Clinical Trial

Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS

Name: Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS — clinical trial record (NCT07743268)
Creator: DataLookout
License: https://creativecommons.org/licenses/by/4.0/

Enrolling by Invitation Phase 1/2

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Summary

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.

Trial Details

NCT Number	NCT07743268
Lead Sponsor	n-Lorem Foundation Collaborators: Columbia University, Washington University School of Medicine
Conditions	Amyotrophic Lateral Sclerosis (ALS)
Enrollment	6 participants
Start Date	2026-02-19
Primary Completion	2028-02 (estimated)
Study Completion	2028-02 (estimated)
Updated on ClinicalTrials.gov	2026-08-03