Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS

Running, not enrolling · Phase 1/Phase 2

Conditions studied: Amyotrophic Lateral Sclerosis

In brief

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

Key facts

Study ID

NCT06977451

Run by

n-Lorem Foundation

People needed

1

Starts

2024-06-24

Expected to finish

2027-06-01

Last updated by the study team

2026-07-13

Who can join

Age: 63 and older, up to 63. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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

Where it is running

• Columbia University, Irving Medical Center — New York, New York, United States

Full record on ClinicalTrials.gov

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