Parkinson's DiseaseEarly Phase 1NCT07100171
Evaluation of GM101 Injection in Patients With Parkinson's Disease
A Early Phase 1 study testing Biological: GM101 in people with Parkinson's Disease, run by Genemagic Biosciences Co., Ltd at 1 site in China.
Trial summary
The objective of this study is to assess the safety of GM101 in participants with Parkinson's disease (PD) Read more about this study
- Who is it for?
- Ages 40 to 70 years
- Conditions the study is for
- Parkinson's Disease
- What is being tested?
- Biological: GM101
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 29 May 2025to 17 Feb 2030
- Phase
- Early Phase 1
- 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 40 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Clinically diagnosed patients with primary Parkinson's Disease [in accordance with the "Diagnostic Criteria for Parkinson's Disease in China" released in 2016];
- Participants must be able to comprehend (fully understand the details of the clinical trial and the potential risks and benefits of the study) and voluntarily sign the informed consent form. When participants are unable to read, the informed consent form and other written materials may be read by a legal representative or an impartial witness, who will also witness the consent process;
- Age between 40 and 70 years old, inclusive, regardless of gender;
- Body weight between 40 kg and 110 kg, and Body Mass Index (BMI) between 18 kg/m² and 34 kg/m²;
- A history of Parkinson's Disease for >= 5 years;
- Able to undergo surgical anesthesia, suitable for neurosurgical procedures under anesthesia, and capable of undergoing CT/MRI examinations;
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- Hoehn-Yahr stage (Appendix IV Hoehn-Yahr Staging) at screening in the "off" state is 4-5 (including borderline values);
- MDS-Unified Parkinson's Disease Rating Scale Part III Motor Examination (MDS-UPDRS-III) score in the "off" state >= 30; and a positive levodopa challenge test (improvement in UPDRS-III score from "off" to "on" state >30%);
- Patients whose PD symptoms cannot be effectively controlled or who experience intolerable adverse drug reactions despite treatment with medications recommended by the "Chinese Guidelines for the Treatment of Parkinson's Disease (4th Edition)-2020";
- Receiving a stable dose of anti-Parkinson's medication for at least 4 weeks prior to administration;
- Acceptable laboratory values during the screening period and prior to administration (Day 0): a) Hemoglobin >= 100 g/L; b) Platelets >= 100×10^9/L; c) Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) <= 1.5 times the upper limit of normal; d) Total bilirubin <= 1.5 times the upper limit of normal; e) Serum creatinine (Cr) <= 1.5 times the upper limit of normal;
- Participants agree not to receive any other therapeutic intervention studies during the main study phase;
- Participants agree not to participate in any other clinical studies during the study period;
- Participants agree not to receive any other vaccines within 30 days after administration;
- Participants must have the ability to live independently or the ability to clearly delegate relevant caregivers, with good compliance, and be able to attend regular follow-ups. During follow-up, participants must accurately complete the PD patient diary, and family members, guardians, or caregivers may assist in filling out the patient diary;
- Participants must have a pre-existing neutralizing AAV8 antibody titer <= 1:90 as determined by testing;
- Based on the judgment of the Principal Investigator (PI), cancer-related screening will be conducted for patients who meet the inclusion criteria.
Who cannot join
- Patients with atypical parkinsonian syndromes (Parkinson-plus syndromes, secondary Parkinson's syndrome, or hereditary Parkinson's syndrome).
- Patients who have previously undergone pallidotomy, deep brain stimulation (DBS) surgery, striatal/extrapyramidal system surgery, stereotactic brain surgery, or other cerebral surgeries that might affect the observation of this trial's outcomes; or those who have undergone other surgeries deemed by investigators to interfere with study participation.
- Patients with intracranial lesions revealed by previous CT/MRI scans, such as cerebral trauma, cerebrovascular malformations, hydrocephalus, brain tumors, or striatal/other brain region abnormalities that significantly increase surgical risks.
