NCT06025032

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

Withdrawn EARLY_PHASE1 Last updated 10 April 2025

What this trial tests

EARLY_PHASE1 trial testing HG205 in Congenital Hearing Loss. Withdrawn.

Timeline

30 March 2023

Primary endpoint
8 April 2025

8 April 2025

Quick facts

Lead sponsor	HuidaGene Therapeutics Co., Ltd.
Phase	EARLY_PHASE1
Status	Withdrawn
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Start date	30 March 2023
Primary completion	8 April 2025
Estimated completion	8 April 2025
Sites	1 location across China

Drugs / interventions tested

• HG205

Conditions studied

• Congenital Hearing Loss — all drugs for Congenital Hearing Loss →

Sponsor

HuidaGene Therapeutics Co., Ltd. — full company profile →

Who can join

Adults 1 to 16, any sex, with Congenital Hearing Loss. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Recent Therapeutic Gene Editing Applications to Genetic Disorders.
   Deneault E. · · 2024 · cited 25× · PMID 38785523 · DOI 10.3390/cimb46050255
2. AAV-mediated Gene Therapy for Hereditary Deafness: Progress and Perspectives.
   Zhang L, Tan F, Qi J, Lu Y, et al · · 2024 · cited 17× · PMID 39556694 · DOI 10.1002/advs.202402166
3. Recent Therapeutic Progress and Future Perspectives for the Treatment of Hearing Loss.
   Lye J, Delaney DS, Leith FK, Sardesai VS, et al · · 2023 · cited 16× · PMID 38137568 · DOI 10.3390/biomedicines11123347
4. Inner Ear Gene Therapy: An Overview from Bench to Bedside.
   Gadenstaetter AJ, Krumpoeck PE, Landegger LD. · · 2025 · cited 8× · PMID 39625555 · DOI 10.1007/s40291-024-00759-1
5. Engineering adeno-associated viral vectors for CRISPR/Cas based in vivo therapeutic genome editing.
   Moyo B, Brown LBC, Khondaker II, Bao G. · · 2025 · cited 6× · PMID 40203649 · DOI 10.1016/j.biomaterials.2025.123314
6. Expanding Horizons of CRISPR/Cas Technology: Clinical Advancements, Therapeutic Applications, and Challenges in Gene Therapy.
   Bairqdar A, Karitskaya PE, Stepanov GA. · · 2024 · cited 6× · PMID 39769084 · DOI 10.3390/ijms252413321
7. CRISPR-Cas technologies in neurodegenerative disorders: mechanistic insights, therapeutic potential, and translational challenges.
   Yashooa RK, Nabi AQ, Smail SW, Azeez SS, et al · · 2025 · cited 3× · PMID 41674784 · DOI 10.3389/fneur.2025.1737468
8. Advancements in CRISPR/Cas systems for disease treatment.
   Xu Y, Le H, Wu Q, Wang N, et al · · 2025 · cited 2× · PMID 40654372 · DOI 10.1016/j.apsb.2025.05.007