Amyotrophic Lateral Sclerosis (ALS)Early Phase 1NCT06849609
A Study to Evaluate the Tolerability, Safety and Efficacy of VGN-R13 in Patients with ALS
A Early Phase 1 study testing VGN-R13 in people with Amyotrophic Lateral Sclerosis (ALS), run by Hong Chen at 1 site in China.
Trial summary
The purpose of this trial is to evaluate safety and efficacy of intrathecal delivery of VGN-R13 as a treatment of Amyotrophic Lateral Sclerosis (ALS). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- VGN-R13
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteChina
- Study dates
- 16 Jan 2025to Jun 2026
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Fully understand the purpose and risks of the study and voluntarily provide a signed and dated informed consent form.
- Aged ≥18 years, male or female;
- A diagnosis of ALS according to the World Federation of Neurology El Escorial criteria (revised according to the Airlie House Conference 1998 [Brooks 2000]), and confirmed through genetic diagnosis to exclude pathogenic mutations in the superoxide dismutase 1 (SOD1) and/or FUS genes;
- The duration of the disease from the first symptom (any ALS symptom) prior to the screening must be less than 2 years (inclusive);
- Forced Vital Capacity (FVC) adjusted for gender, age, and height (sitting position) ≥50% of the predicted value.
- Discontinued riluzole for more than five half-lives prior to screening (and is not expected to resume during the study) or has been on a stable dose of riluzole for ≥30 days and continue to maintain this dose during the study period.
- Discontinued edaravone for more than five half-lives prior to screening (and is not expected to resume edaravone during the study) or is receiving the standard edaravone treatment regimen at screening and has maintained a stable dose for ≥60 days (two treatment cycles) prior to administration, and continue to maintain this dose during the study period.
Who cannot join
- 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.
- Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that is not managed optimally and could place a participant at an increased risk for intraoperative or postoperative bleeding. These could include, but are not limited to, anatomical factors at or near the LP site (e.g., vascular abnormalities, neoplasms, or other abnormalities) and underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., hemophilia, Von Willebrand's disease, liver disease).
- Current severe liver or kidney or cardiovascular disease or coagulation dysfunction, autoimmune deficiency, or uncontrolled autoimmune disease or need immunosuppressive long-term treatment, poorly controlled diabetes (HBA1C ≥7% at screening) or high blood pressure, presence of sever gastrointestinal ulcers or history of gastrointestinal bleeding;
- Presence of active infection that needs system treatment;
- Presence or history of malignant tumors within 5 years prior to screening;
- Significant cognitive impairment, clinical dementia, or unstable psychiatric illness, including psychosis, suicidal ideation, suicide attempt, or untreated major depression ≤ 90 days, as determined by the Investigator.
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- Current using medications including, drugs, herbal or OTC medications that strongly inhibit or induce CYP3A4 or P-glycoprotein (P-gp), e.g., metoclopramide, grapefruit juice, ketoconazole, erythromycin;
- Received another drug for the treatment of ALS disease (including but not limited to sodium phenylbutyrate (PB), taurine diol (TURSO), tauroursodeoxycholic acid (TUDCA) within 1 month before the first dose) or ursodeoxycholic acid (UDCA), biologics or Other investigational drugs with a discontinuation period shorter than five half-lives;
- Anticipated need, in the opinion of the Investigator, for administration of any antiplatelet or anticoagulant medication (e.g., clopidogrel) for 7 days before or 48 hours after an LP；
- Received systemic immunosuppressive therapy within 3 months prior to screening;
- Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter；
- Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter；
- Participation in gene therapy or stem cell transduction therapy at any time prior to screening；
- Positive test result for HIV, positive treponemal antibody for syphilis, Active hepatitis B or hepatitis C infection, Active tuberculosis infection;
- Clinically significant abnormalities in hematology or clinical chemistry parameters, as determined by the Investigator, which would render the participant unsuitable for enrollment;
- Clinically significant, as determined by the Investigator, 12-lead ECG abnormalities, including corrected QT interval using Fridericia's correction method of > 450 ms for males and > 470 ms for females;
- History of systemic hypersensitivity reaction to investigational product, the excipients contained in the formulation, or prophylactic immunosuppressant;
- Contraindicated use of corticosteroids and sirolimus;
- History of drug abuse or alcoholism within ≤ 6 months of study enrollment that would limit participation in the study, as determined by the Investigator;
- Alcohol abuse (drinking more than 14 units of alcohol per week) or smoking more than 5 cigarettes per day on average in the 6 months prior to screening;
- Pregnant or breastfeeding women;
- Other unspecified reasons that, in the opinion of the Investigator or Sponsor, make the participant unsuitable for enrollment;
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: An Open, Dose-Escalation Early Phase Clinical Study to Evaluate the Tolerability, Safety, and Efficacy of Intrathecal of VGN-R13 in Patients with Amyotrophic Lateral Sclerosis (ALS)
The purpose of this trial is to evaluate safety and efficacy of intrathecal delivery of VGN-R13 as a treatment of Amyotrophic Lateral Sclerosis (ALS).
