Angelman Syndrome
45
14
15
23
Key Insights
Highlights
Success Rate
96% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
2.2%
1 terminated out of 45 trials
95.8%
+9.3% vs benchmark
9%
4 trials in Phase 3/4
26%
6 of 23 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 23 completed trials
Clinical Trials (45)
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
Long-term Extension of GTX-102 in Angelman Syndrome
REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
A Natural History Study of Angelman Syndrome
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman Syndrome
Italian Angelman Syndrome Registry
IDMet (RaDiCo Cohort) (RaDiCo-IDMet)
Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)
A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome
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.
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)
Expanded Access/Compassionate Use of Rugonersen in Patients With Angelman Syndrome
A Study in Adults and Adolescents With Angelman Syndrome (STARS)
Parent and Infant Inter(X)Action Intervention (PIXI)
An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome
Natural History Study for Patients With Angelman Syndrome
Early Check: Expanded Screening in Newborns