NCT07095712

Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS

An Open-label Multicenter, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to TARDBP (TDP-43) Genetic Mutation

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: n-Lorem Foundation · Academic / Other
• Sex: Female
• Age: 49 Years – 49 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 TARDBP.

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 TARDBP

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

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

Timeline

• Start date: 2024-11-25
• Primary completion: 2025-12-16
• Completion: 2025-12-16
• First posted: 2025-07-31
• Last updated: 2026-04-09

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study