"A Randomized, Double-Blind, Single Center, Phase 2, Efficacy and Safety Study of Autologous HB-adMSCs vs Placebo for the Treatment of Patients With Parkinson's Disease"
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 24
- 试验地点
- 1
- 主要终点
- Laboratory Values. CMP (mg/dL)
研究概览
简要总结
This is a randomized, double-blind, single center, phase 2 study to assess efficacy and safety of multiple HB-adMSCs vs Placebo for the treatment of Parkinson's disease.
The trial includes a screening period of up to 4 weeks, a 32-week treatment period, and a safety Follow-up period of 20 weeks after the last investigational product administration.
This clinical trial will be open to enroll 24 eligible participants diagnosed with Parkinson's disease. Patients' recruitment will be conducted by the study team, if eligible participants are identified based on eligibility criteria, a screening visit will be scheduled. Informed consent form will be given to the study participants and signed before any study procedures. Informed consent form will include information about the clinical trial and some aspects should be considered during this process.
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)
盲法说明
Study subjects, investigators and study staff will be blinded to the assigned treatment.
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
- 否
入选标准
- •A study participant will be eligible for inclusion in this study only if all of the following criteria apply:
- •Male and female participants 18 - 75 years of age.
- •Study participant must have been diagnosed with early and/or moderate Parkinson's disease at least 6 months before study participation.
- •Study participants must have previously banked their mesenchymal stem cells with Hope Biosciences.
- •Study participants should be able to read, understand and to provide written consent.
- •Voluntarily signed informed consent obtained before any clinical-trial related procedures are performed.
- •Female study participants should not be pregnant or plan to become pregnant during study participation and for 6 months after last investigational product administration.
- •Male participants if their sexual partners can become pregnant should use a method of contraception during study participation and for 6 months after the last administration of the investigated product.
- •Study participant is able and willing to comply with the requirements of this clinical trial.
排除标准
- •A study participant will not be eligible for inclusion in this clinical trial if any of the following criteria apply:
- •Pregnancy, lactation. Women of childbearing age who are not pregnant but do not take effective contraceptive measures.
- •Study participants with advanced Parkinson's disease described as, severe disability, wheelchair bound or bedridden.
- •Study participant has any active malignancy, including evidence of cutaneous basal, squamous cell carcinoma or melanoma.
- •Study participant has known alcoholic addiction or dependency or has current substance use or abuse.
- •Study participant has 1 or more significant concurrent medical conditions (verified by medical records), including the following:
- •Poorly controlled diabetes mellitus (PCDM) defined as history of deficient standard of care treatment and/or pre-prandial glucose >130mg/dl during screening visit or post-prandial glucose >200mg/dl.
- •Medical History of Chronic kidney disease (CKD) diagnosis and/or screening results of eGFR < 59mL/min/1.73m
- •Presence of New York Heart Association (NYHA) Class III/IV heart failure during screening visit.
- •Any medical history of myocardial infarction in any of the different types, such as ST-elevation myocardial infarction (STEMI) or non-ST-elevated myocardial infarction (NSTEMI), coronary spasm, or unstable angina.
- •Medical history of uncontrolled high blood pressure defined as a deficient standard of care treatment and/or blood pressure > 180/120 mm/Hg during screening visit.
- •Medical history of inherited thrombophilias, recent major general surgery, (within 12 months before the Screening), lower extremity paralysis due to spinal cord injury, fracture of the pelvis, hips or femur, cancer of the lung, brain, lymphatic, gynecologic system (ovary or uterus), or gastrointestinal tract (like pancreas or stomach).
- •History of brain surgery for Parkinson's disease.
- •Study participant has received any stem cell treatment within 6 months before first dose of investigational product other than stem cells produced by Hope Biosciences.
- •Receiving any investigational therapy or any approved therapy for investigational use within 1 year prior first dose of the investigational product other than COVID-19 vaccines.
- •Study participant has a laboratory abnormality during screening, including the following:
- •White blood cell count < 3000/mm3
- •Platelet count < 80,000mm3
- •Absolute neutrophil count < 1500/mm3
- •Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) 10 upper limit of normal (ULN) x 1.5
- •Study participant has any other laboratory abnormality or medical condition which, in the opinion of the investigator, poses a safety risk or will prevent the subject from completing the study.
- •Study participant is unlikely to complete the study or adhere to the study procedures.
- •Study participant with known concurrent acute or chronic viral hepatitis B or C or human immunodeficiency virus (HIV) infection.
- •Study participant has a previously diagnosed psychiatric condition which in the opinion of the investigator may affect self-assessments.
- •Study participant with any systemic infection requiring treatment with antibiotics, antivirals, or antifungals within 30 days prior to first dose of the investigational product.
- •Male study participants who plan to donate sperm during the study or within 6 months after the last dose. Female patients who plan to donate eggs or undergo in vitro fertilization treatment during the study or within 6 months after the last dose.
- •Study participants who are determined by the Investigator to be unsuitable for study enrollment for other reasons
研究组 & 干预措施
HB-adMSCs
Autologous Hope Biosciences adipose derived mesenchymal stem cells.
干预措施: HB-adMSCs (Biological)
HB-adMSCs
Autologous Hope Biosciences adipose derived mesenchymal stem cells.
干预措施: Placebo (Other)
Placebo
Sterile Saline Solution 0.9%
干预措施: Placebo (Other)
Placebo
Sterile Saline Solution 0.9%
干预措施: HB-adMSCs (Biological)
结局指标
主要结局
Laboratory Values. CMP (mg/dL)
时间窗: Baseline (Week 0), Week 24, and End of Study (Week 52)
Change from baseline in CMP values with units of mg/dL
Laboratory Values. CMP (Ratio: Urea Nitrogen (mg/dL) to Creatinine (mg/dL))
时间窗: Baseline (Week 0), Week 24, and End of Study (Week 52)
Changes from baseline in CMP laboratory values with units of Ratio: Urea Nitrogen (mg/dL) to Creatinine (mg/dL)
Laboratory Values. CBC (% of WBC)
时间窗: Baseline (Week 0), Week 24, and End of Study (Week 52)
Changes from baseline in CBC laboratory values with unit of % of white blood cell count.
Laboratory Values. CBC (pg)
时间窗: Baseline (Week 0), Week 24, and End of Study (Week 52)
Changes from baseline in CBC laboratory values with unit of pg.
Laboratory Values. CBC (fL)
时间窗: Baseline (Week 0), Week 24, and End of Study (Week 52)
Changes from baseline in CBC laboratory values with unit of fL.
Weight in kg.
时间窗: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Change from baseline in Weight in kg.
Laboratory Values. CMP (Ratio: Albumin (g/dL) to Calc. Globulin (g/dL))
时间窗: Baseline (Week 0), Week 24, and End of Study (Week 52)
Changes from baseline in CMP laboratory values with units of Ratio: Albumin(g/dL) to Calc. Globulin(g/dL)
Laboratory Values. CBC (10^9 Cells/L)
时间窗: Baseline (Week 0), Week 24, and End of Study (Week 52)
Changes from baseline in CBC laboratory values with units of 10\^9 cells/L (Leukocytes, Platelets)
Laboratory Values. CBC (% Difference in Volume and Size of RBC)
时间窗: Baseline (Week 0), Week 24, and End of Study (Week 52)
Changes from baseline in CBC laboratory values with unit of % difference in volume and size of RBC
Laboratory Values. CBC (% of Total Blood Cell Count)
时间窗: Baseline (Week 0), Week 24, and End of Study (Week 52)
Changes from baseline in CBC laboratory values with unit of % of total blood cell count.
Laboratory Values. CMP (g/dL)
时间窗: Baseline (Week 0), Week 24, and End of Study (Week 52)
Changes from baseline in CMP laboratory values with units of g/dL
Laboratory Values. CBC (10^12 Cells/L)
时间窗: Baseline (Week 0), Week 24, and End of Study (Week 52)
Changes from baseline in CBC laboratory values with units of 10\^12 cells/L.
