Amyotrophic Lateral Sclerosis (ALS)Early Phase 1ALSNCT06645197
An IIT Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Injection of SNUG01 in Patients with Amyotrophic Lateral Sclerosis
A Early Phase 1 study testing SNUG01 in people with Amyotrophic Lateral Sclerosis (ALS), run by Peking University Third Hospital at 5 sites in China.
Trial summary
This is a multicenter, open-label, single-arm investigator-initiated clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of SNUG01 in patients 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
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 5 sitesChina
- Study dates
- 16 Oct 2024to 15 Oct 2029
- Phase
- Early Phase 1
- 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
- 1.Able to understand and voluntarily sign the informed consent form, the informed consent form must be signed before performing any clinical trail procedures.
- 2.18~80 years old (including 18 and 80 years old), both male and female. 3.Must have been diagnosed with clinically probable ALS, clinically possible laboratory-supported ALS, or clinically definite ALS according to the revised version of the El Escorial World Federation of Neurology criteria.
- 4.Less than or equal to 36 months (inclusive) after the onset of symptoms of amyotrophic lateral sclerosis.
- 5.Body Mass Index (BMI) ≥19 kg/m2. 6.The forced vital capacity (FVC) in the screening period is greater than or equal to 70% of the estimated vital capacity.
- 7.Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score ≥ 26 during the screening period, with scores of ≥ 4 in the three respiratory items (dyspnea, orthopnea, and respiratory insufficiency) on the ALSFRS-R.
- 8. Adequate organ function: Hematological: Neutrophil count≥1.5 × 10⁹/L, platelet count≥ 100 × 10⁹/L, Hemoglobin ≥90 g/L. Renal: Creatinine ≤1.5×ULN or creatinine clearance (CCr) ≥50 ml/min using the Cockcroft-Gault formula.
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- Hepatic: Total bilirubin (TBIL), alanine aminotransferase (ALT), aspartate aminotransferase (AST)≤2 × ULN, alkaline phosphatase (ALP) ≤ 3 × ULN.
- Coagulation: International Normalized Ratio (INR), Prothrombin Time (PT), Activated Partial Thromboplastin Time (APTT) ≤1.5 × ULN.
- 9.Subjects are not currently receiving riluzole or have been receiving a stable dose of riluzole for at least 4 weeks prior to the screening visit. Subjects treated with riluzole are expected to remain on the same dose throughout the study period.
- 9.Subjects are not currently treated with edaravone or are being treated with the approved standard regimen of edaravone. Subjects being treated with edaravone must have completed at least 1 cycle of treatment prior to the Screening Visit and are expected to continue treatment with edaravone throughout the study period.
- 10.Women of childbearing age must have a negative blood pregnancy test during the screening period and be non-lactating. Female subjects of childbearing age (women of childbearing age include premenopausal women and women within 2 years after menopause, except those who have undergone bilateral tubal ligation, complete oophorectomy or hysterectomy.) and male subjects whose partners are women of childbearing age must Agree to use effective contraceptive methods throughout the study period, such as abstinence, double barrier contraceptive methods, condoms, intrauterine devices and other non-drug contraceptive measures, and are not allowed to donate sperm or eggs.
Who cannot join
- Serum anti-AAV9 neutralizing antibody (Nab) titer \> 1:100 at the time of screening.
- There are contraindications to lumbar puncture during the screening period (including but not limited to skin infection at the administration site and signs or symptoms of increased intracranial pressure), receiving any active intrathecal therapy, and having implants for drainage of cerebrospinal fluid (CSF). Inserted shunt, presence of implanted central nervous system (CNS) cannula, or any condition that prevents CSF collection.
- There are other diseases related to motor neuron dysfunction (progressive bulbar palsy, primary lateral sclerosis, cervical spondylosis, lumbar spondylosis, etc., idiopathic inflammatory myopathy), which may confuse or cover up the diagnosis of ALS.
- Previously required invasive ventilation or tracheotomy due to ALS disease, or currently using non-invasive ventilation support with an average of ≥16 hours/day.
- Previous history of gene therapy, hematopoietic stem cell transplantation, and solid organ transplantation.
