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

Primary Purpose

Parkinson's Disease

Status
Not yet recruiting
Phase
Early Phase 1
Locations
China
Study Type
Interventional
Intervention
BBM-P002
Sponsored by
Xiangya Hospital of Central South University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Parkinson's Disease focused on measuring Parkinson's Disease, Gene therapy, Adeno-Associated Virus

Eligibility Criteria

40 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Xiangya Hospital of Central South University

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Arm of BBM-P002

Arm Description

single-arm

Outcomes

Primary Outcome Measures

Incidence of Adverse Events and Serious Adverse Events
Safety and Tolerability of BBM-P002 assessed by Adverse Events and Serious Adverse Event
Post-treatment MRI assessment
Incidence of parenthetical toxicity in the infusion region or elsewhere throughout the brain on MRI

Secondary Outcome Measures

Efficacy of BBM-P002
Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS)

Full Information

First Posted
March 1, 2023
Last Updated
April 9, 2023
Sponsor
Xiangya Hospital of Central South University
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1. Study Identification

Unique Protocol Identification Number
NCT05822739
Brief Title
Safety and Efficacy Study of Parkinson's Disease Gene Therapy Drug (BBM-P002)
Official Title
A Single-arm, Open-label, Single-center Trial, Evaluating the Safety and Efficacy of BBM-P002 for Primary Advanced Parkinson's Disease
Study Type
Interventional

2. Study Status

Record Verification Date
February 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
April 28, 2023 (Anticipated)
Primary Completion Date
May 30, 2025 (Anticipated)
Study Completion Date
December 30, 2028 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Xiangya Hospital of Central South University

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Parkinson's Disease
Keywords
Parkinson's Disease, Gene therapy, Adeno-Associated Virus

7. Study Design

Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Arm of BBM-P002
Arm Type
Experimental
Arm Description
single-arm
Intervention Type
Drug
Intervention Name(s)
BBM-P002
Other Intervention Name(s)
BBM003
Intervention Description
Genetic: single- dose intracranial injection of BBM-P002 Neurosurgical delivery of BBM-P002 to the brain
Primary Outcome Measure Information:
Title
Incidence of Adverse Events and Serious Adverse Events
Description
Safety and Tolerability of BBM-P002 assessed by Adverse Events and Serious Adverse Event
Time Frame
Day 1 through week 52
Title
Post-treatment MRI assessment
Description
Incidence of parenthetical toxicity in the infusion region or elsewhere throughout the brain on MRI
Time Frame
Day 1 through week 52
Secondary Outcome Measure Information:
Title
Efficacy of BBM-P002
Description
Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
Time Frame
Change from Baseline to Week 52

10. Eligibility

Sex
All
Minimum Age & Unit of Time
40 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Lu Shen, M.D, Ph.D
Phone
86-731-84327216
Email
shenlu@csu.edu.cn
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Lu Shen, M.D, Ph.D
Organizational Affiliation
Xiangya Hospital of Central South University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Xiangya Hospital of Central South University
City
Changsha
State/Province
Hunan
ZIP/Postal Code
410008
Country
China
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Lu Shen, M.D, Ph.D
Phone
86-731-84327216
Email
shenlu@csu.edu.cn

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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