Umbilical Cord Blood Therapy in a Child With Eosinophilic Duodenitis and Autism Spectrum Disorder: a Case Study
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 1
- 主要终点
- Feasibility and Safety of Umbilical Cord Blood Infusion in a Child with Eosinophilic Duodenitis and Autism Spectrum Disorder
研究概览
简要总结
This single-case exploratory clinical study aims to evaluate the therapeutic potential of umbilical cord blood (UCB) infusion in a pediatric patient diagnosed with both eosinophilic duodenitis (ED) and autism spectrum disorder (ASD). ED is a rare inflammatory gastrointestinal condition characterized by excessive eosinophil infiltration in the duodenal mucosa, often associated with immune hypersensitivity and allergic responses. ASD is a neurodevelopmental disorder marked by deficits in social interaction, communication, and behavioral flexibility. Recent evidence suggests a link between gastrointestinal inflammation and neurodevelopmental symptoms via the gut-brain axis, especially in patients with co-occurring ASD and eosinophilic gastrointestinal disorders (EGIDs).
In this study, the patient will receive three UCB infusions: one autologous and two allogeneic. The first (autologous) UCB is stored at a certified cord blood bank and will be administered intravenously. Subsequently, two allogeneic UCB infusions will be administered six weeks apart using HLA-matched donor units selected from a hospital-based cord blood repository. The cell product will contain a minimum of 3 × 10⁷ total nucleated cells per kg, and donor-recipient compatibility for HLA A, B, and DRB1 will be considered.
To support immune tolerance and reduce potential adverse responses, a 7-day course of low-dose oral cyclosporine will be administered with each allogeneic infusion. All cord blood handling, thawing, and infusion will be performed in a cell therapy center under standardized protocols.
The primary aim is to explore the immune regulatory effects and symptom relief following UCB therapy in this rare comorbid case. Assessments will include brain MRI with DTI, EEG, fNIRS, sensory profiles (SP), social communication questionnaires (SCQ), autism rating scales (K-CARS-2), behavioral checklists (CBCL), gastrointestinal endoscopy, and developmental/cognitive/language assessments (e.g., WISC, WPPSI, GMFM, VMI, SELSI, PRES, FIM). Blood samples will be analyzed for eosinophil counts and gene/protein expression related to inflammation, neuroendocrine function, and gut-brain signaling (e.g., TNF-α, IL-6, serotonin, dopamine, GABA, CRH, BDNF).
This case study will also track safety indicators including vital signs, laboratory panels, and adverse events. The data may inform the feasibility of future therapeutic use of UCB in children with complex immune-neurodevelopmental conditions.
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
- 4 Years 至 4 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Child aged 4 years at the time of enrollment
- •Diagnosed with both eosinophilic duodenitis (ED) and autism spectrum disorder (ASD)
- •Actively receiving outpatient care at the Department of Rehabilitation Medicine, CHA Bundang Medical Center
- •Autologous cord blood available and stored at an accredited cord blood bank
- •Written informed consent provided by a legally authorized representative (parent or guardian) after receiving a full explanation of the study
排除标准
- •Presence of a severe uncontrolled medical condition that may interfere with cord blood infusion or study assessments
- •History of severe allergic reaction to components of the investigational product or immunosuppressive agents (e.g., cyclosporine)
- •Current or recent participation (within 30 days) in another interventional clinical trial
- •Any contraindication to MRI, EEG, or fNIRS assessments (e.g., implanted metal device, severe behavioral intolerance)
- •Determined by the principal investigator to be unsuitable for participation due to safety concerns or noncompliance
研究组 & 干预措施
umbilical cord blood
This is a single-arm, open-label, exploratory case study involving one pediatric participant diagnosed with both eosinophilic duodenitis (ED) and autism spectrum disorder (ASD). The intervention includes a series of three intravenous umbilical cord blood (UCB) infusions: one autologous UCB infusion followed by two allogeneic UCB infusions at 6-week intervals. Each infusion is performed under sterile conditions at a hospital-based cell therapy center. The donor UCB units are selected based on HLA compatibility (minimum 3/6 match for HLA-A, B, DRB1) and total nucleated cell count (≥ 3×10⁷ cells/kg). The allogeneic infusions are accompanied by a 7-day low-dose oral cyclosporine regimen to reduce the risk of immune rejection. The total study period includes baseline evaluation, three infusion sessions, and multiple post-infusion follow-up assessments over three months.
干预措施: Umbilical Cord Blood Infusion (Autologous and Allogeneic) (Biological)
结局指标
主要结局
Feasibility and Safety of Umbilical Cord Blood Infusion in a Child with Eosinophilic Duodenitis and Autism Spectrum Disorder
时间窗: From baseline (within 6 months prior to first infusion) through 3 months after the final (third) UCB infusion (approximately 4-5 months total)
The number and severity of adverse events (AEs) and serious adverse events (SAEs) related to cord blood infusion will be assessed and categorized using MedDRA and CTCAE v5.0. Unit of measure is number of participants with AE or SAE
次要结局
- Change in Sensory Processing Scores (Sensory Profile)(Baseline (within 6 months prior to first infusion), 7 days after each infusion, and 3 months after final infusion)
- Change in Social Communication (SCQ Score)(Baseline, 7 days after each infusion, and 3 months after final infusion)
- Change in Behavioral Functioning (CBCL)(Baseline, 7 days after each infusion, and 3 months after final infusion)
- Change in Brain Connectivity and White Matter Integrity (MRI with DTI)(Baseline and 3 months after final infusion)
- Change in Cortical Hemodynamic Response (fNIRS)(Baseline and 3 months after final infusion)
- Change in EEG Patterns(Baseline and 3 months after final infusion)
- Duodenal Eosinophil Count (if clinically indicated)(Baseline, 7 days after each infusion, and 3 months after final infusion)
- Change in Autism Severity (K-CARS-2)(Baseline, 7 days after each infusion, and 3 months after final infusion)
- Peripheral Blood Eosinophil Count(Baseline, 7 days after each infusion, and 3 months after final infusion)
研究者
MinYoung Kim, MD, PhD
Principle Investigator
Bundang CHA Hospital