Clinical Assessment on the Safety and Potential Efficacy of Mesenchymal Stem Cells Preconditioned With Ethionamide (ET-STEM) in Patients With Frontotemporal Dementia (FTD)
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- To determine DLT (Dose limiting toxicity)
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 40 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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)
排除标准
- •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)
- •A subject in whom Ommaya reservoir insertion and general anesthesia are considered difficult
- •Abnormal Laboratory findings at Screening
- •Suspected active lung disease based on chest X-ray at Screening
- •Positive hepatitis B nuclear antibody and hpatitis C antibody
- •Subjects who the principal investigator considers inappropriate for participation in the study due to the possible harmful effect on the subjects,difficulty in study completion, or previous or current medical conditions that may disturb evaluation of study results
- •Subjects who the principal investigator considers impossible to comply with clinical research procedures.
研究组 & 干预措施
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
干预措施: ET-STEM (Drug)
结局指标
主要结局
To determine DLT (Dose limiting toxicity)
时间窗: First 3-week cycle of treatment
incidence rate of DLT (Dose limiting toxicity)
adverse events as assessed by CTCAE v5.0
时间窗: up to 5years
all potentially treated subjects to assess the safety
次要结局
- 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)
研究者
Hee Jin Kim
assistant professor
Samsung Medical Center