NCT07095686

Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS

An Open-Label Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to an Arg15Leu Pathogenic Variant in CHCHD10

• Status: Enrolling By Invitation
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: n-Lorem Foundation · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	nL-CHCHD-001	Personalized antisense oligonucleotide

Timeline

• Start date: 2024-10-02
• Primary completion: 2026-04-01
• Completion: 2026-04-01
• First posted: 2025-07-31
• Last updated: 2025-07-31

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study