18 active trials 11 recruiting 5 Phase 3 13 sponsors 1 changes in 30 days
| Phase 1/2 | 2 | |
| Phase 2 | 1 | |
| Phase 3 | 5 |
| NCT ID | Title | Sponsor | Status | Updated |
|---|---|---|---|---|
| Phase 3 | ||||
| NCT06914609 | REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome | Ionis Pharmaceuticals, Inc. | Recruiting | 2026-08-12 |
| NCT07605429 | BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome. | OHB Pediatrics Ltd. | Recruiting | 2026-07-28 |
| NCT07782827 | CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children… | Ionis Pharmaceuticals, Inc. | Not Yet Recruiting | 2026-08-24 |
| NCT06617429 | Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome… | Ultragenyx Pharmaceutical Inc | Active, Not Recruiting | 2026-08-06 |
| NCT06415344 | Long-term Extension of GTX-102 in Angelman Syndrome | Ultragenyx Pharmaceutical Inc | Enrolling by Invitation | 2026-07-27 |
| Phase 2 | ||||
| NCT07157254 | A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type… | Ultragenyx Pharmaceutical Inc | Recruiting | 2026-08-07 |
| Phase 1/2 | ||||
| NCT07181837 | A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome | MavriX Bio, LLC | Recruiting | 2026-07-24 |
| NCT05127226 | HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple… | Ionis Pharmaceuticals, Inc. | Active, Not Recruiting | 2026-08-20 |
| Not applicable | ||||
| NCT06139172 | Web Intervention for Parents of Youth With Genetic Syndromes (WINGS) | Rush University Medical Center | Active, Not Recruiting | 2026-07-13 |
| NCT03836300 | Parent and Infant Inter(X)Action Intervention (PIXI) | RTI International | Enrolling by Invitation | 2026-07-13 |
| Not specified | ||||
| NCT07417137 | A Natural History Study of Angelman Syndrome | Massachusetts General Hospital | Recruiting | 2026-08-21 |
| NCT06737718 | Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman… | Assistance Publique - Hôpitaux de Paris | Recruiting | 2026-03-16 |
| NCT05945576 | IDMet (RaDiCo Cohort) (RaDiCo-IDMet) | Institut National de la Santé Et de la Recherche Médicale, France | Recruiting | 2026-02-12 |
| NCT06229769 | Natural History Study for Patients With Angelman Syndrome | Centre Hospitalier Universitaire de Liege | Recruiting | 2025-05-20 |
| NCT04507997 | Angelman Syndrome Natural History Study | Boston Children's Hospital | Recruiting | 2025-04-03 |
| NCT06353620 | Structural-functional Connectome in Drug-resistant Epilepsies and Neurodevelopmental… | IRCCS Eugenio Medea | Recruiting | 2024-04-09 |
| NCT05293184 | The Global Angelman Syndrome Registry | Foundation for Angelman Syndrome Therapeutics, Australia | Recruiting | 2024-02-23 |
| NCT03655223 | Early Check: Expanded Screening in Newborns | RTI International | Active, Not Recruiting | 2026-07-01 |
Ionis Pharmaceuticals, Inc. (3) · Ultragenyx Pharmaceutical 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)