This Phase 1/2 study by the n-Lorem Foundation involves delivering nL-CHCHD-001, a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) caused by a pathogenic variant in CHCHD10. Eligibility requires informed consent/assent from the participant and/or their parent(s) or legally authorized representative(s), the ability to travel to the study site, and compliance with study-related follow-up examinations and procedures. The trial is active but no longer recruiting.
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 stie 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