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)
Incidence of Treatment Emergent Adverse Events (TEAEs) — Up to 5 Years
Trial sites (23)
Facility
City
Region
Status
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
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.
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