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

Parkinson's Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05822739
• Other study ID #: BBM003- IIT1003
• Status: Not yet recruiting
• Phase: Early Phase 1
• Start date: April 28, 2023
• Est. completion date: December 30, 2028

Study information

• Verified date: February 2023
• Source: Xiangya Hospital of Central South University
• Contact: Lu Shen, M.D, Ph.D
• Phone: 86-731-84327216
• Email: shenlu[@]csu.edu.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 6
• Est. completion date: December 30, 2028
• Est. primary completion date: May 30, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 40 Years to 70 Years

Eligibility

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.

Related Conditions & MeSH terms

• Parkinson Disease
• Parkinson's Disease

Intervention

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

Locations

Country	Name	City	State
China	Xiangya Hospital of Central South University	Changsha	Hunan

Sponsors (1)

Lead Sponsor	Collaborator
Xiangya Hospital of Central South University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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	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