Safety and Efficacy of Intravenous Administration of SHED-CM for ALS
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- All Adverse Events
研究概览
简要总结
This study evaluates the safety and efficacy of Stem Cell from Human Exfoliated Deciduous teeth Conditioned Media (SHED-CM) in patients with Amyotrophic Lateral Sclerosis (ALS), using the Japanese version of the revised ALS Functional Rating Scale (ALSFRS-R) as an indicator.
详细描述
The main objective of this study is to evaluate the safety and efficacy of Stem Cell from Human Exfoliated Deciduous teeth Conditioned Media in patients with Amyotrophic Lateral Sclerosis (ALS), Particular focus will be placed on improving neurological function, slowing the rate of symptom progression, and improving the patient's quality of life.
What are the advantages of this therapy over standard therapy? and for which patients it is most effective ?
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
- 20 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who meet the following inclusion criteria (1) to (6) at the time of Informed consent
- •Patients who have provided written informed consent to participate in the study.
- •Patients who are at least 20 years of age at the time of obtaining informed consent.
- •Patients diagnosed with isolated or familial ALS and diagnosed as definite, the probable, or the probable laboratory-supported by updated Awaji criteria.
- •Patients with severity 1 or 2 on ALS severity criteria.
- •Outpatients.
- •Patients residing in Japan who can communicate in Japanese.
排除标准
- •Patients who do not meet any of the exclusion criteria (1) to (15) at the time of Informed consent
- •Patients with a tracheostomy
- •Patients with a history of non-invasive respiratory support
- •Patients with a percent FVC of 60 or less
- •Patients with chronic obstructive pulmonary disease (COPD)
- •Patients newly treated with edaravone or riluzole (oral) within 4 weeks prior to Informed consent
- •Patients receiving HAL medical leg type treatment
- •Patients receiving intravenous edaravone
- •Patients with cognitive impairment
- •Pregnant women or patients who may be pregnant
- •Patients with serious respiratory, cardiovascular, hepatic, or renal disease
- •Patients with malignant tumors
- •Patients with uncontrolled infection
- •Patients who have participated in other clinical trials within 12 weeks prior to obtaining consent
- •Patients with a history of drug allergy or severe allergic disease (e.g., anaphylactic shock) or concomitant history
- •Patients who are deemed inappropriate to participate in the study by the principal investigator or research coordinator.
研究组 & 干预措施
Receiving the study drug
The study drug will be administered intravenously at 120 ml once a week for 12 week.
干预措施: The study drug is SHED-CM manufactured by U-Factor (Biological)
结局指标
主要结局
All Adverse Events
时间窗: Immediately after each administration and up to 4 weeks post-treatment
This measure will check the total number of adverse events (AEs) reported during the study, categorized by CTCAE Ver 5.0 .
Number of Clinically Significant Changes in Laboratory Test Results
时间窗: Baseline, Week 4, Week 8, Week 12, and at follow-up (Week 16).
This measure will track the number of events where clinically significant abnormalities are detected in laboratory test parameters, including blood count, biochemistry, and coagulation function tests. Changes from baseline to follow-up are evaluated based on predefined clinical thresholds.
Number of Clinically Significant Changes in Vital Signs
时间窗: Baseline, Week 4, Week 8, Week 12, and at follow-up (Week 16).
This measure will evaluate the number of events where clinically significant changes in vital signs occur. Parameters include systolic blood pressure, diastolic blood pressure, pulse rate, body temperature, and SpO2. Each event will be assessed at two time points: immediately after treatment and after follow-up session, comparing values to baseline.
Safety assessment during the study period: Adverse events - Self- and other findings
时间窗: Immediately after each administration and up to 4 weeks post-treatment
Medical examination, subjective findings, Other findings
次要结局
- Efficacy assessment: ALSFRS-R(Change from baseline ALSFRS-R at follow-up session)
- Efficacy assessment: %FVC(Change from baseline %FVC at follow-up session)
- Efficacy assessment: grip strength(Change from baseline grip strength at follow-up session)