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NCT05788536 · ClinicalTrials.gov registry record · Phase 1
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations
A Phase 1 study of Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF), sponsored by Regeneron Pharmaceuticals.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 30
- Enrollment target
- 15
- Study locations
NCT05788536: Recruiting Phase 1 study of Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF), sponsored by Regeneron Pharmaceuticals.
NCT05788536 is a Phase 1 study of Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF) that is actively recruiting participants, run by Regeneron Pharmaceuticals. The registered enrollment target is 30 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (50% lower). The trial reports 15 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05788536, a Phase 1 study of Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF), is actively recruiting participants, sponsored by Regeneron Pharmaceuticals.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 30 participants
- Enrollment target
- 15
- Study locations
Study Summary
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)
Conditions Studied
Interventions
- GENETIC DB-OTO
Study Locations (15)
Other
- University Hospital Tubingen - Tübingen
- Hospital Universitario Materno Infantil en las Palmas de Gran Canaria - Las Palmas de Gran Canaria
- Ramon y Cajal University Hospital - Madrid
- Addenbrooke's Hospital, Cambridge University Hospitals NHS FT - Cambridge
- Great Ormond Street Hospital For Children NHS Foundation Trust - London
California
- University of California Los Angeles Medical Center - Los Angeles
- Rady Children's Hospital - San Diego
Florida
- Nemours Children s Clinic - Jacksonville
- Nemours Childrens Hospital - Orlando
Massachusetts
- Boston Children's Hospital - Main - Boston
New York
- Columbia University Irving Medical Center - New York
Ohio
- Cincinnati Children's Hospital Medical Center - Cincinnati
Washington
- Seattle Children's Hospital - Seattle
Wisconsin
- Medical College of Wisconsin - Milwaukee
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 30 participants |
| Start Date | 2023-06-27 |
| Est. Completion | 2031-04-19 |
| Phase | Phase 1 |
What NCT05788536 shows while recruiting
NCT05788536 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 30 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (50% lower).
The record links to 1 condition, with Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF) appearing as the primary indexed condition, and to 1 intervention - of which DB-OTO is the first listed.
NCT05788536 lists 15 locations in 9 states (Other, California, Florida).
Frequently Asked Questions
What is clinical trial NCT05788536 about?
NCT05788536 is a clinical study titled "A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations". 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-O...
What is the current status of trial NCT05788536?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 30 participants. The study started on 2023-06-27. Estimated completion is 2031-04-19.
What conditions does trial NCT05788536 study?
This clinical trial studies the following conditions: Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF).
What interventions are being tested in trial NCT05788536?
The interventions under investigation include: DB-OTO (GENETIC).
Who is sponsoring clinical trial NCT05788536?
This trial is sponsored by Regeneron Pharmaceuticals, which has 280 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05788536 being conducted?
This trial has 15 study locations across California, Florida, Massachusetts, New York, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT05788536's enrollment target sits among peer trials
30 1333rd of 2000 higher than 561 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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