Clinical Trial

Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS

Completed Phase 1/2
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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.
Protocol Amendment History 1 amendment
This ClinicalTrials.gov record has been amended once since 2025-07-23.
Status change: Active, Not Recruiting → Completed 2026-04-06
Trial Details
NCT Number NCT07095712
Lead Sponsor n-Lorem Foundation
Collaborators: The Methodist Hospital Research Institute, Columbia University
Conditions Amyotrophic Lateral Sclerosis
Enrollment 1 participants
Start Date 2024-11-25
Primary Completion 2025-12-16 (estimated)
Study Completion 2025-12-16 (estimated)
Updated on ClinicalTrials.gov 2026-04-09