Status:
ACTIVE_NOT_RECRUITING
Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS
Lead Sponsor:
n-Lorem Foundation
Collaborating Sponsors:
Columbia University
Conditions:
Amyotrophic Lateral Sclerosis
Eligibility:
All Genders
63-63 years
Phase:
PHASE1
PHASE2
Brief 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 ...
Eligibility Criteria
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 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
- 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
Key Trial Info
Start Date :
June 24 2024
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 1 2026
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT06977451
Start Date
June 24 2024
End Date
June 1 2026
Last Update
July 25 2025
Active Locations (1)
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1
Columbia University, Irving Medical Center
New York, New York, United States, 10032