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自闭症谱系障碍儿童的 hCT-MSC (IMPACT)

2026年5月28日 更新者：Joanne Kurtzberg, MD

HCT-MSC（一种脐带来源的间充质基质细胞产物）在自闭症谱系障碍儿童中的 II 期研究

这项 II 期研究的目的是确定人脐带组织来源的间充质基质细胞 (hCT-MSC) 对改善自闭症谱系障碍 (ASD) 儿童社交沟通能力的功效。

研究概览

地位

完全的

条件

干预/治疗

详细说明

这项双盲 II 期研究的目的是确定人脐带组织来源的间质间质细胞 (hCT-MSC) 以两种不同的给药策略对患有自闭症谱系障碍 (ASD) 的儿童的疗效。

这项研究将招募 4-11 岁的 ASD 儿童。 合格的受试者将接受神经心理学评估、脑电图测试、眼动追踪、CVA 评估和研究产品输注。 受试者将被随机分配到两个研究组之一； 1) 在基线时单次输注 6.0x106 个细胞/Kg，然后在六个月时进行盲法安慰剂输注，或 2) 在基线时进行安慰剂输注，然后在六个月时静脉注射 6x106 个细胞/Kg。

这项研究的主要终点是社会沟通技巧从基线到六个月的变化。 与输注 MSCs 相关的潜在风险包括对产品的反应（皮疹、呼吸急促、喘息、呼吸困难、低血压、口腔、喉咙或眼睛周围肿胀、心动过速、出汗）、感染传播和 HLA 致敏。

研究类型

介入性

注册 (实际的)

137

阶段

  • 阶段2

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习地点

  • 美国
    • North Carolina
      • Durham、North Carolina、美国、27705
        • Duke University Medical Center

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

4年 至 11年 (孩子)

接受健康志愿者

不

描述

纳入标准

  1. 同意时年龄 ≥ 4 岁至 < 12 岁（11 岁 364 天）
  2. 根据自闭症症状简要观察 (BOSA) 和自闭症诊断访谈修订版 (ADI-R) 提供的信息，使用 DSM-5 检查表确认自闭症谱系障碍的临床 DSM-5 诊断
  3. 进行了脆性 X 测试且呈阴性；进行了 CMA 和/或全外显子组测序，结果与自闭症诊断无关
  4. 在输注研究产品前至少 2 个月在目前的精神科药物治疗方案（剂量和给药方案）上保持稳定
  5. 正常绝对淋巴细胞计数（非裔美国人参与者≥1200/uL，所有其他参与者≥1500/uL）
  6. GAI ≥ 65 通过研究人员的认知测试
  7. 参与者和家长/监护人会说英语
  8. 能够两次前往杜克大学（基线，六个月），并且家长/监护人能够参与中期调查和访谈
  9. 至少一位父母/监护人的父母/监护人同意书

排除标准

  1. 一般的：

    1. 医疗记录审查和/或筛查评估表明 ASD 诊断和/或 GAI > 65 不自信
    2. 已知诊断为以下任何并存的精神疾病：抑郁症、双相情感障碍、精神分裂症、与双相情感障碍相关的强迫症、图雷特综合征
    3. 筛选数据表明，参与者将无法遵守研究团队评估的研究程序要求
    4. 家人不愿意或不能承诺参与所有与研究相关的评估，包括方案跟进
    5. 兄弟姐妹参加了这项 (Duke IMPACT) 研究

  2. 遗传：

    1. 记录表明孩子患有已知的遗传综合征，例如（但不限于）脆性 X 综合征、神经纤维瘤病、Rett 综合征、结节性硬化症、PTEN 突变、囊性纤维化、肌肉萎缩症或明确已知与 ASD 相关的遗传缺陷
    2. 与 ASD 相关的已知致病突变或拷贝数变异 (CNV)（例如 16p11.2、 15q13.2, 2q13.3)

  3. 传染性：

    1. 已知活动性中枢神经系统感染
    2. 基于记录或临床评估的不受控制的感染证据
    3. 已知的 HIV 阳性
    4. 在过去 14 天内接触过 COVID-19 或在过去 28 天内 COVID-19 测试呈阳性。 有 COVID-19 感染史的受试者在初次就诊前 14 天必须无症状。

