Phase I Randomized, Double-blind Study to Evaluate Feasibility, Safety, and Clinical Responses of Implanting Autologous Peripheral Nerve Tissue Into the Nucleus Basalis of Meynert or Substantia Nigra for Non-motor or Motor Symptoms in Patients With Parkinson's Disease Undergoing DBS Surgery
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 24
- 试验地点
- 2
- 主要终点
- Successful deployment of bilateral peripheral nerve tissue (PNT) into the brain
研究概览
简要总结
The investigators propose a Phase I single surgical-center, double-blinded randomized parallel clinical trial involving bilateral autologous peripheral nerve tissue (PNT) delivery into the NBM or the alternate target also affecting cognition in this population, the substantia nigra (SN), to address "repair cell" support of these areas. Twenty-four participants with idiopathic Parkinson's Disease (PD) who have selected, qualified and agreed to receive as standard of care deep brain stimulation (DBS) will be enrolled and randomly allocated to receive bilateral PNT deployment to either the NBM or SN at the time of DBS surgery. Participants will be allocated equally among both assignments over the course of three years (8 Year 1, 10 Year 2, 6 Year 3). Participants will be evaluated for neurocognitive, motoric function, activities of daily living, and quality of life at enrollment before surgery, two-weeks after surgery, and 6, 12, and 24 months after surgery.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 45 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Undergoing DBS
- •Diagnosis of clinically established or clinically probably PD as defined by MDS criteria
- •Age 45-75, inclusive
- •Able to tolerate the surgical procedure
- •Able to undergo all planned assessments
- •Available access to the sural nerve
排除标准
- •Any condition that would not make the subject a candidate for DBS
- •Dementia diagnosis
- •Previous PD surgery or intracranial surgery
- •Unable to undergo an MRI
- •An obstructed trajectory path to the substantia nigra and nucleus basalis of Meynert
研究组 & 干预措施
Peripheral nerve tissue (PNT) deployment to the Substantia Nigra
干预措施: Reparative Autologous peripheral nerve tissue (Procedure)
Peripheral nerve tissue (PNT) deployment to the nucleus basalis of Meynert
干预措施: Reparative Autologous peripheral nerve tissue (Procedure)
结局指标
主要结局
Successful deployment of bilateral peripheral nerve tissue (PNT) into the brain
时间窗: Intraoperative
Number of participants in each study arm who successfully receive bilateral PNT delivery.
Number of participants completing 12 month study visit
时间窗: 12-month study visit
Total number of participants to complete study visit by arm assignment
Study-related adverse events as assessed by MedDRA v27
时间窗: Enrollment to 24-month study visit
Total number of study-related adverse events experienced by participants.
Study-related serious adverse events as assessed by MedDRA v.27
时间窗: Enrollment to 24-month study visit
Total number of serious adverse events experienced by participants
次要结局
- PNT deployment attempts(During surgery)
- Mean change in Montreal Cognitive Assessment (MoCA) scores(Baseline 6, 12 and 24 months after surgery)
- Mean change in Neuropsychological assessment scores(Baseline, 12 and 24 months)
- Change in Neuropsychological diagnosis(Baseline, 12 and 24 months)
- Change in Neuropsychological domains with impairment(At 12, 24 months compared to baseline)
- Mean change in Modified Schwab and England Scale of Activities of Daily Living scores(At 6, 12, 24 months compared to baseline)
- Mean change in Parkinson's Disease Questionnaire-8 (PDQ-8) quality of life scores(At 12 and 24 months compared to baseline)
- Mean change in Non-motor symptom scale scores(At 12 and 24 months compared to baseline)
- Mean change of the Movement Disorder Society - Unified Parkinsons Disease Rating Scale (MDS-UPDRS) Part I scores(6, 12, and 24 months as compared to baseline)
- Mean change of the MDS-UPDRS Part II scores(At 6, 12, 24 months compared to baseline)
- Mean change in MDS-UPDRS Part III scores(At 6, 12, 24 months compared to baseline)
- Mean change in MDS-UPDRS Part IV scores(At 6, 12, and 24 months compared to baseline)
- Number of participants completing 24-month study visit(24-month study visit)
- Mean change in Dementia Rating Scale (DSRS)(Baseline 6, 12 and 24 months after surgery)
研究者
Craig van Horne, MD, PhD
Department of Neurosurgery Chair
University of Kentucky