drugset
drugset / Trial / NCT07095686

Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS

NCT07095686 ↗

Phase 1/2 Enrolling by invitation 9 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 individual participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10

Timeline

Start
2024-10-02
Primary completion
2027-04
Completion
2027-04

Drugs

EvaluationDrugModalityDoseRoute
Subject nL-CHCHD-001 Antisense oligonucleotide — —

Indications

amyotrophic lateral sclerosis