Study design & results
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Trial intelligence / Profile preview
Aurora study of GTX-102 in deletion- and nondeletion-type Angelman syndrome
2024-519393-39-00Phase 2Completed
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Feb 2026
- 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 2, open-label trial of apazunersen in Angelman syndrome (AS) (4 arms), sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoints: Safety, Bayley-4 Cognitive raw score and 1 more. Started Feb 2026; status: completed.
02
Drugs
apazunersen
03
Conditions
Angelman syndrome (AS)
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