Amyotrophic Lateral Sclerosis (ALS)Phase 1/2NCT07703462
Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS
A Phase 1/2 study testing nL-TARDB-002 in people with Amyotrophic Lateral Sclerosis (ALS), run by n-Lorem Foundation at 1 site in United States.
Trial summary
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. 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-002
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2026to Dec 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
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 a single participant 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 a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- nL-TARDB-002
- 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-002Drug
Personalized antisense oligonucleotide
| Study group | Role | What they receive |
|---|---|---|
| Open Label | Experimental | Not described Receives: Drug: nL-TARDB-002 |
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-002 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-002 administration in Slow Vital Capacity (SVC)
Measured: Baseline to 12 months
Clinical Functioning
Change from baseline at 12-months post nL-TARDB-002 administration in Handheld Dynamometry (HHD)
Measured: Baseline to 12 months
Clinical Functioning
Change from baseline at 12-months post nL-TARDB-002 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-002 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-002 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-002 administration in survival status
Measured: Baseline to 12 months
Disease Biomarkers
Change from baseline at 12-months post nL-TARDB-002 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 posted14 Jul 2026
- Study startDec 2026 (estimated)
- Primary completionDec 2028 (estimated)
- Study completionDec 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
- 1 participants (estimated)
Sponsor and investigators
- Lead sponsor
- n-Lorem Foundation
- Sponsor type
- University, hospital or other
- Collaborators
- Thomas Jefferson University
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07703462
- Sponsor's protocol ID
- NLF-TARDB-002-A
- Status last verified
- Jul 2026
- First submitted
- 9 Jul 2026
- Last updated
- 14 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to TARDBP (TDP-43) Genetic Mutation
- ClinicalTrials.gov ID
- NCT07703462
- Acronym / sponsor ID
- NLF-TARDB-002-A
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 1 participants (estimated)
- Lead sponsor
- n-Lorem FoundationUniversity, hospital or other
- Collaborators
- Thomas Jefferson University
- Study start
- Dec 2026 (estimated)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 14 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open Label | Experimental | Drug: nL-TARDB-002 |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| nL-TARDB-002Personalized antisense oligonucleotide | Drug | Open Label |
Primary outcome
Clinical Functioning
Time frame: Baseline to 12 months
Change from baseline at 12-months post nL-TARDB-002 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).
Clinical Functioning
Time frame: Baseline to 12 months
Change from baseline at 12-months post nL-TARDB-002 administration in Slow Vital Capacity (SVC)
Clinical Functioning
Time frame: Baseline to 12 months
Change from baseline at 12-months post nL-TARDB-002 administration in Handheld Dynamometry (HHD)
Clinical Functioning
Time frame: Baseline to 12 months
Change from baseline at 12-months post nL-TARDB-002 administration in scores on Edinburgh Cognitive and Behavioral ALS Screen (ECAS).
Clinical Functioning
Time frame: Baseline to 12 months
Change from baseline at 12-months post nL-TARDB-002 administration in scores on ALS Cognitive Behavioral Screen (ALS-CBS).
Clinical Functioning
Time frame: Baseline to 12 months
Change from baseline at 12-months post nL-TARDB-002 administration in scores on Amyotrophic Lateral Sclerosis Assessment Questionnaire 5 (ALSAQ-5).
Survival
Time frame: Baseline to 12 months
Change from baseline at 12-months post nL-TARDB-002 administration in survival status
Disease Biomarkers
Time frame: Baseline to 12 months
Change from baseline at 12-months post nL-TARDB-002 administration in serum/plasma and CSF neurofilament light chain levels
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jul 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing nL-TARDB-002 in people with Amyotrophic Lateral Sclerosis (ALS), run by n-Lorem Foundation at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 14 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinical Functioning (time frame: Baseline to 12 months). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jul 2026.