A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations

Conditions

• Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)

Eligibility Criteria

Sex

ALL

Age

N/A - 17 Years

Healthy Volunteers

Not accepted

Interventions

• DB-OTO — GENETIC
  DB-OTO will be administered as a single intracochlear injection into one ear (Part A). * LD Cohort (lower dose) * HD Cohort (high dose) - not implemented
• DB-OTO — GENETIC
  DB-OTO will be administered as a single intracochlear injection into both ears (Part B). For bilateral injections (Part B), patients will receive DB-OTO in 1 surgical session.

Study Details

Regeneron is conducting a study of an investigational new drug called DB-OTO. DB-OTO is a gene therapy that is being developed to treat children who have hearing loss due to changes in the otoferlin gene. The purpose of this study is to: * Learn about the safety of DB-OTO * Determine how well DB-OTO is tolerated (does not cause ongoing discomfort) * Evaluate the efficacy of DB-OTO (how well DB-OTO works)

Key Dates

Start date

Jun 27, 2023

Status verified

Apr 2026

Primary completion

Feb 28, 2032

Completion

Feb 28, 2032

Study Design

Enrollment

30 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: DB-OTO - Unilateral Dose Escalation
  Part A: Unilateral intracochlear dosing
• Experimental: DB-OTO - Bilateral Dose Expansion
  Part B: Bilateral intracochlear dosing using the dose selected based on safety and efficacy data from Part A.

Primary Outcome Measure

Incidence and severity of Treatment-Emergent Adverse Events (TEAEs) [ Time Frame: Up to week 104 ]

Central Contacts

• Clinical Trials Administrator
  844-734-6643
  [email protected]

Locations (9)

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