Amyotrophic Lateral SclerosisPhase 1/2NCT06392126
Personalized Antisense Oligonucleotide Therapy 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 48 years and older, men only
- 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
- 16 Apr 2024to Apr 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 48 years and older
- Men only
- No healthy volunteers
- Adult, Older 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 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.
About this study
Official title: An Open-label Study of an Experimental Antisense Oligonucleotide Treatment for a Single Participant With Amyotrophic Lateral Sclerosis (ALS) Due to a 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
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 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).
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
Clinical Functioning
Change from baseline at 12-months post nL-CHCHD-001 administration in scores on vital capacity performance.
Measured: Baseline to 12 months
Disease biomarkers
Change from baseline at 12-months post nL-CHCHD-001 administration in serum/plasma and CSF neurofilament light chain levels
Measured: Baseline to 12 months
Survival
Change from baseline at 12-months post nL-CHCHD-001 administration in survival status
Measured: Baseline to 12 months
Secondary outcomes (4)
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 tolerability
Emergent abnormalities in neurological exam
Measured: Baseline to 12 months
Safety and tolerability
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 posted30 Apr 2024
- Study start16 Apr 2024
- Primary completionApr 2027 (estimated)
- Study completionApr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Mayo Clinic
- Responsible party
- Sponsor
- Investigators
- Björn E. Oskarsson, M.D., Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06392126
- Sponsor's protocol ID
- 23-011476
- Status last verified
- Jul 2026
- First submitted
- 24 Apr 2024
- Last updated
- 13 Jul 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Cousin MA, Dagli AI, Mignon L, Shah JS, Prudencio M, Gendron TF, Jain A, Udine E, van Blitterswijk M, Middlebrooks EH, Donahue MH, Dagher JE, Watzlawik JO, Springer W, Parekh T, McEachin ZT, Petrucelli L, Oskarsson B. N-of-1 investigational study of a novel antisense oligonucleotide drug in CHCHD10-related ALS shows early signs of efficacy. Med. 2026 Sep 14:101295. doi: 10.1016/j.medj.2026.101295. Online ahead of print.
PubMed 42735682
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 Study of an Experimental Antisense Oligonucleotide Treatment for a Single Participant With Amyotrophic Lateral Sclerosis (ALS) Due to a Pathogenic Variant in CHCHD10
- ClinicalTrials.gov ID
- NCT06392126
- Acronym / sponsor ID
- 23-011476
- 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
- Mayo Clinic
- Study start
- 16 Apr 2024 (actual)
- Primary completion
- Apr 2027 (estimated)
- Study completion
- Apr 2027 (estimated)
- First posted
- 30 Apr 2024 (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
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 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
- 48 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older 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 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-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).
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).
Clinical Functioning
Time frame: Baseline to 12 months
Change from baseline at 12-months post nL-CHCHD-001 administration in scores on vital capacity performance.
Disease biomarkers
Time frame: Baseline to 12 months
Change from baseline at 12-months post nL-CHCHD-001 administration in serum/plasma and CSF neurofilament light chain levels
Survival
Time frame: Baseline to 12 months
Change from baseline at 12-months post nL-CHCHD-001 administration in survival status
4 secondary outcomes
Safety and tolerability
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 tolerability
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.
- Derived from this study: PubMed 42735682Cousin MA, Dagli AI, Mignon L, Shah JS, Prudencio M, Gendron TF, Jain A, Udine E, van Blitterswijk M, Middlebrooks EH, Donahue MH, Dagher JE, Watzlawik JO, Springer W, Parekh T, McEachin ZT, Petrucelli L, Oskarsson B. N-of-1 investigational study of a novel antisense oligonucleotide drug in CHCHD10-related ALS shows early signs of efficacy. Med. 2026 Sep 14:101295. doi: 10.1016/j.medj.2026.101295. Online ahead of print.
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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.
Björn E. Oskarsson, M.D. principal investigator
Mayo Clinic
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?
On the ClinicalTrials.gov record for NCT06392126, which also lists 1 linked publication.
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.