Read the detailed description
The study will evaluate safety and efficacy of gene therapy in Amyotrophic Lateral Sclerosis (ALS) patients. ALS is a fatal central nervous system neurodegenerative disease. There is no effective treatment for ALS and current drug therapy has been unsuccessful in stabilizing or reversing this disease. Only supportive care is currently possible.
This study consists of screening period, treatment period and follow-up period. Eligible subjects will be enrolled. The day of administration set to be D1. Prophylactic immunosuppressive therapy will be initiated on D1. During the follow-up period (up to 52 weeks after administration), all the examinations will be completed based on the evaluation time point specified in the Schedule of Assessments table for efficacy and safety assessments until the End of Trial. Unscheduled visits may occur if the PI determines that they are necessary.
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- VGN-R13
- 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.
VGN-R13Biological
An adeno-associated viral vector 9 gene therapy product.
| Study group | Role | What they receive |
|---|---|---|
| VGN-R13 | Experimental | Up to two sequential VGN-R13 dose levels are planned. Each subject will receive one-time administration of VGN-R13 through intrathecal injection. Receives: Biological: VGN-R13 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of adverse events
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a product; the event will not need to have a causal relationship with the treatment.
Measured: up to 52 weeks
Secondary outcomes (6)
Change From Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score
The ALSFRS-R measures 4 functional domains, including respiratory, bulbar function, gross motor skills, and fine motor skills. There are 12 questions, each scored from 0 (no function) to 4 (full function), for a total possible score of 48. Scores decline with disease progression. ALSFRS-R scores calculated at diagnosis can be compared to scores throughout time to determine the speed of progression. Higher scores represent better function, negative change from baseline indicates disease progression.
Measured: up to 52 weeks
Change From Baseline in Percent Predicted Forced Vital Capacity (FVC)
Vital capacity is measured by means of an FVC test.
Measured: up to 52 weeks
Time to Death or Permanent Ventilation
Time to Death or Permanent Ventilation is defined as the time to the earliest occurrence of one of the following events that were adjudicated by an independent committee: Death; Permanent ventilation (≥22 hours of mechanical ventilation [invasive or noninvasive] per day for ≥21 consecutive days).
Measured: up to 52 weeks
Change From Baseline in Handheld Dynamometry (HHD) Megascore as Measured by the HHD Device
Quantitative muscle strength is evaluated using HHD, which tests isometric strength of multiple muscles using standard participant positioning.
Measured: up to 52 weeks
The antibody titer
Immunogenicity
Measured: up to 52 weeks
Immunogenicity
the number of subjects with positive anti-AAV9 in the blood and CSF.
Measured: up to 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2025
- Study start16 Jan 2025
- Primary completionMay 2026 (estimated)
- Study completionJun 2026 (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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hong Chen
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Hong Chen, Tongji Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06849609
- Sponsor's protocol ID
- VGN-R13-001
- Status last verified
- Dec 2024
- First submitted
- 23 Feb 2025
- Last updated
- 17 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: IPD will be shared with other researchers when VGN-R13 is fully approved.
