NCT06977451Active, not recruitingPhase 1/2
Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS
An Open-Label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to an Arg15Leu Pathogenic Variant in CHCHD10
Source: ClinicalTrials.gov · Last updated 13 Jul 2026
Brief summary (registry, in English)
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
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 1 (actual)
- First submitted
- 9 May 2025
- Start date
- 24 Jun 2024
- Primary completion date
- 30 Jun 2027
- Lead sponsor
- n-Lorem Foundation
- Diseases
- ALS (motor neurone disease)
- NCT number
- NCT06977451
What you commit to
- Study type: Interventional · Phase 1/2
- Participants enrolled: 1
- From 24 Jun 2024 to the primary completion date, 30 Jun 2027
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Use of an investigational medication within less than 5 half-lives of the drug at enrollment
Other illness
- Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures.
Who the study is looking for
- 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.
Age in the registry: 63 years to 63 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Columbia University Irving Medical Center Coordinator of this site? Confirm its status | New York | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.