Angelman syndrome is a rare genetic neurodevelopmental disorder that affects nervous system function and is characterized by severe developmental delay, limited speech, movement and balance problems, seizures, and a typically happy demeanor. It is caused by abnormalities affecting the UBE3A region on chromosome 15.
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| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07417137 |
A Natural History Study of Angelman Syndrome |
RECRUITING | |
| NCT07181837 |
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome |
RECRUITING | PHASE1/PHASE2 |
| NCT07157254 |
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) |
RECRUITING | PHASE2 |
| NCT07136454 |
Expanded Access/Compassionate Use of Rugonersen in Patients With Angelman Syndrome |
AVAILABLE | |
| NCT06914609 |
REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome |
RECRUITING | PHASE3 |
| NCT06737718 |
Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman Syndrome |
RECRUITING | |
| NCT06617429 |
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT06415344 |
Long-term Extension of GTX-102 in Angelman Syndrome |
ENROLLING_BY_INVITATION | PHASE3 |
| NCT06353620 |
Structural-functional Connectome in Drug-resistant Epilepsies and Neurodevelopmental Syndromes With Epilepsy |
RECRUITING | |
| NCT06337383 |
Study of the Prevalence of Autistic Traits in Angelman Syndrome |
RECRUITING |
