Observational Study of Long-term Safety and Efficacy of Subjects Who Participated in CS10BR05 Inj. Phase 1 Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- Safety assessment(evaluation)
研究概览
简要总结
The study purpose is conducting follow-up surveillance for the incidence of adverse events and efficacy of subjects participated in phase 1 trial to evaluate the safety and tolerability of autologous bone marrow-derived mesenchymal stem cells (CS10BR05) in subjects with Multiple System Atrophy until 60 months from administering investigational product (IP).
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 and erectile dysfunction), Parkinson's symptoms (movement decreases and limb tremors) and cerebellar ataxia symptoms (grogginess and incorrect pronunciation). 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 purpose is conducting follow-up surveillance for the incidence of adverse events and efficacy of subjects participated in phase 1 trial to evaluate the safety and tolerability of autologous bone marrow-derived mesenchymal stem cells (CS10BR05) in subjects with Multiple System Atrophy until 60 months from administering investigational product (IP).
Because it is the following observational study for subjects participated in phase 1 trial, there is no additional administration of the investigational product in the study.
If the subjects who participated in phase 1 trial voluntarily consent in writing to take part in this observational study, the subjects visit the institution according to the protocol that designed the follow-up visits every 3 months (Visit 1, Visit 2) until 6 months from administration of investigational product and since then 6 months later (Visit 3), every 12 months (Visit 4~7) to observe the incidence of adverse events and efficacy.
As a result of evaluating severity of AE divided into subjective, objective in accordance with the CTCAE (Version 4.0) standards.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects who participated in the phase 1 clinical trial(Protocol No.: CS10BR05- MSA101) at 3 months after administering investigational product
- •Subjects who give a written, signed and dated informed consent spontaneously
排除标准
- •Subjects who decided as inappropriate cases to participate in the observational study by investigator
结局指标
主要结局
Safety assessment(evaluation)
时间窗: 5 years
1. Adverse events(AE): After the completion of the CS10BR05-MSA101 phase 1 trial, new adverse events, including tumor are assessed and detailed information is collected at each visit. Whole body PET test is performed for tumor observation at visit 7. 2. Laboratory test: The following laboratory tests are performed at each visit. * Hematology test: WBC, RBC, Hemoglobin, Hematocrit, Platelets count, WBC Diffcount * Chemistry test: Na, K, Ca, Cl, BUN, Creatinine, Uric acid, Total bilirubin, Albumin, Total protein, Creatine Kinase, Cystatin-C, ALT, AST, r-GT, ALP, LDH, Glucose, Total cholesterol, Triglyceride * Urine test: Protein, Glucose, Urobilinogen, WBC, RBC 3. Measuring vital signs (blood pressure in mmHg, heart rate in bpm, body temperature in Celsius) is performed at each visit. 4. Physical examination is performed at each visit. If clinically significant abnormal findings are recorded in the CRF and collected as AEs.
Efficacy assessment(evaluation)
时间窗: 5 years
The following tests are conducted at each visit to exploratively evaluate the efficacy. Evaluate the change of score of each tools. -UMSARS(Unified Multiple System Atrophy rating scale) The scale comprises the following components: Part I, historical, 12 items; Part II, motor examination, 14 items; Part III, autonomic examination; and Part IV, global disability scale (total UMSARS with scores ranging from 1 to 109) -K-MMSE(Korea Mini-Mental Status Examination) The scale comprises the following components: Orientation, memory, attention and calculation, naming and language, and drawing (total K-MMSE score of 30) -DSM-Ⅳ(Diagnostic and Statistical Manual of Mental Disorders)
次要结局
未报告次要终点