Trials / Withdrawn
WithdrawnNCT06025032
a Study in Subjects With Otoferlin Mutation-related Hearing Loss Using RNA Base-eDiting Therapy(SOUND)
An Open-label, Multiple-cohort, Dose-finding, Investigator-initiated Trial to Evaluate the Safety, Tolerability, and Efficacy of HG205 RNA Base-Editing Therapy in Subjects With OTOF-p.Q829X Mutation-associated Hearing Loss
- Status
- Withdrawn
- Phase
- EARLY_Phase 1
- Study type
- Interventional
- Enrollment
- 0 (actual)
- Sponsor
- HuidaGene Therapeutics Co., Ltd. · Industry
- Sex
- All
- Age
- 1 Year – 16 Years
- Healthy volunteers
- Not accepted
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
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | HG205 | The study will enroll up to 2 cohorts, evaluating a starting dose plus a higher or lower dose |
Timeline
- Start date
- 2023-03-30
- Primary completion
- 2025-04-08
- Completion
- 2025-04-08
- First posted
- 2023-09-06
- Last updated
- 2025-04-10
Locations
1 site across 1 country: China
Source: ClinicalTrials.gov record NCT06025032. Inclusion in this directory is not an endorsement.