Long-term Extension of GTX-102 in Angelman Syndrome

Key facts

Sponsor

Ultragenyx Pharmaceutical Inc

Principal Investigator

Medical Director

Ultragenyx Pharmaceuticals Inc.

Intervention

GTX-102(drug)

Enrollment

255 enrolled

Eligibility

All sexes

Timeline

2024 – 2029

Study locations (23)

• 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
• Boston Children's Hospital, Boston, Massachusetts, United States
• Weill Cornell Medicine, New York, New York, United States
• Queensland Children's Hospital, South Brisbane, Queensland, Australia
• The Royal Children's Hospital, Melbourne, Victoria, Australia
• MAGIC Clinic Ltd, Calgary, Alberta, Canada
• British Columbia Children's Hospital, Vancouver, British Columbia, Canada
• Childrens Hospital London Health Sciences Centre, London, Ontario, Canada
• Childrens Hospital of Eastern Ontario, Ottawa, Ontario, Canada
• The Research Institute of the McGill University Health Centre, Montreal, Quebec, Canada
• AP-HM - Hopital de la Timone, Marseille, France
• AP-HP Hopital Necker-Enfants Malades, Paris, France
• +8 more locations on ClinicalTrials.gov

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Other trials for Angelman syndrome due to a point mutation

Additional recruiting or active studies for the same condition.

• RECRUITINGNCT07417137
  A Natural History Study of Angelman Syndrome
  Massachusetts General Hospital
• RECRUITINGPHASE1, PHASE2NCT07181837
  A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
  MavriX Bio, LLC
• RECRUITINGPHASE2NCT07157254
  A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
  Ultragenyx Pharmaceutical Inc
• RECRUITINGNCT06737718
  Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman Syndrome
  Assistance Publique - Hôpitaux de Paris
• ACTIVE NOT RECRUITINGPHASE3NCT06617429
  Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
  Ultragenyx Pharmaceutical Inc
• RECRUITINGPHASE1, PHASE2NCT05127226
  HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
  Ionis Pharmaceuticals, Inc.
• RECRUITINGNCT06229769
  Natural History Study for Patients With Angelman Syndrome
  Centre Hospitalier Universitaire de Liege
• RECRUITINGNCT05293184
  The Global Angelman Syndrome Registry
  Foundation for Angelman Syndrome Therapeutics, Australia

See all trials for Angelman syndrome due to a point mutation →