NCT07423494
Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576)
Not Yet Recruiting · Phase 1, Phase 2 · n-Lorem Foundation · registry updated 2026-02-20
Inclusion and exclusion lines below are quoted from ClinicalTrials.gov. No match score is shown, because a score needs a person's age, biomarkers, and treatment dates. Confirm the record with the study team.
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10
Inclusion
- 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
- 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