Clinical Trial

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

Study acronym: ASCEND-AS
Recruiting Phase 1/2
View on ClinicalTrials.gov →
Summary
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.
Protocol Amendment History 2 changes
critical Trial status changed: Active, Not Recruiting → Recruiting 2026-07-25
critical Trial status changed: Recruiting → Active, Not Recruiting 2026-06-19
Trial Details
NCT Number NCT07181837
Lead Sponsor MavriX Bio, LLC
Conditions Angelman Syndrome
Enrollment 12 participants
Start Date 2025-10-29
Primary Completion 2028-03-31 (estimated)
Study Completion 2031-05-31 (estimated)
Updated on ClinicalTrials.gov 2026-07-24