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Long-term Study of GTX-102 for Angelman Syndrome

This study is looking at the long-term safety and effectiveness of a medicine called GTX-102 for people with Angelman Syndrome (a genetic disorder affecting development). You can join this study if you have already participated in a previous clinical trial involving GTX-102 and completed your final visit. The main goal is to see how safe GTX-102 is over a long period, up to five years. This study is currently open to participants who have been in a prior GTX-102 study.

Study design
This is a Phase 3, long-term extension study. It is open to participants who have already been in a previous GTX-102 clinical study, with a planned enrollment of 255 people.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The study will measure side effects for up to 5 years.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06415344

Long-term Extension of GTX-102 in Angelman Syndrome

Enrolling by Invitation
PHASE3All AgesInterventionalTreatment
Ultragenyx Pharmaceutical Inc
~255 participants
Updated 2026-07-27 on ClinicalTrials.gov
What's tested:GTX-102

At a glance

Recruiting sites
0 of 23 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of Treatment Emergent Adverse Events (TEAEs)
Measured over Up to 5 Years

Conditions

Angelman Syndrome

Explore related trials

Angelman Syndrome

Where it's being run

23 sites across 16 states
United Kingdom3
California2
Ontario2
France2
Germany2
Spain2
Georgia1
Illinois1

Study leadership

  • Medical Director · STUDY_DIRECTOR · Ultragenyx Pharmaceuticals Inc.

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

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

What this trial measures

  • Incidence of Treatment Emergent Adverse Events (TEAEs)Up to 5 Years