Phase 2 Study of GTX-102 in Subjects with Deletion- or Nondeletion-type Angelman Syndrome

• Research type

  Research Study

• Full title

  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

• IRAS ID

  1012549

• Contact name

  Vanessa Horne

• Sponsor organisation

  Ultragenyx Pharmaceutical Inc

• Research summary

  Angelman syndrome (AS) is a rare genetic condition that affects the brain and nervous system. It causes severe disabilities with movement, learning and understanding. At the moment there are no approved medicines to treat AS. This study is testing a new medicine called GTX-102 to see if it is safe and helpful for people with AS. GTX-102 is still being researched and has not yet been approved by health authorities like the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK. The study, called GTX-102-CL210, includes three groups of participants:
  - Children between 1 and under 4 years old who have a specific type of AS called deletion-type
  - Children between 4 and under 18 years old who have other types of AS caused by changes in the gene called nondeletion-type
  - Adults between 18 and under 65 years old who have any type of AS.

• REC name

  East of England - Cambridge South Research Ethics Committee

• REC reference

  25/EE/0204

• Date of REC Opinion

  31 Oct 2025

• REC opinion

  Further Information Favourable Opinion