nL-TARD-001 for ALS
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new drug, nL-TARD-001, for individuals with amyotrophic lateral sclerosis (ALS), a severe neurological disorder affecting nerve cells in the brain and spinal cord. The focus is on a specific genetic type of ALS linked to a TARDBP gene variant. The trial aims to determine if this personalized treatment can help manage the condition. Ideal participants have a genetically confirmed neurological disorder and can travel to the study site for follow-ups. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the chance to be among the first to receive this new treatment.
Do I need to stop taking my current medications for the trial?
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 current medications with the trial team.
Do I need to stop taking my current medications for the trial?
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 current medications with the trial team.
Is there any evidence suggesting that nL-TARD-001 is likely to be safe for humans?
Research shows that certain treatments, like nL-TARD-001, are under study for their potential to treat amyotrophic lateral sclerosis (ALS). These treatments, known as antisense oligonucleotides (ASOs), target specific genetic changes linked to ALS. Past studies have generally found similar ASO treatments to be safe for patients, with usually manageable side effects.
Although no direct data exists yet for nL-TARD-001, it is helpful to note that another ASO, Tofersen, has already received FDA approval for a different type of ALS and has some known side effects. This approval suggests that nL-TARD-001 might also be safe and tolerable. However, as this is an early-phase trial, researchers are closely monitoring its safety. Early-phase trials emphasize safety, so any serious side effects will be identified and addressed.
Participants are encouraged to discuss potential risks and benefits with their healthcare providers to make informed decisions about joining the trial.12345Why do researchers think this study treatment might be promising for ALS?
Researchers are excited about nL-TARD-001 for ALS because it offers a novel approach compared to existing treatments like riluzole and edaravone. Unlike current options that primarily aim to slow disease progression, nL-TARD-001 targets a specific protein pathway involved in ALS, potentially addressing the underlying cause more directly. This unique mechanism could lead to more effective management of symptoms and improved quality of life for patients.
What evidence suggests that nL-TARD-001 might be an effective treatment for ALS?
Research has shown that special molecules called antisense oligonucleotides (ASOs), such as nL-TARD-001, can target specific genetic changes linked to amyotrophic lateral sclerosis (ALS). These ASOs attach to faulty genetic material and stop its harmful effects. nL-TARD-001 is designed for individuals with a specific change in the TARDBP gene, known to cause ALS. Early studies suggest that this method could slow the disease by reducing harmful proteins produced by this genetic change. Although direct human data remains limited, the personalized approach of nL-TARD-001 offers a promising new way to treat genetic forms of ALS.12367
Are You a Good Fit for This Trial?
This trial is specifically for one person with a rare form of ALS caused by a TARDBP gene mutation. The participant's eligibility will be determined based on specific inclusion and exclusion criteria set by the researchers.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a personalized antisense oligonucleotide (ASO) treatment
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- nL-TARD-001
Trial Overview
The study is testing an individualized antisense oligonucleotide (ASO) drug, nL-TARD-001, tailored to treat ALS in this single participant with a genetic variation in TARDBP.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Find a Clinic Near You
Who Is Running the Clinical Trial?
n-Lorem Foundation
Lead Sponsor
The Methodist Hospital Research Institute
Collaborator
Columbia University
Collaborator
Citations
Study Details | NCT07095712 | Personalized Antisense ...
Change from baseline at 12-months post nL-TARD-001 administration in ALS Cognitive Behavioral Screen (ALS-CBS) score, Baseline to 12 months.
nL-TARD-001 for ALS · Info for Participants
This Phase 1 & 2 medical study run by n-Lorem Foundation needs participants to evaluate whether nL-TARD-001 will have tolerable side effects & efficacy for ...
N=1 Collaborative: advancing customized nucleic acid ...
amyotrophic lateral sclerosis, frontotemporal dementia (FTD), 2, nL-TARD-001, Allele selective RNase H, IT, n-Lorem, Unpublished and provided ...
Top Amyotrophic Lateral Sclerosis Clinical Trials | Power
This study intends to evaluate the safety and efficacy of STR04 administered intravenously in participants with Amyotrophic Lateral Sclerosis. ... nL-TARD-001 for ...
5.
htsyndication.com
htsyndication.com/us-fed-news/article/clinical-trial%3A-personalized-antisense-oligonucleotide-therapy-for-a-single-participant-with-tardbp-als/555746486Personalized Antisense Oligonucleotide Therapy for A ...
Condition: Amyotrophic Lateral Sclerosis Intervention: DRUG: nL-TARD-001. Personalized antisense oligonucleotide. Recruitment Status ...
Nano-rare Patient Colloquium
nL-TARD-001. TARDBP. Allele ... Antisense oligonucleotide as a therapeutic approach in amyotrophic lateral sclerosis, Nature Medicine (2022).
Serious Neurologic Adverse Events in Tofersen Clinical Trials ...
Tofersen is approved for the treatment of amyotrophic lateral sclerosis (ALS) due to superoxide dismutase 1 mutations (SOD1‐ALS).
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