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Trial intelligence / Profile preview
Phase 1b study of intrathecal ION356 in Pelizaeus-Merzbacher disease
NL-OMON60602Phase 1Recruiting
- Sponsor
- Ionis Pharmaceuticals
- Start
- Apr 2024
- Modality
- Gene Therapies, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of ION356 in Pelizaeus-Merzbacher disease (PMD), sponsored by Ionis Pharmaceuticals. Primary endpoints: Treatment-emergent adverse events and serious adverse events, Laboratory safety assessments and 3 more. Started Apr 2024; status: recruiting.
02
Drugs
ION356
03
Conditions
Pelizaeus-Merzbacher disease (PMD)
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