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Phase 1b intrathecal ION356 in Pelizaeus-Merzbacher disease
jRCT2031240449Phase 1Recruiting
- Sponsor
- Ionis Pharmaceuticals
- Start
- Apr 2025
- Modality
- Gene Therapies, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
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Overview
Phase 1, single-arm trial of ION356 in Pelizaeus-Merzbacher disease (PMD), sponsored by Ionis Pharmaceuticals. Primary endpoints: Number of participants with treatment-emergent adverse events and serious adverse events, Number of participants with clinically significant change from baseline in laboratory assessments and 4 more. Started Apr 2025; status: recruiting.
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Drugs
ION356
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Conditions
Pelizaeus-Merzbacher disease (PMD)
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