This research project involves delivering nL-TARD-001, a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) caused by a pathogenic variant in TARDBP. The Phase 1/2 study is an individualized (n-of-1) treatment program. Participation requires informed consent from the participant and/or their parents or legally authorized representatives, along with 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.
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 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