drugset
drugset / Trial / NCT06977451

Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS

NCT06977451 ↗

Phase 1/2 Active not recruiting 1 enrolled n-Lorem Foundation Columbia University · collab
NaSingle-groupOpen-labelTreatment

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

Timeline

Start
2024-06-24
Primary completion
2027-06
Completion
2027-06

Drugs

EvaluationDrugModalityDoseRoute
Subject nL-CHCHD-001 Antisense oligonucleotide — —

Indications

amyotrophic lateral sclerosis