Browse recruiting and upcoming clinical trials for Angelman syndrome.
Patient-friendly, AI-assisted summaries based on official ClinicalTrials.gov ↗ records. Last updated: Oct 10, 2026
About this list Hide ClinicalTrials.gov is the complete and most up-to-date source. Trials may be missing, and trial information may have changed since our last check. Always verify important information in the official registry.
Sex All Female (includes ALL) Male (includes ALL) All sexes only
Age All ages Pediatric (under 18) Adult (18+)
Clear Who can join AI People with Angelman syndrome due to paternal uniparental disomy (UPD) or imprinting defects.
What is being studied AI Tests obudanersen (ION582) by spinal injection for expressive communication in Angelman syndrome due to UPD/imprinting defects
Study type Treatment (drug) · Phase 3
Who can join AI People aged 1–50 years with Angelman syndrome and a confirmed UBE3A mutation or maternally inherited 15q11–q13 deletion.
What is being studied AI Tests intrathecal rugonersen versus sham on development, EEG measures, and safety in Angelman syndrome.
Study type Treatment (drug) · Phase 3
Who can join AI People with Angelman syndrome.
What is being studied AI Tests the safety and effects of MVX-220 gene therapy delivered by cisterna magna injection.
Study type Treatment (drug) · Phase 1/2
Who can join AI Infants (15 months or younger) with a confirmed diagnosis of one of several rare neurogenetic conditions and their parent/primary caregiver (English-speaking household).
What is being studied AI Tests a parent-focused early intervention (PIXI) for infants diagnosed with rare neurogenetic disorders.
Study type Treatment (other)
Who can join AI People aged 1 year and older with a genetically confirmed diagnosis of Angelman syndrome.
What is being studied AI Tracks development, behavior, sleep, and brain activity over one year using tests, EEGs, and actigraphy.
Who can join AI Children aged 3–17 with Angelman syndrome, and healthy control children without neurological/genetic/psychiatric conditions.
What is being studied AI Uses eye tracking to measure social perception abnormalities in children with Angelman syndrome.
Who can join AI People with epilepsy, Angelman syndrome, or Dup15q syndrome.
What is being studied AI Uses high-density EEG and MRI data to study brain network organization in epilepsy and syndromes
Who can join AI People with genetically confirmed Angelman syndrome whose carer can give consent (ages 0–99, any sex).
What is being studied AI Collects clinical, developmental and movement-monitoring data (ActiMyo®).
Who can join AI Children and people with imprinting disorders (examples: Silver‑Russell, Beckwith‑Wiedemann, Angelman, Prader‑Willi, and others).
What is being studied AI Collects clinical, genetic, metabolic and morphometric data to describe natural history and metabolic profiles.
Who can join AI People with Angelman syndrome who have a molecular (genetic) diagnosis.
What is being studied AI Collects clinical history, developmental and communication measures over time using clinician and parent assessments.
Countries Canada, United States
Who can join AI People of any age who have a diagnosis of Angelman Syndrome.
What is being studied AI Collects ongoing health, diagnosis, and caregiver-reported data about people with Angelman Syndrome.