Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS

Plain-language summary

This Phase 1/2 research project involves delivery of nL-TARDB-002, a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) caused by a pathogenic variant in TARDBP. The study is run by the n-Lorem Foundation. Enrollment is by invitation and requires informed consent or assent and the ability to travel to the study site and adhere to study-related follow-up examinations and procedures.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Enrolling by invitation

Sponsor

n-Lorem Foundation

Intervention

nL-TARDB-002

Start date

12/1/2026

Estimated completion

12/1/2028

Countries

United States

Related genes

Locations

• Philadelphia, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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