Umbilical Cord Blood Therapy in a Child With Eosinophilic Duodenitis and Autism Spectrum Disorder: a Case Study

Обзор

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.

Вмешательства

• Биопрепарат Umbilical Cord Blood Infusion (Autologous and Allogeneic)
  The intervention consists of three intravenous infusions of umbilical cord blood (UCB) in a pediatric patient diagnosed with both eosinophilic duodenitis (ED) and autism spectrum disorder (ASD). The first infusion uses autologous UCB stored at a certified cord blood bank. The second and third infusions use allogeneic UCB units selected from a hospital-based donor cord blood bank. Donor units are matched at a minimum of 3 out of 6 HLA loci (HLA-A, B, DRB1) and contain ≥ 3×10⁷ total nucleated cell

Первичные конечные точки

• Feasibility and Safety of Umbilical Cord Blood Infusion in a Child with Eosinophilic Duodenitis and Autism Spectrum Disorder [Срок оценки: From baseline (within 6 months prior to first infusion) through 3 months after the final (third) UCB infusion (approximately 4-5 months total)]

Вторичные конечные точки (9)

• Change in Sensory Processing Scores (Sensory Profile) [Срок оценки: 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) [Срок оценки: Baseline, 7 days after each infusion, and 3 months after final infusion]
• Change in Behavioral Functioning (CBCL) [Срок оценки: Baseline, 7 days after each infusion, and 3 months after final infusion]
• Change in Brain Connectivity and White Matter Integrity (MRI with DTI) [Срок оценки: Baseline and 3 months after final infusion]
• Change in Cortical Hemodynamic Response (fNIRS) [Срок оценки: Baseline and 3 months after final infusion]
• Change in EEG Patterns [Срок оценки: Baseline and 3 months after final infusion]
• Duodenal Eosinophil Count (if clinically indicated) [Срок оценки: Baseline, 7 days after each infusion, and 3 months after final infusion]
• Change in Autism Severity (K-CARS-2) [Срок оценки: Baseline, 7 days after each infusion, and 3 months after final infusion]
• Peripheral Blood Eosinophil Count [Срок оценки: Baseline, 7 days after each infusion, and 3 months after final infusion]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT06995274 · 2025-03-057

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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