A Phase 1 Study to Evaluate the Safety and Tolerability of Autologous Bone Marrow Derived Mesenchymal Stem Cells in Subjects with Multiple System Atrophy
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- Adverse events, frequency and rate of vitals sign, laboratory test, physical examination, ECG, etc.
研究概览
简要总结
The purpose of conducting phase 1 trial is to evaluate the safety and tolerability of autologous bone marrow-derived mesenchymal stem cells(CS10BR05) in subjects with Multiple System Atrophy.
Evaluation of DLT by carotid artery(intra-arterial) injection according to dose-escalating in Multiple System Atrophy.
详细描述
Multiple system atrophy is a neurodegenerative disease of the central nervous system which is accompanied by signs of autonomic imbalance (orthostatic hypotension, urinary problems, erectile dysfunction), Parkinson's symptoms (movement decreases, limb tremors) and cerebellar ataxia symptoms (grogginess, pronounced incorrectly). It shows signs similar to Parkinson's disease, however, it doesn't show improvement of symptoms by dopaminergic drugs and occurs at any age.
The clinical trial was designed as a single center, open-label, public phase 1 clinical trials.
If that subject's written consent to participate in this clinical trial will be conducted the required examinations and tests in accordance with the study protocol.
Then, the register for each dose step. For the final 3 subjects in the inclusion / exclusion criteria and be administered a drug test.
And check the adverse events for more than four hours after administration of the investigational drug, and will determine whether the expression of adverse drug reaction(ADR) by the visit after 1 days, 14 days and 28 days.
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
- 30 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and Female patients aged ≥30 years and ≤ 75 years
- •Meet criteria for diagnosis of probable MSA -cerebellar type according to the Gilman Criteria(2008)
- •MRI or PET imaging showed a decrease in the cerebellar atrophy or cerebral metabolism
- •Patients who are less than 4 years from the time of documented MSA diagnosis
- •Patients unified MSA rating scale 30~50
- •Those who have no hematologic abnormalities and who are not suspected of failing bone marrow function
- •Patients who consented to participate in the study in writing by themselves or their legal representatives
排除标准
- •Suspected clear Dementia (K-MMSE < 24)
- •DSM-IV criteria for Dementia
- •Radiologic imaging findings suggest that vascular encephalopathy coexist
- •Other central nervous system diseases except MSA (Parkinsons disease etc.)
- •Patients with Stroke or Brain surgery
- •If there is a coexistence of severe medical illness, or if it is in a severe pyrexia state
- •Serum SGOT / SGPT measures above three times of upper limit of normal or creatine levels were above 1.5 times of upper limit of normal levels were more than 1.5 times normal
- •disease that affects the patient's long-term survival(Tumor, Serious Heart failure)
- •Patients with genetic tests showed that spinocerebellar ataxia 1, 2, 3, 6, 7, 17
- •Patients with unstable vital signs
- •Patients with uncontrolled comorbidities such as moderate to severe infections, bleeding
- •Those who are found to be active to viral infection(HBV, HCV, HIV, CMV, HTLVⅠ/Ⅱ, VDRL)
- •Patients who are hypersensitive to bovine protein or antibiotics such as penicillin and streptomycin
- •Patients with difficult catheter insertion(bleeding disorder, artery hardening narrowness, Patients who are at risk of stroke in cerebral angiography)
- •Patients with cardiovascular disease(for example, hypertension, myocardial infarction etc;)
- •Severe disease uncontrolled (diabetes)
- •Those who are using drug likely to affect bone marrow functions
- •Pregnant women or nursing women
- •Women of childbearing age and male who do not consent to use proper contraception to prevent his partner from being pregnant during participation in the study
- •Now clinical trials treated with other drugs and in clinical trials the previous 4 weeks
- •Subjects who by the investigator to make them ineligible for participation in this clinical study
研究组 & 干预措施
CS10BR05
The single injection of CS10BR05 Inj. in the carotid artery
干预措施: CS10BR05 (Biological)
结局指标
主要结局
Adverse events, frequency and rate of vitals sign, laboratory test, physical examination, ECG, etc.
时间窗: up to 28days
To evaluate the safety and tolerability of CS10BR05 Inj. in patients with Multiple System Atrophy.
Dose-Limiting Toxicity and Maximum Tolerated Dose of CS10BR05 Inj.
时间窗: up to 28days
To evaluate the safety and tolerability of CS10BR05 Inj. in patients with Multiple System Atrophy including determination of its Dose Limiting Toxicity (DLT) and Maximum Tolerated Dose (MTD).
次要结局
- Unified Multiple System Atrophy Rating Scale(UMSARS)(-35days, 28days)