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NCT07181837: ASCEND-AS

A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome

Recruiting now Phase 1, PHASE2 Last updated 16 March 2026
What this trial tests

Phase 1, PHASE2 trial testing MVX-220 in Angelman Syndrome in 12 participants. Currently enrolling.

Timeline
29 October 2025
Primary endpoint
31 March 2028
31 May 2031

Quick facts

Lead sponsorMavriX Bio, LLC
PhasePhase 1, PHASE2
StatusRecruiting now
Study typeINTERVENTIONAL
Allocationnon randomized
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment12
Start date29 October 2025
Primary completion31 March 2028
Estimated completion31 May 2031
Sites3 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

MavriX Bio, LLC — full company profile →

Who can join

Adults 4 to 50, any sex, with Angelman Syndrome. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

  1. Crossing the finish line towards a disease-modifying treatment for Angelman syndrome.
    Judson MC, de Almeida LP, Burdine RD, Chamberlain SJ, et al · · 2026 · PMID 41795090 · DOI 10.1186/s11689-026-09681-5

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Other recruiting trials for Angelman Syndrome

Currently open trials in the same condition.

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Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT07181837.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing