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About
A phase Ⅰ/Ⅱ study to evaluate the tolerability, safety, and efficacy of VGN-R09b in pa-tients with Parkinson's disease
Full description
In the open-label dose escalation part, 3 dose cohorts will be explored, with 3 subjects per cohort.
Cohort 1: 3 subjects on 8.0×10^11 vg for at least 4 weeks post infusion Cohort 2: 3 subjects on 1.6×10^12 vg for at least 4 weeks post infusion Cohort 3: 3 subjects on 3.2×10^12 vg for at least 4 weeks post infusion In the dose-escalation part, each cohort follows the principle of sentinel administration (i.e., one subject will be enrolled and dosed first in each cohort). If no significant safety risk is observed within 4 weeks after administra-tion, the remaining 2 subjects will be dosed.
Additional cohort(s) and/or a safe low and high dose will be determined by the safety review committee (SRC) to initiate Part II
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
Subjects must meet all the following inclusion criteria:
Exclusion criteria
Subject has any of the following diseases or disease history
Atypical or secondary parkinsonism, including but not limited to symptoms believed to be due to trauma, brain tumor, infection, cerebrovascular disease, or other neurological disease, or to drugs, chemicals, or tox-ins, as determined by the Investigator.
Known pathogenic gene mutations of GBA1, PINK1, and Parkin
MoCA score ≤16
Currently active infection or a severe infection (e.g., pneumonia, septicemia, central nervous system infec-tions [e.g. meningitis, encephalitis]) within 12 weeks prior to Screening
Active infection of HBV, HCV or TP, or with HIV-positive at screening.
Unstable autoimmune disease within 6 months prior to Screening, or requiring chronic immunosuppression.
Poorly controlled diabetes (Screening glycosylated hemoglobin [HbA1C] ≥ 7%), or uncontrolled hyperten-sion.
History of stroke or transient ischemic attack, unstable angina, myocardial infarction, chronic heart failure (New York Heart Association Class III or IV), or clinically significant conduction abnormalities (e.g., un-stable atrial fibrillation) within 1 year prior to Screening.
New or unstable psychiatric conditions (e.g. psychosis, severe depression, or with current suicidal ideation or suicide attempt) within 1 year of screening.
History of malignancy within 3 years of screening, other than fully excised non-melanoma skin cancers, non-metastatic prostate cancer, and fully treated carcinoma in situ, provided it has been stable for at least 6 months.
Any medical conditions that, in the opinion of the Investigator, could interfere with study-related proce-dures (including the safe performance of intraparenchymal injection), such as severe dyskinesia/impulse control disorders/tremor that may interfere with drug injection, brain implants, bleeding diathesis, clinically significant coagulopathy, thrombocytopenia, or increased intracranial pressure.
Subject who is receiving or has a history of any of the following medications
Any type of prior gene or cell therapy.
Prior brain surgery for deep brain stimulation, focused ultrasound, infusion therapies or any other brain sur-gery for PD, or planned brain surgery for PD within 1 year after study entering.
Any anticoagulant or antiplatelet therapies that could not be stopped before study drug injection.
Any live vaccine within 4 weeks prior to Screening. Subject who meets any of the following test endpoints at screening
An MRI showed a significant structural abnormality or lesion, bleeding, or >1 cm3 infarct, which, in the opinion of the Investigator, are contraindications to intraparenchymal injection.
Clinically significant abnormalities in laboratory test values at Screening are in the opinion of the Investiga-tor, unsuitable for enrollment immediately.
Subject under any of the following general conditions
Contraindications to MRI/PET and/or agents used in PET.
Contraindications to general anesthesia or deep sedation.
Woman who is pregnant or breastfeeding.
Participation within 3 months prior to Screening in another therapeutic investigational drug or device study, unless it can be documented that the patient received a placebo.
Presence of substance (drug, alcohol) abuse within 2 years prior to Screening.
The patient is generally frail or has any condition for which, in view of the Investigator, participation in the study would not be in the best interest of the patient or is likely to prohibit further participation during the study.
Primary purpose
Allocation
Interventional model
Masking
39 participants in 3 patient groups
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Data sourced from clinicaltrials.gov
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