Antisense Oligonucleotide Therapy for ALS

Age: 18+

Sex: Male

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 explores a new treatment for individuals with a specific genetic type of amyotrophic lateral sclerosis (ALS), a serious condition affecting nerve cells in the brain and spinal cord. The treatment, nL-CHCHD-001, is an antisense oligonucleotide therapy that targets a genetic issue linked to ALS using a personalized approach. It is designed for those with a genetic variant in the CHCHD10 gene. Individuals genetically diagnosed with a neurological disorder related to ALS who can travel to the study site for follow-ups might be suitable candidates for this trial. As a Phase 1, Phase 2 trial, the research aims to understand how the treatment works in people and measure its effectiveness in an initial, smaller group, offering participants a chance to contribute to groundbreaking ALS research.

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 study team.

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

Research shows that treatments called antisense oligonucleotides (ASOs), such as nL-CHCHD-001, are under investigation for their potential to treat amyotrophic lateral sclerosis (ALS). ASO treatments target specific genetic mutations, like the CHCHD10 variant, which can lead to ALS. Although detailed safety information for nL-CHCHD-001 is not yet available, other ASO drugs have shown promise and have been generally well-tolerated by patients in similar studies.

This trial is in its early stages, focusing primarily on assessing the treatment's safety. Early trials typically involve small groups of participants, allowing researchers to closely monitor their responses to the treatment. While ASO treatments approved for other conditions suggest potential safety, each new treatment may differ. Therefore, participants will be carefully monitored to ensure their safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about nL-CHCHD-001 for ALS because it represents a novel approach to tackling this challenging condition. Unlike standard treatments like riluzole and edaravone, which primarily aim to slow disease progression, nL-CHCHD-001 is an antisense oligonucleotide therapy that targets specific genetic mutations linked to ALS. This precision targeting has the potential to modify the disease directly at its genetic source, which could lead to more effective management of symptoms and possibly alter the disease's course. This groundbreaking mechanism of action is what makes nL-CHCHD-001 stand out and why there is hope for its success.

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

Research has shown that antisense oligonucleotides (ASOs) may help treat amyotrophic lateral sclerosis (ALS), particularly in patients with the CHCHD10 gene variant. In this trial, participants will receive the personalized ASO drug, nL-CHCHD-001. Early results suggest it has slowed ALS progression in some patients. Specifically, one study found that nine out of eleven patients with CHCHD10-ALS who received nL-CHCHD-001 showed improvements in movement and survival. This treatment targets the disease at the genetic level, aiming to correct the faulty gene causing ALS symptoms. Although more research is needed, these early findings offer hope for individuals with this specific genetic mutation. ^{1 2 3 4 6}

Who Is on the Research Team?

Björn E. Oskarsson, M.D.

Principal Investigator

Mayo Clinic

Are You a Good Fit for This Trial?

This trial is specifically for one person with a rare form of ALS (Lou Gehrig's disease) caused by a CHCHD10 gene mutation. The participant must have this genetic variant and meet other undisclosed criteria.

Inclusion Criteria

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

My neurological disorder is confirmed through genetic testing.

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

Exclusion Criteria

Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures

I haven't taken any experimental drugs recently.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Administration of personalized antisense oligonucleotide (ASO) treatment

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 study is testing an individualized drug called nL-CHCHD-001, which is an antisense oligonucleotide designed to target the genetic cause of 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+

Mayo Clinic

Collaborator

Trials

3,427

Recruited

3,221,000+

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

nL-CHCHD-001 - Drug Targets, Indications, Patents 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. 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 ...

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

ALS News: Breakthrough Drug Shows Promising Results ... Recent ALS news reveals a groundbreaking personalized drug therapy that has demonstrated remarkable efficacy in slowing ALS progression.

6. globalgenes.org globalgenes.org/raredaily/nih-grant-funds-n-lorem-treatments-for-multiple-patients-with-ultra-rare-form-of-als/

NIH Grant Funds n-Lorem Treatments for Multiple Patients ... Mutant CHCHD10 protein is thought to be toxic to neurons, leading to ALS and related disorders like frontotemporal dementia. As a therapeutic ...