Status: Recruiting
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
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.
Participants needed: 12
Trial details
Phase: Phase 1, Phase 2Age: 4-50Biological sex: AllType: InterventionalSponsor: MavriX Bio, LLCUpdated: Jul 24, 2026Locations: 3
Eligibility criteria
The participant's parent/legal guardian must provide written informed consent. [+6]
Clinically significant medical finding other than AS, that, in the judgment of t... [+19]