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Active Not Recruiting
NCT06977451
Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS
Conditions: Amyotrophic Lateral Sclerosis
Sex: All
Ages: 63 Years – 63 Years
Healthy volunteers: No
Phase: PHASE1, PHASE2
Enrollment: 1
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 a single participant 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 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
Source: ClinicalTrials.gov (NCT06977451). StuddyBuddy aggregates publicly available trial information.
