drugset
drugset / Trial / NCT07423494

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

NCT07423494 ↗

Phase 1/2 Not yet recruiting 1 enrolled n-Lorem Foundation Mayo Clinic · 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
2026-03
Primary completion
2027-03
Completion
2027-03

Drugs

EvaluationDrugModalityDoseRoute
Subject nL-CHCHD-001 Antisense oligonucleotide — —

Indications

amyotrophic lateral sclerosis