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Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

Amyotrophic Lateral Sclerosis Clinical Trial

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

Clinical Trial 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

Clinical Trial Description

This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10 ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06392126
Study type Interventional
Source n-Lorem Foundation
Contact
Status Active, not recruiting
Phase Phase 1/Phase 2
Start date April 16, 2024
Completion date April 2025
View Details
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