Autism Spectrum DisorderEarly Phase 1NCT07433517
VGN-Ex05e in Patients With Autism Spectrum Disorder Associated With Severe Self-Injurious and Aggressive Behaviors
A Early Phase 1 study testing VGN-Ex05e in people with Autism Spectrum Disorder, run by Shanghai Jiao Tong University School of Medicine.
Trial summary
To evaluate the safety and tolerability of intracerebral injection of VGN-Ex05e in the nucleus accumbens of patients with autism spectrum disorder. Read more about this study
- Who is it for?
- Ages 7 to 18 years
- Conditions the study is for
- Autism Spectrum Disorder
- What is being tested?
- VGN-Ex05e
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- No sites listed yet
- Study dates
- Apr 2026to Oct 2027
- 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 7 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male or Female, age >7, ≤18
- DSM-5 diagnosis of Autism Spectrum Disorder, confirmed by ADOS-2
- Diagnosis ≥ 2 years, with at least 6 months of unsuccessful systematic behavioral interventions or training, or those unable to undergo training;
- Target symptoms and severity: Presence of recurrent self-injurious or aggressive behaviors within the last 3 months, with at least one of the following documented (meeting at least one criterion): Any self-injury item on the RBS-R scoring ≥ 3; Self-injury/aggression subscale of the BPI reaching one of the following criteria: frequency score ≥ 3, or severity score ≥ 3;
- Previous use of multiple medications (one or more antipsychotics and one or more mood stabilizers) with inadequate response, or those with severe adverse reactions, or those who cannot tolerate，and have discontinued medication for more than 5 half-lives or 1 month (whichever is longer) prior to screening, with the expectation that these medications will not be re-administered during the study period;
- Subjects/parents/guardians are able to understand the trial information, objectives, and risks as described in the informed consent form, are willing to comply with the clinical trial protocol, and can authorize the use of the subject's health information, voluntarily providing the signed and dated informed consent form;
- Subjects/parents/guardians are willing to act as information providers for the study, providing information on the subject's health status, cognitive, and physical abilities (including providing information for scales).
- Male and female subjects of childbearing potential must continuously and correctly use at least one highly effective method of contraception from the screening period through at least 1 year after administration.
Who cannot join
- Refractory epilepsy within the last 12 months prior to screening (≥2 seizures per month or status epilepticus), or significant EEG discharges during the screening period with a high-risk assessment;
- Co-occurring schizophrenia, bipolar disorder, or severe depressive disorder;
- Severe cardiac, hepatic, renal, or hematologic diseases;
- Coagulation disorders or bleeding tendencies (affecting surgical drug administration);
- Active infection at the time of screening, requiring systemic treatment;
- Co-occurring malignant tumors or history of tumors within the past 5 years；
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- Positive HIV antibody, active hepatitis C infection, active hepatitis B infection, syphilis positive, or active tuberculosis at the time of screening;
- clinically significant abnormal ECG;
- Any of the following laboratory test indicators meet the following criteria (those who meet the criteria and have a clear reason for retesting can recheck and confirm within one week): 1) Hemoglobin count is below the detection limit, or thrombocytopenia (< 100 × 10^9/L); 2) Alanine aminotransferase ≥ 3 × ULN and/or Aspartate aminotransferase ≥ 3 × ULN and/or Total bilirubin ≥ 2 × ULN; 3) Those with renal function impairment, defined as eGFR < 60 ml/min/1.73 m2; 4) Myocardial enzyme spectrum (Creatine kinase CK and Creatine kinase isoenzyme CK-MB) > 3 × ULN; 5) Any laboratory abnormal value with significant clinical significance that other researchers consider may interfere with the efficacy and safety data analysis of this study.
- previous history of stem cell therapy or gene therapy;
- pregnant or lactating.
- Any history of intracranial infection or traumatic brain injury within 6 months prior to screening.
- MRI evidence of space-occupying lesions, arteriovenous malformations, active intracranial hemorrhage or infarction, diffuse cerebral atrophy, or severe developmental malformations.
- Prior intracranial surgical scarring that interferes with the planned trajectory to the nucleus accumbens (NAc).
- Participation in another clinical trial within 3 months prior to screening.
- History of severe hypersensitivity or allergic reactions to anesthetic agents or any components of the cell product.
- Presence of cranial metallic materials or implanted devices that contraindicate MRI, cause significant imaging artifacts affecting surgical navigation, or other conditions that preclude MRI examination.
- Any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in the clinical trial.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: An Early-Phase Clinical Study to Evaluate the Tolerability, Safety, and Efficacy of VGN-Ex05e in Patients With Autism Spectrum Disorder (ASD) Associated With Severe, Treatment-Resistant Self-Injurious and Aggressive Behaviors
To evaluate the safety and tolerability of intracerebral injection of VGN-Ex05e in the nucleus accumbens of patients with autism spectrum disorder.
Read the detailed description
This study is a single-center, open-label, Investigator-Initiated Trial. The entire trial is expected to enroll 6 subjects. The initial plan of this study is to explore two doses: 2.0×10^6 cells and 6.0×10^6 cells. During the trial, the SRC (Safety Review Committee) will conduct a comprehensive assessment and may adjust the dose as necessary.
