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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Arm | Experimental | injected with 3x10^7 cells/2mL of ET-STEM to intraventricular space via an Ommaya reservoir. repeated 3 times at 4 week intervals |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ET-STEM | Drug | mesenchymal stem cells preconditioned with ethionamide |
|
| Measure | Description | Time Frame |
|---|---|---|
| To determine DLT (Dose limiting toxicity) | incidence rate of DLT (Dose limiting toxicity) | First 3-week cycle of treatment |
| adverse events as assessed by CTCAE v5.0 | all potentially treated subjects to assess the safety | up to 5years |
| Measure | Description | Time Frame |
|---|---|---|
| ADAS-Cog 13 response rate | response rate, no change or improvement on ADAS cog 13 score | Screening, after the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks |
| The Clinical Dementia Rating Sum of Boxes |
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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
â‘ Probable bvFTD (behavior variant FTD)
â‘¡ svPPA (semantic variant primary progressive aphasia)
â‘¢ 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:
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| Name | Affiliation | Role |
|---|---|---|
| HeeJin Kim | Samsung Medical Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Samsung Medical Center | Seoul | Gangnam-gu | 06351 | South Korea |
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| ID | Term |
|---|---|
| D057180 | Frontotemporal Dementia |
| ID | Term |
|---|---|
| D057174 | Frontotemporal Lobar Degeneration |
| D003704 | Dementia |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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Single Group
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Change from the baseline in CDR-SB, min 0, max 24, higher scores mean a worse outcome
| 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 | 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 | the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks |
| preliminary efficacy | Change from the baseline in CSF biomarkers | up to 12weeks |
| K-MMSE | Korean Mini-Mental State Examination(MMSE), min 0, max 30, higher scores mean a better outcome | the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks |
| D009422 | Nervous System Diseases |
| D057177 | TDP-43 Proteinopathies |
| D019636 | Neurodegenerative Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D019965 | Neurocognitive Disorders |
| D001523 | Mental Disorders |