A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
Part of Brain & nervous system, Genetic & congenital clinical trials.
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.
Summary from the official registry record.
Who can take part
- The participant's parent/legal guardian must provide written informed consent.
- Full maternal UBE3A gene deletion causing AS in the region of 15q11.2-q13
- Uniparental disomy
- Imprinting center defect
- The participant must be 18 to 50 years of age, inclusive (for adult participants), or 4 to 8 years of age, inclusive (for pediatric participants), at Screening.
- The participant must have the ability to ambulate independently.
See the full eligibility criteria below for the complete list.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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