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ASCEND: Phase 1-2 Open-Label Study of Intrathecal ION337 in Pediatric Dravet Syndrome
jRCT2051260142Phase 2Recruiting
- Sponsor
- Ionis Pharmaceuticals
- Start
- Aug 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
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Overview
Phase 2, open-label, single-arm trial of ION337 in Dravet syndrome, sponsored by Ionis Pharmaceuticals. Primary endpoints: Treatment-emergent adverse events, Clinically significant change in safety laboratory values and 4 more. Started Aug 2026; status: recruiting.
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Drugs
ION337
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Conditions
Dravet syndrome
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