Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS

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

Status

Enrolling by invitation

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT07743268

Enrollment

Registered

2026-08-03

Start date

2026-02-19

Completion date

2028-02-01

Last updated

2026-08-03

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

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Brief summary

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for participants 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 participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP

Interventions

DRUGnL-TARDB-006

Personalized antisense oligonucleotide

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Healthy volunteers

Inclusion criteria

* Informed consent/assent provided by the participant (when appropriate), and/or participants 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-TARDB-006 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).
Survival	Baseline to 12 months	Change from baseline at 12-months post nL-TARDB-006 administration in survival status
Disease Biomarkers	Baseline to 12 months	Change from baseline at 12-months post nL-TARDB-006 administration in serum/plasma and CSF neurofilament light chain levels

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Aug 4, 2026