Antisense Oligonucleotide for ALS

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: n-Lorem Foundation

Must not be taking: Investigational drugs

Disqualifiers: Neurological disorder, 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 tests a new treatment called nL-CHCHD-001, an antisense oligonucleotide, for individuals with a specific genetic form of ALS, a serious disease affecting the nerves that control muscles. The goal is to determine if this personalized drug can slow down or improve symptoms of ALS caused by a genetic variant in the CHCHD10 gene. It suits those with a genetically confirmed neurological disorder like ALS who can travel to the study site for regular check-ups. As a Phase 1, Phase 2 trial, the study focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group, offering participants a chance to be among the first to receive this potentially groundbreaking therapy.

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 nL-CHCHD-001 is likely to be safe for humans?

Research shows that drugs like nL-CHCHD-001, known as antisense oligonucleotides (ASOs), could offer a promising treatment for ALS. These drugs target and reduce harmful proteins in the body. Studies have found that ASOs are generally well-tolerated by people with similar conditions. Some patients have experienced mild side effects, such as headaches or reactions at the injection site, but serious side effects are rare.

In trials for other conditions, ASOs have been used safely, indicating a good safety record. However, since nL-CHCHD-001 remains in the early testing stages for ALS, researchers are closely monitoring its safety. This vigilance helps ensure that any potential risks are quickly identified and managed. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for ALS, which primarily include medications like riluzole and edaravone that aim to slow disease progression, nL-CHCHD-001 is unique because it uses antisense oligonucleotides to target the genetic causes of the disease directly. This innovative approach works by binding to specific RNA sequences to alter gene expression, potentially addressing the root cause of ALS rather than just managing symptoms. Researchers are excited about this treatment because it offers a new way to tackle the disease at the molecular level, which could lead to more effective outcomes for patients.

What evidence suggests that nL-CHCHD-001 might be an effective treatment for ALS?

Research has shown that certain treatments can target specific genetic issues related to amyotrophic lateral sclerosis (ALS). In this trial, participants will receive an open-label treatment with nL-CHCHD-001, designed for individuals with a particular change in the CHCHD10 gene. Early results from using this treatment have demonstrated improvements in movement and survival for those with this gene change. Specifically, a study with several patients showed it could slow the disease's progress. While more information is still being gathered, these early findings suggest that nL-CHCHD-001 might offer hope for people with this rare type of ALS. ^{1 3 4 6 7}

Are You a Good Fit for This Trial?

This trial is for individuals with ALS caused by a specific genetic change in CHCHD10. Participants must be able to travel to the study site, follow the study's procedures, and allow access to their medical records. Those who have used investigational drugs recently or have conditions that may hinder completing the study cannot join.

Inclusion Criteria

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

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

My neurological disorder is confirmed through 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

Participants receive a personalized antisense oligonucleotide (ASO) drug designed for individual participants with ALS due to a pathogenic variant in CHCHD10

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 nL-CHCHD-001, a personalized antisense oligonucleotide (ASO) drug tailored for each participant with ALS due to a mutation in CHCHD10. The ASO aims to target and modify gene expression related to ALS.

How Is the Trial Designed?

Group I: Open Label nL-CHCHD-001 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) ...