A Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander Disease
Latest Information Update: 18 Nov 2024
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At a glance
- Drugs Zilganersen (Primary)
- Indications Alexander disease
- Focus First in man; Registrational; Therapeutic Use
- Sponsors Ionis Pharmaceuticals
- 02 Aug 2024 Planned End Date changed from 1 Mar 2029 to 1 Sep 2029.
- 02 Aug 2024 Planned primary completion date changed from 1 Mar 2025 to 1 Sep 2025.
- 18 Jul 2024 According to an Ionis Pharmaceuticals media release, topline data are anticipated in the second half of 2025.