Safety and Efficacy Study of Parkinson's Disease Gene Therapy Drug (BBM-P002)

Study Overview
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Brief Summary
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Safety and Efficacy Study of BBM-P002 in subjects with primary advanced Parkinson's disease

Detailed Description
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Parkinson's disease is a neurodegenerative disorder involving loss of dopaminergic neurons in the substantia nigra. This study is a single-center, single-arm, open-label, treatment clinical study to evaluate the safety, tolerability and efficacy of an adeno-associated virus (AAV)-based gene therapy, termed BBM-P002, will be injected into the brain via a neurosurgical procedure.

Official Title
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A Single-arm, Open-label, Single-center Trial, Evaluating the Safety and Efficacy of BBM-P002 for Primary Advanced Parkinson's Disease

Conditions
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Parkinson's Disease

Intervention / Treatment
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* Drug: BBM-P002


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosed with idiopathic Parkinson's disease. Males and females, 40 to 70 years of age (inclusive). Disease duration from diagnosis of ≥5 years. Responsiveness to dopamine. Good compliance and regular follow-up. Completion of Parkinson's disease patient diary accurately during follow-up, and family members, guardians or caregivers can help subjects fill in patient diary. Exclusion Criteria: Atypical or secondary parkinsonism. History of coagulopathy, abnormal bleeding or hemopathy family history. Other bleeding risk which increases risk of surgery determined by investigator. Any type of prior gene therapy. Clinically significant electrocardiogram (ECG) abnormalities. Concomitant disease including unstable cardiovascular and cerebrovascular diseases within 3 months, uncontrolled hypertension, sever postural hypotension, poorly controlled diabetes, history of malignancy. Acute or chronic: hepatitis B (HBV); hepatitis C (HCV) infection must have completed curative antiviral treatment with HBV/HCV viral load below the limit of quantification or be negative due to prior treatment or natural resolution to be eligible for enrollment. Subject with Human Immunodeficiency Virus (HIV) or Syphilitic serum positive should be excluded.

Ages Eligible for Study
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Minimum Age: 40 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Arm of BBM-P002<br>single-arm | Drug: BBM-P002<br>* Genetic: single- dose intracranial injection of BBM-P002 Neurosurgical delivery of BBM-P002 to the brain<br>* Other names: BBM003;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Adverse Events and Serious Adverse Events | Safety and Tolerability of BBM-P002 assessed by Adverse Events and Serious Adverse Event | Day 1 through week 52 | 
| Post-treatment MRI assessment | Incidence of parenthetical toxicity in the infusion region or elsewhere throughout the brain on MRI | Day 1 through week 52 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy of BBM-P002 | Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) | Change from Baseline to Week 52 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Parkinson's Disease, Gene therapy, Adeno-Associated Virus