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open, Dose-Escalation Early Phase Clinical Study to Evaluate the Tolerability, Safety, and Efficacy of Intrathecal of VGN-R13 in Patients with Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT06849609
- Acronym / sponsor ID
- VGN-R13-001
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Hong ChenUniversity, hospital or other
- Study start
- 16 Jan 2025 (actual)
- Primary completion
- May 2026 (estimated)
- Study completion
- Jun 2026 (estimated)
- First posted
- 27 Feb 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 purpose of this trial is to evaluate safety and efficacy of intrathecal delivery of VGN-R13 as a treatment of Amyotrophic Lateral Sclerosis (ALS).
Detailed description
The study will evaluate safety and efficacy of gene therapy in Amyotrophic Lateral Sclerosis (ALS) patients. ALS is a fatal central nervous system neurodegenerative disease. There is no effective treatment for ALS and current drug therapy has been unsuccessful in stabilizing or reversing this disease. Only supportive care is currently possible.
This study consists of screening period, treatment period and follow-up period. Eligible subjects will be enrolled. The day of administration set to be D1. Prophylactic immunosuppressive therapy will be initiated on D1. During the follow-up period (up to 52 weeks after administration), all the examinations will be completed based on the evaluation time point specified in the Schedule of Assessments table for efficacy and safety assessments until the End of Trial. Unscheduled visits may occur if the PI determines that they are necessary.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VGN-R13 | Experimental | Biological: VGN-R13Up to two sequential VGN-R13 dose levels are planned. Each subject will receive one-time administration of VGN-R13 through intrathecal injection. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Fully understand the purpose and risks of the study and voluntarily provide a signed and dated informed consent form.
- Aged ≥18 years, male or female;
- A diagnosis of ALS according to the World Federation of Neurology El Escorial criteria (revised according to the Airlie House Conference 1998 [Brooks 2000]), and confirmed through genetic diagnosis to exclude pathogenic mutations in the superoxide dismutase 1 (SOD1) and/or FUS genes;
- The duration of the disease from the first symptom (any ALS symptom) prior to the screening must be less than 2 years (inclusive);
- Forced Vital Capacity (FVC) adjusted for gender, age, and height (sitting position) ≥50% of the predicted value.
Key exclusion criteria (22 listed)
- 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.
- Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that is not managed optimally and could place a participant at an increased risk for intraoperative or postoperative bleeding. These could include, but are not limited to, anatomical factors at or near the LP site (e.g., vascular abnormalities, neoplasms, or other abnormalities) and underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., hemophilia, Von Willebrand's disease, liver disease).
- Current severe liver or kidney or cardiovascular disease or coagulation dysfunction, autoimmune deficiency, or uncontrolled autoimmune disease or need immunosuppressive long-term treatment, poorly controlled diabetes (HBA1C ≥7% at screening) or high blood pressure, presence of sever gastrointestinal ulcers or history of gastrointestinal bleeding;
- Presence of active infection that needs system treatment;
- Presence or history of malignant tumors within 5 years prior to screening;
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 |
|---|---|---|
| VGN-R13An adeno-associated viral vector 9 gene therapy product. | Biological | VGN-R13 |
Primary outcome
Incidence and severity of adverse events
Time frame: up to 52 weeks
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a product; the event will not need to have a causal relationship with the treatment.
6 secondary outcomes
Change From Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score
Time frame: up to 52 weeks
Change From Baseline in Percent Predicted Forced Vital Capacity (FVC)
Time frame: up to 52 weeks
Time to Death or Permanent Ventilation
Time frame: up to 52 weeks
Change From Baseline in Handheld Dynamometry (HHD) Megascore as Measured by the HHD Device
Time frame: up to 52 weeks
The antibody titer
Time frame: up to 52 weeks
Immunogenicity
Time frame: up to 52 weeks
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 17 Mar 2025.
- Individual participant dataSharing planned: Yes. IPD will be shared with other researchers when VGN-R13 is fully approved.
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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.
1 site 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 VGN-R13 in people with Amyotrophic Lateral Sclerosis (ALS), run by Hong Chen at 1 site 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 7 inclusion and 22 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: looking for participants now, as of the sponsor's last update on 17 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 Hong Chen (13296508243, chenhong1129@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of adverse events (time frame: up to 52 weeks). 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 17 Mar 2025.