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Clinical trial condition

Angelman Syndrome

Explore clinical trials studying Angelman Syndrome. Study availability and eligibility vary by location and protocol.

40 public trials 9 recruiting studies
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Angelman Syndrome trial results

Active, not recruiting

Long-term Extension of GTX-102 in Angelman Syndrome

NCT06415344

Conditions
Abnormalities, Multiple, Angelman Syndrome, Central Nervous System Diseases, +7 more
Locations
  • University of California, Los Angeles (UCLA) Los Angeles, California, United States
  • University of California, San Diego - Rady Children's Hospital San Diego, California, United States
  • Rare Disease Research, LLC Atlanta, Georgia, United States
  • Rush University Medical Center Chicago, Illinois, United States
View all 23 locations
View Trial Details
Active, not recruiting

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

NCT07157254

Conditions
Abnormalities, Multiple, Angelman Syndrome, Central Nervous System Diseases, +7 more
Locations
  • Cedars Sinai Medical Center Los Angeles, California, United States
  • Rush University Medical Center Chicago, Illinois, United States
  • Clinical Trial Site Kansas City, Missouri, United States
  • Rare Disease Research Hillsborough, North Carolina, United States
View all 21 locations
View Trial Details
Active, not recruiting

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

NCT05127226

Conditions
Abnormalities, Multiple, Angelman Syndrome, Central Nervous System Diseases, +7 more
Locations
  • Rady Children's Hospital San Diego, California, United States
  • Colorado Children's Hospital Research Institute Aurora, Colorado, United States
  • Rush University Medical Center Chicago, Illinois, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
View Trial Details
Active, not recruiting

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

NCT06617429

Conditions
Abnormalities, Multiple, Angelman Syndrome, Central Nervous System Diseases, +7 more
Locations
  • Cedars Sinai Los Angeles, California, United States
  • UCSD, Rady Children's Hospital San Diego, California, United States
  • UCSF San Francisco, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
View all 28 locations
View Trial Details
Active, not recruiting

Early Check: Expanded Screening in Newborns

NCT03655223

Conditions
17 Alpha-Hydroxylase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, +456 more
Locations
  • RTI International Research Triangle Park, North Carolina, United States
View Trial Details
Completed

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

NCT04259281

Conditions
Abnormalities, Multiple, Angelman Syndrome, Central Nervous System Diseases, +7 more
Locations
  • UCLA Medical Center Los Angeles, California, United States
  • Rady Children's Hospital San Diego, California, United States
  • Rare Disease Research Atlanta, Georgia, United States
  • Rush University Medical Center Chicago, Illinois, United States
View all 25 locations
View Trial Details
Completed

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.

NCT05630066

Conditions
Abnormalities, Multiple, Angelman Syndrome, Central Nervous System Diseases, +7 more
Locations
  • Rush Medical Center Chicago, Illinois, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
  • Columbia University Medical Center New York, United States
  • Carolina Institute for Development DisabilitiesUniversity of North Carolina/School of Medicine Carrboro, North Carolina, United States
View all 19 locations
View Trial Details

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