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Enrolling by invitation NCT07743268

Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS

Phase I / Phase II Interventional Amyotrophic Lateral Sclerosis (ALS)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: nL-TARDB-006.
Who it may be relevant to
Registry conditions: Amyotrophic Lateral Sclerosis (ALS). Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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

  • Drug nL-TARDB-006
    Personalized antisense oligonucleotide

Primary outcome measures

  • Clinical Functioning [Time frame: Baseline to 12 months]
  • Clinical Functioning [Time frame: Baseline to 12 months]
  • Clinical Functioning [Time frame: Baseline to 12 months]
  • Clinical Functioning [Time frame: Baseline to 12 months]
  • Clinical Functioning [Time frame: Baseline to 12 months]
  • Clinical Functioning [Time frame: Baseline to 12 months]
  • Survival [Time frame: Baseline to 12 months]
  • Disease Biomarkers [Time frame: Baseline to 12 months]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 2 centers
  • Washington University — St Louis
  • Columbia University, Irving Medical Center — New York

Identifiers

NCT: NCT07743268 · NLF-TARDB-006-A

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