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Trial intelligence / Profile preview
Expanded open-label trial of ION363 for FUS-associated ALS
jRCT2031260639Not yet recruiting
- Sponsor
- Otsuka Pharmaceutical Co., Ltd.
- Start
- Mar 2027
- 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
- 1
01
Overview
Open-label, single-arm trial of ulefnersen in amyotrophic lateral sclerosis (ALS) and Amyotrophic lateral sclerosis 6 (ALS6), sponsored by Otsuka Pharmaceutical Co., Ltd. Primary endpoints: Safety, Columbia-Suicide Severity Rating Scale and 5 more. Planned start Mar 2027; status: not yet recruiting.
02
Drugs
ulefnersen
03
Conditions
amyotrophic lateral sclerosis (ALS)Amyotrophic lateral sclerosis 6 (ALS6)
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