AzTrials
AzTrials

Parkinson Disease (PD)Phase 1/2NCT07414290

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

A Phase 1/2 study testing VGN-R08b 4.2×10^13 vg, VGN-R08b 8.4×10^13 vg and VGN-R08b 1.68×10^14 vg in people with Parkinson Disease (PD), run by Shanghai Vitalgen BioPharma Co., Ltd. at 1 site in China.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
Search all trials

Trial 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 Read more about this study

Who is it for?
Ages 30 to 70 years
Conditions the study is for
Parkinson Disease (PD)
What is being tested?
VGN-R08b 4.2×10^13 vg, VGN-R08b 8.4×10^13 vg and VGN-R08b 1.68×10^14 vg
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
17 participants
Where
1 siteChina
Study dates
25 May 2026to 1 Sep 2031
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 30 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Subjects must meet all the following inclusion criteria:
  • Male or female, aged 30 to 70 years (inclusive) at the time of signing the informed consent form.
  • 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).
  • Glucocerebrosidase (GCase) enzyme activity below the normal range, as measured from past or screening dried blood spot tests.
  • 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.
  • 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) .
Show all 12Show fewer
  • 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).
  • 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.
  • 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.
  • 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.
  • 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.
  • The patient has a reliable study partner (e.g., family member, friend, caregiver) who is willing and able to assist with study visits when needed, and to help provide information regarding the patient's health status, and cognitive and physical abilities (including providing input for rating scales).

Who cannot join

  • Subject has any of the following diseases or disease history
  • 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.
  • 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).
  • Patients with severe liver disease, severe immunodeficiency, or autoimmune diseases within 6 months prior to screening, or those requiring long-term immunosuppressive therapy.
  • 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.
  • 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.
Show all 12Show fewer
  • Patients with a history of epileptic seizures or unexplained coma, deemed unsuitable by the investigator for participation in the trial.
  • 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.
  • 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.
  • 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.
  • Patients with a history of malignancy within 3 years prior to screening, except for completely resected non-melanoma skin cancer, non-metastatic prostate cancer, or fully cured carcinoma in situ that has remained stable for at least 6 months.
  • Patients with any other contraindications deemed by the investigator to potentially affect trial-related procedures, including lumbar puncture or intracerebral injection, such as spinal disorders, bleeding diathesis, clinically significant coagulation dysfunction, thrombocytopenia, or elevated intracranial pressure.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

China1 site

  • Xiangya Hospital, Central South University

    Changsha, Hunan, 410008

    Clinical Medical Ethics Committee of Xiangya Hospital, 0731-84327919, xyyyllwyh@126.com

Other trials in China

About this study

Official title: 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

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

Read the 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

Being tested
VGN-R08b 4.2×10^13 vg, VGN-R08b 8.4×10^13 vg, VGN-R08b 1.68×10^14 vg
Medical subject headings
Parkinson Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • VGN-R08b 4.2×10^13 vgDrug

    Intracerebroventricular injection

  • VGN-R08b 8.4×10^13 vgDrug

    Intracerebroventricular injection

  • VGN-R08b 1.68×10^14 vgDrug

    Intracerebroventricular injection

Study groups in NCT07414290
Study groupRoleWhat they receive
Group (1)Experimental3 subjects on 4.2×10^13 vg for at least 4 weeks post infusion
Receives: Drug: VGN-R08b 4.2×10^13 vg
Group (2)Experimental3 subjects on 8.4×10^13 vg for at least 4 weeks post infusion
Receives: Drug: VGN-R08b 8.4×10^13 vg
Group (3)Experimental3 subjects on 1.68×10^14 vg for at least 4 weeks post infusion
Receives: Drug: VGN-R08b 1.68×10^14 vg

What is being measured

The primary outcome is the main question the study is designed to answer.

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

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

    Measured: up to Week 52

Secondary outcomes (6)

  1. Pharmacodynamic indicator

    Changes in glucocerebrosidase (GCase) activity after medication.

    Measured: Up to Year 5

  2. Changes in Glucosylceramide (Lyso-GL1) Levels

    changes in glucosylceramide (Lyso-GL1) levels in peripheral blood and cerebrospinal fluid (CSF) after medication

    Measured: Up to 5 years

  3. Disease indicators

    Diary: Daily medication dosage (equivalent dose of levodopa), changes in high-quality "on" time (referring to the time without dyskinesia or dyskinesia not affecting daily life) and "off" time;

    Measured: Up to Year 5

  4. Unified Parkinson's Disease Rating Scale (UPDRS) and Hoehn-Yahr (H-Y) Staging

    The Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS) consists of four parts: Part I (Non-motor Experiences of Daily Living), Part II (Motor Experiences of Daily Living), Part III (Motor Examination), and Part IV (Motor Complications). Each item on the scale is scored on a five-point scale of 0, 1, 2, 3, and 4, with higher scores indicating more severe PD symptoms. The scores of each part and the total score of the four parts will specifically reflect the disease progression of Parkinson's disease. The scoring sheet will include MDS-UPDRS-III "off" and "on" scores, and Hoehn-Yahr "off" staging.

    Measured: Up to 5 Years

  5. Viral shedding

    Changes in VGN-R08b vector genome levels in peripheral blood, urine, and feces after medication.

    Measured: Up to Year 5

  6. Immunogenicity

    Number of subjects developing antibodies against AAV9 and GCase and antibody titers, including in serum and CSF

    Measured: Up to Year 5

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Feb 2026
  2. Study start25 May 2026 (estimated)
  3. Primary completion1 Sep 2027 (estimated)
  4. Study completion1 Sep 2031 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
17 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07414290
Sponsor's protocol ID
VGN-R08b-103
Status last verified
Feb 2026
First submitted
9 Feb 2026
Last updated
1 May 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing VGN-R08b 4.2×10^13 vg, VGN-R08b 8.4×10^13 vg and VGN-R08b 1.68×10^14 vg in people with Parkinson Disease (PD), run by Shanghai Vitalgen BioPharma Co., Ltd. at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 1 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Adverse Events (AEs), Serious Adverse Events (SAEs)Vital signs (time frame: up to Week 52). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07414290.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 May 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in