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Personalized nL-TARDB-002 for TARDBP ALS

This study is testing a personalized medicine called nL-TARDB-002 for a single participant with Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease. This specific type of ALS is caused by a change in the TARDBP gene. The goal is to see if nL-TARDB-002 is safe and how well it works to improve clinical functioning over 12 months. To join, you would need to have genetically confirmed ALS related to the TARDBP gene, provide informed consent, and be able to travel to the study site for follow-up appointments. The study is currently unclear on its recruitment status.

Study design
This is an interventional study designed for a single participant. It is not specified if it is randomized, blinded, or what phase it is in.
What's involved
You would need to travel to the study site for follow-up examinations and procedures, and allow access to your medical records.
Compensation
Not stated in the trial record.
Follow-up
Your clinical functioning will be measured for up to 12 months after starting treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07703462

Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS

Enrolling by Invitation
PHASE1All AgesInterventionalTreatment
n-Lorem Foundation
~1 participants
Updated 2026-07-14 on ClinicalTrials.gov
What's tested:nL-TARDB-002

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Clinical Functioning
Measured over Baseline to 12 months
+7 more outcomes measured

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Explore related trials

Amyotrophic Lateral SclerosisTARDBP Gene

NCT07703462

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Jefferson Health

    Philadelphia, Pennsylvaniano site contact published

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

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Eligibility criteria

Inclusion

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

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

What this trial measures

  • Clinical FunctioningBaseline to 12 months

    Change from baseline at 12-months post nL-TARDB-002 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).

  • Clinical FunctioningBaseline to 12 months

    Change from baseline at 12-months post nL-TARDB-002 administration in Slow Vital Capacity (SVC)

  • Clinical FunctioningBaseline to 12 months

    Change from baseline at 12-months post nL-TARDB-002 administration in Handheld Dynamometry (HHD)

  • Clinical FunctioningBaseline to 12 months

    Change from baseline at 12-months post nL-TARDB-002 administration in scores on Edinburgh Cognitive and Behavioral ALS Screen (ECAS).

  • Clinical FunctioningBaseline to 12 months

    Change from baseline at 12-months post nL-TARDB-002 administration in scores on ALS Cognitive Behavioral Screen (ALS-CBS).

  • Clinical FunctioningBaseline to 12 months

    Change from baseline at 12-months post nL-TARDB-002 administration in scores on Amyotrophic Lateral Sclerosis Assessment Questionnaire 5 (ALSAQ-5).

  • SurvivalBaseline to 12 months

    Change from baseline at 12-months post nL-TARDB-002 administration in survival status

  • Disease BiomarkersBaseline to 12 months

    Change from baseline at 12-months post nL-TARDB-002 administration in serum/plasma and CSF neurofilament light chain levels