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New treatment option for Developmental Delay Disorder
Official title Clinical Trial of the Dual Vector Base Editor for the Treatment of the CHD3-R1025W Mutation
ClinicalTrials.gov ID: NCT06860672
What this study is testing
What is Dual vector DNA base editor?
Dual vector DNA base editor is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for developmental delay disorder.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 10
You may be able to join if
- 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 likely can't join if
- 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
See the full eligibility 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
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Shanghai, Shanghai Municipality, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 10 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shanghai, Shanghai Municipality, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.