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

Registry ID

MVX-AAV-101

Source registry

HEALTH_CANADA

Status

Pending

Phase

PHASE1

Study type

INTERVENTIONAL

Sponsor

MAVRIX BIO, LLC

Last update

2025-10-29

Conditions

• ANGELMAN SYNDROME (AS)

Related clinical trials

• PHASE 3 STUDY OF THE EFFICACY AND SAFETY OF ION582 IN CHILDREN AND ADULTS WITH ANGELMAN SYNDROME
• A PHASE 3, RANDOMIZED, DOUBLE-BLIND, SHAM-CONTROLLED STUDY INVESTIGATING THE EFFICACY AND SAFETY OF GTX-102 IN PEDIATRIC SUBJECTS WITH ANGELMAN SYNDROME
• A LONG-TERM EXTENSION TRIAL INVESTIGATING THE SAFETY AND EFFICACY OF GTX-102 IN PATIENTS WITH ANGELMAN SYNDROME

View on source registry