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

Central South University

Status and phase: Not yet enrolling; Early Phase 1

Conditions: Parkinson's Disease

Treatments: Drug: BBM-P002

Study type: Interventional

Funder types: Other

Identifiers

NCT05822739

BBM003- IIT1003

Details and patient eligibility

About

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

Full 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.

Enrollment: 6 estimated patients

Sex: All

Ages: 40 to 70 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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6 participants in 1 patient group

Arm of BBM-P002

Experimental group

Description:

single-arm

Treatment:

Drug: BBM-P002