Personalized Antisense Oligonucleotide Therapy for a Participant 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 a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.

Key facts

Study ID

NCT07703462

Run by

n-Lorem Foundation

People needed

1

Starts

2026-12-01

Expected to finish

2028-12-01

Last updated by the study team

2026-07-14

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

• Jefferson Health — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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