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Safety and Feasibility of Bilateral Striatal Transplantation of DopaCell in Parkinson's Disease

2026年6月16日 更新者:Royan Institute

Evaluation of the Safety and Feasibility of a Single Transplantation of 10 Million Human Embryonic Stem Cell-Derived Dopaminergic Progenitor Cells Into the Bilateral Striatum of Patients With Moderately Severe Parkinson's Disease: a Multicenter, Open-label, Single-arm Phase I Clinical Trial

Dopason is a phase I, open-label, multicenter, single-arm clinical trial designed to evaluate the safety and feasibility of intraputaminal transplantation of human embryonic stem cell-derived dopaminergic progenitor cells (DopaCells) in patients with moderately severe Parkinson's disease.

研究概览

详细说明

During this trial phase, a total of six participants will be included and distributed between two clinical centers. Each participant will undergo one standardized surgical procedure consisting of bilateral stereotactic intraputaminal transplantation of approximately 10 × 10⁶ cells in total (about 5 × 10⁶ cells per putamen), aiming to achieve an estimated survival of 200,000 neurons. All procedures will be performed using the same surgical delivery system and an almost identical operative protocol across both sites to ensure procedural consistency. After transplantation, patients will receive immunosuppressive therapy for a duration of one year and will be systematically followed for study outcomes for at least 12 months.

研究类型

介入性

注册 (估计的)

6

阶段

  • 阶段1

联系人和位置

本节提供了进行研究的人员的详细联系信息,以及有关进行该研究的地点的信息。

学习联系方式

学习地点

      • Shiraz、伊朗
        • 招聘中
        • Shiraz University of Medical Sciences
        • 接触:
      • Tehran、伊朗
        • 招聘中
        • Royan Institute
        • 接触:
      • Tehran、伊朗
        • 招聘中
        • Iran University of Medical Sciences
        • 接触:

参与标准

研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

  • 成人
  • 年长者

接受健康志愿者

描述

Inclusion Criteria:

  • Age: 30-70 years
  • Diagnosis of PD: MDS clinical Diagnostic Criteria for Parkinson's disease
  • The disease duration more than 5 years
  • Moderate Parkinson's disease, defined as a Hoehn and Yahr stage of 2 or 3 during the OFF period.
  • The patient is receiving oral pharmacological therapy, and in the opinion of the Principal Investigator, the patient's symptoms remain inadequately controlled despite optimal medical management, or the patient is experiencing adverse effects related to their current treatment
  • No history or only mild levodopa-induced dyskinesia, defined as a score of 2 or less on the UDysRS scale in any body region during the ON state.
  • The patient demonstrates a clinically meaningful response to a therapeutic dose of levodopa, as determined by the Principal Clinical Investigator or a trained specialist under the supervision of the Principal Investigator.
  • The performance of different organs based on laboratory evaluations:

    • Number of neutrophils ≥2000 / microliter
    • Platelet count ≥100,000 / microliter
    • AST / ALT: less than or equal to three times the maximum normal value at the intervention site
    • Total bilirubin less than or equal to 1.5 times the maximum normal amount at the intervention site
    • eGFR * rate: greater than or equal to 60 ml / min / 1.73 m2 * eGFR (mL / min / 1.73 m2) = 194 X Cr ^ -1.094 X age ^ -0.287 (X 0.739 for females)
  • Informed consent

Exclusion Criteria:

