Amyotrophic Lateral SclerosisPhase 1/2NCT06977451
Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS
A Phase 1/2 study testing nL-CHCHD-001 in people with Amyotrophic Lateral Sclerosis, 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 CHCHD10 Read more about this study
- Who is it for?
- Ages 63 to 63 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- nL-CHCHD-001
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 24 Jun 2024to Jun 2027
- 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 63 to 63 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
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.
United States1 site
Columbia University, Irving Medical Center
New York, New York, 10032
About this study
Official title: 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
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
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- nL-CHCHD-001
- 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-CHCHD-001Drug
Personalized Antisense Oligonucleotide
| Study group | Role | What they receive |
|---|---|---|
| Open Label | Experimental | Not described Receives: Drug: nL-CHCHD-001 |
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-CHCHD-001 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R). A maximum score of 48 represents normal functioning, and the minimum score is 0.
Measured: Baseline to 12 months
Motor Functioning
Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Slow Vital Capacity (SVC) Performance.
Measured: Baseline to 12 months
Motor Functioning
Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Handheld Dynamometry (HHD).
Measured: Baseline to 12 months
Clinical Functioning
Change from baseline at 12-months post nL-CHCHD-001 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-CHCHD-001 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-CHCHD-001 administration in scores on Amyotrophic Lateral Sclerosis Assessment Questionnaire 5 (ALSAQ-5)
Measured: Baseline to 12 months
Secondary outcomes (5)
Disease Biomarkers
Change from baseline at 12-months post nL-CHCHD-001 administration in serum and cerebrospinal fluid neurofilament light chain levels
Measured: Baseline to 12 months
Safety and Tolerability
Incidence and Severity of Adverse Events
Measured: Baseline to 12 months
Safety and Tolerability
Emergent abnormalities in physical exam
Measured: Baseline to 12 months
Safety and Efficacy
Emergent abnormalities in neurological exam
Measured: Baseline to 12 months
Safety and Efficacy
Emergent abnormalities in safety labs (CSF, chemistry, hematology, coagulation, and urinalysis)
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 posted18 May 2025
- Study start24 Jun 2024
- Primary completionJun 2027 (estimated)
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies 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 (actual)
Sponsor and investigators
- Lead sponsor
- n-Lorem Foundation
- Sponsor type
- University, hospital or other
- Collaborators
- Columbia University
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06977451
- Sponsor's protocol ID
- AAAV1774
- Status last verified
- Jul 2026
- First submitted
- 9 May 2025
- Last updated
- 13 Jul 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06977451
- Acronym / sponsor ID
- AAAV1774
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 1 participants (actual)
- Lead sponsor
- n-Lorem FoundationUniversity, hospital or other
- Collaborators
- Columbia University
- Study start
- 24 Jun 2024 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 18 May 2025 (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 CHCHD10
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-CHCHD-001 |
Who may qualify
- Ages
- 63 to 63 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- 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.
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-CHCHD-001Personalized Antisense Oligonucleotide | Drug | Open Label |
Primary outcome
Clinical Functioning
Time frame: Baseline to 12 months
Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R). A maximum score of 48 represents normal functioning, and the minimum score is 0.
Motor Functioning
Time frame: Baseline to 12 months
Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Slow Vital Capacity (SVC) Performance.
Motor Functioning
Time frame: Baseline to 12 months
Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Handheld Dynamometry (HHD).
Clinical Functioning
Time frame: Baseline to 12 months
Change from baseline at 12-months post nL-CHCHD-001 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-CHCHD-001 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-CHCHD-001 administration in scores on Amyotrophic Lateral Sclerosis Assessment Questionnaire 5 (ALSAQ-5)
5 secondary outcomes
Disease Biomarkers
Time frame: Baseline to 12 months
Safety and Tolerability
Time frame: Baseline to 12 months
Safety and Tolerability
Time frame: Baseline to 12 months
Safety and Efficacy
Time frame: Baseline to 12 months
Safety and Efficacy
Time frame: Baseline to 12 months
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 13 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. This study is not recruiting at the moment.
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-CHCHD-001 in people with Amyotrophic Lateral Sclerosis, 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: under way, not looking for participants, as of the sponsor's last update on 13 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 6 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 13 Jul 2026.