Clinical Trial of the Dual Vector Base Editor for the Treatment of the CHD3-R1025W Mutation
Recruiting
Early Phase 1
Interventional Study
Developmental Delay Disorder
Intellectual Disability
Rare Diseases
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 2 – 10
- Sex
- Any
- Trial phase
- Early Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 1 (estimated)
- Sponsor
- Yongguo Yu · Other
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About This Trial
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
Trial Locations
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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 le…
Contacts