3 terminated, 2 completed, 2 recruiting, 1 active not recruiting. 8 Phase 3, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 8 | Terminated 3, Completed 2, Recruiting 2, Active not recruiting 1 |
| Phase 2 | 1 | Completed 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 |
| 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 |
| 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 |
| Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) GTX-102, Sham-LP - Angelman Syndrome | Phase 3 | Terminated | Ultragenyx Pharmaceutical Inc | 129 | 2024-12-03 | 2026-11-03 |
| Long-term Extension of GTX-102 in Angelman Syndrome GTX-102 - Angelman Syndrome | Phase 3 | Terminated | Ultragenyx Pharmaceutical Inc | 66 | 2024-07-31 | 2026-11-03 |
| An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome OV101 - Angelman Syndrome | Phase 3 | Terminated | Healx AI | 141 | 2019-01-31 | 2025-07-18 |
| Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities Circadin 2/5/10 mg, Placebo - Sleep Disorders | Phase 3 | Completed | Neurim Pharmaceuticals Ltd. | 125 | 2013-10-01 | 2024-04-23 |
| A Trial of Levodopa in Angelman Syndrome Levodopa, Placebo Oral Capsule - Angelman Syndrome | Phase 2 / Phase 3 | Completed | Wen-Hann Tan | 67 | 2011-01-01 | 2020-07-15 |
Autism Spectrum Disorder (1) · Parasomnias (1) · Sleep Wake Disorders (1) · Smith-Magenis Syndrome (1) · Tuberous Sclerosis (1)
Abnormalities, Multiple (8) · Central Nervous System Diseases (8) · Chromosome Disorders (8) · Congenital Abnormalities (8) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8) · Genetic Diseases, Inborn (8) · Imprinting Disorders (8) · Movement Disorders (8) · Nervous System Diseases (8) · Child Development Disorders, Pervasive (1) · Chronobiology Disorders (1) · Hamartoma (1) · Heredodegenerative Disorders, Nervous System (1) · Malformations of Cortical Development (1) · Malformations of Cortical Development, Group I (1)
Levodopa (1) · Melatonin (1) · carbidopa, levodopa drug combination (1) · gaboxadol (1)
Ionis Pharmaceuticals, Inc. (2) · Ultragenyx Pharmaceutical Inc (2) · Healx AI (1) · Neurim Pharmaceuticals Ltd. (1) · OHB Pediatrics Ltd. (1) · Wen-Hann Tan (1)
United States (8) · Canada (3) · Germany (3) · Israel (3) · Spain (3) · United Kingdom (3) · Australia (2) · France (2) · Japan (2) · Poland (2) · Finland (1) · Italy (1) · Netherlands (1) · Singapore (1) · South Korea (1)
Anne Slavotinek, MD (1) · Brenda Vincenzi, MD (1) · Carlos A. Bacino, MD (1) · Cary Fu, MD (1) · Logan Wink, M.D (1) · Lynne M. Bird, MD (1) · Paul Gringras, PhD (1) · Robert Findling, MD (1) · Steven A. Skinner, MD (1) · Wen-Hann Tan, BMBS (1)