- The patient has poorly controlled acute or chronic respiratory diseases, including but not limited to: chronic obstructive pulmonary disease, severe asthma, severe pneumonia, active tuberculosis.
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- Those who have been implanted or the researchers estimate that they will need to implant a diaphragmatic pacing system during the study period.
- Any thromboembolic events occurred within 6 months before the first administration, such as deep vein thrombosis, pulmonary arteriovenous embolism, jugular vein embolism, etc..
- Received another drug for the treatment of ALS disease (including but not limited to sodium phenylbutyrate (PB), taurine diol (TURSO), tauroursodeoxycholic acid (TUDCA) within 4 weeks before the first dose ) or ursodeoxycholic acid (UDCA), biologics, etc. except riluzole and edaravone).
- Autoimmune disease (e.g., rheumatoid arthritis, systemic lupus erythematosus, inflammatory bowel disease, scleroderma, inflammatory myopathies, mixed connective tissue disease, overlap syndrome, etc.) within 30 days prior to the Screening Period, or ongoing immune-related therapy (e.g., corticosteroids, cyclosporine, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin, rituximab, leflunomide, hydroxychloroquine, interleukin 2 antagonists, etc.), except for intranasal, inhaled, ocular, topical topical, intra-articular corticosteroid therapy, or physiologic replacement therapy with corticosteroids.
- Suffering from active or uncontrolled infection (including but not limited to: infectious pneumonia, sepsis, herpes zoster infection) within 4 weeks before the first administration, or chronic bacterial infection that is considered unacceptable according to the investigator\'s judgment ( medical history such as tuberculosis).
- Have undergone major surgery within 4 weeks before the first administration, or have not recovered from previous treatment-related adverse events (AE) to ≤ grade 1 (CTCAE 5.0), except alopecia.
- Participated in another clinical study within 4 weeks before the first administration, unless it is an observational (non-intervention) clinical study.
- Any febrile illness occurred within 14 days before the first administration.
- Have been vaccinated within 14 days before the first dose.
- Patients with poorly controlled hypertension (refers to resting blood pressure after treatment: systolic blood pressure (SBP) \> 160 mmHg or diastolic blood pressure (DBP) \> 100 mmHg).
- Arterial oxygen saturation and/or finger pulse oxygen saturation \<93% after treatment with oxygen therapy or noninvasive respiratory support (\<16 hrs/day) at the time of screening, as determined by the investigator.
- Hypersensitivity to prednisolone, other glucocorticoids or their excipients.
- Positive hepatitis B surface antigen (HBsAg) or positive hepatitis B core antibody (anti-HBcAb) with peripheral blood HBV DNA titer ≥ 1.0E+02 IU/mL.; positive hepatitis C virus (HCV) antibody with positive HCV RNA results; positive human immunodeficiency virus (HIV) antibody; or positive treponemal antibody for syphilis.
- The following cardiovascular and cerebrovascular diseases, including but not limited to:
- QTc interval (corrected using Fridericia formula) average value: males QTc \> 450 ms, females QTc \> 470 ms.
- Echocardiogram showing left ventricular ejection fraction (LVEF) \< 50% or below the lower limit of normal (whichever is higher).
- History of heart failure of class III-IV (NYHA classification). Myocardial infarction, unstable angina, poorly controlled arrhythmia, cerebrovascular accident or transient ischemic attack occurred within 6 months before the first administration;
- Mental illness that may affect compliance with the test requirements: unstable mental illness, cognitive dysfunction, dementia or alcohol dependence, etc.
- Other conditions that the researcher thinks are not suitable for participating in this study.
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
China5 sites
Peking University Third Hospital
Beijing, Beijing Municipality, 100000
JI Shen, 15710013912, neurbj@126.com
West China Hospital of Sichuan University
Chengdu, Sichuan, 610000
JI Shen, 15710013912, neurbj@126.com
Fujian Medical University Union Hospital
Fujian, Fuzhou, 350000
JI Shen, 15710013912, neurbj@126.com
Second Affiliated Hospital Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310000
JI Shen, 15710013912, neurbj@126.com
JiangSu Provincce Hospital
Nanjing, Jiangsu, 210000
JI Shen, 15710013912, neurbj@126.com
About this study
Official title: A Multicenter, Single-Arm, Open-Label, IIT Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Injection of SNUG01 in Patients with Amyotrophic Lateral Sclerosis (ALS)
This is a multicenter, open-label, single-arm investigator-initiated clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of SNUG01 in patients with Amyotrophic Lateral Sclerosis (ALS).
