A Phase I/II, open-ended, adaptative, open label dose escalation and expansion clinical trial to evaluate the efficacy and safety of unilateral intracochlear injection of SENS-501 using an injection system in children with severe to profound hearing loss due to Otoferlin gene mutations - SENS-501-101

Sensorion0 个研究点目标入组 12 人开始时间: 2023年7月18日最近更新: 2024年7月22日

研究概览

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•Children (male or female) = 6 to = 31 months at the time of inclusion., Severe to profound hearing loss assessed by ABR, Biallelic mutation in the Otoferlin gene, Presence of OAEs, Documented normal cochlea and internal auditory canals, Patients with intact vestibular function

•History of chronic, acute, or major disease, or unspecified reasons, that in the opinion of the Investigator, makes the participant unsuitable for participation in the study or constitutes an unacceptable risk., Have been dosed in a previous gene therapy clinical trial., Patients with a prior or current cochlear implant., Any contraindication to the surgery determined by the surgeon or anesthesia determined by the anesthesiologist, or designee, or history of therapy known as ototoxic (e.g., cisplatin, high dose and long treatment with aminoglycosides, etc.) for an extended period (more than 2 weeks)., Participation in any other interventional clinical trial (in accordance with the definition in Art. 2.2(1) of REGULATION (EU) No 536/2014)., Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the participant or would preclude the participant from successful completion of the study or might interfere with the evaluation of study treatment., Anticipated noncompliance with the protocol requirements

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