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

NCT07433517 | Not Yet Recruiting Early Phase 1

• 1 other identifier: VGN-Ex05e-001
• Study Type: interventional
• Target: 6
• Locations: 0 countries
• Sites: N/A

Brief Summary

To evaluate the safety and tolerability of intracerebral injection of VGN-Ex05e in the nucleus accumbens of patients with autism spectrum disorder.

Conditions

Autism Spectrum Disorder

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of adverse events

  through study completion, an average of 1 year

Secondary Outcomes (5)

• 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

Study Arms (1)

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.

Biological: VGN-Ex05e

Interventions

VGN-Ex05e BIOLOGICAL

Products derived from human embryonic stem cells for the treatment of allogeneic nerve cells

VGN-Ex05e

Eligibility Criteria

• Age: 7 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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；
• 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.

Sponsors & Collaborators

• Shanghai Jiao Tong University School of Medicine: lead
• Shanghai Vitalgen BioPharma Co., Ltd.: collaborator

MeSH Terms

Conditions

Autism Spectrum Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Central Study Contacts

Yunhai Song

CONTACT

18930830752; songyunhai3021@126.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief Physician

Study Record Dates

• First Submitted: February 11, 2026
• First Posted: February 25, 2026
• Study Start: April 1, 2026
• Primary Completion (Estimated): October 1, 2027
• Study Completion (Estimated): October 1, 2027
• Last Updated: February 25, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share