Evaluating a new CRISPR therapy for MECP2 Duplication Syndrome

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial aims to assess the safety, tolerability, and efficacy of HG204, a novel CRISPR RNA-editing therapy designed to target and reduce MECP2 mRNA in the brain. The therapy utilizes high-fidelity Cas13Y technology delivered via a single adeno-associated virus vector, with the goal of alleviating symptoms associated with MECP2 Duplication Syndrome, a severe neurodevelopmental disorder. Preclinical studies have shown promising results in mouse models, including improved motor function and prolonged survival. Participants will undergo an intracerebroventricular injection of the therapy, and their progress will be monitored closely.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Males ≥ 2 and ≤18 years at the time of signing informed consent;
* Genetic test and clinical confirmed diagnosis of MDS;
* Stable pattern of seizures, or has had no seizures while currently receiving medical treatment (including antiepileptics) and physical therapy are stable for at least 2 months before screening;
* Willing to adhere to protocol, including biological samples collection and hospitalization for intracerebroventricular injection surgery;
* Acceptable hematology, clinical chemistry, and urine laboratory parameters.

Exclusion Criteria:

* MECP2 gene triplication;
* Concurrent genetic syndromes other than MDS;
* Significant brain or cerebellar atrophy, or other significant degenerative changes as shown in cranial MRI at screening;
* Prior or current hypertension, cardiomyopathy, myocardial ischemia or atrial fibrillation and other cardiovascular diseases;
* Prior central nervous system surgery within 6 months before enrolment;
* Systemic use of immunosuppressive drugs within 3 months before enrolment;
* Prior gene therapy or oligonucleotide therapy treatments;
* Any other conditions that would not allow the potential subject to complete follow-up examinations during the study and would, in the opinion of the investigator, make the potential subject unsuitable for the study.

Where it's running

• Peking University First Hospital
  Beijing, Beijing Municipality, China Recruiting

Study contacts

• Study coordinator: Study Director
• Email: HG20401@huidagene.com
• Phone: 732-318-9873

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.