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

NCT06995274 | Not Yet Recruiting

• 1 other identifier: 2025-03-057
• Study Type: interventional
• Target: 1
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Autism Spectrum Disorder; Eosinophilic Gastrointestinal Disorders

Outcome Measures

Primary Outcomes (1)

• Feasibility and Safety of Umbilical Cord Blood Infusion in a Child with Eosinophilic Duodenitis and Autism Spectrum Disorder

  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

  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 (9)

• 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

Study Arms (1)

umbilical cord blood

EXPERIMENTAL

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.

Biological: Umbilical Cord Blood Infusion (Autologous and Allogeneic)

Interventions

Umbilical Cord Blood Infusion (Autologous and Allogeneic) BIOLOGICAL

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 cells per kilogram of body weight. Each infusion is administered intravenously via peripheral vein at the Cell Therapy Center of CHA Bundang Medical Center. Prior to each infusion, the participant receives premedication with intravenous chlorpheniramine maleate (0.6 mL/10 kg) to reduce the risk of allergic reactions. For allogeneic infusions, oral cyclosporine (0.07 mL/kg twice daily) is administered for 7 days (3 days before, day of, and 3 days after infusion) to minimize immune-mediated reactions

umbilical cord blood

Eligibility Criteria

• Age: 4 Years - 4 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Bundang CHA Hospital: lead

MeSH Terms

Conditions

Autism Spectrum Disorder; Eosinophilic Esophagitis

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders; Esophagitis; Esophageal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Gastroenteritis; Eosinophilia; Leukocyte Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases

Central Study Contacts

Soyoung Choi

CONTACT

82 031 780 6003; csy505@chamc.co.kr

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principle Investigator

Study Record Dates

• First Submitted: May 11, 2025
• First Posted: May 29, 2025
• Study Start: June 1, 2025
• Primary Completion: December 31, 2025
• Study Completion: December 31, 2025
• Last Updated: May 29, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share