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

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT07095712

Enrollment

Registered

2025-07-31

Start date

2024-11-25

Completion date

2025-12-16

Last updated

2026-04-09

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Amyotrophic Lateral Sclerosis

Brief 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

Interventions

DRUGnL-TARD-001

Personalized antisense oligonucleotide

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

FEMALE

Age

49 Years to 49 Years

Healthy volunteers

Inclusion criteria

* Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s) * Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records * Genetically confirmed neurological disorder

Exclusion criteria

* Participant has any condition that in the opinion of the Site Investigator would ultimately prevent the completion of study procedures * Use of an investigational medication within less than 5 half-lives of the drug at enrollment

Design outcomes

Primary

Measure	Time frame	Description
Clinical Functioning	Baseline to 12 months	Change from baseline at 12-months post nL-TARD-001 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).

Secondary

Measure	Time frame	Description
Disease Biomarkers	Baseline to 12 months	Change from baseline at 12-months post nL-TARD-001 administration in serum/plasma and CSF neurofilament light chain levels
Safety and Tolerability	Baseline to 12 months	Incidence and severity of adverse events

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Apr 10, 2026