A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome

Recruiting now · Phase 1/Phase 2

Conditions studied: Angelman Syndrome

In brief

The purpose of this study is to evaluate the safety and efficacy of MVX-220 gene therapy in children and adults with Angelman syndrome with UBE3A gene deletion, uniparental disomy, or imprinting center defect genotypes.

Key facts

Study ID

NCT07181837

Run by

MavriX Bio, LLC

People needed

12

Starts

2025-10-29

Expected to finish

2031-05-31

Last updated by the study team

2026-07-24

Who can join

Age: 4 and older, up to 50. Sex: any. Healthy volunteers: not accepted.

Where it is running

• Cedars-Sinai Medical Center — Los Angeles, California, United States (enrolling)
• Rush University Medical Center — Chicago, Illinois, United States (enrolling)
• Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)

Full record on ClinicalTrials.gov

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