Angelman Syndrome clinical trials
Data updated daily from ClinicalTrials.gov, last refresh 2026-11-11
What changed in the last 30 days
4 trials changed in the last 30 days: 3 stopped, 0 with notable changes, 1 with a minor change only.
- Minor: BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome Trial sites expanded: 6 → 10 locations
- Stopped: GTX-102 in Angelman Syndrome Trial terminated
- Stopped: Aspire · GTX-102 · Angelman Syndrome (AS) Trial terminated
- Stopped: Aurora · GTX-102 · Deletion- or Nondeletion-type Angelman Syndrome (AS) Trial terminated
- Stopped: terminated, suspended or withdrawn
- Notable: the plan changed
- Minor: routine updates
Active trials by phase
| Phase 1/2 | 2 | |
| Phase 3 | 3 |
Not charted: 10 active trials with no phase listed on ClinicalTrials.gov (typically behavioural, device, procedure and observational studies).
Top sponsors by active trials in this area
Ionis Pharmaceuticals, Inc. (3) · RTI International (2) · Assistance Publique - Hôpitaux de Paris (1) · Boston Children's Hospital (1) · Centre Hospitalier Universitaire de Liege (1) · Foundation for Angelman Syndrome Therapeutics, Australia (1) · Institut National de la Santé Et de la Recherche Médicale, France (1) · IRCCS Eugenio Medea (1) · Massachusetts General Hospital (1) · MavriX Bio, LLC (1)
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