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

NCT ID: NCT06025032

Last Updated: 2025-04-10

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: EARLY_PHASE1
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-03-30
• Study Completion Date: 2025-04-08

Brief 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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

HG205

Method of Administration: Once Unilateral intracochlear injection/subject; The duration of the study for each subject includes a screening period, enrollment visit, treatment visit, a 26-week follow-up period, and a 5-year long-term safety follow-up after the injection

Group Type: EXPERIMENTAL

HG205

Intervention Type: GENETIC

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

Interventions

HG205

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

Intervention Type: GENETIC

Eligibility Criteria

Inclusion Criteria

• Male or females between 1 and 16 years of age at the time the subject/parent/legal guardian signs the informed consent form.
• Willing to adhere to the protocol as evidenced by written informed consent or parental permission and subject assent.
• Molecular diagnosis of biallelic mutations in the OTOF gene with at least one mutation being p.Q829X through collected blood samples at screening;
• Based on auditory brainstem response (ABR), clinically diagnosed sensorineural hearing loss (SNHL) with the following hearing thresholds: severe (65 dB ≤ hearing threshold < 80 dB) or profound (80 dB ≤ hearing threshold < 95 dB) or complete (hearing threshold ≥ 95 dB) hearing loss in both ears.
• Acceptable hematology, clinical chemistry, and urine laboratory parameters.

Exclusion Criteria

• Pre-existing other hearing-loss conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints or complications of surgery.
• Presence of cochlear implants in the study ear.
• Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
• Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter hearing function.
• Prior participation in clinical study with an investigational drug within the past six months.
• Prior gene therapy treatments.
• Any condition which leads the investigator to believe that the participant cannot comply with the protocol requirements or that may place the participant at an unacceptable risk for participation.

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 16 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Eye & ENT Hospital of Fudan University

OTHER

Sponsor Role: collaborator

HuidaGene Therapeutics Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

HuidaGene Therapeutics Co., Ltd.

Locations

Eye & ENT Hospital of Fudan University

Shanghai, , China

Countries

China

Other Identifiers

HG20501

Identifier Type: -

Identifier Source: org_study_id