To evaluate the safety and tolerability of intracerebral injection of VGN-Ex05e in the nucleus accumbens of patients with autism spectrum disorder.
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;
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);
Shanghai Jiao Tong University School of Medicine
Lead sponsor
Shanghai Children's Medical Center
Sponsor institution
Shanghai Vitalgen BioPharma Co., Ltd.
Collaborator