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 are male and between 2 and 18 years old at the time of signing the consent form
- You have been diagnosed with MECP2 Duplication Syndrome (MDS), confirmed by genetic testing and clinical assessment
- Your seizures have been stable, or you have had no seizures, while on your current treatments (including any anti-seizure medicines and physical therapy) for at least 2 months before the study screening
- You and your family are willing to follow the study plan, including providing biological samples and staying in hospital for a procedure to deliver treatment directly into the brain (confirm with trial site)
- Your blood, chemistry, and urine test results are within acceptable ranges
Who may not be able to join:
- You have a triplication (three copies) of the MECP2 gene, rather than a duplication
- You have another genetic condition in addition to MDS
- A brain MRI scan shows significant shrinkage of the brain or cerebellum, or other notable changes to brain structure
- You have, or have had, high blood pressure, heart muscle disease, reduced blood flow to the heart, irregular heart rhythm, or any other significant heart or cardiovascular condition
- You have had brain or nervous system surgery within the 6 months before joining the study
- You have taken medicines that suppress the immune system within the 3 months before joining the study
- You have previously received gene therapy or a type of treatment called oligonucleotide therapy
- There is any other reason the study doctor believes you would not be able to complete the study safely or successfully (confirm with trial site)
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
Phone: 732-318-9873
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 and severity of systemic adverse events
Can't join this trial?
Data last synced from ClinicalTrials.gov: 27 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.