SNUG01 for ALS
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to test the safety and initial effects of a new treatment called SNUG01 for people with ALS, a disease affecting nerve cells in the brain and spinal cord. The study involves a single injection administered at three different dose levels to assess the body's response. It seeks participants who have experienced ALS symptoms for less than two years and can perform daily activities without severe breathing issues. As a Phase 1 trial, this research focuses on understanding how SNUG01 works in people, offering participants the opportunity to be among the first to receive this new treatment.
Will I have to stop taking my current medications?
The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is there any evidence suggesting that SNUG01 is likely to be safe for humans?
Research has shown that SNUG01 appears safe for people with Amyotrophic Lateral Sclerosis (ALS). Early studies have not identified any major safety concerns, and SNUG01 is designed to deliver a protective protein to the brain and spinal cord. The initial safety results are promising, indicating good tolerance among participants. Although more information is needed, these findings support further research in larger studies.12345
Why do researchers think this study treatment might be promising for ALS?
Most treatments for ALS, like riluzole and edaravone, aim to slow disease progression but often have limited impact on symptoms. However, SNUG01 is unique because it is administered through a single intrathecal injection, delivering the treatment directly to the spinal fluid, which may enhance its effectiveness. Researchers are excited because this method could potentially allow for more precise targeting of ALS-related pathways and offer a new mechanism of action that current oral or intravenous treatments don't provide. This innovative approach might lead to more significant improvements in patient outcomes compared to existing options.
What evidence suggests that SNUG01 might be an effective treatment for ALS?
Research has shown that SNUG01 is a gene therapy designed to deliver a protective protein to the central nervous system. Early studies suggest SNUG01 is likely safe and shows initial signs of efficacy, prompting further research. Importantly, SNUG01's wide-ranging protective effects could potentially benefit many ALS patients, not just those with specific genetic mutations. The therapy has received special recognition from health authorities, such as the FDA, highlighting its potential importance in treating ALS.12678
Are You a Good Fit for This Trial?
This trial is for adults with Amyotrophic Lateral Sclerosis (ALS). Specific eligibility criteria are not provided, but typically include factors like disease stage and overall health. People who meet certain conditions that the study outlines may be excluded.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Dose Escalation and Expansion
Participants receive ascending dose levels of SNUG01 via single intrathecal injection
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- SNUG01
Trial Overview
The trial is testing SNUG01's safety, how well patients can tolerate it, and its initial effectiveness in treating ALS. This involves observing any changes in the condition or symptoms of ALS after administering SNUG01.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Find a Clinic Near You
Who Is Running the Clinical Trial?
SineuGene Therapeutics Co., Ltd.
Lead Sponsor
Citations
NCT06645197 | An IIT Clinical Study to Evaluate the Safety ...
SNUG01 is a gene therapy designed to deliver a protective protein gene to the central nervous system. Safety will be the primary focus during the initial 24 ...
SineuGene Reports Positive Clinical Data for ALS Gene ...
“SNUG01 has shown an promising safety profile and early signals of efficacy, which warrant further investigation in larger studies.” Dr. Jia ...
Novel Gene Therapy for ALS cleared for global trials in ...
Crucially, unlike ALS therapies targeting specific genetic mutations, SNUG01's broad neuroprotective mechanism holds the potential to benefit ...
Amyotrophic lateral sclerosis in Mainland China
A Single-Arm, Open-Label Exploratory Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Injection of SNUG01 ...
FDA grants orphan drug status to ALS gene therapy SNUG01
The FDA granted orphan drug designation to Sineugene Therapeutics' SNUG01, a first-in-class experimental gene therapy for people with ALS.
6.
trial.medpath.com
trial.medpath.com/clinical-trial/cd9e082a6bbfe6db/nct07169175-snug01-safety-efficacy-als-adultsA Phase Ⅰ/Ⅱa Study of SNUG01 in Adult Subjects With ALS
The goal of this clinical trial is to evaluate the safety, tolerability and preliminary efficacy of SNUG01 in in adult subjects with Amyotrophic Lateral ...
SineuGene Garners Orphan Drug Designation for ALS ...
SineuGene's SNUG01 receives FDA orphan drug designation for ALS. News release. SineuGene Therapeutics. June 25, 2025. Accessed July 7, 2025 ...
An ongoing trial by SineuGene Therapeutics Co., Ltd.
... Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of SNUG01 in Adult Subjects With Amyotrophic Lateral Sclerosis (ALS). Results ...
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