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

NCT05094011 | Not Yet Recruiting Phase 1

• 1 other identifier: MITOCELL-01
• Study Type: interventional
• Target: 9
• Locations: 0 countries
• Sites: N/A

Brief Summary

Primary Objective: To assess the safety profile of autologous MitoCell administered to subjects with idiopathic Parkinson's disease (PD) Secondary Objective: To explore the efficacy and safety of MitoCell given as the recommended dose by stereotactic intrastriatal implantation

Conditions

Idiopathic Parkinson's Disease

Outcome Measures

Primary Outcomes (13)

• Grading of Adverse Events

  Grading will be assessed using NCI CTCAE, version 5.0.

  within 48 weeks after MitoCell transplantation

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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 Outcomes (8)

• MDS-UPDRS (Movement Disorder Society unified Parkinson's disease rating scale)

  at 12, 24, 48 weeks

• Modified Hoehn & Yahr staging

  at 12, 24, 48 weeks

• 18F-DOPA PET

  at 48 weeks

• levodopa equivalent daily dose (LEDD)

  at 12, 24, 48 weeks

• PDQ-39 (Parkinson's Disease Questionnaire)

  at 12, 24, 48 weeks

Study Arms (1)

Single Arm Study

EXPERIMENTAL

Autologous MitoCell Transplantation in Subjects with Idiopathic Parkinson's Disease

Biological: Aadipose-Derived Mesenchymal Stem Cells

Interventions

Aadipose-Derived Mesenchymal Stem Cells BIOLOGICAL

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).

Also known as: MitoCell

Single Arm Study

Eligibility Criteria

• Age: 45 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Taiwan Mitochondrion Applied Technology Co., Ltd.: lead

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Officials

• Chi-Tang Tu, Ph. D.

  Taiwan Mitochondrion Applied Technology Co., Ltd.

  STUDY DIRECTOR

Central Study Contacts

Kuo-Wei Hsueh, Ph. D.

CONTACT

886-03-5820208; fskenneth@taimito.com

Zong-Han Lu, Master

CONTACT

886-03-5820208; zhlu@taimito.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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)

Study Record Dates

• First Submitted: July 20, 2021
• First Posted: October 26, 2021
• Study Start: March 1, 2026
• Primary Completion (Estimated): September 30, 2027
• Study Completion (Estimated): July 31, 2028
• Last Updated: November 21, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share