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DB-OTO

Phase 1

Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF) | Gene therapy | Rare Disease |Regeneron Pharmaceuticals, Inc.|Last Updated: Aug 5, 2026

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment36

FDA Designations

No designations recorded

Clinical trial landscape

DB-OTO · 1 trial · 1 indication

Phase 1 1
NCT05788536A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin MutationsCongenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
RECRUITING36 Analytics
PHASE1RECRUITING
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
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)Unlock trial analytics

Study Endpoints

Primary Endpoints

Incidence and severity of Treatment-Emergent Adverse Events (TEAEs)
Up to week 104
Achievement of a hearing sensitivity threshold of ≤70 dB assessed by average Pure Tone Audiometry (PTA)
Up to week 104

Secondary Endpoints

Auditory Brainstem Response (ABR) to click stimulus at ≤90 dB normalized Hearing Level (nHL)
Up to week 48
Achievement of hearing sensitivity threshold of ≤45 dB assessed by average PTA
Up to week 104
Achievement of hearing sensitivity threshold of ≤25 dB assessed by average PTA
Up to week 104
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
DB-OTO - Part AEXPERIMENTALUnilateral intracochlear dosing
DB-OTO - Part BEXPERIMENTALBilateral intracochlear dosing using the dose selected based on safety and efficacy data from Part A
DB-OTO - Part CEXPERIMENTALUnilateral or bilateral intracochlear dosing

Interventions

NameTypeDescription
DB-OTOGENETICAdministered per the protocol
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Eligibility Criteria

SexALL
Healthy VolunteersNo
Study Sites17

Key Inclusion Criteria: 1. Willingness to provide written informed consent (by at least one parent/legal guardian for pediatric participants, and with participant to provide assent, when applicable, or by the adult participant) and willingness to comply with trial protocol 2. Willingness to consent...

Countries:United StatesGermanyJapanSpainUnited Kingdom
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Recent Changes (Last 90 Days)

LOWAug 5, 2026NCT05788536lastUpdatePostDate: changed
MEDIUMJul 2, 2026NCT05788536Enrollment: 30 → 36
MEDIUMJul 2, 2026NCT05788536Enrollment: 30 → 36
MEDIUMJul 2, 2026NCT05788536Enrollment: 30 → 36
MEDIUMJul 2, 2026NCT05788536Enrollment: 30 → 36

Frequently asked questions about DB-OTO

What is DB-OTO used for?

DB-OTO is an investigational gene therapy being developed for congenital hearing loss secondary to biallelic mutations of the otoferlin gene (OTOF). It is designed to address hearing loss caused by mutations in this specific gene.

How does DB-OTO work?

DB-OTO is an adeno-associated virus (AAV) based gene therapy. It is designed to deliver a functional copy of the otoferlin gene to target cells, with the goal of restoring hearing in patients with congenital hearing loss due to biallelic OTOF mutations.

Who is developing DB-OTO?

DB-OTO is being developed by Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN). The company is conducting clinical trials to evaluate the safety and efficacy of this gene therapy for congenital hearing loss.

What phase is DB-OTO in?

DB-OTO is currently in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The ongoing Phase 1 trial is recruiting participants to assess the therapy.

What clinical trials is DB-OTO in?

DB-OTO is being studied in a Phase 1 clinical trial (NCT05788536), which is currently recruiting. The trial is evaluating DB-OTO in children, infants, adolescents, and adults with hearing loss due to otoferlin mutations, with an enrollment target of 36 participants.

Is DB-OTO the same as an AAV-based gene therapy?

Yes, DB-OTO is an adeno-associated virus (AAV) based gene therapy. It uses an AAV vector to deliver the therapeutic gene, and it is being investigated for the treatment of congenital hearing loss caused by biallelic otoferlin gene mutations.