  4. 医疗的：

    1. 已知代谢紊乱
    2. 已知的线粒体功能障碍
    3. 不稳定癫痫或不受控制的癫痫病史、婴儿痉挛症、Lennox Gastaut 综合征、Dravet 综合征或其他类似的慢性癫痫病史
    4. 活动性恶性肿瘤或既往接受过化疗的恶性肿瘤
    5. 原发性免疫缺陷病史
    6. 自身免疫性血细胞减少史（即 ITP、AIHA）
    7. 合并症会使孩子在研究过程中出现并发症的风险增加
    8. 可能需要未来干细胞移植的并发遗传或获得性疾病或合并症
    9. 严重的感觉（例如，失明、耳聋、未矫正的听力障碍）或运动（例如，脑瘫）障碍
    10. 由血清肌酐 >1.5mg/dL 或总胆红素 >1.3mg/dL 确定的肾功能或肝功能受损，已知患有吉尔伯特病的患者除外
    11. 显着的血液学异常定义为：血红蛋白 <10.0 g/dL，血小板 <150 x 10e9/uL，WBC <3,000 个细胞/mL，非裔美国人 ALC <1200/uL 或所有其他参与者 <1500/uL。
    12. 由 PI 或其他研究人员（包括接受过识别与神经发育状况相关的畸形特征培训的医学遗传学家和精神病学家）评估的表明遗传综合征的临床相关身体畸形证据。

  5. 当前/之前的治疗：

    A。可用的合格的自体脐带血单位或父母推迟使用合格的自体脐带血单位 b． 既往细胞治疗史 c. 当前或之前使用过 IVIG 或其他抗炎药物，NSAIDs 除外 d. 当前或之前的免疫抑制治疗 i. 没有持续超过 2 周的全身性类固醇治疗，并且在入组前 3 个月内没有全身性类固醇。 允许局部和吸入类固醇。

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：随机化
  • 介入模型：并行分配
  • 屏蔽：四人间

武器和干预

参与者组/臂
干预/治疗
实验性的：海运中心
静脉内施用一剂 6x10e6 细胞/kg。
生物：脐带组织间充质基质细胞
人脐带组织来源的间充质基质细胞 (hCT-MSC)，从同种异体无关供体的脐带组织中分离和扩增。 静脉内施用一剂 6x10e6 细胞/kg。
安慰剂比较：安慰剂输液
其他：安慰剂输液
安慰剂对比输液

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Change on the Average Socialization and Communication Subscale Standard Scores on the Vineland Behavior Scales (VABS-3)
大体时间：Baseline, 6 months
The primary outcome measure is the mean of the Socialization and Communication Subscale Standard Scores on the Vineland Adaptive Behavior Scales (VABS-3) from the Comprehensive Interview form. The primary endpoint is the change in this outcome measure from baseline to six months before the second infusion. A positive change in the scores indicates an improvement in socialization and communication. A standard score of 100 is the mean with a standard deviation of 15 points. A score of 100 should be understood as being similar to the typical population of the same age. Scores greater than or equal to 86 are considered adequate or above adequate. Domain scores less than or equal to 85 are considered moderately low to low and indicate the patient has a significant skill deficit when compared with similarly aged peers.
Baseline, 6 months

次要结果测量

结果测量
措施说明
大体时间
Change in VABS-3 (Vineland Adaptive Behavior Scales) Socialization Standard Score
大体时间：Baseline, 6 months
The change in the Socialization Subscale Standard Scores on the Vineland Adaptive Behavior Scales (VABS-3) from baseline to six months. Higher Socialization Standard scores indicate greater socialization. A positive change in the scores indicates an improvement in socialization. A standard score of 100 is the mean with a standard deviation of 15 points. A score of 100 should be understood as being similar to the typical population of the same age. Scores greater than or equal to 86 are considered adequate or above adequate. Domain scores less than or equal to 85 are considered moderately low to low and indicate the patient has a significant skill deficit when compared with similarly aged peers.
Baseline, 6 months
Change in VABS-3 Communication Standard Score
大体时间：Baseline, 6 months
The change in the Communication Standard Scores on the Vineland Adaptive Behavior Scales (VABS-3) from the Comprehensive Interview form from baseline to six months before the second infusion. Higher scores indicate greater communication. A positive change in the scores indicates an improvement in communication. A standard score of 100 is the mean with a standard deviation of 15 points. A score of 100 should be understood as being similar to the typical population of the same age. Scores greater than or equal to 86 are considered adequate or above adequate. Domain scores less than or equal to 85 are considered moderately low to low and indicate the patient has a significant skill deficit when compared with similarly aged peers.
Baseline, 6 months
CGI-S (Clinical Global Impression - Severity of Illness) Overall Score
大体时间：6 months
The CGI-S Overall Score is a 7-point scale that requires the clinician to rate the severity of the participant's overall functioning and symptoms of autism at the time of assessment, relative to the clinician's experience with participants who have the same diagnosis. The clinician rates the severity of autism symptoms - 1, normal, no symptoms; 2, borderline level of symptoms; 3, mild symptoms; 4, moderate symptoms; 5, marked symptoms; 6, severe symptoms; or 7, extremely severe symptoms. The higher ratings indicate greater severity of overall functioning impairment.
6 months
CGI-I (Clinical Global Impression - Improvement) Overall Score
大体时间：6 months
The CGI-I Overall Score is a 7-point scale that requires the clinician to assess how much the participant's autism overall functioning and symptoms have improved or worsened relative to a baseline assessment. The symptoms are rated as: 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse. The lower scores indicate greater improvement.
6 months
Change in the Pediatric Quality of Life (PedsQL) Total Scale Score
大体时间：Baseline, 6 months
The PedsQL 4.0 Generic Core Scales is a 5-minute parent questionnaire that measures the child's functioning in the dimensions of physical, emotional, social, and school. The items use a Likert rating scale from 0 (Never) to 4 (Almost Always). This 0-4 scale is then transformed to 0=100, 1=75, 2=50, 3=25, and 4=0 for a reverse score. The Total Scale Score is then computed as the sum of all the items over the number of items answered on all the Scales for a total score range of 0 to 100. Higher scores indicate a better quality of life.
Baseline, 6 months

