This study is testing a gene therapy called MVX-220 for people with Angelman Syndrome. MVX-220 aims to provide a working copy of the UBE3A gene, which is often missing or not working correctly in Angelman Syndrome. The study will look at how safe MVX-220 is and how well it works in people aged 4 to 50 years old who have specific genetic types of Angelman Syndrome (deletion, uniparental disomy, or imprinting center defect). Researchers will monitor for any side effects and changes in your health for up to two years after treatment. The study is currently unclear about its recruitment status and plans to enroll 12 participants.

Study design

This is an interventional study with a planned enrollment of 12 participants, including adults and children. All participants will receive a single dose of the MVX-220 gene therapy.

What's involved

You would receive a single injection of MVX-220 into the cisterna magna (a space near the brain). You would also take steroids before and for a short time during the study, and have regular safety checks like lab tests, ECGs, and physical exams for up to two years.

Compensation

Not stated in the trial record.

Follow-up

Participants will be followed for safety and efficacy for an initial 2-year period after treatment, with a total follow-up duration of 5 years.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.