A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
ConditionAngelman Syndrome
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age4-50
SponsorMavriX Bio, LLC
About this trial
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.
Eligibility criteria
Qualifiers
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
Disqualifiers
Clinically significant medical finding other than AS, that, in the judgment of the Investigator would make the participant unsuitable for participation.
Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > upper limit of normal (ULN)
Total and/or fractionated bilirubin (direct and/or indirect) > ULN
Gamma-glutamyl transferase (GGT) > ULN
Trial design
Treatments tested in this trial
- MVX-220
Treatment groups
12 Participants
are divided into 3 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov