Outcome
Type |
Measure |
Description |
Time frame |
Safety issue |
Primary |
Grading of Adverse Events |
Grading will be assessed using NCI CTCAE, version 5.0. |
within 48 weeks after MitoCell transplantation |
|
Primary |
Routine physical examinations |
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. |
within 48 weeks after MitoCell transplantation |
|
Primary |
Changes in physical examinations: clinical standard neurological examination [Safety of Mitocell] |
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 |
within 48 weeks after MitoCell transplantation |
|
Primary |
Changes in vital signs: blood pressure [Safety of Mitocell] |
Changes in blood pressure during the study , measured as systolic and diastolic blood pressure (in mmHg) |
within 48 weeks after MitoCell transplantation |
|
Primary |
Changes in vital signs: pulse rate [Safety of Mitocell] |
Changes in pulse rate during the study (in beats per minute) |
within 48 weeks after MitoCell transplantation |
|
Primary |
Changes in vital signs: body temperature [Safety of Mitocell] |
Changes in body temperature during the study (in degrees celsius) |
within 48 weeks after MitoCell transplantation |
|
Primary |
Changes in clinical laboratory safety screen: haematology - hemoglobin [Safety of Mitocell] |
Changes in laboratory variables for haematology: hemoglobin (g/L). Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance" |
within 48 weeks after MitoCell transplantation |
|
Primary |
Changes in clinical laboratory safety screen: Platelet count [Safety of Mitocell] |
Changes in laboratory variables for haematology: Platelet count (10E9/L). Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance" |
within 48 weeks after MitoCell transplantation |
|
Primary |
Changes in clinical laboratory safety screen: white blood cell (WBC) counts [Safety of Mitocell] |
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" |
within 48 weeks after MitoCell transplantation |
|
Primary |
Changes in clinical laboratory safety screen: International Normalized Ratio (INR) [Safety of Mitocell] |
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" |
within 48 weeks after MitoCell transplantation |
|
Primary |
Changes in clinical laboratory safety screen: activated partial thromboplastin time (aPTT) [Safety of Mitocell] |
Changes in laboratory variables for haematology: aPTT (sec) . Result evaluated as "normal", "abnormal without clinical relevance" or "abnormal with clinical relevance" |
within 48 weeks after MitoCell transplantation |
|
Primary |
Electrocardiogram (ECG) |
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. |
within 48 weeks after MitoCell transplantation |
|
Primary |
Magnetic Resonance Imaging (MRI) |
Safety of Mitocell will be assessed by any clinically significant abnormalities on MRI scans as compared to Baseline. |
within 48 weeks after MitoCell transplantation |
|
Secondary |
MDS-UPDRS (Movement Disorder Society unified Parkinson's disease rating scale) |
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). |
at 12, 24, 48 weeks |
|
Secondary |
Modified Hoehn & Yahr staging |
Change in Modified Hoehn & Yahr staging compared to the baseline. |
at 12, 24, 48 weeks |
|
Secondary |
18F-DOPA PET |
Degree of radiotracer uptake increment/decrement shown on striatum of 18F-DOPA PET compared to the baseline. |
at 48 weeks |
|
Secondary |
levodopa equivalent daily dose (LEDD) |
Reduction of Parkinson's medications consumption by calculating levodopa equivalent daily dose (LEDD) compared to the baseline. |
at 12, 24, 48 weeks |
|
Secondary |
PDQ-39 (Parkinson's Disease Questionnaire) |
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. |
at 12, 24, 48 weeks |
|
Secondary |
Beck Depression Inventory (BDI-II) scores |
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. |
at 48 weeks |
|
Secondary |
Hamilton Depression Rating Scale (HAM-D-17) scores |
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). |
at 48 weeks |
|
Secondary |
Mini Mental State Examination (MMSE) Scores |
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. |
at 48 weeks |
|