- Conditions
- Autism Spectrum Disorder
- Being tested
- VGN-Ex05e
- Medical subject headings
- Autism Spectrum Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VGN-Ex05eBiological
Products derived from human embryonic stem cells for the treatment of allogeneic nerve cells
| Study group | Role | What they receive |
|---|---|---|
| VGN-Ex05e | Experimental | Stereotactic intracerebral injection of VGN-Ex05e solution into the nucleus accumbens with 2.0×10^6 cells or 6.0×10^6 cells. Receives: Biological: VGN-Ex05e |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of adverse events
Measured: through study completion, an average of 1 year
Secondary outcomes (5)
change of Repetitive Behavior Scale-Revised score
Measured: through study completion, an average of 1 year
change of Childhood Autism Rating Scale score
Measured: through study completion, an average of 1 year
change of Autism Diagnostic Observation Schedule-Second Edition score
Measured: through study completion, an average of 1 year
change of Aberrant Behavior Checklist score
Measured: through study completion, an average of 1 year
change of Behavior Problems Index score
Measured: through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2026
- Study startApr 2026 (estimated)
- Primary completionOct 2027 (estimated)
- Study completionOct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- More trials run by Shanghai Jiao Tong University School of MedicineThe 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
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Jiao Tong University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Shanghai Vitalgen BioPharma Co., Ltd.
- Responsible party
- Principal investigator, Yunhai Song, Shanghai Children's Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07433517
- Sponsor's protocol ID
- VGN-Ex05e-001
- Status last verified
- Feb 2026
- First submitted
- 11 Feb 2026
- Last updated
- 25 Feb 2026
- 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-Ex05e is fully approved.
Study at a glance
From the registry record, last updated by the sponsor on 25 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Early-Phase Clinical Study to Evaluate the Tolerability, Safety, and Efficacy of VGN-Ex05e in Patients With Autism Spectrum Disorder (ASD) Associated With Severe, Treatment-Resistant Self-Injurious and Aggressive Behaviors
- ClinicalTrials.gov ID
- NCT07433517
- Acronym / sponsor ID
- VGN-Ex05e-001
- Conditions
- Autism Spectrum Disorder
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Shanghai Jiao Tong University School of MedicineUniversity, hospital or other
- Collaborators
- Shanghai Vitalgen BioPharma Co., Ltd.
- Study start
- Apr 2026 (estimated)
- Primary completion
- Oct 2027 (estimated)
- Study completion
- Oct 2027 (estimated)
- First posted
- 25 Feb 2026 (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.
To evaluate the safety and tolerability of intracerebral injection of VGN-Ex05e in the nucleus accumbens of patients with autism spectrum disorder.
Detailed description
This study is a single-center, open-label, Investigator-Initiated Trial. The entire trial is expected to enroll 6 subjects. The initial plan of this study is to explore two doses: 2.0×10^6 cells and 6.0×10^6 cells. During the trial, the SRC (Safety Review Committee) will conduct a comprehensive assessment and may adjust the dose as necessary.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VGN-Ex05e | Experimental | Biological: VGN-Ex05eStereotactic intracerebral injection of VGN-Ex05e solution into the nucleus accumbens with 2.0×10^6 cells or 6.0×10^6 cells. |
Who may qualify
- Ages
- 7 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Male or Female, age >7, ≤18
- DSM-5 diagnosis of Autism Spectrum Disorder, confirmed by ADOS-2
- Diagnosis ≥ 2 years, with at least 6 months of unsuccessful systematic behavioral interventions or training, or those unable to undergo training;
- Target symptoms and severity: Presence of recurrent self-injurious or aggressive behaviors within the last 3 months, with at least one of the following documented (meeting at least one criterion): Any self-injury item on the RBS-R scoring ≥ 3; Self-injury/aggression subscale of the BPI reaching one of the following criteria: frequency score ≥ 3, or severity score ≥ 3;
- Previous use of multiple medications (one or more antipsychotics and one or more mood stabilizers) with inadequate response, or those with severe adverse reactions, or those who cannot tolerate，and have discontinued medication for more than 5 half-lives or 1 month (whichever is longer) prior to screening, with the expectation that these medications will not be re-administered during the study period;
Key exclusion criteria (18 listed)
- Refractory epilepsy within the last 12 months prior to screening (≥2 seizures per month or status epilepticus), or significant EEG discharges during the screening period with a high-risk assessment;
- Co-occurring schizophrenia, bipolar disorder, or severe depressive disorder;
- Severe cardiac, hepatic, renal, or hematologic diseases;
- Coagulation disorders or bleeding tendencies (affecting surgical drug administration);
- Active infection at the time of screening, requiring systemic treatment;
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-Ex05eProducts derived from human embryonic stem cells for the treatment of allogeneic nerve cells | Biological | VGN-Ex05e |
Primary outcome
Incidence and severity of adverse events
Time frame: through study completion, an average of 1 year
5 secondary outcomes
change of Repetitive Behavior Scale-Revised score
Time frame: through study completion, an average of 1 year
change of Childhood Autism Rating Scale score
Time frame: through study completion, an average of 1 year
change of Autism Diagnostic Observation Schedule-Second Edition score
Time frame: through study completion, an average of 1 year
change of Aberrant Behavior Checklist score
Time frame: through study completion, an average of 1 year
change of Behavior Problems Index score
Time frame: through study completion, an average of 1 year
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 25 Feb 2026.
- Individual participant dataSharing planned: Yes. IPD will be shared with other researchers when VGN-Ex05e 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.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing VGN-Ex05e in people with Autism Spectrum Disorder, run by Shanghai Jiao Tong University School of Medicine.
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 8 inclusion and 18 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 25 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
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 Yunhai Song (18930830752, songyunhai3021@126.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: through study completion, an average of 1 year). 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 25 Feb 2026.