Amyotrophic Lateral Sclerosis (ALS)Phase 1/2NCT07743268
Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS
A Phase 1/2 study testing nL-TARDB-006 in people with Amyotrophic Lateral Sclerosis (ALS), run by n-Lorem Foundation at 2 sites in United States.
Trial summary
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- nL-TARDB-006
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 2 sitesUnited States
- Study dates
- 19 Feb 2026to Feb 2028
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites
Columbia University, Irving Medical Center
New York, New York, 10032
Washington University
St Louis, Missouri, 63110
About this study
Official title: An Open-label Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to TARDBP (TDP-43) Genetic Mutation
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.
Read the detailed description
This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- nL-TARDB-006
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
nL-TARDB-006Drug
Personalized antisense oligonucleotide
| Study group | Role | What they receive |
|---|---|---|
| Open Label | Experimental | Not described Receives: Drug: nL-TARDB-006 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical Functioning
Change from baseline at 12-months post nL-TARDB-006 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).
Measured: Baseline to 12 months
Clinical Functioning
Change from baseline at 12-months post nL-TARDB-006 administration in Slow Vital Capacity (SVC)
Measured: Baseline to 12 months
Clinical Functioning
Change from baseline at 12-months post nL-TARDB-006 administration in Handheld Dynamometry (HHD)
Measured: Baseline to 12 months
Clinical Functioning
Change from baseline at 12-months post nL-TARDB-006 administration in scores on Edinburgh Cognitive and Behavioral ALS Screen (ECAS).
Measured: Baseline to 12 months
Clinical Functioning
Change from baseline at 12-months post nL-TARDB-006 administration in scores on ALS Cognitive Behavioral Screen (ALS-CBS).
Measured: Baseline to 12 months
Clinical Functioning
Change from baseline at 12-months post nL-TARDB-006 administration in scores on Amyotrophic Lateral Sclerosis Assessment Questionnaire 5 (ALSAQ-5).
Measured: Baseline to 12 months
Survival
Change from baseline at 12-months post nL-TARDB-006 administration in survival status
Measured: Baseline to 12 months
Disease Biomarkers
Change from baseline at 12-months post nL-TARDB-006 administration in serum/plasma and CSF neurofilament light chain levels
Measured: Baseline to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Aug 2026
- Study start19 Feb 2026
- Primary completionFeb 2028 (estimated)
- Study completionFeb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- n-Lorem Foundation
- Sponsor type
- University, hospital or other
- Collaborators
- Columbia University, Washington University School of Medicine
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07743268
- Sponsor's protocol ID
- NLF-TARDB-006-A
- Status last verified
- Jul 2026
- First submitted
- 29 Jul 2026
- Last updated
- 3 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No