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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
18
BBM-P002 is an AAV-based gene therapy that delivers a therapeutic gene cassette into putamen for the treatment of Parkinson's Disease
Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China
RECRUITINGIncidence 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
Time frame: Within 4 weeks
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
To evaluate the safety of BBM-P002 by AEs and SAEs.
Time frame: Within 4 weeks
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
To assess the long-term safety of BBM-P002 by AEs and SAEs
Time frame: Within 5 years
Changes in the titers of neutralizing antibodies and binding antibodies in the recombinant adenovirus capsid of blood
To assess changes in the titers of neutralizing antibodies and binding antibodies in the recombinant adenovirus capsid of blood after injecting BBM-P002
Time frame: 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
Time frame: 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
Time frame: Within 5 years
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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.
Time frame: 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.
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: Within 5 years