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The purpose of the study is to evaluate the safety and tolerability of BBM-P002 for stereotactic injection to treat participants with idiopathic Parkinson's Disease (PD) during the dose-limiting toxicity (DLT) observation period.
This is a single-arm, open-label study to evaluate the safety, tolerability, efficacy, pharmacokinetic, pharmacodynamic, and immune response of BBM-P002 within 52 weeks after into the bilateral putamen in Parkinson's disease, as well as the long-term safety and efficacy of BBM-P002 for up to 5 years post injection.
BBM-P002 is an AAV-based gene therapy that delivers a therapeutic gene cassette into putamen for the treatment of Parkinson's Disease.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm of BBM-P002 | Experimental | Single-dose treatment |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Injecting BBM-P002 into the bilateral putamen | Genetic | BBM-P002 is an AAV-based gene therapy that delivers a therapeutic gene cassette into putamen for the treatment of Parkinson's Disease |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of dose limiting toxicity (DLT) events | To evaluate the rate of incidence of DLT events determined by the Safety Data Review Committee (SRC) in DLT observation period after injecting BBM-P002 | Within 4 weeks |
| Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) | To evaluate the safety of BBM-P002 by AEs and SAEs. | Within 4 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) | To assess the long-term safety of BBM-P002 by AEs and SAEs | Within 5 years |
| Changes in the titers of neutralizing antibodies and binding antibodies in the recombinant adenovirus capsid of blood |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Wenshu Luo, Ph.D | Contact | 021-33588288 | luowenshu@beliefbiomed.com |
| Name | Affiliation | Role |
|---|---|---|
| Jun Liu, MD | Ruijin Hospital | Principal Investigator |
| Jing Sun, MD | Ruijin Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine | Recruiting | Shanghai | Shanghai Municipality | 200025 | China |
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To assess changes in the titers of neutralizing antibodies and binding antibodies in the recombinant adenovirus capsid of blood after injecting BBM-P002 |
| Within 5 years |
| Changes in the results of the cranial MRI scan | To assess changes in the results of the cranial MRI scan after injecting BBM-P002 | Within 5 years |
| Changes from baseline in MDS - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) I/II/III/IV scores | The theoretical maximum total score of the MDS-UPDRS is 272 points. It comprehensively assesses the health status of Parkinson's disease patients by evaluating four major aspects: non-motor symptoms, daily activities, motor function, and motor complications. The higher the score, the more severe the condition | Within 5 years |
| Changes from baseline in the Patient Global Impression of Improvement (PGI-I) scores | Choose a number from 1 to 7. The higher the number choosen, the worse the improvement. | Within 5 years |
| Changes from baseline in the Clinical Global Impression of Improvement (CGI-I) scores | Choose a number from 1 to 7. The higher the number choosen, the worse the improvement. | Within 5 years |
| Changes in the activity levels of gene expression products under18F-dopa PET scanning from baseline [striatum - occipital lobe average radioactivity count ratio] | To assess changes in the activity levels of gene expression products under18F-dopa PET scanning from baseline [striatum - occipital lobe average radioactivity count ratio] after injecting BBM-P002 | Within 5 years |
| Changes from baseline in daily levodopa dose or equivalent dose of levodopa | To assess changes from baseline in the levodopa dosage or levodopa equivalent dosage after injecting BBM-P002 | Within 5 years |
| Changes from baseline in the percentage at on time. | To assess changes from baseline in the percentage at on time after injecting BBM-P002 | Within 5 years |