Antisense Oligonucleotide for ALS

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: n-Lorem Foundation

Must not be taking: Investigational drugs

Disqualifiers: Other conditions, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test a new treatment called nL-CHCHD-001, an antisense oligonucleotide, for amyotrophic lateral sclerosis (ALS), a serious condition affecting nerve cells in the brain and spine. The treatment is personalized for individuals with ALS due to a specific genetic change in the CHCHD10 gene. The trial will involve one participant in an open-label setup, where both the researchers and the participant know the treatment being administered. This trial suits someone with a genetically confirmed neurological disorder who can travel for study visits. As a Phase 1, Phase 2 trial, it focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot have used an investigational medication recently. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that antisense oligonucleotides (ASOs) have been used safely in other contexts. An ASO blocks harmful proteins in diseases like ALS. Studies of similar treatments have found that patients generally tolerate them well. Most side effects are mild, such as headaches or nausea, while serious side effects are rare but possible. As this trial is in its early stages, it emphasizes ensuring the treatment's safety. For any concerns, discussing them with the trial team is advisable. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for ALS, which primarily focus on managing symptoms and slowing disease progression, nL-CHCHD-001 offers a novel approach by utilizing antisense oligonucleotides. These molecules can specifically target and modify RNA to reduce the production of harmful proteins associated with ALS. This targeted mechanism has the potential to address the underlying causes of the disease, offering hope for more effective management of ALS. Researchers are excited because this approach could lead to significant improvements in patient outcomes compared to existing therapies.

What evidence suggests that this treatment might be an effective treatment for ALS?

Research has shown that a new treatment, nL-CHCHD-001, might help people with ALS (amyotrophic lateral sclerosis), particularly those with a specific gene variant called CHCHD10. Early results suggest this treatment could improve movement and possibly extend life expectancy. In a small study, nine out of eleven patients with this type of ALS showed positive results after receiving nL-CHCHD-001. While the average survival time after an ALS diagnosis is about three years, some people live longer, and this treatment aims to improve those chances. Although more research is needed, these early findings offer hope for those with this specific form of ALS. ^{1 3 4 6 7}

Are You a Good Fit for This Trial?

This trial is specifically for one person with a rare form of ALS caused by a CHCHD10 gene mutation. There are no detailed eligibility criteria provided, as it's personalized.

Inclusion Criteria

Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s)

I can travel to the study location and follow the study's requirements.

My neurological condition is confirmed by genetic testing.

Exclusion Criteria

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with ALS

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• nL-CHCHD-001

Trial Overview The trial is testing an individualized antisense oligonucleotide (ASO) drug called nL-CHCHD-001, tailored to treat ALS in this single participant.

How Is the Trial Designed?

Group I: Open Label Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

n-Lorem Foundation

Lead Sponsor

Trials

5

Recruited

5+

Columbia University

Collaborator

Trials

1,529

Recruited

2,832,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07095686

Personalized Antisense Oligonucleotide for Participants ... Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised ( ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11651183/

Rethinking antisense oligonucleotide therapeutics for ... This review provides an overview of the current status and discusses the prospects of antisense oligonucleotides treatment for amyotrophic lateral sclerosis.

3. nlorem.org nlorem.org/multi-year-grant-from-national-institutes-of-health-urgent-network-funds-treatment-of-patients-with-ultra-rare-chchd10-als/

Multi-year Grant from National Institutes of Health URGenT ... Beginning in March 2024, nine of eleven CHCHD10-ALS patients enrolled in Silence ALS have been treated with nL-CHCHD-001 – one at the Mayo ...

4. globalrph.com globalrph.com/2025/10/als-news-breakthrough-drug-shows-promising-results-in-clinical-trials/

ALS News: Breakthrough Drug Shows Promising Results ... Notably, while the average post-diagnosis survival remains approximately three years, about 20% of patients live five years or longer, with 5% ...

5. mayo.edu mayo.edu/research/clinical-trials/cls-20566166

Clinical Trials The purpose of this study is to evaluate the clinical efficacy of nL-CHCHD-001 in motor function and survival in a single ALS participant., and ...

6. synapse.patsnap.com synapse.patsnap.com/drug/b9e7df68e8ad4efebd0dcbaf06033747

nL-CHCHD-001 - Drug Targets, Indications, Patents Mutant CHCHD10 protein is thought to be toxic to neurons, leading to ALS and related disorders like frontotemporal dementia. As a therapeutic ...

7. ctv.veeva.com ctv.veeva.com/study/personalized-antisense-oligonucleotide-therapy-for-a-single-participant-with-chchd10-als

Personalized Antisense Oligonucleotide Therapy for A Single ... This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) ...