A phase I/II clinical study to evaluate the tolerance, safety and efficacy of VGN-R08b intracerebroventricular injection in patients with type III Gaucher's disease
The signing of the informed consent form should be completed when the subject is at least 2 years old but less than 18 years old.
The subject has a documented medical history of Gaucher disease confirmed by GCase enzyme activity testing, and has a double GBA1 gene mutation.
According to the investigator's assessment, the neurological signs and/or symptoms are consistent with type III Gaucher disease.
At the time of enrollment, the subject has horizontal eye movement disorders (including gaze paralysis, or delayed or absent saccades), but there is no severe motor dysfunction resulting in bedridden status.
There are other serious neurological disorders that may cause symptoms of Gaucher disease or interfere with the research objectives;
There are severe internal organ damages caused by Gaucher disease, which, after evaluation by the researchers, are considered to pose unacceptable risks to the subjects, or interfere with the subjects' research compliance, or interfere with the execution of the trial;
Long-term ventilation or long-term nasogastric feeding (long-term ventilation is defined as: requiring tracheotomy for respiratory assistance, or continuous 14 days or more of non-invasive respiratory assistance for ≥ 16 hours per day, excluding acute reversible diseases that require assisted ventilation and perioperative ventilation. Long-term nasogastric feeding refers to the use of a nasogastric tube for feeding due to severe loss of swallowing function);
There are severe immunodeficiencies or autoimmune diseases;
Single group
VGN-R08b
Drug6×10\^10 vg/g
VGN-R08b
Drug1.2×10\^11 vg/g
VGN-R08b
Drug1.8×10\^11 vg/g
Number of adverse events (AE), Serious adverse events (SAE)
Vital signs, physical examination, laboratory test results will be monitored after drug injection
Changes in Glucose Gangliosidase (GCase)
Changes in the activities of glucose gangliosidase (GCase) in peripheral blood and cerebrospinal fluid (CSF) after medication administration
Changes in Glucose sialic acid (Lyso-GL1)
Changes in the levels of glucose sialic acid (Lyso-GL1) in peripheral blood and cerebrospinal fluid (CSF) after medication administration
Changes in electrooculogram
Changes in pupillary reflex, horizontal eye movement and vertical eye movement after medication administration compared to the baseline
Changes in Scale for the Assessment and Rating of Ataxia
Changes in ataxia, and the proportion of subjects whose ataxia symptoms showed significant improvement compared to the baseline
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