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Trial intelligence / Profile preview
Aspire Phase 3 study of GTX-102 in pediatric Angelman syndrome
2024-512600-19-00Phase 3Completed
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Apr 2025
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 8
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-4 Cognitive Raw Score Without Caregiver Input. Started Apr 2025; status: completed.
02
Drugs
apazunersen
03
Conditions
Angelman syndrome (AS)
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