A Phase I/II Clinical Study to Evaluate the Tolerability, Safety, and Efficacy of VGN-R08b Intra-cerebroventricular injection in Parkinson's Disease Patients with GBA1 Mutations
Male or female, aged 30 to 70 years (inclusive) at the time of signing the informed consent form.
Documented GBA1-mutant Parkinson's disease, confirmed by medical history: meeting the International Parkinson and Movement Disorder Society (MDS) diagnostic criteria for idiopathic Parkinson's disease, with the presence of at least one pathogenic GBA1 gene mutation (confirmed by investigator interpretation).
Glucocerebrosidase (GCase) enzyme activity below the normal range, as measured from past or screening dried blood spot tests.
Hoehn-Yahr stage of 3 to 4 in the "OFF" state, an MDS-UPDRS Part III (motor examination) score ≥33 points in the "OFF" state, and the ability to walk without relying on a walker or wheelchair.
Subject has any of the following diseases or disease history
Patients with atypical or secondary parkinsonian syndromes, including but not limited to those caused by trauma, brain tumors, infections, cerebrovascular diseases, or other neurological disorders; or symptoms confirmed by the investigator to be induced by drugs, chemicals, or toxins; or those with other serious neurological conditions deemed by the investigator to significantly compromise the safety and efficacy evaluation of the investigational drug.
Patients with active infections (including viral infections such as HBV, HCV, or syphilis) or a history of severe infections within 12 weeks prior to screening (e.g., pneumonia, sepsis, or central nervous system infections such as meningitis or encephalitis).
Patients with severe liver disease, severe immunodeficiency, or autoimmune diseases within 6 months prior to screening, or those requiring long-term immunosuppressive therapy.
Single group
VGN-R08b 4.2×10^13 vg
DrugIntracerebroventricular injection
VGN-R08b 8.4×10^13 vg
DrugIntracerebroventricular injection
VGN-R08b 1.68×10^14 vg
DrugIntracerebroventricular injection
Number of Adverse Events (AEs), Serious Adverse Events (SAEs)Vital signs
Vital signs, physical examination, laboratory test will be monitored after drug injection
Pharmacodynamic indicator
Changes in glucocerebrosidase (GCase) activity after medication.
Changes in Glucosylceramide (Lyso-GL1) Levels
changes in glucosylceramide (Lyso-GL1) levels in peripheral blood and cerebrospinal fluid (CSF) after medication
Disease indicators
Diary: Daily medication dosage (equivalent dose of levodopa), changes in high-quality "on" time (referring to the time without dyskinesia or dyskinesia not affecting daily life) and "off" time;
Unified Parkinson's Disease Rating Scale (UPDRS) and Hoehn-Yahr (H-Y) Staging
The Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS) consists of four parts: Part I (Non-motor Experiences of Daily Living), Part II (Motor Experiences of Daily Living), Part III (Motor Examination), and Part IV (Motor Complications). Each item on the scale is scored on a five-point scale of 0, 1, 2, 3, and 4, with higher scores indicating more severe PD symptoms. The scores of each part and the total score of the four parts will specifically reflect the disease progression of Parkinson's disease. The scoring sheet will include MDS-UPDRS-III "off" and "on" scores, and Hoehn-Yahr "off" staging.
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