Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
ION363/Ulefnersen in FUS-ALS
2024-512163-31-00Phase 3Authorised
- Sponsor
- Ionis Pharmaceuticals
- Start
- Aug 2024
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 16
01
Overview
Phase 3, randomised, double-blind trial in amyotrophic lateral sclerosis (ALS) comparing Ulefnersen with Placebo (2 arms). Primary endpoints: Joint rank analysis of functional impairment, ALSFRS-R component of joint rank analysis and 2 more. Planned start Aug 2024; status: authorised.
02
Drugs
ulefnersen
03
Conditions
amyotrophic lateral sclerosis (ALS)
Beyond the preview
Go deeper on 2024-512163-31-00.
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 2024-512163-31-00.