ASD patients with Severe Self-Injurious / Aggression Behaviours
Conditions
Interventions
Experimental group 1:Stereotactic intracerebral injection of VGN-Ex05e solution into the nucleus accumbens, with 2.0×10^6 cells
Experimental group 2:Stereotactic intracerebral injection of VGN-Ex05e solution into the nucleus accumbens, with 6.0×10^6 cells
Sponsors
Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
Eligibility
Sex/Gender
All
Age
7 Years to 18 Years
Inclusion criteria
Inclusion criteria: 1. Male or Female, age >7, = 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
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 (= 3 × ULN and/or Aspartate aminotransferase >= 3 × ULN and/or Total bilirubin >= 2 × ULN; 3) Those with renal function impairment, defined as eGFR 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| safety and tolerability; | — |
Secondary
| Measure | Time frame |
|---|---|
| change of Repetitive Behavior Scale-Revised score;change of Childhood Autism Rating Scale score;change of Autism Diagnostic Observation Schedule-Second Edition score;change of Aberrant Behavior Checklist score;change of Behavior Problems Index score; | — |
Countries
China
Contacts
Public ContactSong Yunhai
Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
Outcome results
None listed