NCT06860672

Clinical Trial of the Dual Vector Base Editor for the Treatment of the CHD3-R1025W Mutation

Safety, Tolerability and Preliminary Efficacy Study of a Single Intrathecal Injection of the Dual Vector AAV-CHD3-R1025W Base Editor for the Treatment of Developmental Disorders Caused by the R1025W Mutation in the CHD3 Gene

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 1 (estimated)
• Sponsor: Yongguo Yu · Academic / Other
• Sex: All
• Age: 2 Years – 10 Years
• Healthy volunteers: Not accepted

Summary

To evaluate the safety, tolerability and preliminary efficacy study of a single intrathecal injection of the dual vector AAV-CHD3-R1025W base editor for the treatment of developmental disorders caused by the R1025W mutation in the CHD3 gene

Conditions

• Developmental Delay Disorder
• Intellectual Disability
• Rare Diseases

Interventions

Type	Name	Description
GENETIC	Dual vector DNA base editor	The base editor is delivered using a dual vector adeno-associated virus (AAV) system and introduced into the child via intrathecal injection to correct the mutated CHD3 gene. The vital signs of the child will be closely monitored during treatment to assess possible acute adverse effects. The child will be followed up regularly after treatment to monitor the success of gene editing and the neurodevelopmental improvement of the child. Possible long-term adverse events will be closely monitored to assess the safety of the treatment.

Timeline

• Start date: 2025-02-19
• Primary completion: 2025-04-01
• Completion: 2025-09-01
• First posted: 2025-03-06
• Last updated: 2025-03-06

Locations

1 site across 1 country: China