This Phase 1/2 research project involves delivery of nL-CHCHD-001, a personalized antisense oligonucleotide (ASO) drug designed for individual participants with amyotrophic lateral sclerosis (ALS) caused by a pathogenic variant in CHCHD10. 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.
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 representatives(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