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

Sponsor
Xiangya Hospital of Central South University (Other)
Overall Status
Not yet recruiting
CT.gov ID
NCT05822739
Collaborator
(none)
6
Enrollment
1
Location
1
Arm
68.1
Anticipated Duration (Months)
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease Intervention/Treatment Phase
Early Phase 1

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.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
6 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Single-arm, Open-label, Single-center Trial, Evaluating the Safety and Efficacy of BBM-P002 for Primary Advanced Parkinson's Disease
Anticipated Study Start Date :
Apr 28, 2023
Anticipated Primary Completion Date :
May 30, 2025
Anticipated Study Completion Date :
Dec 30, 2028

Arms and Interventions

Arm Intervention/Treatment
Experimental: Arm of BBM-P002

single-arm

Drug: BBM-P002
Genetic: single- dose intracranial injection of BBM-P002 Neurosurgical delivery of BBM-P002 to the brain
Other Names:
  • BBM003

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Incidence of Adverse Events and Serious Adverse Events [Day 1 through week 52]

      Safety and Tolerability of BBM-P002 assessed by Adverse Events and Serious Adverse Event

    2. Post-treatment MRI assessment [Day 1 through week 52]

      Incidence of parenthetical toxicity in the infusion region or elsewhere throughout the brain on MRI

    Secondary Outcome Measures

    1. Efficacy of BBM-P002 [Change from Baseline to Week 52]

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

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    40 Years to 70 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    1. Diagnosed with idiopathic Parkinson's disease.

    2. Males and females, 40 to 70 years of age (inclusive).

    3. Disease duration from diagnosis of ≥5 years. Responsiveness to dopamine.

    4. 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:

    1. Atypical or secondary parkinsonism.

    2. History of coagulopathy, abnormal bleeding or hemopathy family history. Other bleeding risk which increases risk of surgery determined by investigator.

    3. Any type of prior gene therapy.

    4. Clinically significant electrocardiogram (ECG) abnormalities.

    5. Concomitant disease including unstable cardiovascular and cerebrovascular diseases within 3 months, uncontrolled hypertension, sever postural hypotension, poorly controlled diabetes, history of malignancy.

    6. 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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Xiangya Hospital of Central South University Changsha Hunan China 410008

    Sponsors and Collaborators

    • Xiangya Hospital of Central South University

    Investigators

    • Principal Investigator: Lu Shen, M.D, Ph.D, Xiangya Hospital of Central South University

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Xiangya Hospital of Central South University
    ClinicalTrials.gov Identifier:
    NCT05822739
    Other Study ID Numbers:
    • BBM003- IIT1003
    First Posted:
    Apr 21, 2023
    Last Update Posted:
    Apr 21, 2023
    Last Verified:
    Feb 1, 2023
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Xiangya Hospital of Central South University
    Parkinson's Disease
    Gene therapy
    Adeno-Associated Virus
    Additional relevant MeSH terms:
    Parkinson Disease

    Study Results

    No Results Posted as of Apr 21, 2023