A Randomized, Double-Blind, Single Center, Phase 2, Efficacy and Safety Study of Allogeneic 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
- 60
- 试验地点
- 2
- 主要终点
- 1. Changes in the total score MDS-UPDRS Part II.
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 allogeneic HB-adMSCs vs Placebo for the treatment of Parkinson's disease.
详细描述
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 60 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.
After Informed consent has been obtained, each participant should complete the following visits.
- Visit 1 - Screening, during this visit, the principal investigator will make the decision to determine whether the screened participant is eligible and whether the next visit can be scheduled. Once, the principal investigator has evaluated the eligibility of the subject screened (up to 28 days), a randomization process will be conducted in order to assign the eligible subject either allogeneic HB-adMSCs or placebo. Randomization will only apply to eligible subjects. If a study participant does not meet the inclusion and exclusion criteria during the screening process, he/she will be considered Screen Failure (SF) and randomization is not required.
- Visit 2 - Infusion 1, (Baseline): this visit will be used as a starting point for comparison of participant's data. During this visit, eligible study participants will receive his/her first investigational product administration or placebo with monitoring of vital signs for a total of 2 hours after drug exposure. Other study evaluations will be completed as part of this visit.
- Visit 3 - Infusion 2: approximately 4 weeks after the initial investigational product administration this visit should be completed. Other study evaluations will be completed as part of this visit.
- Visit 4 - Infusion 3: approximately 8 weeks after the initial investigational product administration this visit should be completed. Other study evaluations will be completed as part of this visit.
- Visit 5 - Infusion 4: approximately 12 weeks after the initial investigational product administration this visit should be completed. Other study evaluations will be completed as part of this visit.
- Visit 6 - Infusion 5: approximately 16 weeks after the initial investigational product administration this visit should be completed. Other study evaluations will be completed as part of this visit.
- Visit 7 - Infusion 6: approximately 20 weeks after the initial investigational product administration this visit should be completed. Other study evaluations will be completed as part of this visit.
- Phone Call - Safety Follow Up: approximately 24 weeks after the initial investigational product administration, active study participants will complete a phone call follow up.
- Phone Call - Safety Follow Up: approximately 32 weeks after the initial investigational product administration, active study participants will complete a phone call follow up.
- Visit 8 - End of Study, during this final visit (approximately 52 weeks after Week 0) a complete group of study assessments will be performed to evaluate the safety and efficacy of allogeneic HB-adMSCs or Placebo administrations.
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
- 45 Years 至 80 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 the following criteria apply:
- •Male and female participants 45 - 80 years of age.
- •At the screening visit, study participants must have an MDS-UPDRS part II score between 7 and
- •Study participants must have an MDS-UPDRS part III score between 20 and 57 during the screening visit.
- •Carbidopa/Levodopa total dosage must be less than 1200 mg per day for study participants.
- •The total Levodopa equivalent dose for study participants must be less than 1400 mg per day.
- •Study participant must have been diagnosed with early and/or moderate Parkinson's disease at least 2 years prior study participation.
- •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 hepatis 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.
研究组 & 干预措施
Allogeneic HB-adMSCs.
Biological/Vaccine: Allogeneic HB-adMSCs
Allogeneic HB-adMSCs will be administered intravenously to study participants who qualify.
Other Names: Allogeneic Hope Biosciences adipose derived mesenchymal stem cells.
干预措施: Placebo (Other)
Placebo
Placebo will be administered intravenously to study participants who qualify.
Other Names: Sterile Saline Solution 0.9%
干预措施: Placebo (Other)
结局指标
主要结局
1. Changes in the total score MDS-UPDRS Part II.
时间窗: Baseline to Weeks 52.
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II.
2. Changes in the total score MDS-UPDRS Part III.
时间窗: Baseline to Weeks 52.
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III.
3. Incidence of treatment-emergent Adverse Event (TEAEs).
时间窗: Baseline to Weeks 52.
Treatment-emergent Adverse Event.
4. Incidence of treatment-emergent Serious Adverse Events (SAEs).
时间窗: Baseline to Weeks 52.
SSAEs.
5. AEs of special interest (serious or non-serious) - thromboembolic events.
时间窗: Baseline to Weeks 52.
Incidence of thromboembolic events.
6. AEs of special interest (serious or non-serious) - thromboembolism of the extremities
时间窗: Baseline to Weeks 52.
Incidence and risk of AEs of special interest (serious or non-serious), including peripheral events defined as, thromboembolism of the extremities.
7. AEs of special interest (serious or non-serious) - infections
时间窗: Baseline to Weeks 52.
Incidence and risk of AEs of special interest (serious or non-serious), including infections.
8. AEs of special interest (serious or non-serious) - hypersensitivities.
时间窗: Baseline to Weeks 52.
Incidence and risk of AEs of special interest (serious or non-serious), including hypersensitivities.
9. Laboratory value Complete Blood Count (CBC)
时间窗: Baseline to Weeks 52.
Clinically significant changes in CBC values.
10. Laboratory values Chemistry Metabolic Panel (CMP)
时间窗: Baseline to Weeks 52.
Number of Participants with changes in Laboratory CMP values
11. Laboratory values Coagulation Panel; Prothrombin time, Partial Prothrombin time, and INtern
时间窗: Baseline to Weeks 52.
Number of Participants with changes in Laboratory Coagulation Panel values.
12. Vital signs. - Respiratory Rate (breaths per minute)
时间窗: Baseline to Weeks 52.
Number of Participants with Clinically significant changes in Respiratory Rate.
13. Vital signs. - Heart Rate (beats per minute)
时间窗: Baseline to Weeks 52.
Number of Participants with Clinically significant changes in Heart Rate.
14. Vital signs. - Body Temperature (Fahrenheit )
时间窗: Baseline to Weeks 52.
Number of participants with Clinically significant changes in Heart Rate.
15. Vital signs. - Blood Pressure (mmHg)
时间窗: Baseline to Weeks 52.
Number of Participants with Clinically significant changes in Blood Pressure.
16. Weight in lb.
时间窗: Baseline to Weeks 52.
Number of Participants with Clinically significant changes in Weight in lb.
17. Physical examination results. General
时间窗: Baseline to Weeks 52.
Number of Participants with Clinically significant changes in general physical examination results.
18. Physical examination results. Body Systems.
时间窗: Baseline to Weeks 52.
Number of Participants with Clinically significant changes in body systems physical examination results.
次要结局
- 19. Changes in Movement Disorder Society Unified Parkinson's Disease Rating Scale -UPDRS Part I.(Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.)
- 20. Changes in the total score Movement Disorder Society Unified Parkinson's Disease Rating Scale -UPDRS Part II and Part III.(Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.)
- 21. Changes in Movement Disorder Society Unified Parkinson's Disease Rating Scale MDS-UPDRS Part III.(Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.)
- 22. Changes in Movement Disorder Society Unified Parkinson's Disease Rating Scale MDS-UPDRS Part IV.(Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.)
- 23. Changes in Short Form 36 Health Survey Questionnaire (SF-36).(Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.)
- 24. Changes in Parkinson's disease fatigue scale (PFS-16)(Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.)
- 25. Changes in Parkinson's disease Questionnaire (PDQ-39).(Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.)
- 26. Changes in Visual Analog Scale for Pain.(Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.)
- 27. Changes in Visual Analog Scale for Muscle spasms.(Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.)
- 28. Changes in Dosage of medications taken to treat Parkinson's disease.(Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.)