Actively Recruiting
Clinical Trial of the Dual Vector Base Editor for the Treatment of the CHD3-R1025W Mutation
Led by Yongguo Yu · Updated on 2025-03-06
1
Participants Needed
1
Research Sites
27 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
Clinical Trial of the Dual Vector Base Editor for the Treatment of the CHD3-R1025W Mutation
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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
You will not qualify if you...
- Brain tumor or intracranial space-occupying lesion
- Contraindications to lumbar puncture or sheath injection
- Persistent status epilepticus or unstable epilepsy control
- Unstable systemic disease including active bacterial, fungal, HIV, hepatitis A or B infections
- Serum anti-AAV neutralizing antibody titer >1:50
- Treatment with immunological agents other than specified prophylaxis within 3 months
- Prior gene therapy
- Participation in another clinical trial or investigational treatment within 30 days or 5 half-lives
- Known allergy to any investigational product
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Xinhua Hospital affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China, 200092
Actively Recruiting
Research Team
X
Xiaomei Luo, Ms., Master
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here