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Evaluating Safety, Tolerability, and Efficacy of Autologous MitoCell Transplantation in Subjects With Idiopathic Parkinson’s Disease

Studies

Study First Submitted Date 2021-07-20
Study First Posted Date 2021-10-26
Last Update Posted Date 2023-04-21
Start Month Year March 1, 2024
Primary Completion Month Year September 30, 2025
Verification Month Year April 2023
Verification Date 2023-04-30
Last Update Posted Date 2023-04-21

Detailed Descriptions

Sequence: 20639376
Description MitoCell is an autologous stem cell product that cultures with the company's unique patented medium. The mechanism of action of MitoCell is to improve the brain microenvironment in neurodegenerative disease. MitoCell which like mesenchymal stem cells modulate the immune response, and secrete more BDNF and SDF-1 neurotrophic factors than regular stem cell products.Therefore, MitoCell can protect and repair damaged dopamine neurons (DA) and stimulate DA regeneration.This project is a phase I open-label dose-escalation study to evaluate the safety, tolerability, and efficacy of autologous MitoCell intracranial transplantation in subjects with idiopathic Parkinson's disease which rating from stage 3 ~ 4 of modified Hoehn & Yahr staging.

Conditions

Sequence: 51957826
Name Idiopathic Parkinson's Disease
Downcase Name idiopathic parkinson's disease

Id Information

Sequence: 39992996
Id Source org_study_id
Id Value MITOCELL-01

Design Groups

Sequence: 55358411
Group Type Experimental
Title Single Arm Study
Description Autologous MitoCell Transplantation in Subjects with Idiopathic Parkinson's Disease

Interventions

Sequence: 52270169
Intervention Type Biological
Name Aadipose-Derived Mesenchymal Stem Cells
Description Stereotactic intrastriatal implantation of 3×10^7 per hemisphere (total 6×10^7 cells) or 1×10^8 per hemisphere (total 2×10^8 cells) autologous TM01-treated adipose-derived mesenchymal stem cells (MitoCell).

