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Enrolling By Invitation
NCT07095686
Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS
Conditions: Amyotrophic Lateral Sclerosis
Sex: All
Ages: 18 Years – N/A
Healthy volunteers: No
Phase: PHASE1, PHASE2
Enrollment: 9
Sponsor: n-Lorem Foundation
Location: Columbia University, Irving Medical Center New York New York
Summary
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for individual participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10
Eligibility Criteria
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
Source: ClinicalTrials.gov (NCT07095686). StuddyBuddy aggregates publicly available trial information.
