Clinical Assessment on the Safety and Potential Efficacy of Mesenchymal Stem Cells Preconditioned With Ethionamide (ET-STEM) in Patients With Frontotemporal Dementia (FTD)
Overview
- Phase
- Phase 1
- Intervention
- ET-STEM
- Conditions
- Frontotemporal Dementia
- Sponsor
- Samsung Medical Center
- Enrollment
- 12
- Locations
- 1
- Primary Endpoint
- To determine DLT (Dose limiting toxicity)
- Status
- Active, not recruiting
- Last Updated
- 2 years ago
Overview
Brief Summary
The primary purpose of this study is to evaluate the safety and the tolerability of 3 repeated doses of ET-STEM (Mesenchymal stem cells preconditioned with ethionamide) in patients with FTD.
Detailed Description
Subjects with FTD, who signed the informed consent form and meet the eligibility criteria will undergo Ommaya reservoir insertion. 2 weeks after Ommaya reservoir insertion, the subjects will be injected with 3x10\^7 cells/2mL of ET-STEM to intraventricular space via an Ommaya reservoir. The injection will be repeated 3 times at 4 week intervals. The subjects will be hospitalized for 24 hours and observed for acute adverse events. 4 weeks after the 3rd injection, safety and potential efficacy will be assessed.
Investigators
Hee Jin Kim
assistant professor
Samsung Medical Center
Eligibility Criteria
Inclusion Criteria
- •Korean male or female at 40-85 years of age
- •Diagnosis of one of the 3 subtyes of FTD according to the diagnostic criteria for 3 subtypes of FTD
- •① Probable bvFTD (behavior variant FTD)
- •② svPPA (semantic variant primary progressive aphasia)
- •③ nfvPPA (nonfluent/agrammatic variant primary progressive aphasia)
- •K-MMSE ≥ 10
- •Subjects with trusted caregivers who regularly contact the subjects and can accompany the subjects when visiting the hospital.
- •Negative result of amyloid PET imaging
- •A subject who is informed of the clinical trial and signs a consent form (If unable to sign, a consent from a legally acceptable representative is required)
Exclusion Criteria
- •Subjects with dementia cause by other than FTD (i.e. infection of central nervous system, Creutzfeld-Jacob disease, severer head trauma, Huntington's disease, Parkinson's disease, Alzheimer's disease and vascular dementia)
- •Subjects with psychological disorder. (i.e. depression, schizophrenia , bipolar disorder, etc) (except for subjects who were misdiagnosed with psychological disease due to the initial neuropsychiatric symptoms of FTD)
- •Subjects with uncontrolled hypotension, hypertension, diabetes and thyroid disease.
- •Subjects with a cancer (including brain tumor)
- •Subjects with bleeding disorder
- •Woman of childbearing age who refused to practice medically acceptable contraceptive method (post menopausal patient with no menstruation for at least 12 months is considered as infertile)
- •Pregnant or lactating females
- •History of stroke within 3 months prior to study enrollment
- •Substance/alcohol abuse 1
- •Contraindicated for any of the tests performed during the clinical trial period(for example, MRI, CT,PET)
Arms & Interventions
Treatment Arm
injected with 3x10\^7 cells/2mL of ET-STEM to intraventricular space via an Ommaya reservoir. repeated 3 times at 4 week intervals
Intervention: ET-STEM
Outcomes
Primary Outcomes
To determine DLT (Dose limiting toxicity)
Time Frame: First 3-week cycle of treatment
incidence rate of DLT (Dose limiting toxicity)
adverse events as assessed by CTCAE v5.0
Time Frame: up to 5years
all potentially treated subjects to assess the safety
Secondary Outcomes
- ADAS-Cog 13 response rate(Screening, after the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks)
- The Clinical Dementia Rating Sum of Boxes(Screening, after the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks)
- Alzheimer's Disease Cooperative Study- instrumental items of the Activities of Daily Living Inventory(the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks)
- Caregiver-administered Neuropsychiatric Inventory(the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks)
- preliminary efficacy(up to 12weeks)
- K-MMSE(the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks)