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ION373 intrathecal trial in Alexander disease
NL-OMON54391Phase 3Recruiting
- Sponsor
- Ionis Pharmaceuticals
- Start
- Sep 2021
- 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
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial of zilganersen in Alexander disease (3 arms), sponsored by Ionis Pharmaceuticals. Primary endpoint: Percent change from baseline to Week 61 in the 10-meter walk test. Started Sep 2021; status: recruiting.
02
Drugs
zilganersen
03
Conditions
Alexander disease
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