| Recruiting | A Natural History Study of Angelman Syndrome NCT07417137 | Massachusetts General Hospital | — |
| Recruiting | A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome NCT07181837 | MavriX Bio, LLC | Phase 1 / Phase 2 |
| Recruiting | A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) NCT07157254 | Ultragenyx Pharmaceutical Inc | Phase 2 |
| Recruiting | REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome NCT06914609 | Ionis Pharmaceuticals, Inc. | Phase 3 |
| Recruiting | Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman Syndrome NCT06737718 | Assistance Publique - Hôpitaux de Paris | — |
| Active Not Recruiting | Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) NCT06617429 | Ultragenyx Pharmaceutical Inc | Phase 3 |
| Enrolling By Invitation | Long-term Extension of GTX-102 in Angelman Syndrome NCT06415344 | Ultragenyx Pharmaceutical Inc | Phase 3 |
| Recruiting | Structural-functional Connectome in Drug-resistant Epilepsies and Neurodevelopmental Syndromes With Epilepsy NCT06353620 | IRCCS Eugenio Medea | — |
| Recruiting | Angelman Natural History Study - FAST Spain NCT06115109 | Puerta de Hierro University Hospital | — |
| Recruiting | Web Intervention for Parents of Youth With Genetic Syndromes (WINGS) NCT06139172 | Rush University Medical Center | N/A |
| Completed | A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in C NCT05630066 | Hoffmann-La Roche | Phase 2 |
| Completed | Development of a Newborn Screening Assay for Angelman Syndrome and Prader-Willi Syndrome NCT05783791 | University of Wisconsin, Madison | — |
| Completed | An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome NCT05011851 | Neuren Pharmaceuticals Limited | Phase 2 |
| Recruiting | Study of the Prevalence of Autistic Traits in Angelman Syndrome NCT06337383 | IRCCS Eugenio Medea | — |
| Recruiting | HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION5 NCT05127226 | Ionis Pharmaceuticals, Inc. | Phase 1 / Phase 2 |
| Unknown | Angelman Syndrome Natural History Study-FAST UK NCT05100810 | University of Oxford | — |
| Recruiting | Natural History Study for Patients With Angelman Syndrome NCT06229769 | Centre Hospitalier Universitaire de Liege | — |
| Completed | Angelman Syndrome Video Assessment (ASVA) Source Material Study NCT05637697 | The Emmes Company, LLC | — |
| Unknown | Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and/or Overw NCT04768803 | University Hospital, Toulouse | — |
| Completed | A Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses O NCT04863794 | Hoffmann-La Roche | Phase 1 |
| Completed | A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 NCT04428281 | Hoffmann-La Roche | Phase 1 |
| Completed | A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome NCT04259281 | Ultragenyx Pharmaceutical Inc | Phase 1 / Phase 2 |
| Completed | Angelman Syndrome (AS) Biomarker Study NCT04103333 | Biogen | EARLY_Phase 1 |
| Terminated | An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With NCT03882918 | Healx AI | Phase 3 |
| Enrolling By Invitation | Parent and Infant Inter(X)Action Intervention (PIXI) NCT03836300 | RTI International | N/A |
| Completed | Nutritional Formulation for Angelman Syndrome NCT03644693 | University of Colorado, Denver | N/A |
| Enrolling By Invitation | Early Check: Expanded Screening in Newborns NCT03655223 | RTI International | — |
| Recruiting | Angelman Syndrome Natural History Study NCT04507997 | Boston Children's Hospital | — |
| Completed | Italian Angelman Syndrome Registry NCT03650569 | FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS | — |
| Completed | Study on the Brain Network of Angelman Syndrome NCT03358823 | Fudan University | — |
| Completed | Single Dose Pharmacokinetic (PK) Study NCT03109756 | Healx AI | Phase 1 |
| Recruiting | IDMet (RaDiCo Cohort) (RaDiCo-IDMet) NCT05945576 | Institut National de la Santé Et de la Recherche Médicale, France | — |
| Recruiting | The Global Angelman Syndrome Registry NCT05293184 | Foundation for Angelman Syndrome Therapeutics, Australia | — |
| Completed | A Study in Adults and Adolescents With Angelman Syndrome (STARS) NCT02996305 | Healx AI | Phase 2 |
| Completed | SNP-based Microdeletion and Aneuploidy RegisTry (SMART) NCT02381457 | Natera, Inc. | — |
| Completed | Study to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome NCT02056665 | Puerta de Hierro University Hospital | Phase 2 |
| Completed | Clinical Trial of Levodopa/Carbidopa ( Sinemet) Therapy in Angel Man Syndrome NCT03235037 | Bennett Lavenstein | N/A |
| Completed | Minocycline in the Treatment of Angelman Syndrome NCT01531582 | University of South Florida | N/A |
| Completed | Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW NCT02670694 | Baylor College of Medicine | — |
| Completed | A Trial of Levodopa in Angelman Syndrome NCT01281475 | Wen-Hann Tan | Phase 2 / Phase 3 |
| Completed | Study on Tolerability of Levodopa/Carbidopa in Children With Angelman Syndrome NCT00829439 | Boston Children's Hospital | Phase 1 |
| Completed | Dietary Supplements for the Treatment of Angelman Syndrome NCT00348933 | University of California, San Diego | N/A |
| Completed | Characterization of Angelman Syndrome NCT00296764 | Boston Children's Hospital | — |
| Completed | Study of Phenotype and Genotype Correlations in Patients With Contiguous Gene Deletion Syndromes NCT00004351 | National Institute of Neurological Disorders and Stroke (NINDS) | — |
| Available | Expanded Access/Compassionate Use of Rugonersen in Patients With Angelman Syndrome NCT07136454 | Oak Hill Bio Ltd | — |