  • The abnormal function of immune system
  • The symptomatic brain injuries (brain atrophy, cerebral Infarct, trauma, vascular malformation) confirmed by brain MRI
  • Markedly reduced or normal signal in the ventral striatum on TRO-DaT SPECT imaging.
  • Any abnormal findings on brain MRI.
  • Positive GBA mutation test.
  • Diagnosis of dementia based on a MoCA score < 24.
  • The abnormality of thrombotic system or high risk of bleeding
  • Positive for any of the following viral markers or active infections: HBsAg, HBsAb, HBcAb, anti-HIV antibodies, anti-HTLV-1&2 antibodies, active hepatitis C infection, syphilis, or active CMV, VZV, EBV, or COVID-19 infection.
  • Impossibility of MRI imaging for patients with metal in the body, pacemaker in the body, claustrophobia, with artificial heart valves that are incompatible with MRI or body weight is not within the tolerable range for MRI.
  • Patients with contraindications to the study drug: Tacrolimus, Prednisolone, Basiliximab, Cotrimoxazole, MRI contrast agent.
  • Patients undergoing other cell transplants, including embryonic stem cell-derived dopaminergic progenitor cells.
  • Patients with a history of PD at the same time and concurrent: Malignant neoplasm, epilepsy, cerebral hemorrhage or a positive history
  • Psychiatric disorders confirmed by a psychiatrist, including major depression, bipolar disorder, or schizophrenia, that are uncontrolled or treatment resistant.
  • Patients with intellectual disability who, in the judgment of a psychiatrist, are unable to fully comprehend the study requirements.
  • History of pallidotomy, thalamotomy, or deep brain stimulation (DBS).
  • Patients considered high-risk candidates for surgery, particularly neurosurgery or DBS implantation, due to significant cardiovascular, pulmonary, or other systemic comorbidities identified during preoperative evaluation.
  • Patients who have a history of taking the following in the three months prior to enrollment: Immunosuppressant, antipsychotic drug, anticonvulsant drugs or anticoagulant therapy (if discontinuation or perioperative adjustment is not feasible), botulinum toxin (within 6 months), phenol injections, or other treatments for dystonia or muscle spasm
  • History of Apomorphine use
  • History of chronic alcohol use or illicit drug abuse.
  • Patients who are pregnant, lactating, or people who did not avoid pregnancy during the study.
  • Patients who, according to the researchers' opinions, are not suitable for safe study.

学习计划

本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。

研究是如何设计的?

设计细节

  • 主要用途:治疗
  • 分配:不适用
  • 介入模型:单组作业
  • 屏蔽:无(打开标签)

武器和干预

参与者组/臂
干预/治疗
实验性的:Transplantation
Experimental: DopaCell Biological: DopaCell, 5 million cells per putamen Immunosuppressive Regimen: Basiliximab, Tacrolimus, Prednisolone Device: Customized microinjection device
DopaCells are dopamine-producing progenitor cells generated from human embryonic stem cells through differentiation under GMP-compliant conditions.
Basiliximab, Tacrolimus, Prednisolone
For intraputaminal delivery of DopaCell

研究衡量的是什么?

主要结果指标

结果测量
措施说明
大体时间
Safety
大体时间:Baseline to 12 Months Post-Transplant
Incidence and severity of treatment-emergent adverse events (TEAEs)
Baseline to 12 Months Post-Transplant
Safety
大体时间:Baseline to 12 Months Post-Transplant
Evidence of any post-transplantation anatomical changes at the transplant site suggestive of tumor formation, as assessed by MRI imaging.
Baseline to 12 Months Post-Transplant
Feasibility
大体时间:Baseline to 12 Months Post-Transplant
Successful completion of a single-session intraputaminal transplantation with of the intended cell dose
Baseline to 12 Months Post-Transplant

次要结果测量

结果测量
措施说明
大体时间
Efficacy
大体时间:Baseline to 12 Months Post-Transplant
Changes in the duration of OFF and ON periods following intracerebral DopaCells transplantation as assessed by the Hauser diary
Baseline to 12 Months Post-Transplant
Efficacy
大体时间:Baseline to 12 Months Post-Transplant
Changes in the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III score from baseline following intracerebral DopaCells transplantation during ON and OFF periods ( MDS-UPDRS Part III ranges from 0 to 132, where higher scores indicate worse motor impairment.)
Baseline to 12 Months Post-Transplant
Efficacy
大体时间:Baseline to 12 Months Post-Transplant
Graft survival following intracerebral DopaCells transplantation based on TRODAT SPECT imaging findings.
Baseline to 12 Months Post-Transplant
Continuous Safety
大体时间:Baseline to 60 Months Post-Transplant
Continuous Safety: The number and severity of treatment-emergent adverse events
Baseline to 60 Months Post-Transplant

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2025年8月1日

初级完成 (估计的)

2028年8月1日

研究完成 (估计的)

2030年8月1日

研究注册日期

首次提交

2026年4月7日

首先提交符合 QC 标准的

2026年5月2日

首次发布 (实际的)

2026年5月7日

研究记录更新

最后更新发布 (实际的)

2026年6月18日

上次提交的符合 QC 标准的更新

2026年6月16日

最后验证

2026年6月1日

更多信息

与本研究相关的术语

药物和器械信息、研究文件

研究美国 FDA 监管的药品

研究美国 FDA 监管的设备产品

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