NCT07703462Enrolling by invitationPhase 1/2
Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS
An Open-label Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to TARDBP (TDP-43) Genetic Mutation
Source: ClinicalTrials.gov · Last updated 14 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 TARDBP.
- Status (registry)
- Enrolling by invitation
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 1 (estimated)
- First submitted
- 9 Jul 2026
- Start date
- Dec 2026
- Primary completion date
- Dec 2028
- Lead sponsor
- n-Lorem Foundation
- Diseases
- ALS (motor neurone disease)
- NCT number
- NCT07703462
What you commit to
- Study type: Interventional · Phase 1/2
- Planned participants: 1
- From Dec 2026 to the primary completion date, Dec 2028
- 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 participants 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
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 |
|---|---|---|---|
| Jefferson Health Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.