其他结果措施

结果测量
措施说明
大体时间
Number of Participants Experiencing an Infusion Reaction
大体时间：up to 12 months
To assess safety, participants were considered according to the treatment received at each time point, not what they were randomized. Patients received the infusion to which they were randomized at baseline and the alternate treatment at 6 months. Therefore, adverse events are reported by randomized treatment arm broken into periods: 0-6 months (post-baseline infusion, pre-six month infusion) and 6-12 months (post-six month infusion).
up to 12 months
Number of Participants Experiencing Product-related Infections
大体时间：up to 12 months
To assess safety, participants were considered according to the treatment received at each time point, not to what they were randomized. Patients received the infusion to which they are randomized at baseline, and the alternate treatment at 6 months. AEs are reported by randomized treatment arm broken into periods: 0-6 months (post-baseline infusion, pre-six month infusion) and 6-12 months (post-six month infusion). 6-12 month evaluation time points are further broken into whether or not the participant received a second infusion, since three MSC patients and one Placebo patient did not receive the 6-month incentive infusion but were still evaluated.
up to 12 months
Evidence of Formation of Anti-HLA Antibodies
大体时间：Baseline, 6 months, 12 months
Assess for anti-HLA antibodies
Baseline, 6 months, 12 months
Number of Participants Experiencing Graft Versus Host Disease (GVHD)
大体时间：up to 12 months
To assess safety, participants were considered according to the treatment received at each time point, not to what they were randomized. Patients received the infusion to which they are randomized at baseline, and the alternate treatment at 6 months. AEs are reported by randomized treatment arm broken into periods: 0-6 months (post-baseline infusion, pre-six month infusion) and 6-12 months (post-six month infusion). 6-12 month evaluation time points are further broken into whether or not the participant received a second infusion, since three MSC patients and one Placebo patient did not receive the 6-month incentive infusion but were still evaluated.
up to 12 months
Number of Participants Experiencing Unexpected Adverse Events Related to the Study Product
大体时间：up to 12 months
To assess safety, participants were considered according to the treatment received at each time point, not to what they were randomized. Patients received the infusion to which they are randomized at baseline, and the alternate treatment at 6 months. AEs are reported by randomized treatment arm broken into periods: 0-6 months (post-baseline infusion, pre-six month infusion) and 6-12 months (post-six month infusion). 6-12 month evaluation time points are further broken into whether or not the participant received a second infusion, since three MSC patients and one Placebo patient did not receive the 6-month incentive infusion but were still evaluated.
up to 12 months
Change on the Adaptive Behavior Composite Subscale Standard Score on the Vineland Behavior Scales (VABS-3)
大体时间：Baseline, 6 months
The change in the Vineland Adaptive Behavior Scales (VABS-3) Adaptive Behavior Composite Subscale from baseline to 6 months before the second infusion. Higher Adaptive Behavior Composite Standard scores indicate greater adaptive behavior. A positive change in the scores indicates an improvement in adaptive behavior. A standard score of 100 is the mean with a standard deviation of 15 points. A score of 100 should be understood as being similar to the typical population of the same age. Scores greater than or equal to 86 are considered adequate or above adequate. Domain scores less than or equal to 85 are considered moderately low to low and indicate the patient has a significant skill deficit when compared with similarly aged peers.
Baseline, 6 months
Change in VABS-3 (Vineland Adaptive Behavior Scales) Daily Living Skills Standard Score
大体时间：Baseline, 6 months
The change in the Daily Living Skills Subscale Standard Scores on the Vineland Adaptive Behavior Scales (VABS-3) from baseline to six months before the second infusion. Higher Daily Living Skills Standard scores indicate greater daily living skills. A positive change in the scores indicates an improvement in daily living skills. A standard score of 100 is the mean with a standard deviation of 15 points. A score of 100 should be understood as being similar to the typical population of the same age. Scores greater than or equal to 86 are considered adequate or above adequate. Domain scores less than or equal to 85 are considered moderately low to low and indicate the patient has a significant skill deficit when compared with similarly aged peers.
