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Clinical Trials in the USA / NCT06415344
Active, not recruiting Phase 3

Long-term Extension of GTX-102 in Angelman Syndrome

NCT06415344 · tracked via the Priya Life Science USA tracker
Sponsor
Ultragenyx Pharmaceutical Inc
Phase
Phase 3
Started
2024-07-31
Last updated
2026-09-09

Condition(s) studied

Angelman Syndrome

Investigational drug(s) / intervention(s)

GTX-102

GTX-102: Antisense Oligonucleotide

Study summary

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

Eligibility

Sex
ALL
Min age
Max age
Healthy volunteers
No
Inclusion Criteria: * 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. Exclusion Criteria: * 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

Primary outcome measure(s)

Trial sites (23)

FacilityCityRegionStatus
University of California, Los Angeles (UCLA) Los Angeles California
University of California, San Diego - Rady Children's Hospital San Diego California
Rare Disease Research, LLC Atlanta Georgia
Rush University Medical Center Chicago Illinois
Boston Children's Hospital Boston Massachusetts
Weill Cornell Medicine New York New York
Queensland Children's Hospital South Brisbane Queensland
The Royal Children's Hospital Melbourne Victoria
MAGIC Clinic Ltd Calgary Alberta
British Columbia Children's Hospital Vancouver British Columbia
Childrens Hospital London Health Sciences Centre London Ontario
Childrens Hospital of Eastern Ontario Ottawa Ontario
The Research Institute of the McGill University Health Centre Montreal Quebec
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

More Ultragenyx Pharmaceutical Inc trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06415344 on ClinicalTrials.gov ↗ ← All trials in the USA