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

Taiwan Mitochondrion Applied Technology

Status and phase

Begins enrollment this month

Phase 1

Conditions

Idiopathic Parkinson's Disease

Treatments

Biological: Aadipose-Derived Mesenchymal Stem Cells

Study type

Interventional

Funder types

Industry

Identifiers

NCT05094011

MITOCELL-01

Details and patient eligibility

About

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

Full 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.

Enrollment

9 estimated patients

Sex

All

Ages

45 to 70 years old

Volunteers

No Healthy Volunteers

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