A Clinical Study to Evaluate the Safety, and Tolerability of BBM-P002 in the Treatment of Parkinson's Disease

Shanghai Xinzhi BioMed Co., Ltd.

Status and phase

Enrolling

Phase 1

Conditions

Treatments

Genetic: Injecting BBM-P002 into the bilateral putamen

Study type

Interventional

Funder types

Industry

Identifiers

NCT07195825

BBM003-CLN001

Details and patient eligibility

About

The purpose of the study is to evaluate the safety and tolerability of BBM-P002 for stereotactic injection to treat participants with idiopathic Parkinson's Disease (PD) during the dose-limiting toxicity (DLT) observation period.

Full description

This is a single-arm, open-label study to evaluate the safety, tolerability, efficacy, pharmacokinetic, pharmacodynamic, and immune response of BBM-P002 within 52 weeks after into the bilateral putamen in Parkinson's disease, as well as the long-term safety and efficacy of BBM-P002 for up to 5 years post injection.

BBM-P002 is an AAV-based gene therapy that delivers a therapeutic gene cassette into putamen for the treatment of Parkinson's Disease.

Enrollment

18 estimated patients

Sex

All

Ages

40 to 70 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Participants with a diagnosis of Parkinson's disease for ≥ 5 years.
The Hoehn-Yahr staging meets the requirements during the OFF periods.
The MDS-UPDRS Part III score met the requirements during the OFF periods. the levodopa challenge test was positive
During the screening period, participants were required to take the same stable dose of dopamine-releasing drugs (such as levodopa) regularly
The participants agreed to postpone the neurosurgical procedures related to the indication treatment during the main study phase
The participants agreed not to participate in any other therapeutic intervention studies during the trial period
The participants agreed not to receive the vaccine during the main study phase
From the screening period until at least 52 weeks after dosing, use a reliable contraceptive method
The participants had good compliance and were able to undergo regular follow-ups. During the follow-up period, they were able to accurately complete the diary cards. Family members, guardians or caregivers could assist the subjects in filling out the diary cards
Voluntarilyvoluntarily participated in the study and signed the informed consent form

Exclusion criteria

Atypical or secondary Parkinsonism
Have contraindications for surgery or have previously undergone brain surgery
The participants has an abnormal brain MRI suggestive of brain pathology other than Parkinson's disease
Those with severe cognitive impairments
Those with severe depression or severe anxiety
Abnormal liver function
Abnormal coagulation function
Abnormalities in infectious disease screening
Currently undergoing antiviral treatment for hepatitis
Suffering from unstable or severe diseases in the cardiovascular, respiratory, digestive, nervous, hematological, immune and other systems
Suffering from malignant tumors or having a history of tumors
Previous history of severe allergies
Exclude those who have participated in other clinical trials within the past three months
Had received gene therapy during the screening period
Select those who have received stem cell treatment within the past 12 months
Exclude those who have used other investigational drugs within the past 4 weeks
During the screening period, if the individual had received live vaccines within the previous 2 months, or had a history of vaccination within the last 30 days, they would be excluded.
Having a history of alcohol dependence or drug addiction
Pregnant or lactating female participants
The situations that were determined by the investigator to be unsuitable for inclusion in the study