A Long-Term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients with Angelman Syndrome

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-510917-14-00

Acronym

GTX-102-CL302

Enrollment

Registered

2024-07-18

Start date

2024-09-03

Completion date

Unknown

Last updated

2025-11-14

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Angelman Syndrome

Brief summary

Treatment-emergent AEs and SAEs, frequency, severity, and relationship to investigational product throughout the study

Detailed description

Change from LTE Month 0 and pretreatment (when available) throughout the study in: • Bayley-4 Cognitive raw score • Bayley-4 Receptive Communication raw score • Bayley-4 Expressive Communication raw score • Bayley-4 Gross Motor raw score • Bayley-4 Fine Motor raw score

Interventions

DRUGELLIOTTS B SOLUTION (buffered intrathecal electrolyte/dextrose injection)

DRUGGTX-102

DRUGGTX/UX Diluent and Flush Solution

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Treatment-emergent AEs and SAEs, frequency, severity, and relationship to investigational product throughout the study	—

Secondary

Measure	Time frame
Change from LTE Month 0 and pretreatment (when available) throughout the study in: • Bayley-4 Cognitive raw score • Bayley-4 Receptive Communication raw score • Bayley-4 Expressive Communication raw score • Bayley-4 Gross Motor raw score • Bayley-4 Fine Motor raw score	—

Countries

France, Germany, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026