A Natural History Study of Angelman Syndrome

Key facts

Sponsor

Massachusetts General Hospital

Principal Investigator

Christopher J Keary, MD

Massachusetts General Hospital

Enrollment

40 enrolled

Eligibility

1 years · All sexes

Timeline

2026 – 2029

Study locations (1)

• MGH Lurie Center for Autism, Lexington, Massachusetts, United States

Collaborators

Astellas Pharma Global Development, Inc.

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.

• 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
• ENROLLING BY INVITATIONPHASE3NCT06415344
  Long-term Extension of GTX-102 in Angelman Syndrome
  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 →