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

NCTID	NCT06860672 (View at clinicaltrials.gov)
Description	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 (Show More)
Development Status 🔄	Active
Indication 🔄	Snijders Blok-Campeau syndrome
Disease Ontology Term 🔄	DOID:0070668
Compound Name 🔄	Dual Vector AAV-CHD3-R1025W Base Editor
Sponsor	Yongguo Yu
Funder Type	Other
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	1 (ESTIMATED)
Results Posted	Not Available

Eligible Age	2 Years - 10 Years
Standard Ages	Child
Sexes Eligible for Study	ALL
Eligibility Criteria	Inclusion Criteria: * Clinical diagnosis of Snijders Blok-Campeau syndrome * Heterozygous mutation of c.3073C\>T, p.(Arg1025Trp) in the CHD3 gene * Normal liver, heart and immune function * Normal coagulation and platelet counts Exclusion Criteria: * Brain tumor or intracranial space-occupying lesion * Contraindications to administration of lumbar puncture or sheath injection administration * Persistent status epilepticus or recurrent epileptic control instability * Presence of unstable systemic disease including active bacterial, fungal or HIV, hepatitis A, hepatitis B infection * Serum anti-AAV neutralizing antibody titer \>1:50 (ELISA immunoassay) * Treatment with immunological agents other than protocol-specified prophylaxis within 3 months * Prior gene therapy * Participation in another clinical trial, or treatment with another investigational product within 30 days or 5 half-lives * Known allergy to any investigational product View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites	1
Locations	China