Angelman Syndrome Clinical Trials

60 clinical trials for Angelman Syndrome across international registries, including 18 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome — RECRUITING · PHASE3
• A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) — RECRUITING · PHASE2
• BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome. — RECRUITING · PHASE3
• Long-term Extension of GTX-102 in Angelman Syndrome — RECRUITING · PHASE3
• A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome — RECRUITING · PHASE1
• A Phase 3, Randomized, Double-blind, Sham-controlled Study Investigating the Efficacy and Safety of GTX-102 in Pediatric Subjects with Angelman Syndrome — RECRUITING · PHASE3
• Parent and Infant Inter(X)Action Intervention (PIXI) — RECRUITING · NA
• A Natural History Study of Angelman Syndrome — RECRUITING
• Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman Syndrome — RECRUITING
• IDMet (RaDiCo Cohort) (RaDiCo-IDMet) — RECRUITING
• HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome — RECRUITING · PHASE1
• Natural History Study for Patients With Angelman Syndrome — RECRUITING
• Angelman Syndrome Natural History Study — RECRUITING
• PHASE 3 STUDY OF THE EFFICACY AND SAFETY OF ION582 IN CHILDREN AND ADULTS WITH ANGELMAN SYNDROME — RECRUITING · PHASE3
• A PHASE 3, RANDOMIZED, DOUBLE-BLIND, SHAM-CONTROLLED STUDY INVESTIGATING THE EFFICACY AND SAFETY OF GTX-102 IN PEDIATRIC SUBJECTS WITH ANGELMAN SYNDROME — RECRUITING · PHASE3
• A LONG-TERM EXTENSION TRIAL INVESTIGATING THE SAFETY AND EFFICACY OF GTX-102 IN PATIENTS WITH ANGELMAN SYNDROME — RECRUITING
• Structural-functional Connectome in Drug-resistant Epilepsies and Neurodevelopmental Syndromes With Epilepsy — RECRUITING
• The Global Angelman Syndrome Registry — RECRUITING
• Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) — ACTIVE_NOT_RECRUITING · PHASE3
• ION582-CS1: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients with Angelman Syndrome — TERMINATED · PHASE2
• Web Intervention for Parents of Youth With Genetic Syndromes (WINGS) — ACTIVE_NOT_RECRUITING · NA
• Early Check: Expanded Screening in Newborns — ACTIVE_NOT_RECRUITING
• REVEAL Study: Phase 3 Study of the Efficacy and Safety of ION582 in Children and Adults with Angelman Syndrome — TERMINATED · PHASE3
• RANDOMIZED, MULTI-CENTER, DOUBLE-BLIND, SHAM-CONTROLLED, PARALLEL GROUP, PHASE III CLINICAL STUDY TO EVALUATE THE EFFICACY AND SAFETY OF INTRATHECALLY ADMINISTERED RUGONERSEN IN PEDIATRIC AND ADULT PARTICIPANTS WITH ANGELMAN SYNDROME — NOT_YET_RECRUITING · PHASE3
• A Phase 2, Open-label, Basket Study Investigating the Safety and Efficacy of GTX-102 in Adult and Pediatric Subjects with Deletion- or Nondeletion-type Angelman Syndrome — TERMINATED · PHASE2
• Italian Angelman Syndrome Registry — COMPLETED
• A Long-Term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients with Angelman Syndrome — TERMINATED · PHASE3
• A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome — COMPLETED · PHASE1
• 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. — COMPLETED · PHASE2
• A MULTI-CENTER, OPEN-LABEL, PHASE 1/2 TRIAL OF THE SAFETY AND EFFICACY OF MVX-220 GENE THERAPY ADMINISTERED BY INTRA-CISTERNA MAGNA INJECTION TO PARTICIPANTS WITH ANGELMAN SYNDROME — NOT_YET_RECRUITING · PHASE1
• A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS) — COMPLETED · PHASE1
• Expanded Access/Compassionate Use of Rugonersen in Patients With Angelman Syndrome — OTHER
• A Study in Adults and Adolescents With Angelman Syndrome (STARS) — COMPLETED · PHASE2
• An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome — TERMINATED · PHASE3
• An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome — COMPLETED · PHASE2
• An Open-Label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO7248824 In Participants with Angelman Syndrome — OTHER · PHASE1
• Study of the Prevalence of Autistic Traits in Angelman Syndrome — OTHER
• Angelman Natural History Study - FAST Spain — OTHER
• Angelman Syndrome Natural History Study-FAST UK — OTHER
• Angelman Syndrome Video Assessment (ASVA) Source Material Study — COMPLETED
• Development of a Newborn Screening Assay for Angelman Syndrome and Prader-Willi Syndrome — COMPLETED
• Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and/or Overweight, and/or Obesity — OTHER
• Minocycline in the Treatment of Angelman Syndrome — COMPLETED · NA
• A Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male Participants — COMPLETED · PHASE1
• Angelman Syndrome (AS) Biomarker Study — COMPLETED · PHASE1
• A STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARMACODYNAMICS OF INTRATHECALLY ADMINISTERED ION582 IN PATIENTS WITH ANGELMAN SYNDROME — COMPLETED
• Study on the Brain Network of Angelman Syndrome — COMPLETED
• Characterization of Angelman Syndrome — COMPLETED
• SNP-based Microdeletion and Aneuploidy RegisTry (SMART) — COMPLETED
• AN OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARMACODYNAMICS OF RO7248824 IN PARTICIPANTS WITH ANGELMAN SYNDROME — COMPLETED

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