NCT07095712CompletedPhase 1/2
Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS
An Open-label Multicenter, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to TARDBP (TDP-43) Genetic Mutation
Source: ClinicalTrials.gov · Last updated 9 Apr 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 TARDBP.
- Status (registry)
- Completed
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 1 (actual)
- First submitted
- 12 May 2025
- Start date
- 25 Nov 2024
- Primary completion date
- 16 Dec 2025
- Lead sponsor
- n-Lorem Foundation
- Diseases
- ALS (motor neurone disease)
- NCT number
- NCT07095712
What you commit to
- Study type: Interventional · Phase 1/2
- Participants enrolled: 1
- From 25 Nov 2024 to the primary completion date, 16 Dec 2025
- 2 sites 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 site 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: 49 years to 49 years · Sex: Female · Does not accept healthy volunteers
Sites (2)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Houston Methodist Coordinator of this site? Confirm its status | Houston | United States | Unknown 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.