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

NCT07181837 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-03-16

No results posted yet for this study

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.

Conditions

Interventions

GENETIC

MVX-220

AAVhu68 viral vector

Sponsors & Collaborators

  • MavriX Bio, LLC

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-10-29
Primary Completion
2028-03-31
Completion
2031-05-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07181837 on ClinicalTrials.gov