Trials Sponsors Conditions Drugs Pricing

Conditions

Congenital Hearing Loss

Keywords

Congenital hearing lossOTOFQ829XHG205CRISPR

Outcome Measures

Primary Outcomes (1)

Incidence of otological and systemic adverse events
Number of AE(Adverse events),SAE(Serious Adverse Events),DLT(Dose Limiting Toxicities)
26 weeks

Secondary Outcomes (2)

Change from baseline in ABR(Auditory Brainstem Response) intensity threshold (decibels normal hearing level [dB nHL])
26 weeks
Change from baseline in hearing performance by behavioral audiometry with pure-tone audiometry
26 weeks

Study Arms (1)

HG205

EXPERIMENTAL

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

Genetic: HG205

Interventions

HG205Genetic

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

HG205

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age1 Year - 16 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

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.

Sponsors & Collaborators

HuidaGene Therapeutics Co., Ltd.
Industrylead
Eye & ENT Hospital of Fudan University
Othercollaborator

Study Sites (1)

Eye & ENT Hospital of Fudan University

Shanghai, China

Study Officials

Study Director
HuidaGene Therapeutics Co., Ltd.
STUDY DIRECTOR

Enrollment

0 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: early phase 1
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

August 21, 2023

First Posted

September 6, 2023

Study Start

March 30, 2023

Actual (reported)

Study Stopped

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

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (2)

Study Arms (1)

HG205

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (2)

Study Arms (1)

HG205

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations