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a Study in Subjects With Otoferlin Mutation-related Hearing Loss Using RNA Base-eDiting Therapy(SOUND)

NCT06025032 · Status: WITHDRAWN · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL

Last updated 2025-04-10

No results posted yet for this study

Summary

The purpose of the study is to determine whether HG205 as CRISPR/Cas13 RNA base-editing therapy is safe and effective for the treatment of hearing loss caused by p.Q829X mutation in OTOF gene.

Conditions

  • Congenital Hearing Loss

Interventions

GENETIC

HG205

The study will enroll up to 2 cohorts, evaluating a starting dose plus a higher or lower dose

Sponsors & Collaborators

  • Eye & ENT Hospital of Fudan University

    collaborator OTHER

  • HuidaGene Therapeutics Co., Ltd.

    lead INDUSTRY

Principal Investigators

  • Study Director · HuidaGene Therapeutics Co., Ltd.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-03-30
Primary Completion
2025-04-08
Completion
2025-04-08

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06025032 on ClinicalTrials.gov