A Phase Ⅰb/Ⅱ Study to Evaluate the Tolerability, Safety, and Efficacy of VGN-R09b in Patients With Parkinson's Disease
Overview
- Phase
- Phase 1
- Intervention
- VGN-R09b
- Conditions
- Parkinson's Disease
- Sponsor
- Shanghai Vitalgen BioPharma Co., Ltd.
- Enrollment
- 39
- Primary Endpoint
- Number of Adverse Events (AEs), Serious Adverse Events (SAEs)Vital signs
- Status
- Not yet recruiting
- Last Updated
- last year
Overview
Brief Summary
A phase Ⅰ/Ⅱ study to evaluate the tolerability, safety, and efficacy of VGN-R09b in pa-tients with Parkinson's disease
Detailed 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
Investigators
Eligibility Criteria
Inclusion Criteria
- •Subjects must meet all the following inclusion criteria:
- •Male or female, ≥40 years and \<75 years of age at Screening.
- •Diagnosis of Idiopathic Parkinson's disease according to the UK Brain Bank.
- •Insufficient control of motor symptoms with an average of ≥2.5 hours of OFF time per day over 3 consecu-tive days despite optimized treatment, as confirmed by the PD patient diary at Screening.
- •Stable Parkinson's symptoms and an optimal regimen of Parkinson's medications for at least 4 weeks prior to screening, with a duration of levodopa treatment of ≥1 year.
- •Hoehn and Yahr Stage 2.5\~4 on "off" state.
- •All men and women of childbearing potential must be willing to use at least one highly effective method of contraception from the signing of informed consent until one year after administration of the study drug.
- •Men must agree not to donate sperm, and women must agree not to donate eggs within at least one year after administration of the study drug.
- •The patient must understand the purpose and risks of the study, sign and date the informed consent, and give authorization to use the protected health information in accordance with national and local privacy regulations.
- •The patient has a reliable study partner/informant (e.g., a family member, friend) willing and able to partici-pate in the study as a source of information on the patient's health status and cognitive and functional abili-ties (including providing input into the rating scales).
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.
Arms & Interventions
8.0×10^11 vg
3 subjects on 8.0×10\^11 vg for at least 4 weeks post infusion
Intervention: VGN-R09b
1.6×10^12 vg
3 subjects on 1.6×1012 vg for at least 4 weeks post infusion
Intervention: VGN-R09b
3.2×10^12 vg
3 subjects on 3.2×1012 vg for at least 4 weeks post infusion
Intervention: VGN-R09b
Outcomes
Primary Outcomes
Number of Adverse Events (AEs), Serious Adverse Events (SAEs)Vital signs
Time Frame: up to Week 52
Vital signs, physical examination, laboratory test will be monitored after drug injection
Secondary Outcomes
- Changes in clinical outcomes(up to Week 52)
- Immunogenicity after injection(up to 52 weeks)