Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS

Enrolling by invitation · Phase 1/Phase 2

Conditions studied: Amyotrophic Lateral Sclerosis (ALS)

In brief

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.

Key facts

Study ID

NCT07743268

Run by

n-Lorem Foundation

People needed

6

Starts

2026-02-19

Expected to finish

2028-02-01

Last updated by the study team

2026-08-03

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Informed consent/assent provided by the participant (when appropriate), and/or participants parent(s) or legally authorized representative(s)
• Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records
• Genetically confirmed neurological disorder

You may not qualify if…

• Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures
• Use of an investigational medication within less than 5 half-lives of the drug at enrollment

Where it is running

• Washington University — St Louis, Missouri, United States
• Columbia University, Irving Medical Center — New York, New York, United States

Full record on ClinicalTrials.gov

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