Clinical Trial

Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS

Active, Not Recruiting 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 CHCHD10
Protocol Amendment History 2 amendments
This ClinicalTrials.gov record has been amended 2 times since 2025-05-09; most recent amendment 2026-07-09.
Trial Details
NCT Number NCT06977451
Lead Sponsor n-Lorem Foundation
Collaborators: Columbia University
Conditions Amyotrophic Lateral Sclerosis
Enrollment 1 participants
Start Date 2024-06-24
Primary Completion 2027-06 (estimated)
Study Completion 2027-06 (estimated)
Updated on ClinicalTrials.gov 2026-07-13