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ENROLLING BY INVITATION
NCT07703462
PHASE1/PHASE2
Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS
Sponsor: n-Lorem Foundation
View on ClinicalTrials.gov
Summary
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.
Official title: An Open-label Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to TARDBP (TDP-43) Genetic Mutation
Key Details
Gender
All
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
1
Start Date
2026-12
Completion Date
2028-12
Last Updated
2026-07-14
Healthy Volunteers
No
Conditions
Interventions
DRUG
nL-TARDB-002
Personalized antisense oligonucleotide
Locations (1)
Jefferson Health
Philadelphia, Pennsylvania, United States