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Intrathecal ION373 in Alexander disease
jRCT2031220465Phase 3Active, not recruiting
- Sponsor
- Ionis Pharmaceuticals
- Start
- Dec 2022
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
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Overview
Phase 3, randomised, double-blind trial in Alexander disease comparing ION373 with Placebo then ION373 (2 arms). Primary endpoint: Percent change from baseline in 10-Meter Walk Test (10MWT). Started Dec 2022; status: active, not recruiting.
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Drugs
zilganersen
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Conditions
Alexander disease
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