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 |
fskenneth@taimito.com | 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 |