NCT07195825招募中1 期
A Single-arm, Open-label, Single-dose Clinical Study to Evaluate the Safety and Tolerability of BBM-P002 for Stereotactic Bilateral Putamen Injection in the Treatment of Idiopathic Parkinson's Disease
Shanghai Xinzhi BioMed Co., Ltd.1 个研究点 分布在 1 个国家目标入组 18 人开始时间: 2025年12月27日最近更新:
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- Incidence of dose limiting toxicity (DLT) events
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 40 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
Arm of BBM-P002
Experimental
Single-dose treatment
干预措施: Injecting BBM-P002 into the bilateral putamen (Genetic)
结局指标
主要结局
Incidence of dose limiting toxicity (DLT) events
时间窗: Within 4 weeks
To evaluate the rate of incidence of DLT events determined by the Safety Data Review Committee (SRC) in DLT observation period after injecting BBM-P002
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Within 4 weeks
To evaluate the safety of BBM-P002 by AEs and SAEs.
次要结局
- Changes in the titers of neutralizing antibodies and binding antibodies in the recombinant adenovirus capsid of blood(Within 5 years)
- Changes in the results of the cranial MRI scan(Within 5 years)
- Changes from baseline in MDS - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) I/II/III/IV scores(Within 5 years)
- Changes from baseline in daily levodopa dose or equivalent dose of levodopa(Within 5 years)
- Changes from baseline in the percentage at on time.(Within 5 years)
- Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)(Within 5 years)
- Changes from baseline in the Patient Global Impression of Improvement (PGI-I) scores(Within 5 years)
- Changes from baseline in the Clinical Global Impression of Improvement (CGI-I) scores(Within 5 years)
- Changes in the activity levels of gene expression products under18F-dopa PET scanning from baseline [striatum - occipital lobe average radioactivity count ratio](Within 5 years)
研究者
研究点 (1)
Loading locations...
相似试验
终止
1 期
Stereotactic Body Radiotherapy (SBRT) and Sorafenib in Patients With Metastatic, Recurrent, or Unresectable Renal Cell Cancer (RCC)Renal Cell CarcinomaNCT00672178Duke University1
尚未招募
1 期
Stereotactic Body Radiation Therapy Followed by NALIRIFOX vs NALIRIFOX for Borderline Resectable Pancreatic CancerPancreatic CancerNCT06259058Tianjin Medical University Cancer Institute and Hospital96
已完成
不适用
Safety and Preliminary Efficacy of SBRT in Patients With Metastatic RCC Treated With Targeted or IO TherapyMetastatic Renal Cell CarcinomaNCT02864615Kidney Cancer Research Bureau17
已完成
不适用
Efficacy and Safety of SBRT in Oligo-metastatic/Persistent/Recurrent Ovarian CancerOvarian NeoplasmsRecurrent Ovarian CarcinomaOligometastatic DiseaseNCT04593381Gemelli Molise Hospital376
招募中
2 期
Positron Emission Tomography (PET) Guided Stereotactic Body Radiation Therapy for the Treatment of Oligoprogressive Non-small Cell Lung Cancer, Melanoma, and Renal Cell CarcinomaLung Non-Small Cell CarcinomaMalignant Solid NeoplasmMelanomaRenal Cell CarcinomaNCT05830058City of Hope Medical Center32