NCT06392126

Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

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

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: n-Lorem Foundation · Academic / Other
• Sex: Male
• Age: 48 Years
• Healthy volunteers: Not accepted

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

Detailed 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

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

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

Timeline

• Start date: 2024-04-16
• Primary completion: 2026-04-01
• Completion: 2026-04-01
• First posted: 2024-04-30
• Last updated: 2025-08-05

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study