- Patients with history of the following cardiovascular/cerebrovascular diseases: 1)Severe heart failure, unstable angina, or myocardial infarction; 2)Severe arrhythmias including but not limited to II/III-degree atrioventricular block or prolonged QT interval; 3)Long QT syndrome; 4)Cardiovascular surgery history; 5)History of stroke or transient ischemic attack (TIA) within 3 months, deemed ineligible by investigators; 6)Subarachnoid hemorrhage history; 7)Major vascular diseases considered ineligible by investigators.
- Patients with history of malignant tumors.
- Patients who have received cell therapy.
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- Patients who have received gene therapy.
- Patients with active disseminated intravascular coagulation or significant bleeding tendency within 3 months prior to consent, or those unable to discontinue antiplatelet/anticoagulant medications >= 7 days pre-operation, or whose coagulation function hasn't normalized >= 10 days after stopping antiplatelet medications.
- Patients who received prolonged >= 14 days) high-dose systemic corticosteroids (prednisone >= 20 mg/day or equivalent) or immunosuppressants within 3 months prior to consent (topical use excluded).
- 10,Patients with psychiatric disorders deemed ineligible by investigators; or those with suicidal ideation/attempts (including actual attempts, interrupted attempts, or aborted attempts) within the past year or currently.
- 11.Patients who received botulinum toxin treatment within 6 months prior to consent.
- 12.Patients with active epilepsy or currently using antiepileptic drugs. 13.Patients with dementia or severe cognitive impairment history; or those showing significant dementia/cognitive dysfunction during screening; MDS-UPDRS Part 1.1 cognitive impairment score >3; dementia affecting compliance, diary accuracy, and/or informed consent capability.
- 14. Individuals with severe depression (Hamilton Depression Rating Scale [HAM-D17] score >= 24) or severe anxiety (Hamilton Anxiety Rating Scale [HAMA] score >= 29) during screening.
- 15. Individuals with the following clinically significant abnormalities during screening: 1) Abnormal coagulation function (prothrombin time >= 1.5 times the upper limit of normal, activated partial thromboplastin time >= 1.5 times the upper limit of normal); 2) Clinically significant immunological abnormalities, deemed unsuitable for participation by the investigator; 3) Poorly controlled hypertension (defined as blood pressure remaining above 160/100 mm Hg despite antihypertensive treatment) and severe orthostatic hypotension; 4) Poorly controlled diabetes (glycated hemoglobin > 9.0%, or fasting plasma glucose [FPG] >= 11.1 mmol/L).
- 16. Individuals with surgical contraindications (e.g., prior cochlear implant, pacemaker, defibrillator, stereotactic neurosurgery, or prior implantation of unilateral or bilateral similar devices) or those who have undergone other surgeries within the past six months that the investigator believes may affect the trial, or those with allergies (e.g., to contrast agents) preventing MRI, or other neurosurgical contraindications.
- 17. Individuals with other severe systemic diseases, such as cor pulmonale, severe chronic obstructive pulmonary disease (COPD) (FEV1% < 50%), etc.
- 18. Individuals with any of the following conditions: positive human immunodeficiency virus (HIV) antibody; positive syphilis antibody; positive hepatitis C virus (HCV) antibody with detectable HCV RNA; positive hepatitis B virus surface antigen with HBV DNA copy number above the normal limit; active tuberculosis; or other active infections deemed by the investigator to potentially affect participation or study outcomes.
- 19. Individuals with alcohol addiction, positive drug abuse screening, or a history of drug dependence.
- 20. Individuals with contraindications or allergies to any components of the drugs used in the study (e.g., tacrolimus, levodopa) or to similar drugs or other macrolide drugs, or those with a history of allergies (e.g., to two or more drugs or foods).
- 21. Women of childbearing potential or breastfeeding women who have not undergone sterilization, are not postmenopausal, or are unwilling to use medically approved effective contraception for two years after receiving the study drug, and men who have not undergone sterilization or are unwilling to use medically approved effective contraception for two years after receiving the study drug.
- 22. Individuals who have received electroconvulsive therapy within 30 days prior to dosing.
- 23. Individuals currently participating in other clinical trials or those who have participated in other clinical studies and received interventional treatment within three months prior to dosing.
