Evaluation of GM101 Injection in Patients With Parkinson's Disease
ConditionParkinson's Disease
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age40-70
SponsorGenemagic Biosciences Co., Ltd
About this trial
The objective of this study is to assess the safety of GM101 in participants with Parkinson's disease (PD)
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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;
Disqualifiers
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.
Trial design
Design model
Sequential
Treatments tested in this trial
Biological: GM101
Other interventionNeurosurgical delivery of GM101 to the putamen
Treatment groups
10 Participants
are divided into 1 treatment groupGroup A: Experimental: GM101 dose escalationExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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
Time frame
Baseline to 5 Years After Gene TransferSecondary outcomes
1
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.
Time frame
Baseline to 5 Years After Gene Transfer2
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.
Time frame
Baseline to 5 Years After Gene Transfer3
Change in occurrence of dyskinesia using Unified Dyskinesia Rating Scale (UDysRS)
Comprehensive rating tool used to assess essential features of dyskinesia in PD.
Time frame
Baseline to 5 Years After Gene Transfer4
Change in Parkinson's medications
Change in Parkinson's medications compared to Baseline.
Time frame
Baseline to 5 Years After Gene TransferSponsors and contacts
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Source ClinicalTrials.gov