# A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

> **NCT04259281** · PHASE1,PHASE2 · COMPLETED · sponsor: **Ultragenyx Pharmaceutical Inc** · enrollment: 74 (actual)

## Conditions studied

- Angelman Syndrome

## Interventions

- **DRUG:** GTX-102

## Key facts

- **NCT ID:** NCT04259281
- **Lead sponsor:** Ultragenyx Pharmaceutical Inc
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2020-02-24
- **Primary completion:** 2025-01-08
- **Final completion:** 2025-01-08
- **Target enrollment:** 74 (ACTUAL)
- **Last updated:** 2026-01-09


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04259281

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04259281, "A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome". Retrieved via AI Analytics 2026-05-28 from https://api.ai-analytics.org/clinical/NCT04259281. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*