26 completed, 10 recruiting, 7 active not recruiting, 4 unknown. 10 Phase 2, 8 Phase 1, 8 Phase 3, 6 Not applicable, 1 Early Phase 1.
Follow Angelman Syndrome: new trials by email Literature Review of Angelman Syndrome trials Research Report on Angelman Syndrome
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS) obudanersen - Angelman Syndrome | Phase 3 | Recruiting | Ionis Pharmaceuticals, Inc. | 30 | 2026-09-30 | 2026-10-20 |
| A Natural History Study of Angelman Syndrome Angelman Syndrome, Neurodevelopmental Conditions | Recruiting | Massachusetts General Hospital | 40 | 2026-09-09 | 2026-10-13 | |
| BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome. rugonersen, Sham procedure - Angelman Syndrome | Phase 3 | Recruiting | OHB Pediatrics Ltd. | 165 | 2026-06-23 | 2026-10-20 |
| A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome MVX-220 - Angelman Syndrome | Phase 1 / Phase 2 | Recruiting | MavriX Bio, LLC | 12 | 2025-10-29 | 2026-07-24 |
| A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) No intervention, GTX-102 - Angelman Syndrome | Phase 2 | Active not recruiting | Ultragenyx Pharmaceutical Inc | 60 | 2025-10-13 | 2026-09-09 |
| REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome obudanersen, Placebo - Angelman Syndrome | Phase 3 | Active not recruiting | Ionis Pharmaceuticals, Inc. | 156 | 2025-06-10 | 2026-09-29 |
| Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman Syndrome Eye tracking, Data collection from patients' medical files, questionnaires - Angelman Syndrome | Recruiting | Assistance Publique - Hôpitaux de Paris | 60 | 2025-02-25 | 2026-03-16 | |
| Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) GTX-102, Sham-LP - Angelman Syndrome | Phase 3 | Active not recruiting | Ultragenyx Pharmaceutical Inc | 129 | 2024-12-03 | 2026-08-06 |
| Long-term Extension of GTX-102 in Angelman Syndrome GTX-102 - Angelman Syndrome | Phase 3 | Active not recruiting | Ultragenyx Pharmaceutical Inc | 255 | 2024-07-31 | 2026-09-09 |
| Structural-functional Connectome in Drug-resistant Epilepsies and Neurodevelopmental Syndromes With Epilepsy High Density EEG recording, Rey Complex Figure Test - Epilepsy, Angelman Syndrome | Recruiting | IRCCS Eugenio Medea | 120 | 2024-02-13 | 2024-04-09 | |
| Angelman Natural History Study - FAST Spain Non intervention, Observational study - Angelman Syndrome | Unknown | Puerta de Hierro University Hospital | 40 | 2023-12-01 | 2024-04-03 | |
| Web Intervention for Parents of Youth With Genetic Syndromes (WINGS) Functional Behavioral Training (FBT), Positive Parenting Strategies-Treatment as Usual - Telomeric 22Q13 Monosomy Syndrome, Tuberous Sclerosis | Not applicable | Active not recruiting | Rush University Medical Center | 79 | 2023-09-15 | 2026-09-18 |
| A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype. Alogabat - Angelman Syndrome | Phase 2 | Completed | Hoffmann-La Roche | 48 | 2023-07-27 | 2025-12-04 |
| Development of a Newborn Screening Assay for Angelman Syndrome and Prader-Willi Syndrome Newborn Screening Assay - Angelman Syndrome, Prader-Willi Syndrome | Completed | University of Wisconsin, Madison | 11 | 2023-04-20 | 2023-09-06 | |
| An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome NNZ-2591 - Angelman Syndrome | Phase 2 | Completed | Neuren Pharmaceuticals Limited | 17 | 2022-07-12 | 2025-01-31 |
| Study of the Prevalence of Autistic Traits in Angelman Syndrome Angelman Syndrome | Unknown | IRCCS Eugenio Medea | 46 | 2022-03-15 | 2024-04-18 | |
| HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome ION582 - Angelman Syndrome | Phase 1 / Phase 2 | Active not recruiting | Ionis Pharmaceuticals, Inc. | 71 | 2021-12-22 | 2026-08-20 |
| Angelman Syndrome Natural History Study-FAST UK Natural History Study - Angelman Syndrome | Unknown | University of Oxford | 40 | 2021-11-01 | 2024-02-28 | |
| Natural History Study for Patients With Angelman Syndrome Angelman Syndrome | Recruiting | Centre Hospitalier Universitaire de Liege | 30 | 2021-10-10 | 2025-05-20 | |
| Angelman Syndrome Video Assessment (ASVA) Source Material Study Angelman Syndrome | Completed | The Emmes Company, LLC | 73 | 2021-08-31 | 2023-12-13 |
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Prader-Willi Syndrome (8) · Fragile X Syndrome (5) · Rett Syndrome (5) · Smith-Magenis Syndrome (5) · Tuberous Sclerosis (4) · Autism Spectrum Disorder (3) · DiGeorge Syndrome (3) · Epilepsy (3) · Parasomnias (3) · Rare Diseases (3) · Beckwith-Wiedemann Syndrome (2) · Creatine deficiency, X-linked (2) · Developmental Disabilities (2) · Down Syndrome (2)
Abnormalities, Multiple (51) · Central Nervous System Diseases (51) · Chromosome Disorders (51) · Congenital Abnormalities (51) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (51) · Imprinting Disorders (51) · Movement Disorders (51) · Genetic Diseases, Inborn (50) · Nervous System Diseases (50) · Neurobehavioral Manifestations (12) · Neurologic Manifestations (12) · Intellectual Disability (11) · Nutritional and Metabolic Diseases (10) · Heredodegenerative Disorders, Nervous System (9) · Mental Disorders (8)
Levodopa (3) · carbidopa, levodopa drug combination (3) · gaboxadol (3) · Melatonin (2) · Minocycline (2) · Betaine (1) · Blood Specimen Collection (1) · Carbidopa (1) · Creatine (1) · Observation (1) · Spinal Puncture (1) · Surveys and Questionnaires (1) · Tetracycline (1) · Vitamin B 12 (1) · alpha-tocotrienol quinone (1)
Ultragenyx Pharmaceutical Inc (4) · Boston Children's Hospital (3) · Hoffmann-La Roche (3) · Ionis Pharmaceuticals, Inc. (3) · Assistance Publique - Hôpitaux de Paris (2) · Healx AI (2) · IRCCS Eugenio Medea (2) · Neurim Pharmaceuticals Ltd. (2) · Puerta de Hierro University Hospital (2) · RTI International (2) · Baylor College of Medicine (1) · Bennett Lavenstein (1) · Biogen (1) · Centre Hospitalier Universitaire de Liege (1) · Edison Pharmaceuticals Inc (1)
United States (32) · France (10) · Spain (9) · Australia (8) · Italy (8) · United Kingdom (8) · Israel (7) · Canada (5) · Germany (5) · Netherlands (4) · Japan (2) · Poland (2) · Argentina (1) · Belgium (1) · Brazil (1)
Wen-Hann Tan, BMBS (5) · Carlos A. Bacino, MD (3) · Steven A. Skinner, MD (3) · Lynne M. Bird, MD (2) · Alan Percy, MD (1) · Amit Rakhit, MD, MBA (1) · Anne Slavotinek, MD (1) · Anne Wheeler, PhD (1) · Arthur L. Beaudet, MD (1) · Bennett Lavenstein, MD (1)
PMID 10417280, PMID 12820840, PMID 16814942, PMID 20375405, PMID 21310373, PMID 21519036, PMID 21827860, PMID 22002941, PMID 22005709, PMID 22281937, PMID 22464072, PMID 22617594, PMID 22742782, PMID 23215555, PMID 23613152, PMID 23712453, PMID 24135152, PMID 24388429, PMID 24785862, PMID 25004354, PMID 25071871, PMID 25111587, PMID 25283752, PMID 25479548, PMID 25491305, PMID 29096777, PMID 30126448, PMID 30826071, PMID 31315600, PMID 31640736, PMID 31918761, PMID 31982581, PMID 32792659, PMID 33395098, PMID 33443117, PMID 34510212, PMID 35033576, PMID 35085538, PMID 35142618, PMID 35797778, PMID 36052644, PMID 36522453, PMID 36947593, PMID 36965866, PMID 38479041, PMID 38879552, PMID 39222563, PMID 40646322, PMID 9647154
2019 (1), U54RR019478 (1)