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Aspire: GTX-102 in Pediatric Subjects With Angelman Syndrome
NCT06617429Phase 3Terminated
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Dec 2024
- Primary completion
- Jul 2026
- Modality
- Modified DNA Oligonucleotides, Single-strand DNA, Antisense Oligonucleotides (ASOs)
- Countries
- 6
01
Overview
Phase 3, randomised, double-blind trial of apazunersen in Angelman syndrome (AS) (2 arms), sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoint: Change from Baseline in Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4) Cognitive Raw Score Without Caregiver Input at Day 338. Started Dec 2024; status: terminated.
02
Drugs
apazunersen
03
Conditions
Angelman syndrome (AS)
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