NCT06415344Phase 3Active not recruiting

Long-term Extension of GTX-102 in Angelman Syndrome

Study sponsor: Ultragenyx Pharmaceutical Inc · RARE

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

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Primary completion2029-02Estimated

Enrollment · participants255Estimated

Source updated2026-09-09Last update listed in the registry record

Version history

Changes to this trial

Last 12 months

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Posted 2026-09-09 · 1 field changed

Recruitment status changed

− Before

~~Enrolling by invitation~~

+ After

Active not recruiting

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Posted 2026-07-27 · 1 field changed

Main study measure wording changed

Text changed; whether the study measures changed has not been verified. Saved text may be shortened.

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− Before

Measure: ~~Frequency, Severity, and Relationship to Investigational Drug~~ of Treatment Emergent Adverse Events (~~AEs~~) ~~and Serious Adverse Events (SAEs)~~

+ After

Measure: Incidence of Treatment Emergent Adverse Events (TEAEs)

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Posted 2025-10-08 · 1 field changed

Planned enrollment increased by 180 participants

− Before

~~75 participants (planned)~~

+ After

255 participants (planned)

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