear.
- •Subject's parent(s)/guardian(s) provide informed consent before the initiation of study-related procedures.
- •Subject is able and willing to comply (or provide assent if old enough) will all study requirements.
排除标准
- •Subject has non-GJB2 mediated hearing loss including genetic, syndromic, or non-syndromic hearing loss that is not associated with GJB2 mutations.
- •Subject has autosomal dominant nonsyndromic hearing loss due to GJB2 mutation.
- •No response on ABR testing.
- •Bilateral Cochlear Implants.
研究组 & 干预措施
Older
Four subjects aged 2 to 7 years old (inclusive) to receive an intracochlear infusion of SKY-GJB2 in the study ear using a sterile, one-time use investigational medical device.
干预措施: SKY-GJB2 (Combination Product)
Younger
Six subjects aged 9 months to less than 2 years old to receive an intracochlear infusion of SKY-GJB2 in the study ear using a sterile, one-time use investigational medical device.
干预措施: SKY-GJB2 (Combination Product)