Read the detailed description
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 weeks post-treatment (primary study period). After the primary study period, participants will be assessed for both safety and efficacy for up to a total of 5 years following SNUG01 treatment.
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- SNUG01
- Keywords
- Gene therapy, AAV (adeno-associated virus), ALS, Neuromuscular Diseases, Motor Neuron Disease, Nervous System Diseases
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Neuromuscular Diseases, Motor Neuron Disease, Nervous System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SNUG01Drug
Recombinant adeno-associated virus serotype 9 capsid containing human SG001 expression cassette
| Study group | Role | What they receive |
|---|---|---|
| 3 single Ascending Dose Levels | Experimental | The study will be open-label with an initial plan to explore 3 dose levels of SNUG01 in approximately 7 to 10 Participants in total. Each Participant will receive a single dose of SNUG01 delivered via intrathecal (IT) infusion and will be followed for up to 5 years after administration. Receives: Drug: SNUG01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety and tolerability of ascending doses of intrathecally administered SNUG01 in Participants with ALS
Safety and tolerability, including adverse events (AE), vital signs, physical examination, clinical laboratory tests (hematology, blood chemistry, urinalysis, coagulation, cardiac enzymes, etc.) and 12 electrocardiograms,etc.
Measured: Each visit in 1 year after administration
Secondary outcomes (5)
Characterization of Immune Response to SNUG01
Development of ADAs (antidrug antibodies) and neutralizing antibodies of SNUG01 and AAV9 in blood and cerebrospinal fluid ( CSF )
Measured: up to 1 years
Characterization of the Effect of intrathecally administered SNUG01
Change from Baseline in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), the Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale（ROADS）and the Amyotrophic Lateral Sclerosis Assessment Questionnaire 40 (ALSAQ 40) total scores to Month 3 and Month 6
Measured: 3 months, 6months
Characterization of the Effect of intrathecally administered SNUG01
The Change from baseline in forced vital capacity (FVC) to month 3, month 6 and month 12
Measured: 3 months, 6 months,12 months
Characterization of the Effect of intrathecally administered SNUG01
The change from baseline in Medical Research Council (MRC) on M3, M6 and M12 after IT administration of SNUG01.
Measured: 3 months, 6 months,12 months
Evaluate the long-term safety and tolerability of intrathecally administered SNUG01 in Participants with ALS
Number of participants with treatment-related adverse events and serious adverse events,including vital signs, physical examination, clinical laboratory tests (hematology, blood chemistry, urinalysis, coagulation, cardiac enzymes, etc.) and 12 electrocardiograms,etc.
Measured: up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Oct 2024
- Study start16 Oct 2024 (estimated)
- Primary completion15 Apr 2025 (estimated)
- Study completion15 Oct 2029 (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.
- More trials run by Peking University Third HospitalThe sponsor's other registered studies.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 7 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University Third Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- SineuGene Therapeutics Co., Ltd.
- Responsible party
- Principal investigator, Fan Dongsheng, Peking University Third Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06645197
- Sponsor's protocol ID
- CTP-SNUG01-IIT-02
- Acronym
- ALS
- Status last verified
- Sep 2024
- First submitted
- 11 Oct 2024
- Last updated
- 16 Oct 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Single-Arm, Open-Label, IIT Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Injection of SNUG01 in Patients with Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT06645197
- Acronym / sponsor ID
- ALS / CTP-SNUG01-IIT-02
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Keywords
- Gene therapy, AAV (adeno-associated virus), ALS, Neuromuscular Diseases, Motor Neuron Disease, Nervous System Diseases
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 7 participants (estimated)
- Lead sponsor
- Peking University Third HospitalUniversity, hospital or other
- Collaborators
- SineuGene Therapeutics Co., Ltd.
- Study start
- 16 Oct 2024 (estimated)
- Primary completion
- 15 Apr 2025 (estimated)
- Study completion
- 15 Oct 2029 (estimated)
- First posted
- 16 Oct 2024 (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.
