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A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R08b in Parkinson's Disease Patients With GBA1 Mutations | Clinical Trials | Clareo Health

NOT_YET_RECRUITINGPHASE1/PHASE2

A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R08b in Parkinson's Disease Patients With GBA1 Mutations

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

NCT07414290•Shanghai Vitalgen BioPharma Co., Ltd.

View on ClinicalTrials.gov

Summary

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

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 4.2×10\^13 vg for at least 4 weeks post infusion Cohort 2: 3 subjects on 8.4×10\^13 vg for at least 4 weeks post infusion Cohort 3: 3 subjects on 1.68×10\^14 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

Eligibility

Age

30 - 70 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Male or female, aged 30 to 70 years (inclusive) at the time of signing the informed consent form.
•2. 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).
•3. Glucocerebrosidase (GCase) enzyme activity below the normal range, as measured from past or screening dried blood spot tests.
•4. 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.
•5. Montreal Cognitive Assessment (MoCA) score meeting the following criteria: \>13 (for ≤6 years of education), \>15 (for 7-12 years of education), or \>16 (for \>12 years of education) .
•6. On an optimized levodopa regimen at screening (defined as a regimen optimized with at least a combination of levodopa preparations plus a dopamine agonist or MAO-B inhibitor, with levodopa administered ≥3 times per day and at a total daily dose of ≥300 mg), yet still experiencing suboptimal symptom control or significant "wearing-off" (as evidenced by a diary documenting a daily "OFF" time of ≥2.5 hours for three consecutive days during screening).
•7. Stable Parkinson's disease symptoms and stable optimized anti-Parkinson's medication regimen for ≥4 weeks prior to screening; patients with GD-PD receiving Gaucher disease (GD) therapy must have been on stable enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 3 months prior to screening.
•8. Men and women of childbearing potential must agree to consistently and correctly use a highly effective method of contraception from the screening period until at least 1 year after dosing.
•9. Men must agree not to donate sperm, and women must agree not to donate eggs, from the screening period until at least 1 year after dosing.
•10. The patient and/or the patient's legal guardian demonstrates understanding of the trial information, purpose, and risks described in the informed consent form, and is able to authorize the use of the patient's health information by providing a signed and dated informed consent form.
+1 more criteria

Exclusion Criteria

•Subject has any of the following diseases or disease history
•1. 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.
•2. 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).
•3. Patients with severe liver disease, severe immunodeficiency, or autoimmune diseases within 6 months prior to screening, or those requiring long-term immunosuppressive therapy.
•4. Patients with poorly controlled diabetes or hypertension, judged by the investigator as unsuitable for dosing or likely to substantially impact the efficacy and safety analysis of the investigational drug.
•5. Patients with a history of stroke or transient ischemic attack (TIA), unstable angina, myocardial infarction, chronic heart failure (NYHA Class III or IV), or clinically significant conduction abnormalities (e.g., unstable atrial fibrillation) within 1 year prior to screening.
•6. Patients with a history of epileptic seizures or unexplained coma, deemed unsuitable by the investigator for participation in the trial.
•7. Patients with a history of severe allergic reactions, or hypersensitivity to any inactive ingredient of the investigational drug or to immunosuppressants required by the trial protocol.
•8. Patients with contraindications to corticosteroids or sirolimus, including but not limited to osteoporosis with vertebral fractures within 1 year prior to screening, poorly controlled hyperlipidemia or hypercholesterolemia, renal insufficiency, or interstitial lung disease.
•9. Patients with newly diagnosed or unstable psychiatric disorders within 1 year prior to screening that may interfere with trial procedures and evaluations, including confusion, severe depression (HAMD score \>35), or suicidal/self-harm tendencies.
+2 more criteria

Locations (1)

Xiangya Hospital, Central South University

Changsha, Hunan, China

Key Dates

Start Date

March 1, 2026

Primary Completion Date

September 1, 2027

Completion Date

September 1, 2031

Last Updated

March 2, 2026

Enrollment

ESTIMATED participants

Conditions

Parkinson Disease (PD)

Interventions

VGN-R08b 4.2×10^13 vg

DRUG

VGN-R08b 8.4×10^13 vg

DRUG

VGN-R08b 1.68×10^14 vg

DRUG

Robotic Apparel to Prevent Freezing of Gait in Parkinson Disease

NCT06602544

Parkinson Disease (PD)

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ENROLLING_BY_INVITATION

Prodromal Model of Parkinson's Disease Confined to The Peripheral Nervous System

NCT06745011

PolyneuropathyAlpha-Synucleinopathy+9 more

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 2, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R08b in Parkinson's Disease Patients With GBA1 Mutations

Inclusion Criteria

Exclusion Criteria

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