Long-term Extension of GTX-102 in Angelman Syndrome

Enrolling by invitation · Phase 3

Conditions studied: Angelman Syndrome

In brief

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

Key facts

Study ID

NCT06415344

Run by

Ultragenyx Pharmaceutical Inc

People needed

255

Starts

2024-07-31

Expected to finish

2029-02-01

Last updated by the study team

2026-07-27

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Signed informed consent from parent(s) or legal guardian(s).
• Completed a final study visit in a prior clinical trial with GTX-102. If the subject has an ongoing adverse event, medical monitor approval is required to rollover. The Screening visit must occur within 6 months of the last visit in the prior GTX-102 trial.
• From the time of informed consent through the end of the study and for at least 6 months after the final dose of GTX-102, females of childbearing potential who are sexually active must use highly effective contraception or abstinence. Males are able to participate if they agree to remain abstinent (refrain from heterosexual intercourse) or use acceptable contraceptive methods during the study and for at least 3 months after the final dose of GTX-102.

You may not qualify if…

• Discontinued from study or withdrew from study treatment in a prior GTX-102 trial.
• History or evidence of any other medical, neurological, or psychological condition that would expose the subject to an undue risk of a significant AE or interfere with study assessments during the course of the trial as determined by the clinical judgment of the Investigator.
• Known hypersensitivity to GTX-102 or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects.
• Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study

Where it is running

• 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
• Universitaetsklinikum Hamburg-Eppendorf — Hamburg, Germany
• Universitaetsklinikum Leipzig — Leipzig, Germany
• Sheba Medical Center — Ramat Gan, Israel
• Hospital Universitario Puerta de Hierro — Majadahonda, Spain
• Corporacio Sanitaria Parc Tauli - Hospital de Sabadell — Sabadell, Spain
• University of Cambridge — Cambridge, United Kingdom
• Great Ormond Street Hospital for Children — London, United Kingdom
• Oxford University Hospitals NHS Foundation Trust — Oxford, United Kingdom

Full record on ClinicalTrials.gov

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