Clinical Study on the Safety, Tolerance and Preliminary Efficacy of Human Mesenchymal Stem Cell Therapy for Parkinson's Disease
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 20
- 主要终点
- Incidence of Treatment-Emergent Adverse Events (TEAEs)
研究概览
简要总结
This study, through different administration methods, adopted a randomized, double-blind, placebo-controlled trial design to evaluate the safety and tolerability of human umbilical cord mesenchymal stem cells (hUC-MSCs) in patients with Parkinson's disease, explore their initial effectiveness and the relationship between biological active factors and therapeutic efficacy. The "Clinical Study on the Treatment of Parkinson's Disease with Human Umbilical Cord Mesenchymal Stem Cells" of this study is expected to provide clinical trial evidence for the development of safe and effective clinical cell therapies for patients with Parkinson's disease.
详细描述
Research Design and Implementation
(1) Inclusion, Exclusion and Withdrawal Criteria for Participants
- Case Inclusion Criteria:
1.1 Male and female individuals aged between 40 and 70 years old, excluding adolescent patients with Parkinson's disease.
1.2 According to the 2015 MDS Parkinson's disease diagnostic criteria, confirmed by a PI or other movement disorder experts based on medical history, physical examination and neurological examination.
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
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •The participants must meet all of the following criteria to be included in this study:
- •The participants must fully understand and comply with the research procedures, voluntarily participate in the study and sign the informed consent form;
- •At the time of signing the informed consent, the participants must be aged 18 or above and under 75 years old, with no gender restrictions;
- •During the screening process, the participants must have had primary Parkinson's disease for at least 5 years, have a confirmed medical history record and meet the diagnostic criteria for primary Parkinson's disease as defined by the International Parkinson and Movement Disorder Society (MDS);
- •During the screening, according to the MDS-UPDRS scoring scale, the Hoehn-Yahr classification of the "off" period of drug treatment is 2 to 4;
- •During the screening, the score of the third part of the MDS-UPDRS in the "off" period must be greater than 30;
- •During the screening, the stable duration of Parkinson's disease and the stable duration of the optimized drug dosage must be at least 4 weeks, and the duration of levodopa use must be at least 1 year;
- •During the screening, the participants must have a response to levodopa treatment, and the levodopa loading test must be positive;
- •The participants must experience a decline in the efficacy of anti-Parkinson's disease treatment, which affects their quality of life;
- •The participants must have good compliance and be able to cooperate with the completion of the assessment items of the trial; For participants with reproductive potential and their partners, they must be free from pregnancy plans for at least 2 weeks before the screening to at least 1 year after the administration of the drug, and must agree to take effective non-drug contraceptive measures during the trial (such as condoms, non-drug intrauterine devices, etc.), except for those who have taken permanent contraceptive measures, such as bilateral tubal ligation, vasectomy, etc.
排除标准
- •If the subjects meet any of the following criteria, they will not be included in this study:
- •Allergic to the study drug or its excipients, or allergic to similar drugs of the study drug, or have a history of severe allergies (including any food allergy or drug allergy);
- •Have a previous history of mental disorders, serious diseases, or other significant diseases that may affect safety;
- •Have a known history of human immunodeficiency virus (HIV) infection (HIV 1/2 antibody positive), or acquired immunodeficiency syndrome-related diseases, or positive HIV serological test results;
- •Positive for hepatitis B surface antigen (HBsAg), or hepatitis C antibody (HCV-Ab), or Treponema pallidum antibody positive;
- •Previously diagnosed with secondary or atypical Parkinson's syndrome caused by drugs, metabolic disorders, or other reasons;
- •Previously diagnosed with epilepsy, stroke, multiple sclerosis, poorly controlled or progressive neurological diseases;
- •Have new or unstable mental symptoms within 1 year before screening (such as mental confusion, severe depression, or tendencies towards self-harm/suicide);
- •Have a history of dementia or severe cognitive dysfunction; or have obvious dementia or cognitive dysfunction at screening; the 1.1 part of the MDS-UPDRS score at screening is > 3; due to dementia, the subject's compliance is affected, diary cannot be accurately recorded, and/or the informed consent cannot be signed;
- •Have other serious systemic diseases at the time of screening;
- •Have any history of malignant tumors in the past;
- •Are participating in other clinical trials, or have participated in other clinical studies within 3 months before administration and received intervention treatment;
- •Have active infections at the screening period, and still need systemic application of antibiotics, antifungal, antiviral treatment at baseline and the infection has not been controlled;
- •Have a history of stroke, unstable angina pectoris, or myocardial infarction attack within 6 months before screening;
- •Have a history of schizophrenia or other severe mental disorders, drug or alcohol abuse;
- •Pregnant or lactating women; Subjects deemed unsuitable for participation in the study by the investigator's comprehensive assessment.
研究组 & 干预措施
Human-derived stromal cells
Administer human matrix cells by intravenous infusion or nasal drip once every two weeks, for a total of 5 administrations.
干预措施: Human-derived stromal cells (Biological)
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events (TEAEs)
时间窗: From baseline up to 48 weeks
The safety and tolerability of hUC-MSCs will be assessed by monitoring the frequency of all adverse events (AEs) and serious adverse events (SAEs). Measure: Number of participants with AEs and SAEs
Severity of Adverse Events
时间窗: From baseline up to 48 weeks
Severity of AEs and SAEs will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) Version 5.0. Measure: Grade on NCI-CTCAE v5.0 scale
Safety Assessments: Vital Signs
时间窗: From baseline up to 48 weeks
Changes in vital signs including body temperature, heart rate, blood pressure, and oxygen saturation. Measure: Number of participants with clinically significant changes in vital signs
Safety Assessments: Laboratory Parameters
时间窗: From baseline up to 48 weeks
Changes in laboratory tests including complete blood count, coagulation profile, liver and kidney function, BNP, D-dimer, and serum troponin. Measure: Number of participants with clinically significant abnormal laboratory values
Safety Assessments: Physical Examination
时间窗: From baseline up to 48 weeks
Findings from physical examinations, including skin lesions/rashes. Measure: Number of participants with clinically significant physical examination findings
次要结局
- Change in Motor Function as Assessed by MDS-UPDRS Part III(Baseline, 4, 16, 28, and 48 weeks)
- Change in Disease Staging as Assessed by Hoehn & Yahr Staging(Baseline, 4, 16, 28, and 48 weeks)
- Change in Non-Motor Symptoms as Assessed by NMSS(Baseline, 4, 16, 28, and 48 weeks)
- Change in Depressive Symptoms as Assessed by HAMD(Baseline, 4, 16, 28, and 48 weeks)
- Change in Anxiety Symptoms as Assessed by HAMA(Baseline, 4, 16, 28, and 48 weeks)