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Study identifier: NCT06415344 Synced from ClinicalTrials.gov · August 07, 2026
● Study status: Enrolling By Invitation

Long-term Extension of GTX-102 in Angelman Syndrome

Condition: Angelman Syndrome  ·  Sponsor: Ultragenyx Pharmaceutical Inc

PhasePhase 3
Planned participants255
Who can joinAll sexes, N/A to no upper limit
Healthy volunteersNo

About this study

The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)

This description comes directly from the study's public registry record.

Talk to the study team

No contact information is available for this study as per the public registry record.

Always discuss trial participation with your own doctor first.

Locations (23)

University of California, Los Angeles (UCLA)Los Angeles, California, United States
University of California, San Diego - Rady Children's HospitalSan Diego, California, United States
Rare Disease Research, LLCAtlanta, Georgia, United States
Rush University Medical CenterChicago, Illinois, United States
Boston Children's HospitalBoston, Massachusetts, United States
Weill Cornell MedicineNew York, New York, United States
Queensland Children's HospitalSouth Brisbane, Queensland, Australia
The Royal Children's HospitalMelbourne, Victoria, Australia
MAGIC Clinic LtdCalgary, Alberta, Canada
British Columbia Children's HospitalVancouver, British Columbia, Canada
Childrens Hospital London Health Sciences CentreLondon, Ontario, Canada
Childrens Hospital of Eastern OntarioOttawa, Ontario, Canada

+ 11 more locations — full list on the registry record.

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