VGN-Ex05e in Patients With Autism Spectrum Disorder Associated With Severe Self-Injurious and Aggressive Behaviors

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Autism Spectrum Disorder
• Intervention / Treatment: Biological: VGN-Ex05e

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 Type: Interventional
• Enrollment (Estimated): 6
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Yunhai Song; Phone Number: 18930830752; Email: songyunhai3021@126.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Male or Female, age >7, ≤18
2. DSM-5 diagnosis of Autism Spectrum Disorder, confirmed by ADOS-2
3. Diagnosis ≥ 2 years, with at least 6 months of unsuccessful systematic behavioral interventions or training, or those unable to undergo training;
4. 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;
5. 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;
6. 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;
7. 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).
8. 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.

Exclusion Criteria:

1. 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;
2. Co-occurring schizophrenia, bipolar disorder, or severe depressive disorder;
3. Severe cardiac, hepatic, renal, or hematologic diseases;
4. Coagulation disorders or bleeding tendencies (affecting surgical drug administration);
5. Active infection at the time of screening, requiring systemic treatment;
6. Co-occurring malignant tumors or history of tumors within the past 5 years；
7. Positive HIV antibody, active hepatitis C infection, active hepatitis B infection, syphilis positive, or active tuberculosis at the time of screening;
8. clinically significant abnormal ECG;
9. 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.
10. previous history of stem cell therapy or gene therapy;
11. pregnant or lactating.
12. Any history of intracranial infection or traumatic brain injury within 6 months prior to screening.
13. MRI evidence of space-occupying lesions, arteriovenous malformations, active intracranial hemorrhage or infarction, diffuse cerebral atrophy, or severe developmental malformations.
14. Prior intracranial surgical scarring that interferes with the planned trajectory to the nucleus accumbens (NAc).
15. Participation in another clinical trial within 3 months prior to screening.
16. History of severe hypersensitivity or allergic reactions to anesthetic agents or any components of the cell product.
17. 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.
18. Any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in the clinical trial.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: VGN-Ex05e; Stereotactic intracerebral injection of VGN-Ex05e solution into the nucleus accumbens with 2.0×10^6 cells or 6.0×10^6 cells.	Biological: VGN-Ex05e; Products derived from human embryonic stem cells for the treatment of allogeneic nerve cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence and severity of adverse events	through study completion, an average of 1 year

Secondary Outcome Measures

Outcome Measure	Time Frame
change of Repetitive Behavior Scale-Revised score	through study completion, an average of 1 year
change of Childhood Autism Rating Scale score	through study completion, an average of 1 year
change of Autism Diagnostic Observation Schedule-Second Edition score	through study completion, an average of 1 year
change of Aberrant Behavior Checklist score	through study completion, an average of 1 year
change of Behavior Problems Index score	through study completion, an average of 1 year

Collaborators and Investigators

• Sponsor: Shanghai Jiao Tong University School of Medicine
• Collaborators: Shanghai Vitalgen BioPharma Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Estimated): April 1, 2026
• Primary Completion (Estimated): October 1, 2027
• Study Completion (Estimated): October 1, 2027

Study Registration Dates

• First Submitted: February 11, 2026
• First Submitted That Met QC Criteria: February 21, 2026
• First Posted (Actual): February 25, 2026

Study Record Updates

• Last Update Posted (Actual): February 25, 2026
• Last Update Submitted That Met QC Criteria: February 21, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Child Development Disorders, Pervasive; Autism Spectrum Disorder
• Other Study ID Numbers: VGN-Ex05e-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: IPD will be shared with other researchers when VGN-Ex05e is fully approved.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No