- 24. Individuals deemed by the investigator to have poor compliance. 25. Patients currently receiving treatment such as apomorphine or levodopa infusion therapy (Duodopa®).
- 26. Individuals with severe movement disorders in both medicated and unmedicated states.
- 27. Individuals with major illnesses or any other conditions deemed by the investigator to jeopardize the safety of the participant or affect the investigator's assessments.
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, 1 recruiting
- Recruiting
Xiangya Hospital, Central South University
Changsha
Zhiquan Yang, MD, PhD, +86 731 8975 3036, y66406914@163.com
About this study
Official title: A Single-Center, Open-Label, Single-Arm Exploratory Study Evaluating GM101 Injection in Patients With Mid-to-Late Stage Parkinson's Disease
The objective of this study is to assess the safety of GM101 in participants with Parkinson's disease (PD)
Read the detailed description
We utilize adeno-associated viruses (AAVs) to deliver gene to reprogram astrocytes in the putamen into dopaminergic neurons to treat patients with Parkinson's disease.
- Conditions
- Parkinson's Disease
- Being tested
- Biological: GM101
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biological: GM101Other
Neurosurgical delivery of GM101 to the putamen
| Study group | Role | What they receive |
|---|---|---|
| Experimental: GM101 dose escalation | Experimental | This is a dose-escalation study with two dose cohorts under a single-group assignment framework Receives: Other: Biological: GM101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Grading of adverse Events/Serious Adverse Events (AE's/SAE's)
Grading will be assessed using NCI CTCAE, version 4.03. Grade 1 = Mild Grade 2 = Moderate Grade 3 = Severe Grade 4 = Potentially life-threatening Grade 5 = Death
Measured: Baseline to 5 Years After Gene Transfer
Secondary outcomes (5)
Change in motor function using Unified Parkinson's Disease Rating Scale (UPDRS);
Standard assessment scale used to quantify signs and symptoms of PD. The total UPDRS score is calculated by summing the scores from each section, ranging from 0 (no disability) to 176 (severe disability), the higher scores indicate the greater severity of symptoms.
Measured: Baseline to 5 Years After Gene Transfer
Change in motor function using Modified Hoehn and Yahr Scale
Scale used to measure overall level of disability due to PD. The scale includes stages 1 through 5, the higher stage means the worse condition.
Measured: Baseline to 5 Years After Gene Transfer
Change in occurrence of dyskinesia using Unified Dyskinesia Rating Scale (UDysRS)
Comprehensive rating tool used to assess essential features of dyskinesia in PD.
Measured: Baseline to 5 Years After Gene Transfer
Change in Parkinson's medications
Change in Parkinson's medications compared to Baseline.
Measured: Baseline to 5 Years After Gene Transfer
Change in dopamine transporter binding assessed by regional brain using [¹¹C]CFT-PET (DAT imaging)
Change in Parkinson's DAT imaging compared to Baseline.