Design Outcomes

Sequence: 176622657 Sequence: 176622658 Sequence: 176622659 Sequence: 176622654 Sequence: 176622655 Sequence: 176622656 Sequence: 176622660 Sequence: 176622661 Sequence: 176622662 Sequence: 176622663 Sequence: 176622664 Sequence: 176622665 Sequence: 176622666 Sequence: 176622667 Sequence: 176622668 Sequence: 176622669 Sequence: 176622670 Sequence: 176622671 Sequence: 176622672 Sequence: 176622673 Sequence: 176622674
Outcome Type primary Outcome Type primary Outcome Type primary Outcome Type primary Outcome Type primary Outcome Type primary Outcome Type primary Outcome Type primary Outcome Type primary Outcome Type primary Outcome Type primary Outcome Type primary Outcome Type primary Outcome Type secondary Outcome Type secondary Outcome Type secondary Outcome Type secondary Outcome Type secondary Outcome Type secondary Outcome Type secondary Outcome Type secondary
Measure Changes in vital signs: blood pressure [Safety of Mitocell] Measure Changes in vital signs: pulse rate [Safety of Mitocell] Measure Changes in vital signs: body temperature [Safety of Mitocell] Measure Grading of Adverse Events Measure Routine physical examinations Measure Changes in physical examinations: clinical standard neurological examination [Safety of Mitocell] Measure Changes in clinical laboratory safety screen: haematology – hemoglobin [Safety of Mitocell] Measure Changes in clinical laboratory safety screen: Platelet count [Safety of Mitocell] Measure Changes in clinical laboratory safety screen: white blood cell (WBC) counts [Safety of Mitocell] Measure Changes in clinical laboratory safety screen: International Normalized Ratio (INR) [Safety of Mitocell] Measure Changes in clinical laboratory safety screen: activated partial thromboplastin time (aPTT) [Safety of Mitocell] Measure Electrocardiogram (ECG) Measure Magnetic Resonance Imaging (MRI) Measure MDS-UPDRS (Movement Disorder Society unified Parkinson's disease rating scale) Measure Modified Hoehn & Yahr staging Measure 18F-DOPA PET Measure levodopa equivalent daily dose (LEDD) Measure PDQ-39 (Parkinson's Disease Questionnaire) Measure Beck Depression Inventory (BDI-II) scores Measure Hamilton Depression Rating Scale (HAM-D-17) scores Measure Mini Mental State Examination (MMSE) Scores
Time Frame within 48 weeks after MitoCell transplantation Time Frame within 48 weeks after MitoCell transplantation Time Frame within 48 weeks after MitoCell transplantation Time Frame within 48 weeks after MitoCell transplantation Time Frame within 48 weeks after MitoCell transplantation Time Frame within 48 weeks after MitoCell transplantation Time Frame within 48 weeks after MitoCell transplantation Time Frame within 48 weeks after MitoCell transplantation Time Frame within 48 weeks after MitoCell transplantation Time Frame within 48 weeks after MitoCell transplantation Time Frame within 48 weeks after MitoCell transplantation Time Frame within 48 weeks after MitoCell transplantation Time Frame within 48 weeks after MitoCell transplantation Time Frame at 12, 24, 48 weeks Time Frame at 12, 24, 48 weeks Time Frame at 48 weeks Time Frame at 12, 24, 48 weeks Time Frame at 12, 24, 48 weeks Time Frame at 48 weeks Time Frame at 48 weeks Time Frame at 48 weeks
Description Changes in blood pressure during the study , measured as systolic and diastolic blood pressure (in mmHg) Description Changes in pulse rate during the study (in beats per minute) Description Changes in body temperature during the study (in degrees celsius) Description Grading will be assessed using NCI CTCAE, version 5.0. Description Safety of Mitocell will be assessed by routine physical examinations. Physical examination conducted in this study will include general appearance, skin, eyes, ears, nose,throat, head and neck, heart, chest and lungs, abdomen, extremities, lymph nodes, musculoskeletal,neurological, etc. Description Clinical standard neurological examination by study investigator. Changes in motor function, sensory function, cranial nerve function (visual fields), cortical functions and reflexes are followed in the examination, scored as normal – abnormal without clinical relevance – abnormal with clinical relevance Description Changes in laboratory variables for haematology: hemoglobin (g/L). Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance" Description Changes in laboratory variables for haematology: Platelet count (10E9/L). Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance" Description Changes in laboratory variables for haematology: Cell counts (10E9/L) for total WBC, neutrophils, lymphocytes, monocytes, eosinophils and basophils. Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance" Description Changes in laboratory variables for haematology: INR (standardized prothrombin time) to determine the effects of oral anticoagulants on the clotting system. Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance" Description Changes in laboratory variables for haematology: aPTT (sec) . Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance" Description Safety of Mitocell will be assessed by any clinically significant abnormalities on ECG results as compared to Baseline. A standard 12-lead ECG was measured by using ECG machine that automatically measured PR, QRS, QT, and QTcF intervals. Description Safety of Mitocell will be assessed by any clinically significant abnormalities on MRI scans as compared to Baseline. Description Change in MDS-UPDRS motor (Part III) "OFF" score compared to the baseline. All items have 5 response options with uniform anchors of: 0 = normal, 1 = slight (symptoms/signs with sufficiently low frequency or intensity to cause no impact on function), 2 = mild (symptoms/signs of frequency or intensity sufficient to cause a modest impact on function, 3 = moderate (symptoms/signs sufficiently frequent or intense to impact considerably, but not prevent function), 4 = severe (symptoms/signs that prevent function). Description Change in Modified Hoehn & Yahr staging compared to the baseline. Description Degree of radiotracer uptake increment/decrement shown on striatum of 18F-DOPA PET compared to the baseline. Description Reduction of Parkinson's medications consumption by calculating levodopa equivalent daily dose (LEDD) compared to the baseline. Description Change in PDQ-39 scale compared to the baseline. The PDQ-39 is a 39 questions Parkinson's Disease self-completed questionnaire that comprises 8 domains: mobility (10 items), activities of daily living (6 items), emotional well-being (6 items), stigma (4 items), social support (3 items), cognition (4 items), communication (3 items) and bodily discomfort (3 items). All items are assumed to impact QoL and must be answered to compute scores for each dimension. Questions are answered based on experiences from the preceding month using a 5-point ordinal scoring system: 0 = never, 1 = occasionally, 2 = sometimes, 3 = often, 4 = always. Each scores range from 0 = never have difficulty to 100 = always have difficulty. Description Net change from baseline in BDI-II scores. BDI-II Scale is a 21-item self-reported questionnaire which measures the existence and severity of symptoms of depression. Each of the 21 items on BDI-II tool represents a depressive symptom. The symptoms are each scored on a 4-point Likert scale of 0 to 3 (0=symptom is absent; 3=symptom is severe). Scores for each symptom are added up to obtain the total scores for all 21 items. Total score ranges from 0-63; of which 0-8 is considered no depression, 0-13 is minimal depression, 14-19 is mild depression, 20-28 is moderate depression and 29-63 is severe depression. Description Net change from baseline in HAM-D-17 scores. The HAMD-17 is a 17-item assessment used to assess the severity of depression and its improvement during the course of therapy. Each item was evaluated and scored using either a 5-point scale of 0 (not present/absent) to 4 (very severe) or a 3-point scale of 0 (not present/absent) to 2 (marked). Higher scores indicate greater symptom severity. The total score was the sum of the scores from HAMD-17 Items 1 through 17 and ranged from 0 (not at all depressed) to 52 (severely depressed). Description Net change from baseline in MMSE scores. The MMSE uses a 30 point questionnaire to measure cognitive impairment. The MMSE is scored from 0 to 30,with a score equal to or greater than 24 points indicating normal cognition, a score of 19-23 points indicating mild cognitive impairment, 10-18 points indicating moderate impairment and a score equal to or below 9 indicating severe impairment.

