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

Key facts

Sponsor

Ultragenyx Pharmaceutical Inc

Principal Investigator

Medical Director

Ultragenyx Pharmaceuticals Inc.

Intervention

GTX-102(drug)

Enrollment

129 enrolled

Eligibility

4-17 years · All sexes

Timeline

2024 – 2027

Study locations (28)

• 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
• Nicklaus Children's Hospital, Miami, Florida, United States
• Rare Disease Research, Atlanta, Georgia, United States
• Rush University, Chicago, Illinois, United States
• Boston Children's Hospital, Boston, Massachusetts, United States
• Children's Mercy, Kansas City, Missouri, United States
• Columbia University Medical Center, New York, New York, United States
• UNC Chapel Hill Pediatrics, Chapel Hill, North Carolina, United States
• Rare Disease Research, Hillsborough, North Carolina, United States
• The University of Texas, Austin, Texas, United States
• Carum Research Inc, Dallas, Texas, United States
• McGill University Health Centre, Montreal, Quebec, Canada
• +13 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.

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• RECRUITINGPHASE1, PHASE2NCT07181837
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• RECRUITINGPHASE2NCT07157254
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• RECRUITINGNCT06737718
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• ENROLLING BY INVITATIONPHASE3NCT06415344
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• RECRUITINGPHASE1, PHASE2NCT05127226
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• RECRUITINGNCT06229769
  Natural History Study for Patients With Angelman Syndrome
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• RECRUITINGNCT05293184
  The Global Angelman Syndrome Registry
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See all trials for Angelman syndrome due to a point mutation →