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ION356 in Pelizaeus-Merzbacher Disease
NCT06150716Phase 1Recruiting
- Sponsor
- Ionis Pharmaceuticals
- Start
- Apr 2024
- Primary completion
- Jun 2028
- Modality
- Gene Editing, Gene Silencing, Modified DNA Oligonucleotides
- Countries
- 5
01
Overview
Phase 1, open-label trial of ION356 in Pelizaeus-Merzbacher disease (PMD) (2 cohorts), sponsored by Ionis Pharmaceuticals. Primary endpoints: Treatment-Emergent Adverse Events at 145 weeks, Clinically Significant Change from Baseline in Laboratory Assessments at 145 weeks and 4 more. Started Apr 2024; status: recruiting.
02
Drugs
ION356
03
Conditions
Pelizaeus-Merzbacher disease (PMD)
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