Browse Conditions

Sequence: 192641130 Sequence: 192641131 Sequence: 192641132 Sequence: 192641133 Sequence: 192641134 Sequence: 192641135 Sequence: 192641136 Sequence: 192641137 Sequence: 192641138
Mesh Term Parkinson Disease Mesh Term Parkinsonian Disorders Mesh Term Basal Ganglia Diseases Mesh Term Brain Diseases Mesh Term Central Nervous System Diseases Mesh Term Nervous System Diseases Mesh Term Movement Disorders Mesh Term Synucleinopathies Mesh Term Neurodegenerative Diseases
Downcase Mesh Term parkinson disease Downcase Mesh Term parkinsonian disorders Downcase Mesh Term basal ganglia diseases Downcase Mesh Term brain diseases Downcase Mesh Term central nervous system diseases Downcase Mesh Term nervous system diseases Downcase Mesh Term movement disorders Downcase Mesh Term synucleinopathies Downcase Mesh Term neurodegenerative diseases
Mesh Type mesh-list Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor

Sponsors

Sequence: 48119776
Agency Class INDUSTRY
Lead Or Collaborator lead
Name Taiwan Mitochondrion Applied Technology Co., Ltd.

Overall Officials

Sequence: 29163448
Role Study Director
Name Chi-Tang Tu, Ph. D.
Affiliation Taiwan Mitochondrion Applied Technology Co., Ltd.

Central Contacts

Sequence: 11960707 Sequence: 11960708
Contact Type primary Contact Type backup
Name Kuo-Wei Hsueh, Ph. D. Name Zong-Han Lu, Master
Phone 886-03-5820208 Phone 886-03-5820208
Email fskenneth@taimito.com Email zhlu@taimito.com
Role Contact Role Contact

