Tundra Space

Tundra Space

Clinical Research Directory

Browse clinical research sites, groups, and studies.

Back to Studies
RECRUITING
NCT07181837
PHASE1/PHASE2

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

Sponsor: MavriX Bio, LLC

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.

Official title: A Multi-Center, Open-label, Phase 1/2 Trial of the Safety and Efficacy of MVX-220 Gene Therapy Administered by Intra-Cisterna Magna Injection to Participants With Angelman Syndrome

Key Details

Gender

All

Age Range

4 Years - 50 Years

Study Type

INTERVENTIONAL

Enrollment

12

Start Date

2025-10-29

Completion Date

2031-05-31

Last Updated

2026-03-16

Healthy Volunteers

No

Interventions

GENETIC

MVX-220

AAVhu68 viral vector

Locations (3)

Cedars-Sinai Medical Center

Los Angeles, California, United States

Rush University Medical Center

Chicago, Illinois, United States

Boston Children's Hospital

Boston, Massachusetts, United States