Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
ION373 for Alexander disease
2024-510603-11-00Phase 2Authorised
- Sponsor
- Ionis Pharmaceuticals
- Start
- May 2024
- 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
- 8
01
Overview
Phase 2, randomised, double-blind trial in Alexander disease comparing ION373 with Placebo (2 arms). Primary endpoint: Percent change from baseline to Week 61 in the 10-Meter Walk Test. Planned start May 2024; status: authorised.
02
Drugs
zilganersen
03
Conditions
Alexander disease
Beyond the preview
Go deeper on 2024-510603-11-00.
Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on 2024-510603-11-00.