Baseline, 6 months
Change in VABS-3 Motor Skills Standard Score
大体时间：Baseline, 6 months
The change in the Motor Skills Standard Scores on the Vineland Adaptive Behavior Scales (VABS-3) from the Comprehensive Interview form from baseline to six months before the second infusion. Higher scores indicate greater motor skills. A positive change in the scores indicates an improvement in motor skills. A standard score of 100 is the mean with a standard deviation of 15 points. A score of 100 should be understood as being similar to the typical population of the same age. Scores greater than or equal to 86 are considered adequate or above adequate. Domain scores less than or equal to 85 are considered moderately low to low and indicate the patient has a significant skill deficit when compared with similarly aged peers.
Baseline, 6 months
Change in Aberrant Behavior Checklist-Community (ABC-C) Social Withdrawal
大体时间：Baseline, 6 months
The change in the Aberrant Behavior Checklist-Community (ABC-C) Social Withdrawal scale from baseline to six months before the second infusion. Higher scores indicate greater problems with social withdrawal. A positive change in the scores indicates a worsening in social withdrawal. This scale has 16 items that are scored on a 4-point Likert scale: 0 = not a problem, 1 = slight problem, 2 = moderately serious problem, 3 = severe problem. The individual items are added together to calculate the Social Withdrawal scale score.
Baseline, 6 months
Change in Pervasive Developmental Disorder Behavior Inventory (PDDBI) Total Score
大体时间：Baseline, 6 months
The change in the Pervasive Developmental Disorder Behavior Inventory (PDDBI) from baseline to 6 months before the second infusion. The PDDBI is a measure of problem behaviors and social, language, and learning or memory skills of children who have been diagnosed with autism spectrum disorder. Raw scores are converted to T-scores. The T-score has a mean of 50 with a standard deviation of 10 points with a range of 10-100. Higher scores indicate greater behavioral issues and a positive change in score indicates worsening.
Baseline, 6 months
Change in the Autism Impact Measure (AIM)
大体时间：Baseline, 6 months
The change in the Autism Impact Measure (AIM) from baseline to 6 months before second infusion. AIM is a measure of core Autism Spectrum Disorder Symptoms. It is a parent-report questionnaire that includes 41 core-symptom items rated on two corresponding 5-point scales: frequency (ranging from "never" to "always") and impact (ranging from "not at all" to "severely") over the previous two weeks. Frequency and impact ratings are combined, yielding a total score range of 82 to 410. Higher scores indicate greater symptom severity; a positive change in score indicates worsening of symptoms.
Baseline, 6 months
Change in the Behavior Rating Inventory of Executive Function (BRIEF) Emotional Control Subscale
大体时间：Baseline, 6 months
The change in the Behavior Rating Inventory of Executive Function (BRIEF) Emotional Control subscale from baseline to month 6 before the second infusion. BRIEF is a survey that assesses executive function and self-regulation with the following subscales: Inhibit, Shift, Emotional Control, Working Memory, and Planning and Organization. Higher scores indicate greater levels of dysfunction each respective domain. A positive change in score indicates worsening the respective domain. Raw scores for each domain are converted to t-scores (Mean = 50, SD = 10) and percentiles. T scores 60-64 are considered to be mildly elevated, 65-69 are considered to be potentially clinically elevated, and >= 70 are considered to be clinically elevated.
Baseline, 6 months
Change in the Behavior Rating Inventory of Executive Function (BRIEF) Working Memory Subscale
大体时间：Baseline, 6 months
The change in the Behavior Rating Inventory of Executive Function (BRIEF) Working Memory subscale from baseline to month 6 before the second infusion. The BREIF is a survey that assesses executive function and self-regulation with the following subscales: Inhibit, Shift, Emotional Control, Working Memory, and Planning and Organization. Higher scores indicate greater levels of dysfunction each respective domain. A positive change in score indicates worsening the respective domain. Raw scores for each domain are converted to t-scores (Mean = 50, SD = 10) and percentiles. T scores 60-64 are considered to be mildly elevated, 65-69 are considered to be potentially clinically elevated, and >= 70 are considered to be clinically elevated.
