Personalized antisense drug for one person with ALS caused by a TARDBP mutation
Tests the antisense drug nL-TARDB-002 in one person with TARDBP-related ALS.
Plain-language title generated from the registry record
Registry title: Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS
Key facts
- Where
- Philadelphia, United StatesSee the site
- Phase
- Phase 1/2
Where in United States
- Jefferson HealthPhiladelphia, United StatesNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- n-Lorem Foundation
- Trial code
- NCT07703462
- Record updated
- 14 July 2026
- Original description
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.Read full original descriptionShow less
An Open-label Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to TARDBP (TDP-43) Genetic Mutation
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.
Inclusion: 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.
Exclusion: Use of an investigational medication within less than 5 half-lives of the drug at enrollment; Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures.