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Phase 1/2a Study of SNUG01 for ALS

This study is testing a new gene therapy called SNUG01 for adults with Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease. The main goals are to see if SNUG01 is safe and well-tolerated when given into the spinal fluid (intrathecal administration), and to find the best dose that shows some early signs of helping with ALS. You might be able to join if you are between 18 and 80 years old and have a confirmed diagnosis of ALS. The study plans to enroll 21 participants, but the current recruitment status is unclear.

Study design
This is a single-arm, open-label study, meaning all participants will receive SNUG01 and everyone involved will know what treatment is being given. It's a Phase 1/2a study, which typically means it's an early-stage study looking at safety and initial effectiveness.
What's involved
Participants will receive SNUG01 and be monitored for up to one year after the treatment. Specific details about visits, procedures, or tests are not provided.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for safety and preliminary effectiveness for up to one year after receiving SNUG01.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07169175

A Phase Ⅰ/Ⅱa Study of SNUG01 in Adult Subjects With ALS

Not Yet Recruiting
PHASE1Ages 18–80InterventionalTreatment
SineuGene Therapeutics Co., Ltd.
~21 participants
Updated 2025-09-11 on ClinicalTrials.gov
What's tested:SNUG01

At a glance

Recruiting sites
0 of 4 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
The safety and tolerability of SNUG01 administered by intrathecal.
Measured over up to 1 year after administration
+1 more outcome measured

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Explore related trials

Amyotrophic Lateral Sclerosis

NCT07169175

Where you'd take part

This study runs at 4 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Fujian Medical University Union Hospital

    Fuzhou, Fujian, Chinano site contact published

  • Massachusetts General Hospital

    Boston, Massachusettsno site contact published

  • Peking University Third Hospital

    Beijing, Beijing Municipality, Chinano site contact published

  • Second Affiliated Hospital Zhejiang University School of Medicine

    Hangzhou, Zhejiang, Chinano site contact published

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

Ji Shen
[email protected]
+8617274855306
Email the study team

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Eligibility criteria

Inclusion

Subjects who are able to provide written informed consent form (ICF).
Subjects who are males or females must be ≥ 18 years and ≤ 80 years of age at the screening visit.
Subjects who have clinically definite ALS, clinically probable ALS, or clinically probable-laboratory supported ALS as specified in the revised version of the El Escorial World Federation of Neurology criteria.
Subjects must have an ALS disease duration (from first symptom onset to the screening visit) ≤ 2 years.
Subjects with a body mass index (BMI) ≥ 19 kg/m2 at the screening visit.
Subjects whose percent-predicted Forced Vital Capacity (%FVC) is ≥ 70% or percent-predicted Slow Vital Capacity (%SVC) is ≥ 60%, adjusted for sex, age, and height at the screening visit.
The ALSFRS-R score ≥ 30 during the screening period, and the three respiratory scores (dyspnea, upright respiration, and respiratory insufficiency) must be full marks.

Exclusion

Serum Anti-AAV9 neutralizing antibody titer ≥ 1:100.
Current or previous exposure to gene therapy, stem cell products, and solid organ transplantation.
Subjects who have implanted or are estimated to require a diaphragmatic pacing system during the study period.
Any thromboembolic event, such as deep vein thrombosis, pulmonary arteriovenous embolism, and jugular vein embolism, has occurred within 6 months before the administration.
Suffering from autoimmune diseases or ongoing immune-related therapy, except intranasal, inhalation, ocular, topical, intra-articular corticosteroid therapy or corticosteroid physiological replacement therapy.
Active or chronic uncontrolled infection within 4 weeks before the administration, deemed unacceptable in the discretion of the investigator.
Evidence of human immunodeficiency virus (HIV) and treponema pallidum (TP) infection, as documented by the treatment for HIV or TP, or by HIV or TP antibodies positivity at the screening visit.
Has a positive serum pregnancy test at screening (females of childbearing potential only), a positive urine or serum pregnancy test at baseline (Day -1. females of childbearing potential only), or is nursing.

What this trial measures

  • The safety and tolerability of SNUG01 administered by intrathecal.up to 1 year after administration

    Dose-limiting toxicity (DLT), adverse events (AEs), clinically significant changes in vital signs, physical examination, clinical laboratory tests (hematology, biochemistry, urinalysis, coagulation, cardiac enzymes, etc.) and 12-lead ECG, etc.

  • To recommend the optimal expansion dose of SNUG01 that demonstrates acceptable safety with maximum preliminary efficacy administered by IT.up to 1 years