A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome (NCT07181837) | Clinical Trial Compass
RecruitingPhase 1/2
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
United States12 participantsStarted 2025-10-29
Plain-language 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.
Who can participate
Age range4 Years – 50 Years
SexALL
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Inclusion criteria
✓. The participant's parent/legal guardian must provide written informed consent.
✓. Symptoms consistent with AS and documented genetic confirmation of one of the following genotypes resulting in a diagnosis of AS:
✓. 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.
✓. The participant must be on stable antiepileptic medications (with no changes within 1 month prior to the Screening visit, except for weight associated dose adjustments).
Exclusion criteria
✕. Clinically significant medical finding other than AS, that, in the judgment of the Investigator would make the participant unsuitable for participation.
✕. Laboratory abnormalities including but not limited to:
✕. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> upper limit of normal (ULN)
✕. Total and/or fractionated bilirubin (direct and/or indirect) \> ULN
What they're measuring
1
Incidence of Adverse Events, Serious Adverse Events, and Adverse Events of Special Interest as assessed through clinical safety, laboratory tests, ECG, vital sign measurements, and physical examinations