A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R09b in Patients With Parkinson's Disease

NCT06480461 | Active Not Recruiting Phase 1

• 1 other identifier: VGN-R09b-101
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 1

Brief Summary

A phase Ⅰ/Ⅱ study to evaluate the tolerability, safety, and efficacy of VGN-R09b in pa-tients with Parkinson's disease

Conditions

Parkinson's Disease

Keywords

VGN-R09b

Outcome Measures

Primary Outcomes (1)

• Number of Adverse Events (AEs), Serious Adverse Events (SAEs)Vital signs

  Vital signs, physical examination, laboratory test will be monitored after drug injection

  up to Week 52

Secondary Outcomes (3)

• Changes in clinical outcomes

  up to Week 52

• Changes in clinical outcomes

  up to Week 52

• Immunogenicity after injection

  up to 52 weeks

Study Arms (3)

8.0×10^11 vg

EXPERIMENTAL

3 subjects on 8.0×10\^11 vg for at least 4 weeks post infusion

Drug: VGN-R09b

1.6×10^12 vg

EXPERIMENTAL

3 subjects on 1.6×1012 vg for at least 4 weeks post infusion

Drug: VGN-R09b

3.2×10^12 vg

EXPERIMENTAL

3 subjects on 3.2×1012 vg for at least 4 weeks post infusion

Drug: VGN-R09b

Interventions

VGN-R09b DRUG

This is a Phase I/II trial, with safety as the primary measure. Sample size is not determined through statistical justification. There will be 3 subjects in each dose cohort in the dose escalation part. In the dose expansion part, a sham surgery group and 2 dose groups will be designed; 10 subjects will be enrolled in each group.

1.6×10^12 vg; 3.2×10^12 vg; 8.0×10^11 vg

Eligibility Criteria

• Age: 40 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Shanghai Vitalgen BioPharma Co., Ltd.: lead

Study Sites (1)

Shanghai

Shanghai, Shanghai Municipality, 200120, China

Location

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 14, 2024
• First Posted: June 28, 2024
• Study Start: September 2, 2024
• Primary Completion (Estimated): September 10, 2026
• Study Completion (Estimated): September 10, 2030
• Last Updated: March 31, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)