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Phase 3 GTX-102 Efficacy and Safety in Pediatric Angelman Syndrome (Aspire)
jRCT2011250004Phase 3Active, not recruiting
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- May 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
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Overview
Phase 3, randomised trial of apazunersen in Angelman syndrome (AS) (2 arms), sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoint: Change in Bayley-4 Cognitive raw score. Started May 2025; status: active, not recruiting.
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Drugs
apazunersen
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Conditions
Angelman syndrome (AS)
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