Key Insights

Highlights

Success Rate

96% trial completion (above average)

Clinical Risk Assessment

Based on trial outcomes

Moderate Risk

Score: 40/100

Termination Rate

2.2%

1 terminated out of 46 trials

Success Rate

95.8%

+9.3% vs benchmark

Late-Stage Pipeline

11%

5 trials in Phase 3/4

Results Transparency

26%

6 of 23 completed with results

Key Signals

6 with results96% success

Data Visualizations

Phase Distribution

25Total
Not Applicable (6)
Early P 1 (1)
P 1 (7)
P 2 (6)
P 3 (5)

Trial Status

Completed23
Recruiting14
Active Not Recruiting3
Unknown3
Enrolling By Invitation2
Terminated1

Trial Success Rate

95.8%

Benchmark: 86.5%

Based on 23 completed trials

Clinical Trials (46)

Showing 20 of 20 trials
NCT06914609Phase 3RecruitingPrimary

REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome

NCT07605429Phase 3RecruitingPrimary

BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome.

NCT07157254Phase 2RecruitingPrimary

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

NCT06415344Phase 3Enrolling By InvitationPrimary

Long-term Extension of GTX-102 in Angelman Syndrome

NCT07181837Phase 1RecruitingPrimary

A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome

NCT03836300Not ApplicableEnrolling By Invitation

Parent and Infant Inter(X)Action Intervention (PIXI)

NCT06139172Not ApplicableActive Not Recruiting

Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)

NCT03655223Active Not Recruiting

Early Check: Expanded Screening in Newborns

NCT07417137RecruitingPrimary

A Natural History Study of Angelman Syndrome

NCT06617429Phase 3Active Not RecruitingPrimary

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

NCT06737718RecruitingPrimary

Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman Syndrome

NCT03650569CompletedPrimary

Italian Angelman Syndrome Registry

NCT05945576Recruiting

IDMet (RaDiCo Cohort) (RaDiCo-IDMet)

NCT04259281Phase 1CompletedPrimary

A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

NCT05630066Phase 2CompletedPrimary

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.

NCT05127226Phase 1RecruitingPrimary

HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome

NCT04428281Phase 1CompletedPrimary

A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)

NCT07136454UnknownPrimary

Expanded Access/Compassionate Use of Rugonersen in Patients With Angelman Syndrome

NCT02996305Phase 2CompletedPrimary

A Study in Adults and Adolescents With Angelman Syndrome (STARS)

NCT03882918Phase 3TerminatedPrimary

An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome

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