This is a multicenter, open-label, single-arm investigator-initiated clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of SNUG01 in patients with Amyotrophic Lateral Sclerosis (ALS).
Detailed description
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 weeks post-treatment (primary study period). After the primary study period, participants will be assessed for both safety and efficacy for up to a total of 5 years following SNUG01 treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 3 single Ascending Dose Levels | Experimental | Drug: SNUG01The study will be open-label with an initial plan to explore 3 dose levels of SNUG01 in approximately 7 to 10 Participants in total. Each Participant will receive a single dose of SNUG01 delivered via intrathecal (IT) infusion and will be followed for up to 5 years after administration. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 1.Able to understand and voluntarily sign the informed consent form, the informed consent form must be signed before performing any clinical trail procedures.
- 2.18~80 years old (including 18 and 80 years old), both male and female. 3.Must have been diagnosed with clinically probable ALS, clinically possible laboratory-supported ALS, or clinically definite ALS according to the revised version of the El Escorial World Federation of Neurology criteria.
- 4.Less than or equal to 36 months (inclusive) after the onset of symptoms of amyotrophic lateral sclerosis.
- 5.Body Mass Index (BMI) ≥19 kg/m2. 6.The forced vital capacity (FVC) in the screening period is greater than or equal to 70% of the estimated vital capacity.
- 7.Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score ≥ 26 during the screening period, with scores of ≥ 4 in the three respiratory items (dyspnea, orthopnea, and respiratory insufficiency) on the ALSFRS-R.
Key exclusion criteria (25 listed)
- Serum anti-AAV9 neutralizing antibody (Nab) titer \> 1:100 at the time of screening.
- There are contraindications to lumbar puncture during the screening period (including but not limited to skin infection at the administration site and signs or symptoms of increased intracranial pressure), receiving any active intrathecal therapy, and having implants for drainage of cerebrospinal fluid (CSF). Inserted shunt, presence of implanted central nervous system (CNS) cannula, or any condition that prevents CSF collection.
- There are other diseases related to motor neuron dysfunction (progressive bulbar palsy, primary lateral sclerosis, cervical spondylosis, lumbar spondylosis, etc., idiopathic inflammatory myopathy), which may confuse or cover up the diagnosis of ALS.
- Previously required invasive ventilation or tracheotomy due to ALS disease, or currently using non-invasive ventilation support with an average of ≥16 hours/day.
- Previous history of gene therapy, hematopoietic stem cell transplantation, and solid organ transplantation.
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 |
|---|---|---|
| SNUG01Recombinant adeno-associated virus serotype 9 capsid containing human SG001 expression cassette | Drug | 3 single Ascending Dose Levels |
Primary outcome
To evaluate the safety and tolerability of ascending doses of intrathecally administered SNUG01 in Participants with ALS
Time frame: Each visit in 1 year after administration
Safety and tolerability, including adverse events (AE), vital signs, physical examination, clinical laboratory tests (hematology, blood chemistry, urinalysis, coagulation, cardiac enzymes, etc.) and 12 electrocardiograms,etc.
5 secondary outcomes
Characterization of Immune Response to SNUG01
Time frame: up to 1 years
Characterization of the Effect of intrathecally administered SNUG01
Time frame: 3 months, 6months
Characterization of the Effect of intrathecally administered SNUG01
Time frame: 3 months, 6 months,12 months
Characterization of the Effect of intrathecally administered SNUG01
Time frame: 3 months, 6 months,12 months
Evaluate the long-term safety and tolerability of intrathecally administered SNUG01 in Participants with ALS
Time frame: up to 5 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 16 Oct 2024.
- Individual participant dataSharing planned: No
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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.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing SNUG01 in people with Amyotrophic Lateral Sclerosis (ALS), run by Peking University Third Hospital at 5 sites in China.
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 11 inclusion and 25 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 16 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in China. 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 phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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 Peking University Third Hospital (15710013912, neurbj@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the safety and tolerability of ascending doses of intrathecally administered SNUG01 in Participants with ALS (time frame: Each visit in 1 year after administration). 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 16 Oct 2024.