Clinical trial NCT06995274: Umbilical Cord Blood Therapy in a Child With Eosinophilic Duodenitis and Autism Spectrum Disorder: a Case Study
Official title: Umbilical Cord Blood Therapy in a Child With Eosinophilic Duodenitis and Autism Spectrum Disorder: a Case Study
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: NA
Conditions: Autism Spectrum Disorder
Eosinophilic Gastrointestinal Disorders
Lead sponsor: Bundang CHA Hospital
Sponsor class: OTHER
First posted: 2025-05-29
Enrollment: 1
Sex: MALE
Minimum age (years): 4
Maximum age (years): 4
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

1. Child aged 4 years at the time of enrollment
2. Diagnosed with both eosinophilic duodenitis (ED) and autism spectrum disorder (ASD)
3. Actively receiving outpatient care at the Department of Rehabilitation Medicine, CHA Bundang Medical Center
4. Autologous cord blood available and stored at an accredited cord blood bank
5. Written informed consent provided by a legally authorized representative (parent or guardian) after receiving a full explanation of the study

Exclusion Criteria:

1. Presence of a severe uncontrolled medical condition that may interfere with cord blood infusion or study assessments
2. History of severe allergic reaction to components of the investigational product or immunosuppressive agents (e.g., cyclosporine)
3. Current or recent participation (within 30 days) in another interventional clinical trial
4. Any contraindication to MRI, EEG, or fNIRS assessments (e.g., implanted metal device, severe behavioral intolerance)
5. Determined by the principal investigator to be unsuitable for participation due to safety concerns or noncompliance

Primary outcomes:
measure: Feasibility and Safety of Umbilical Cord Blood Infusion in a Child with Eosinophilic Duodenitis and Autism Spectrum Disorder
timeFrame: From baseline (within 6 months prior to first infusion) through 3 months after the final (third) UCB infusion (approximately 4-5 months total)
Secondary outcomes:
measure: Change in Sensory Processing Scores (Sensory Profile)
timeFrame: Baseline (within 6 months prior to first infusion), 7 days after each infusion, and 3 months after final infusion
measure: Change in Social Communication (SCQ Score)
timeFrame: Baseline, 7 days after each infusion, and 3 months after final infusion
measure: Change in Behavioral Functioning (CBCL)
timeFrame: Baseline, 7 days after each infusion, and 3 months after final infusion
measure: Change in Brain Connectivity and White Matter Integrity (MRI with DTI)
timeFrame: Baseline and 3 months after final infusion
measure: Change in Cortical Hemodynamic Response (fNIRS)
timeFrame: Baseline and 3 months after final infusion
measure: Change in EEG Patterns
timeFrame: Baseline and 3 months after final infusion
measure: Duodenal Eosinophil Count (if clinically indicated)
timeFrame: Baseline, 7 days after each infusion, and 3 months after final infusion
measure: Change in Autism Severity (K-CARS-2)
timeFrame: Baseline, 7 days after each infusion, and 3 months after final infusion
measure: Peripheral Blood Eosinophil Count
timeFrame: Baseline, 7 days after each infusion, and 3 months after final infusion
Other outcomes:
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