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
Condition(s) studied
Angelman Syndrome
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
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 |
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Other trials for the same condition
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
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A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
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Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
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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