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ACTIVE NOT RECRUITING
NCT06392126
PHASE1/PHASE2
Personalized Antisense Oligonucleotide Therapy for A Single Participant 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 a single participant 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 a Single Participant With Amyotrophic Lateral Sclerosis (ALS) Due to a Pathogenic Variant in CHCHD10
Key Details
Gender
MALE
Age Range
48 Years - Any
Study Type
INTERVENTIONAL
Enrollment
1
Start Date
2024-04-16
Completion Date
2026-04
Last Updated
2025-08-05
Healthy Volunteers
No
Conditions
Interventions
DRUG
nL-CHCHD-001
Personalized antisense oligonucleotide
Locations (1)
Mayo Clinic
Jacksonville, Florida, United States