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NCT06617429Phase 3ACTIVE NOT RECRUITING

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

Study Summary

The primary objective of this study is to evaluate the effect of GTX-102 in cognitive function in participants with deletion-type Angelman Syndrome (AS).

Conditions
Angelman Syndrome
Interventions / Drugs
GTX-102Sham-LP
View on ClinicalTrials.gov ↗
Sponsor
RARE
Ultragenyx Pharmaceutical
Rare Disease
D49/100
Study Details
Enrollment129 participants
Primary Completion—
Lead SponsorUltragenyx Pharmaceutical Inc
Data Retrieved2026-04-11 04:25:35.045689+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.

Status & Expected Data

Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.

Enrolled: 129 participants
Sponsor Catalyst Profile

Ultragenyx Pharmaceutical (RARE) is the sponsoring company for this trial. BiotechSign currently grades this company D (49/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

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Not financial advice. BiotechSign data is sourced from ClinicalTrials.gov, SEC EDGAR, and other public databases for research and informational purposes only. It does not constitute investment recommendations. Always do your own due diligence before making investment decisions.