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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
mesenchymal stem cells preconditioned with ethionamide
Samsung Medical Center
Seoul, Gangnam-gu, South Korea
To determine DLT (Dose limiting toxicity)
incidence rate of DLT (Dose limiting toxicity)
Time frame: First 3-week cycle of treatment
adverse events as assessed by CTCAE v5.0
all potentially treated subjects to assess the safety
Time frame: up to 5years
ADAS-Cog 13 response rate
response rate, no change or improvement on ADAS cog 13 score
Time frame: Screening, after the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
The Clinical Dementia Rating Sum of Boxes
Change from the baseline in CDR-SB, min 0, max 24, higher scores mean a worse outcome
Time frame: Screening, after the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
Alzheimer's Disease Cooperative Study- instrumental items of the Activities of Daily Living Inventory
Change from the baseline in ADCS-iADL, min 0, max78, higher scores mean a better outcome
Time frame: the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
Caregiver-administered Neuropsychiatric Inventory
Change from the baseline in CGA-NPI, min 0, max 144, higher scores mean a worse outcome
Time frame: the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
preliminary efficacy
Change from the baseline in CSF biomarkers
Time frame: up to 12weeks
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
K-MMSE
Korean Mini-Mental State Examination(MMSE), min 0, max 30, higher scores mean a better outcome
Time frame: the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks