A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults 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 - N/A

Healthy Volunteers

Not accepted

Interventions

• DB-OTO — GENETIC
  Administered per the protocol

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 pediatric and adult participants who have severe-to profound and profound 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

First listed

Mar 29, 2023

Start date

Jun 27, 2023

Status verified

Jun 2026

Primary completion

Feb 26, 2029

Completion

Feb 25, 2032

Study Design

Enrollment

36 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: DB-OTO - Part A
  Unilateral intracochlear dosing
• Experimental: DB-OTO - Part B
  Bilateral intracochlear dosing using the dose selected based on safety and efficacy data from Part A
• Experimental: DB-OTO - Part C
  Unilateral or bilateral intracochlear dosing

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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