Umbilical Cord Blood Therapy for Global Developmental Delay

Study Overview
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Brief Summary
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This open-label study aims to evaluate the safety and efficacy of autologous or allogeneic umbilical cord blood therapy for children with global developmental delay.

Detailed Description
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Global developmental delay is defined as significant delay in two or more of the following developmental domains: gross/fine motor, speech/language, cognition, social/personal, and activities of daily living. Umbilical cord blood has been used for inherited metabolic diseases that feature global developmental delay and many experimental animal studies have revealed umbilical cord blood is useful to repair neurological impairments in brain.

Official Title
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Umbilical Cord Blood Therapy for Children With Global Developmental Delay

Conditions
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Global Developmental Delay

Intervention / Treatment
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* Procedure: Umbilical cord blood transplantation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Global developmental delay Willing to comply with all study procedure Exclusion Criteria: Medical instability including pneumonia or renal function at enrollment Poor cooperation of guardian,including inactive attitude for rehabilitation and visits for follow-up Uncontrolled persistent epilepsy Not eligible according to the principal investigator

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 15 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: allogeneic cord blood transplantation for children with developmental delay
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Umbilical cord blood transplantation<br>Allogeneic umbilical cord blood will be administered intravenously or intraarterially under non-myeloablative immunosuppression. In case of autologous umbilical cord blood, immunosuppression is not required. | Procedure: Umbilical cord blood transplantation<br> <br> * Other names: Autologous or Allogeneic Umbilical Cord Blood;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in Cognition | Korean Wechsler Preschool and Primary Scale of Intelligence (K-WPPSI) (Higher value means better cognitive function (K-WPPSI: below 70 - worst, over 130 - best) | Baseline - 6 months - 12 months | 
| Changes in Cognitive Neurodevelopmental Outcome | Korean version of Bayley Scale of Infant Development-II Mental Scales (Higher value means better cognitive function: raw score 0 - worst, 178 - best.) | Baseline - 1 month - 3 months - 6 months - 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in Motor Performance | GMPM (Gross Motor Performance Measure) as a standardized measurement tool for assessing quality of movement regarding 3 properties of 5 ones; alignment, coordination, dissociated movement, stability, and weight shift (range: 0 100, Higher value means better motor quality.) | Baseline - 1 month - 3 months - 6 months - 12 months | 
| Changes in Standardized Gross Motor Function | GMFM (Gross Motor Function Measure) as a standardized measurement tool for assessing Gross Motor Function consisting of sub-scales; lying & rolling, sitting, crawling & kneeling, standing, walking, running & jumping (range: 0 100 , Higher value means better gross motor function.) | Baseline - 1 month - 3 months - 6 months - 12 months | 
| Changes in Motor Neurodevelopmental Outcome | Korean version of Bayley Scale of Infant Development-II Motor Scales (Higher value means better motor function: raw score 0 - worst, 112 - best) | Baseline - 1 month - 3 months - 6 months - 12 months | 
| Changes in Functional Independence in Daily Activities | WeeFIM (Functional Independence Measure for Children) measures functional independence in daily activities. WeeFIM contains 18 items and each item is ranked from complete dependence (scored as 1) to complete independence (scored as 7). The range is from 18 to 126 and higher scores mean more independent performance in daily activities. | Baseline - 1 month - 3 months - 6 months - 12 months | 
| Changes in Visual Perception Test | Visual perception function is evaluated with one of three measures: DTVP (Developmental Test of Visual Perception), MVPT (Motor-free Visual Perception Test), and VMI (Visual-Motor Integration). All can be scored as percentile rank from 0 to 100. Higher values mean better visual perception ability. | Baseline - 1 month - 3 months - 6 months - 12 months | 
| Changes in Muscle Strength | Summation of MMT (manual muscle testing): summated scores of the manual muscle strength test (zero=0, trace=1, poor=2, fair=3, good=4, normal=5) for flexors, extensors, abductors, and adductors of bilateral shoulder and hip joints; flexors and extensors of bilateral elbow, wrist, and knee; dorsiflexors and plantar flexors of the ankles (range: 0   160). Higher scores mean better muscle strength. | Baseline - 1 month - 3 months - 6 months - 12 months | 
| Changes in Functional Performance in Daily Activities | Pediatric Evaluation of Disability Inventory (PEDI) for assessing functional performance in daily activities in children (All values are adjusted and higher value means better functional performance, 0 - worst, 100 - best.) | Baseline - 1 month - 3 months - 6 months - 12 months | 
| Changes in Language Evaluation | Sequenced Language Scale for Infant (SELSI), Preschool Receptive-Expressive Language Scale (PRES) or Korean Western Aphasia Battery (K-WAB) | Baseline - 6 months - 12 months | 
| Changes in Brain glucose metabolism | FDG-PET | Baseline - 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Global developmental delay, Umbilical cord blood, Rehabilitation