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

NCT06860672 · Status: RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2025-03-06

No results posted yet for this study

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

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.

Sponsors & Collaborators

  • Yongguo Yu

    lead OTHER

Principal Investigators

  • Yongguo Yu, Dr, MD, PhD · Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

  • Zilong Qiu, PhD · Shanghai Jiao Tong University School of Medicine Songjiang Research Institute

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
10 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-02-19
Primary Completion
2025-04-30
Completion
2025-09-30

Countries

  • China

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06860672 on ClinicalTrials.gov