Active Not Recruiting PHASE3
Long-term Extension of GTX-102 in Angelman Syndrome
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)
Drug
- GTX-102
Study locations
Australia
Queensland
South Brisbane (1)
- Queensland Children's Hospital — Site status not reported
Victoria
Melbourne (1)
- The Royal Children's Hospital — Site status not reported
Canada
Alberta
Calgary (1)
- MAGIC Clinic Ltd — Site status not reported
British Columbia
Vancouver (1)
- British Columbia Children's Hospital — Site status not reported
Ontario
London (1)
- Childrens Hospital London Health Sciences Centre — Site status not reported
Ottawa (1)
- Childrens Hospital of Eastern Ontario — Site status not reported
Quebec
Montreal (1)
- The Research Institute of the McGill University Health Centre — Site status not reported
France
State / region not reported
Marseille (1)
- AP-HM - Hopital de la Timone — Site status not reported
Paris (1)
- AP-HP Hopital Necker-Enfants Malades — Site status not reported
Germany
State / region not reported
Hamburg (1)
- Universitaetsklinikum Hamburg-Eppendorf — Site status not reported
Leipzig (1)
- Universitaetsklinikum Leipzig — Site status not reported
Israel
State / region not reported
Ramat Gan (1)
- Sheba Medical Center — Site status not reported
Spain
State / region not reported
Majadahonda (1)
- Hospital Universitario Puerta de Hierro — Site status not reported
Sabadell (1)
- Corporacio Sanitaria Parc Tauli - Hospital de Sabadell — Site status not reported
United Kingdom
State / region not reported
Cambridge (1)
- University of Cambridge — Site status not reported
London (1)
- Great Ormond Street Hospital for Children — Site status not reported
Oxford (1)
- Oxford University Hospitals NHS Foundation Trust — Site status not reported
United States
California
Los Angeles (1)
- University of California, Los Angeles (UCLA) — Site status not reported
San Diego (1)
- University of California, San Diego - Rady Children's Hospital — Site status not reported
Georgia
Atlanta (1)
- Rare Disease Research, LLC — Site status not reported
Illinois
Chicago (1)
- Rush University Medical Center — Site status not reported
Massachusetts
Boston (1)
- Boston Children's Hospital — Site status not reported
New York
New York (1)
- Weill Cornell Medicine — Site status not reported
Full registry eligibility criteria
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
Important information
Registry information may change. FAST context and classifications are editorial additions. Inclusion does not confirm eligibility or availability at a specific site. Review the official record and contact the study team for participation requirements.