Design Group Interventions

Sequence: 67863606
Design Group Id 55358411
Intervention Id 52270169

Eligibilities

Sequence: 30639844
Gender All
Minimum Age 45 Years
Maximum Age 70 Years
Healthy Volunteers No
Criteria Inclusion Criteria: Provision of signed and dated informed consent form Aged 45 to 70 years old (inclusive) at Screening Idiopathic Parkinson's disease patients who meet the diagnostic criteria of the "Movement Disorder Society Clinical Diagnostic Criteria for Parkinson's disease" With at least 5 years since the diagnosis of Parkinson's disease With responsiveness to levodopa or dopa agonist. This is defined as improvement between ''Off'' and ''On'' MDS-UPDRS by at least 33% of the Motor MDS-UPDRS Idiopathic Parkinson's disease of Stage 3 ~ 4 of modified Hoehn & Yahr staging during ''ON'' time Stable Parkinsonian medications for at least 2 months prior to the Screening Visit MRI not showing gross atrophy or any brain pathology other than PD Mini-Mental State Examination (MMSE) ≧ 24 With score of the Beck Depression Inventory (BDI-II) < 29 and Hamilton Rating Scale for Depression (HAM-D-17) < 25 Exclusion Criteria: Atypical or secondary Parkinsonism With neurodegenerative disorders other than PD Unable to receive MRI or PET scanning With any concomitant disorder that would contraindicate coagulation, general anesthesia, or stereotactic neurosurgery Received any other investigational agent within 4 weeks prior to Screening History of intracranial surgeries or implantation of a device for Parkinson's disease 2 years prior to Screening Major surgery within the previous 6 months at Screening Significant cardiovascular disease, including: New York Heart Association (NYHA) class III or IV congestive heart failure Uncontrolled hypertension: Blood pressure >140/90 mmHg History of serious ventricular arrhythmia Malignancy within 2 years prior to Screening Any diagnosis of autoimmune disease or immune compromised state and requiring systemic steroid or immunosuppressive treatment Any other severe systemic disorder, including history of schizophrenia or other psychotic disorders, stroke, seizure, traumatic brain injury, or central nervous system infection, which judged by the investigator that entering the trial may be detrimental to the subject Psychiatric, addictive or any other disorder that compromises ability to give a truly informed consent and perform all study assessments Positive in any of the following regulatory authority-licensed screening tests: HIV antigen/antibody combo test Anti-HCV test Hepatitis B surface antigen (HBsAg) test Rapid plasma reagin (RPR) test HIV-1 nucleic acid test (NAT) HBV NAT HCV NAT Any of the following hematologic abnormalities: Hemoglobin < 9.0 g/dL, ANC < 1,500/μL Platelets < 100,000/μL Any of the following serum biochemistry abnormalities: Total bilirubin > 1.5 × ULN AST or ALT > 2.5 × ULN r-GT > 2.5 × ULN ALP > 2.5 × ULN serum albumin < 3.0 g/dL creatinine > 1.5 × ULN Female subject who is lactating or has positive serum or urine pregnancy test at Screening Visit Female subject with childbearing potential or male subject with female spouse/partner with childbearing potential who refuses to adopt at least two forms of birth control (at least one of which must be a barrier method) from Screening until Final/Early Termination Visit. Acceptable forms include: Established use of oral, injected or implanted hormonal methods of contraception Placement of an intrauterine device (IUD) or intrauterine system (IUS) Barrier methods of contraception: condom, or occlusive cap (diaphragm or cervical/vault caps) With any condition judged by the investigator that entering the trial may be detrimental to the subject
Adult True
Child False
Older Adult True

Calculated Values

Sequence: 254172713
Registered In Calendar Year 2021
Were Results Reported False
Has Single Facility False
Minimum Age Num 45
Maximum Age Num 70
Minimum Age Unit Years
Maximum Age Unit Years
Number Of Primary Outcomes To Measure 13
Number Of Secondary Outcomes To Measure 8

Designs

Sequence: 30386764
Allocation N/A
Intervention Model Sequential Assignment
Observational Model
Primary Purpose Treatment
Time Perspective
Masking None (Open Label)
Intervention Model Description This study plans to enroll approximately 4 subjects to obtain 3 evaluable subjects successfully receiving 3×10^7 cells/hemisphere (total 6×10^7 cells, Cohort 1) and approximately 8 subjects to obtain 6 evaluable subjects for 1×10^8 cells/hemisphere (total 2×10^8 cells, Cohort 2)

Intervention Other Names

Sequence: 26561635
Intervention Id 52270169
Name MitoCell

Responsible Parties

Sequence: 28753364
Responsible Party Type Sponsor