Laboratory Values. CBC (g/dL)
时间窗: Baseline (Week 0), Week 24, and End of Study (Week 52)
Changes from baseline in CBC laboratory values with unit of g/dL.
Vital Signs. - Blood Pressure (mmHg)
时间窗: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Change from baseline in Blood Pressure.
Laboratory Values. CMP (IU/L)
时间窗: Baseline (Week 0), Week 24, and End of Study (Week 52)
Changes from baseline in CMP laboratory values with units of IU/L.
Laboratory Values. CMP (mL/Min/1.73m^2)
时间窗: Baseline (Week 0), Week 24, and End of Study (Week 52)
Changes from baseline in CMP laboratory values with units of mL/min/1.73m\^2.
Laboratory Values. CMP (mmol/L)
时间窗: Baseline (Week 0), Week 24, and End of Study (Week 52)
Changes from baseline in CMP laboratory values with units of mmol/L.
Vital Signs. - Body Temperature (Celsius )
时间窗: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Change from baseline in Body Temperature.
Laboratory Values. Coagulation Panel (Seconds)
时间窗: Baseline (Week 0), Week 24, and End of Study (Week 52)
Changes from baseline in Coagulation Panel values with units of seconds.
Vital Signs. - Respiratory Rate (Breaths Per Minute)
时间窗: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Changes from Baseline in Respiratory Rate.
Laboratory Values. Coagulation Panel (Ratio: Prothrombin Time (Seconds) / Mean Normal Prothrombin Time (Seconds))
时间窗: Baseline (Week 0), Week 24, and End of Study (Week 52)
Changes from baseline in Coagulation laboratory values with units of Ratio: Prothrombin time (seconds) / Mean normal prothrombin time (seconds).
Vital Signs. - Heart Rate (Beats Per Minute)
时间窗: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Change from baseline in Heart Rate.
Change From Baseline in MDS-UPDRS Part II.
时间窗: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
The MDS-UPDRS scale refers to Movement Disorder Society - Unified Parkinson Disease Rating Scale, and it is a rating tool used to gauge the course of Parkinson's disease in patients. The MDS-UPDRS scale consists of 4 segments. Part II tests "Motor Aspects of Experiences of Daily Living". Each answer to the scale is evaluated by the principal investigator during the study visit. Some sections of the MDS-UPDRS scale require multiple grades assigned to each extremity. There are 13 items included in Part II. Part II score ranges from 0 - 52; 12 and below is mild, 30 and above is severe. Each item has 0-4 ratings: 0 (normal), 1 (slight), 2 (mild), 3 (moderate), and 4 (severe). The total score ranges from 0 to 260, with 0 indicating no disability and 260 indicating total disability. Higher values represent a worse outcome.
次要结局
- Change From Baseline in Neuro-QOL. - Depression(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in Neuro-QOL. - Mobility(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in Neuro-QOL. - Well-Being(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in Neuro-QOL. - Cognition(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in Neuro-QOL. - Communication(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Changes From Baseline in Neuro-QOL. - Anxiety(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in Neuro-QOL. - Dyscontrol(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in Neuro-QOL. - Fine Motor(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in Dosage of Carbidopa/Levodopa(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in MDS-UPDRS Part I.(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in MDS-UPDRS Part III.(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in Neuro-QOL. - Social Roles and Activities(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Changes From Baseline in Neuro-QOL. - Fatigue(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in Neuro-QOL. - Sleep(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in Neuro-QOL. - Stigma(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in MDS-UPDRS Part IV.(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in Neuro-QOL. - Social Roles(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in Parkinson's Disease Fatigue Scale (PFS-16).(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in Total Visual Analog Scale(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in Parkinson's Disease Questionnaire (PDQ-39).(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)
- Change From Baseline in Vital Signs. - Oxygen Saturation.(Baseline through Week 32, Follow-up at week 42 and End of Study at week 52)