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ENROLLING BY INVITATION
NCT07095686
PHASE1/PHASE2
Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS
Sponsor: n-Lorem Foundation
View on ClinicalTrials.gov
Summary
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for individual participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10
Official title: An Open-Label Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to an Arg15Leu Pathogenic Variant in CHCHD10
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
9
Start Date
2024-10-02
Completion Date
2026-04
Last Updated
2025-07-31
Healthy Volunteers
No
Conditions
Interventions
DRUG
nL-CHCHD-001
Personalized antisense oligonucleotide
Locations (1)
Columbia University, Irving Medical Center
New York, New York, United States