Actively Recruiting
A Single-Center, Open-Label, Single-Arm Exploratory Study Evaluating GM101 Injection in Patients With Mid-to-Late Stage Parkinson's Disease
Led by Genemagic Biosciences Co., Ltd · Updated on 2025-08-03
10
Participants Needed
1
Research Sites
156 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety of GM101 injection in participants with Parkinson's disease, focusing on those in mid-to-late stages of the condition. This early-phase 1 study uses adeno-associated viruses to deliver a gene that reprograms brain cells called astrocytes into dopamine-producing neurons in a specific brain region called the putamen. The goal is to explore a potential new treatment approach for Parkinson's disease. The study involves a single group of participants undergoing a dose-escalation of the GM101 injection delivered through neurosurgery directly to the putamen. Participants receive one of two dose levels in this open-label design, where both researchers and participants know the treatment given. The trial is non-randomized and includes careful monitoring after the gene transfer. Participants will be closely monitored from baseline up to five years after the gene transfer. Researchers will assess safety by tracking adverse events and serious adverse events. They will also measure changes in motor function using various Parkinson's disease rating scales, medication use, and brain imaging to observe dopamine transporter binding. Participants must attend regular follow-up visits, complete patient diaries, and undergo imaging, laboratory tests, and neurological evaluations throughout the study.
CONDITIONS
Brief Title
Evaluation of GM101 Injection in Patients With Parkinson's Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Clinically diagnosed with primary Parkinson's Disease according to the 2016 Chinese diagnostic criteria
- Able to understand the study details and voluntarily sign informed consent
- Age between 40 and 70 years inclusive
- Body weight between 40 kg and 110 kg and BMI between 18 and 34 kg/m²
- Parkinson's disease history of 5 years or more
- Able to undergo surgical anesthesia and suitable for neurosurgery and CT/MRI exams
- Hoehn-Yahr stage 4-5 in the "off" state at screening
- MDS-UPDRS-III motor score >= 30 in the "off" state and positive levodopa challenge test (>30% improvement)
- PD symptoms not controlled or intolerable side effects despite recommended medications
- Stable anti-Parkinson's medication dose for at least 4 weeks before treatment
- Acceptable lab values including hemoglobin >= 100 g/L, platelets >= 100x10^9/L, liver and kidney function within limits
- Agree not to participate in other therapeutic studies or clinical trials during this study
- Agree not to receive other vaccines within 30 days after treatment
- Able to live independently or have caregivers and attend regular follow-ups
- Pre-existing neutralizing AAV8 antibody titer <= 1:90
- Cancer screening as judged necessary by the Principal Investigator
You will not qualify if you...
- Atypical Parkinsonian syndromes or secondary/hereditary Parkinson's
- Previous brain surgeries affecting study outcomes like pallidotomy, DBS, or stereotactic surgeries
- Intracranial lesions increasing surgical risks (e.g., trauma, tumors, malformations)
- History of severe cardiovascular or cerebrovascular diseases including recent stroke or heart failure
- History of malignant tumors
- Previous cell or gene therapy
- Active bleeding disorders or inability to stop anticoagulant medications before surgery
- Recent use of high-dose corticosteroids or immunosuppressants
- Psychiatric disorders including recent suicidal ideation or attempts
- Recent botulinum toxin treatment
- Active epilepsy or use of antiepileptic drugs
- Dementia or severe cognitive impairment affecting consent or compliance
- Severe depression or anxiety as measured during screening
- Significant coagulation, immune, or metabolic abnormalities
- Neurosurgical contraindications or allergies preventing MRI
- Severe systemic diseases like cor pulmonale or severe COPD
- Active infections including HIV, hepatitis, tuberculosis
- Alcohol or drug dependence
- Allergies to study drugs or related compounds
- Women of childbearing potential or breastfeeding without sterilization or contraception plans
- Recent electroconvulsive therapy
- Participation in other interventional clinical trials within 3 months
- Poor compliance or inability to complete study requirements
- Current treatment with apomorphine or levodopa infusion therapy
- Severe movement disorders unresponsive to medication
- Other major illnesses or conditions deemed unsafe by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person) for eligibility assessment including medical history, physical exams, and imaging.
Duration - Single administration with follow-up lasting up to 5 years
Participants receive a neurosurgical delivery of GM101 to the putamen to treat Parkinson's disease.
1 surgery visit followed by frequent visits for safety and motor function assessments over 5 years
Duration - Up to 5 years after gene transfer
Participants are monitored for safety, motor function changes, and adverse events after treatment.
Regular follow-up visits including motor assessments and imaging studies over 5 years
Trial Site Locations
Total: 1 location
1
Xiangya Hospital, Central South University
Changsha, China
Actively Recruiting
Research Team
K
Kaikai Li, Master
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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