An Open-Label, Multicenter Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of Intracochlear Ascending Dose Injections of SENS-601 Followed by Expansion Cohort in Pediatric Population With Hearing Loss Related to Biallelic Pathogenic Variation(s) of GJB2, As Well As to Demonstrate the Performance of the Administration System, Its Safety, Its Clinical Benefit, and Its Usability.

Condition studied

Hearing Loss Related to biallelic pathogenic variation (s) of the GJB2 Gene.

Investigational medicinal product(s)

Eligibility

Primary endpoint

For Part I: The incidence and severity of Treatment-Emergent Adverse Events. The incidence and severity of Adverse Device Events, Serious Adverse Device Events, and Device Deficiencies., For Part 2: Change in auditory brainstem response and pure-tone audiometry from baseline.

Endpoint detail

1. Auditory brainstem response, 2. Pure-tone audiometry, 3. Patient-Reported Outcomes and Clinician-Reported Outcomes via questionnaires, 4. Otoacoustic emissions, 5. Vector shedding, 6. Inflammatory biomarkers, 7. Humoral immune biomarkers, 8. Cellular immune biomarkers