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Trial intelligence / Profile preview
GTX-102 long-term extension in Angelman syndrome
2024-510917-14-00Phase 3Completed
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Sep 2024
- 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, open-label, single-arm trial of apazunersen in Angelman syndrome (AS), sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoint: Treatment-emergent adverse events and serious adverse events. Started Sep 2024; status: completed.
02
Drugs
apazunersen
03
Conditions
Angelman syndrome (AS)
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Competitive context
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