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NOT YET RECRUITING
NCT07423494
PHASE1/PHASE2

Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576)

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 Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to CHCHD10 Gene Mutation

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

1

Start Date

2026-03

Completion Date

2027-03

Last Updated

2026-02-20

Healthy Volunteers

No

Interventions

DRUG

nL-CHCHD-001

Personalized antisense oligonucleotide

Locations (1)

Mayo Clinic

Rochester, Minnesota, United States