Amyotrophic Lateral Sclerosis (ALS)Phase 1/2NCT07169175
A Phase Ⅰ/Ⅱa Study of SNUG01 in Adult Subjects With ALS
A Phase 1/2 study testing SNUG01 in people with Amyotrophic Lateral Sclerosis (ALS), run by SineuGene Therapeutics Co., Ltd. at 4 sites in 2 countries.
Trial summary
The goal of this clinical trial is to evaluate the safety, tolerability and preliminary efficacy of SNUG01 in in adult subjects with Amyotrophic Lateral Sclerosis (ALS). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- SNUG01
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 1 Dec 2025to 30 Sep 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
4 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
4 sites in 2 countries
China3 sites
Peking University Third Hospital
Beijing, Beijing Municipality, 100000
Fujian Medical University Union Hospital
Fuzhou, Fujian, 350000
Second Affiliated Hospital Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310000
United States1 site
Massachusetts General Hospital
Boston, Massachusetts, 02467
About this study
Official title: A Multicenter, Open-label, Single-arm, Dose Escalation and Expansion, Phase I/IIa Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of SNUG01 in Adult Subjects With Amyotrophic Lateral Sclerosis (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 Sclerosis (ALS).
Read the detailed description
This is a 52-week, multicenter, open-label, single-arm, dose escalation and expansion Phase Ⅰ/Ⅱa study of SNUG01 in adults with ALS. Safety will be the primary focus, with secondary emphasis on immunogenicity, PK and preliminary clinical efficacy of SNUG01.
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- SNUG01
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SNUG01Drug
AAV (adeno-associated virus) Gene therapy
| Study group | Role | What they receive |
|---|---|---|
| 3 Ascending Dose Levels, Single Injection by Intrathecal(IT) | Experimental | Not described Receives: Drug: SNUG01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The safety and tolerability of SNUG01 administered by intrathecal.
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.
Measured: up to 1 year after administration
To recommend the optimal expansion dose of SNUG01 that demonstrates acceptable safety with maximum preliminary efficacy administered by IT.
Measured: up to 1 years
Secondary outcomes (3)
Immunogenicity of SNUG01
Change of anti-SG001 antibody and anti-AAV9 neutralizing antibody in serum, change of anti-AAV9 neutralizing antibody in CSF.
Measured: up to 1 years
PK of SNUG01 (Biodistribution and Viral Shedding)
SG001 protein level in CSF, viral vector DNA in serum, viral shedding in saliva, urine, and feces.
Measured: up to 1 years
Preliminary Clinical Efficacy of SNUG01
Change in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) , the Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) , the percent-predicted Forced Vital Capacity (%FVC) / percent-predicted Slow Vital Capacity (%SVC) and muscle strength, the Patient Health Questionnaire (PHQ-9) and NfL level in CSF and serum from baseline to Week 48 after administration.
Measured: up to 1 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Sep 2025
- Study start1 Dec 2025 (estimated)
- Primary completion30 Mar 2028 (estimated)
- Study completion30 Sep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 21 participants (estimated)
Sponsor and investigators
- Lead sponsor
- SineuGene Therapeutics Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07169175
- Sponsor's protocol ID
- SNUG01-101
- Status last verified
- Sep 2025
- First submitted
- 8 Aug 2025
- Last updated
- 11 Sep 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-label, Single-arm, Dose Escalation and Expansion, Phase I/IIa Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of SNUG01 in Adult Subjects With Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT07169175
- Acronym / sponsor ID
- SNUG01-101
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- SineuGene Therapeutics Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 1 Dec 2025 (estimated)
- Primary completion
- 30 Mar 2028 (estimated)
- Study completion
- 30 Sep 2028 (estimated)
- First posted
- 11 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical trial is to evaluate the safety, tolerability and preliminary efficacy of SNUG01 in in adult subjects with Amyotrophic Lateral Sclerosis (ALS).
Detailed description
This is a 52-week, multicenter, open-label, single-arm, dose escalation and expansion Phase Ⅰ/Ⅱa study of SNUG01 in adults with ALS. Safety will be the primary focus, with secondary emphasis on immunogenicity, PK and preliminary clinical efficacy of SNUG01.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 3 Ascending Dose Levels, Single Injection by Intrathecal(IT) | Experimental | Drug: SNUG01 |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
Key exclusion criteria (8 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SNUG01AAV (adeno-associated virus) Gene therapy | Drug | 3 Ascending Dose Levels, Single Injection by Intrathecal(IT) |
Primary outcome
The safety and tolerability of SNUG01 administered by intrathecal.
Time frame: 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.
Time frame: up to 1 years
3 secondary outcomes
Immunogenicity of SNUG01
Time frame: up to 1 years
PK of SNUG01 (Biodistribution and Viral Shedding)
Time frame: up to 1 years
Preliminary Clinical Efficacy of SNUG01
Time frame: up to 1 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing SNUG01 in people with Amyotrophic Lateral Sclerosis (ALS), run by SineuGene Therapeutics Co., Ltd. at 4 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 11 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in China, United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ji Shen (+8617274855306, patient@sineugene.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The safety and tolerability of SNUG01 administered by intrathecal. (time frame: up to 1 year after administration). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Sep 2025.