Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS

NCT07095712 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: PRO00038978
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 2

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.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (5)

• Clinical Functioning

  Change from baseline at 12-months post nL-TARD-001 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).

  Baseline to 12 months

• Clinical Functioning

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

  Baseline to 12 months

• Clinical Functioning

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

  Baseline to 12 months

• Clinical Functioning

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

  Baseline to 12 months

• Clinical Functioning

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

  Baseline to 12 months

Secondary Outcomes (5)

• Disease Biomarkers

  Baseline to 12 months

• Safety and Tolerability

  Baseline to 12 months

• Safety and Tolerability

  Baseline to 12 months

• Safety and Tolerability

  Baseline to 12 months

• Safety and Tolerability

  Baseline to 12 months

Study Arms (1)

Open Label

EXPERIMENTAL

Drug: nL-TARD-001

Interventions

nL-TARD-001 DRUG

Personalized antisense oligonucleotide

Open Label

Eligibility Criteria

• Age: 49 Years - 49 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• n-Lorem Foundation: lead
• The Methodist Hospital Research Institute: collaborator
• Columbia University: collaborator

Study Sites (2)

Columbia University, Irving Medical Center

New York, New York, 10032, United States

Location

Houston Methodist

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 12, 2025
• First Posted: July 31, 2025
• Study Start: November 25, 2024
• Primary Completion: December 16, 2025
• Study Completion: December 16, 2025
• Last Updated: April 9, 2026

Record last verified: 2026-04

Locations

US (2)