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OpenTrials
Completed

NCT Number: NCT07095712

Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS

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.

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Key information

Conditions

Amyotrophic Lateral Sclerosis Central Nervous System Diseases Metabolic Diseases Motor Neuron Disease Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Proteostasis Deficiencies Spinal Cord Diseases TDP-43 Proteinopathies

Age range

49 year–49 year

Sex eligibility

Female

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Columbia University, Irving Medical Center, New York, United States

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About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

nL-TARD-001

Drug

Personalized antisense oligonucleotide

Primary outcomes

  1. Clinical Functioning

    Time frame: 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).

  2. Clinical Functioning

    Time frame: Baseline to 12 months

    Change from baseline at 12-months post nL-TARD-001 administration in Slow Vital Capacity (SVC)

  3. Clinical Functioning

    Time frame: Baseline to 12 months

    Change from baseline at 12-months post nL-TARD-001 administration in Edinburgh Cognitive and Behavioral ALS Screen (ECAS) score

  4. Clinical Functioning

    Time frame: Baseline to 12 months

    Change from baseline at 12-months post nL-TARD-001 administration in ALS Cognitive Behavioral Screen (ALS-CBS) score

  5. Clinical Functioning

    Time frame: Baseline to 12 months

    Change from baseline at 12-months post nL-TARD-001 administration in Amyotrophic Lateral Sclerosis Assessment Questionnaire 5 (ALSAQ-5)

Secondary outcomes

  1. Disease Biomarkers

    Time frame: Baseline to 12 months

    Change from baseline at 12-months post nL-TARD-001 administration in serum/plasma and CSF neurofilament light chain levels

  2. Safety and Tolerability

    Time frame: Baseline to 12 months

    Incidence and severity of adverse events

  3. Safety and Tolerability

    Time frame: Baseline to 12 months

    Emergent abnormalities in physical exam (changes in appearance, skin, neck, ears, nose, throat, heart/lungs, abdomen, lymph nodes, and extremities compared to baseline)

  4. Safety and Tolerability

    Time frame: Baseline to 12 months

    Emergent abnormalities in neurological exam (changes in mental status, gait, cerebellar, cranial nerve, motor, reflex, and sensations compared to baseline)

  5. Safety and Tolerability

    Time frame: Baseline to 12 months

    Emergent abnormalities in laboratory analyses (results outside of normal range for CSF, chemistry, hematology, coagulation, and urinalysis)

Sponsors and collaborators

Lead sponsor

n-Lorem Foundation

Other

Collaborators

  • Columbia University
  • The Methodist Hospital Research Institute

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2025
Study completion
2025
First posted
Jul 31, 2025
Registry last updated
Apr 9, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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