Measured: Baseline to 5 Years After Gene Transfer
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Aug 2025
- Study start29 May 2025
- Primary completion17 Feb 2027 (estimated)
- Study completion17 Feb 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- This is a dose-escalation study with two dose cohorts under a single-group assignment framework
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Genemagic Biosciences Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07100171
- Sponsor's protocol ID
- 202411203-4
- Status last verified
- Jul 2025
- First submitted
- 18 Feb 2025
- Last updated
- 3 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Center, Open-Label, Single-Arm Exploratory Study Evaluating GM101 Injection in Patients With Mid-to-Late Stage Parkinson's Disease
- ClinicalTrials.gov ID
- NCT07100171
- Acronym / sponsor ID
- 202411203-4
- Conditions
- Parkinson's Disease
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Genemagic Biosciences Co., LtdIndustry (pharma, biotech, devices)
- Study start
- 29 May 2025 (actual)
- Primary completion
- 17 Feb 2027 (estimated)
- Study completion
- 17 Feb 2030 (estimated)
- First posted
- 3 Aug 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of this study is to assess the safety of GM101 in participants with Parkinson's disease (PD)
Detailed description
We utilize adeno-associated viruses (AAVs) to deliver gene to reprogram astrocytes in the putamen into dopaminergic neurons to treat patients with Parkinson's disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is a dose-escalation study with two dose cohorts under a single-group assignment framework
| Arm | Type | Receives |
|---|---|---|
| Experimental: GM101 dose escalation | Experimental | Other: Biological: GM101This is a dose-escalation study with two dose cohorts under a single-group assignment framework |
Who may qualify
- Ages
- 40 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Clinically diagnosed patients with primary Parkinson's Disease [in accordance with the "Diagnostic Criteria for Parkinson's Disease in China" released in 2016];
- Participants must be able to comprehend (fully understand the details of the clinical trial and the potential risks and benefits of the study) and voluntarily sign the informed consent form. When participants are unable to read, the informed consent form and other written materials may be read by a legal representative or an impartial witness, who will also witness the consent process;
- Age between 40 and 70 years old, inclusive, regardless of gender;
- Body weight between 40 kg and 110 kg, and Body Mass Index (BMI) between 18 kg/m² and 34 kg/m²;
- A history of Parkinson's Disease for >= 5 years;
Key exclusion criteria (25 listed)
- Patients with atypical parkinsonian syndromes (Parkinson-plus syndromes, secondary Parkinson's syndrome, or hereditary Parkinson's syndrome).
- Patients who have previously undergone pallidotomy, deep brain stimulation (DBS) surgery, striatal/extrapyramidal system surgery, stereotactic brain surgery, or other cerebral surgeries that might affect the observation of this trial's outcomes; or those who have undergone other surgeries deemed by investigators to interfere with study participation.
- Patients with intracranial lesions revealed by previous CT/MRI scans, such as cerebral trauma, cerebrovascular malformations, hydrocephalus, brain tumors, or striatal/other brain region abnormalities that significantly increase surgical risks.
- Patients with history of the following cardiovascular/cerebrovascular diseases: 1)Severe heart failure, unstable angina, or myocardial infarction; 2)Severe arrhythmias including but not limited to II/III-degree atrioventricular block or prolonged QT interval; 3)Long QT syndrome; 4)Cardiovascular surgery history; 5)History of stroke or transient ischemic attack (TIA) within 3 months, deemed ineligible by investigators; 6)Subarachnoid hemorrhage history; 7)Major vascular diseases considered ineligible by investigators.
- Patients with history of malignant tumors.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Biological: GM101Neurosurgical delivery of GM101 to the putamen | Other | Experimental: GM101 dose escalation |
Primary outcome
Grading of adverse Events/Serious Adverse Events (AE's/SAE's)
Time frame: Baseline to 5 Years After Gene Transfer
Grading will be assessed using NCI CTCAE, version 4.03. Grade 1 = Mild Grade 2 = Moderate Grade 3 = Severe Grade 4 = Potentially life-threatening Grade 5 = Death
5 secondary outcomes
Change in motor function using Unified Parkinson's Disease Rating Scale (UPDRS);
Time frame: Baseline to 5 Years After Gene Transfer
Change in motor function using Modified Hoehn and Yahr Scale
Time frame: Baseline to 5 Years After Gene Transfer
Change in occurrence of dyskinesia using Unified Dyskinesia Rating Scale (UDysRS)
Time frame: Baseline to 5 Years After Gene Transfer
Change in Parkinson's medications
Time frame: Baseline to 5 Years After Gene Transfer
Change in dopamine transporter binding assessed by regional brain using [¹¹C]CFT-PET (DAT imaging)
Time frame: Baseline to 5 Years After Gene Transfer
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Aug 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Biological: GM101 in people with Parkinson's Disease, run by Genemagic Biosciences 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 17 inclusion and 25 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: looking for participants now, as of the sponsor's last update on 3 Aug 2025. 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 phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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?
Contact Kaikai Li, Master (+86 159 0069 2632, kaikaili@genemagicbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Grading of adverse Events/Serious Adverse Events (AE's/SAE's) (time frame: Baseline to 5 Years After Gene Transfer). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 3 Aug 2025.