Baseline, 6 months
Change in the Behavior Rating Inventory of Executive Function (BRIEF) Inhibit Subscale
大体时间：Baseline, 6 months
The change in the Behavior Rating Inventory of Executive Function (BRIEF) Inhibit subscale from baseline to month 6 before the second infusion. The BRIEF is a survey that assesses executive function and self-regulation with the following subscales: Inhibit, Shift, Emotional Control, Working Memory, and Planning and Organization. Higher scores indicate greater levels of dysfunction each respective domain. A positive change in score indicates worsening the respective domain. Raw scores for each domain are converted to t-scores (Mean = 50, SD = 10) and percentiles. T scores 60-64 are considered to be mildly elevated, 65-69 are considered to be potentially clinically elevated, and >= 70 are considered to be clinically elevated.
Baseline, 6 months
Change in the Behavior Rating Inventory of Executive Function (BRIEF) Plan/Organization Subscale
大体时间：Baseline, 6 months
The change in the Behavior Rating Inventory of Executive Function (BRIEF) Plan/Organization subscale from baseline to month 6 before the second infusion. The BRIEF is a survey that assesses executive function and self-regulation with the following subscales: Inhibit, Shift, Emotional Control, Working Memory, and Planning and Organization. Higher scores indicate greater levels of dysfunction each respective domain. A positive change in score indicates worsening the respective domain. Raw scores for each domain are converted to t-scores (Mean = 50, SD = 10) and percentiles. T scores 60-64 are considered to be mildly elevated, 65-69 are considered to be potentially clinically elevated, and >= 70 are considered to be clinically elevated.
Baseline, 6 months
Change in the Behavior Rating Inventory of Executive Function (BRIEF) Shift Subscale
大体时间：Baseline, 6 months
The change in the Behavior Rating Inventory of Executive Function (BRIEF) Shift subscale from baseline to month 6 before the second infusion. The BRIEF is a survey that assesses executive function and self-regulation with the following subscales: Inhibit, Shift, Emotional Control, Working Memory, and Planning and Organization. Higher scores indicate greater levels of dysfunction each respective domain. A positive change in score indicates worsening the respective domain. Raw scores for each domain are converted to t-scores (Mean = 50, SD = 10) and percentiles. T scores 60-64 are considered to be mildly elevated, 65-69 are considered to be potentially clinically elevated, and >= 70 are considered to be clinically elevated.
Baseline, 6 months
Change in the Expressive Vocabulary Test (Third Edition, EVT-3)
大体时间：Baseline, 6 months
The change in the Expressive Vocabulary Test (Third Edition, EVT-3) from baseline to 6 months before the second infusion. The EVT-3 is a measure of a participant's ability to match a spoken word with an image of an object, action, or concept. The number of words that are retrieved is converted to a Standard Score, where 100 is the mean with a standard deviation of 15 points. A score of 100 should be understood as being similar to the typical population of the same age. Scores greater than or equal to 86 are considered adequate or above adequate. Scores less than or equal to 85 are considered moderately low to low and indicate the patient has a significant skill deficit when compared with similarly aged peers. A positive change in EVT-3 score indicates improvement.
Baseline, 6 months

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Joanne Kurtzberg, MD

合作者

The Marcus Foundation
Cryo-Cell International

调查人员

  • 首席研究员：Lauren Franz, MBChB、Duke University
  • 首席研究员：Beth Shaz, MD、Duke University

出版物和有用的链接

负责输入研究信息的人员自愿提供这些出版物。这些可能与研究有关。

一般刊物

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2020年10月12日

初级完成 (实际的)

2023年5月2日

研究完成 (实际的)

2024年2月1日

研究注册日期

首次提交

2019年9月12日

首先提交符合 QC 标准的

2019年9月12日

首次发布 (实际的)

2019年9月13日

研究记录更新

最后更新发布 (实际的)

2026年6月24日

上次提交的符合 QC 标准的更新

2026年5月28日

最后验证

2026年5月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • Pro00113011
  • Pro00102894 (其他标识符：Duke IRB)

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

不

药物和器械信息、研究文件

研究美国 FDA 监管的药品

是的

研究美国 FDA 监管的设备产品

不

在美国制造并从美国出口的产品

不

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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