Early Phase 1 Rare Disease Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You have been diagnosed with Snijders Blok-Campeau syndrome
- You have a specific genetic change (mutation) in the CHD3 gene identified as c.3073C>T, p.(Arg1025Trp)
- Your liver, heart, and immune system are all functioning normally
- Your blood clotting and platelet levels are within normal range
Who may not be able to join:
- You have a brain tumor or any growth or mass inside the skull
- You have a medical reason why a lumbar puncture (spinal tap) or injection into the spinal canal cannot be safely performed
- You have ongoing or hard-to-control epileptic seizures
- You have an unstable or active infection, including bacterial, fungal, HIV, hepatitis A, or hepatitis B
- A blood test shows a high level of antibodies against the therapy's delivery vehicle (AAV), above a specific threshold (confirm with trial site)
- You have received immune-system-affecting medications (other than those allowed by the study) within the past 3 months
- You have previously received any gene therapy treatment
- You are currently in another clinical trial or have taken an experimental treatment within the past 30 days (or within 5 half-lives of that treatment)
- You have a known allergy to any of the products used in this trial
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Yongguo Yu, Dr, MD, PhD, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Phone: +86-25-25076466
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Incidence of drug-related serious adverse events
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.