Enrolling by invitation NCT06415344
Long-term Extension of GTX-102 in Angelman Syndrome
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: GTX-102.
- Who it may be relevant to
- Registry conditions: Angelman Syndrome. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Australia, Canada, France, Germany +3
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients With Angelman Syndrome
Overview
The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
Detailed description
This study is a phase 3, long term extension (LTE) which is open to participants rolling over from a prior GTX-102 clinical study. The LTE study will evaluate the long-term safety and efficacy of GTX-102 in participants with AS.
Interventions
- Drug GTX-102
Antisense Oligonucleotide
Primary outcome measures
- Incidence of Treatment Emergent Adverse Events (TEAEs) [Time frame: Up to 5 Years]
Secondary outcome measures (2)
- Change from Baseline in the Bayley-4 Raw Score [Time frame: Baseline, Up to 5 Years]
- Net Response in Multidomain Responder Index (MDRI) [Time frame: Month 12, Month 24]
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 6 centers
- University of California, Los Angeles (UCLA) — Los Angeles
- University of California, San Diego - Rady Children's Hospital — San Diego
- Rare Disease Research, LLC — Atlanta
- Rush University Medical Center — Chicago
- Boston Children's Hospital — Boston
- Weill Cornell Medicine — New York
Canada · 5 centers
- MAGIC Clinic Ltd — Calgary
- British Columbia Children's Hospital — Vancouver
- Childrens Hospital London Health Sciences Centre — London
- Childrens Hospital of Eastern Ontario — Ottawa
- The Research Institute of the McGill University Health Centre — Montreal
United Kingdom · 3 centers
- University of Cambridge — Cambridge
- Great Ormond Street Hospital for Children — London
- Oxford University Hospitals NHS Foundation Trust — Oxford
Australia · 2 centers
- Queensland Children's Hospital — South Brisbane
- The Royal Children's Hospital — Melbourne
France · 2 centers
- AP-HM - Hopital de la Timone — Marseille
- AP-HP Hopital Necker-Enfants Malades — Paris
Germany · 2 centers
- Universitaetsklinikum Hamburg-Eppendorf — Hamburg
- Universitaetsklinikum Leipzig — Leipzig
Spain · 2 centers
- Hospital Universitario Puerta de Hierro — Majadahonda
- Corporacio Sanitaria Parc Tauli - Hospital de Sabadell — Sabadell
Israel · 1 center
- Sheba Medical Center — Ramat Gan
Identifiers
NCT: NCT06415344 · GTX-102-CL302 · 2024-510917-14-00