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

NCT05822739 | Completed Early Phase 1

• 1 other identifier: BBM003- IIT1003
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 2

Brief Summary

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

Conditions

Parkinson's Disease

Keywords

Parkinson's Disease; Gene therapy; Adeno-Associated Virus

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (4)

• Efficacy of BBM-P002

  Change from Baseline to week 4，week 52

• Efficacy of BBM-P002

  Change from Baseline to Week 52

• Efficacy of BBM-P002

  Change from Baseline to Week 52

• Efficacy of BBM-P002

  Change from Baseline to Week 52

Study Arms (1)

Arm of BBM-P002

EXPERIMENTAL

single-arm

Drug: BBM-P002

Interventions

BBM-P002 DRUG

Genetic: single- dose intracranial injection of BBM-P002 Neurosurgical delivery of BBM-P002 to the brain

Also known as: BBM003

Arm of BBM-P002

Eligibility Criteria

• Age: 40 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Clinically confirmed diagnosis of idiopathic Parkinson's Disease (PD) in accordance with the 2015 Movement Disorder Society (MDS) diagnostic criteria.
• Participants aged between 40 and 65 years, inclusive, of any gender.
• A disease history of five years or more.
• A Hoehn and Yahr (H-Y) stage of 2.5 to 4 in the Of-state.
• An adeno-associated virus (AAV) neutralizing antibody titer of fit thecriteria.
• A Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III score greater than 35 in the Off-state, with an improvement of at least 30% following an acute levodopa challenge test.
• Stable anti-Parkinsonian medication regimen prior to the screening period for at least four weeks.
• The subject must agree to postpone any other neurosurgical procedures (including deep brain stimulation) during the main study phase, with the exception of emergency neurosurgery for life-threatening conditions.
• The subject must agree not to participate in other therapeutic interventional studies during the main study phase.
• The subject must agree not to undergo vaccination during the main study phase.
• Commitment to using a reliable method of contraception from the screening period until at least 52 weeks post-infusion.
• The subject must be compliant and capable of attending regular follow-up appointments. The subject must be able to accurately complete a PD patient diary during the follow-up period, with assistance from family members, guardians, or caregivers permitted.
• The subject must be fully informed about the nature, objectives, procedures, and potential adverse effects of the clinical trial, and must voluntarily consent to participate by signing the informed consent form. In cases where the subject is unable to read, a legally authorized representative or an impartial witness may read the informed consent form and other written materials to them and witness the consent process.

You may not qualify if:

• Diagnosis of atypical or secondary parkinsonian syndromes (e.g., Parkinson-plus syndromes, hereditary parkinsonian syndromes, drug-induced parkinsonism).
• Presence of surgical contraindications, a history of prior brain surgery such as deep brain stimulation (DBS), pallidotomy, or extrapyramidal surgery, or any other neurosurgical procedure that the investigator deems would interfere with participation in this trial.
• Previous cranial imaging showing structural brain abnormalities, cerebrovascular malformations, intracranial tumors, risk of intracranial hemorrhage, traumatic brain injury, or other significant anomalies.
• A Mini-Mental State Examination (MMSE) score of \<24.
• A Patient Health Questionnaire-9 (PHQ-9) score of ≥16.
• Abnormal liver or kidney function, defined as: Aspartate aminotransferase (AST) or Alanine aminotransferase (ALT) \>1.5 times the upper limit of normal (ULN), or serum creatinine (Cr) \>1.5 times the ULN.
• Coagulation dysfunction or current use of anticoagulant agents.
• Positive screening for infectious diseases: positive for Hepatitis B surface antigen (HBsAg) or Hepatitis B virus DNA (HBV-DNA), positive for Hepatitis C virus RNA (HCV-RNA), positive for Human Immunodeficiency Virus (HIV), or seropositive for syphilis.
• Currently undergoing antiviral treatment for Hepatitis B or C.
• Presence of unstable or severe concomitant systemic diseases, including but not limited to active tuberculosis, or diseases of the cardiovascular, respiratory, digestive, urinary, neuropsychiatric (e.g., epilepsy), hematologic, or immune systems, or abnormal laboratory values that, in the judgment of the investigator, would make the subject unsuitable for the trial.
• Current or prior history of a malignant tumor.
• History of a severe allergic reaction, allergy to contrast agents, or inability to tolerate surgical anesthesia.
• Current participation in another clinical trial, or participation in any other clinical trial within 3 months prior to the screening period.
• Prior history of gene therapy before screening.
• Receipt of stem cell therapy within 6 months prior to the screening period.

Sponsors & Collaborators

• Xiangya Hospital of Central South University: lead

Study Sites (2)

Xiangya Hospital of Central South University

Changsha, Hunan, 410008, China

Location

Ruijin Hospital, Shanghai Jiao Tong University School of Medicine

Shanghai, China

Location

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 1, 2023
• First Posted: April 21, 2023
• Study Start: April 14, 2023
• Primary Completion: October 30, 2025
• Study Completion: October 30, 2025
• Last Updated: January 15, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

CN (2)