Umbilical Cord Blood Therapy for Children With 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 umbilical cord blood therapy for children with global developmental delay.

Detailed Description
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Global developmental delay (GDD) is a subset of developmental disabilities 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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Safety and Efficacy of Allogeneic Umbilical Cord Blood Therapy for Children With Global Developmental Delay

Conditions
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Global Developmental Delay

Intervention / Treatment
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* Biological: Umbilical Cord Blood administration
* Other: Active Rehabilitation


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
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Umbilical Cord Blood and Rehabilitation<br>Allogeneic Umbilical Cord Blood infusion and Active Rehabilitation | Biological: Umbilical Cord Blood administration<br>* The subjects will be undertaken allogeneic umbilical cord blood infusion intravenously or intraarterially under non-myeloablative immunosuppression.<br>* Other names: Donated Umbilical Cord Blood Units from Affiliated Cord Blood Bank;Other: Active Rehabilitation<br>* All subjects should participate in active rehabilitation. They will receive two physical and occupational therapy sessions per day. Post discharge, each participant should continue to receive rehabilitation therapy at least 3 days per week until the study completion.<br>|

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 Primay Scale of Intelligence (K-WPPSI) (Higher value means better cognitive function (K-WPPSI: below 70 - worst, over 130 - best). The investigators will report K-WPPSI at each assessment time points. | Baseline - 6 months - 12 months | 
| Changes in Cognitive Neurodevelopmental Outcome | Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Mental Scales (Higher value means better cognitive function, K-BSID-II mental scale raw score: 0 - worst, 178 - best). The investigators will report K-BSID-II Mental Scale raw scores at each assessment time points. | 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). The investigators will report GMPM scores at each assessment time points. | 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). The investigators will report GMFM scores at each assessment time points. | Baseline - 1 month - 3 months - 6 months - 12 months | 
| Changes in Motor Neurodevelopmental Outcome | Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Motor Scales (higher value means better motor function: 0 - worst, 111 - best). The investigators will report K-BSID-II Motor Scale raw scores at each assessment time points. | 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. The investigators will report total WeeFIM scores measured at each assessment time points. | Baseline - 1 month - 3 months - 6 months - 12 months | 
| Changes in Visual Perception Test | We will evaluate visual perception function with one of three measures: DTVP (Developmental Test of Visual Perception), MVPT (Motor-free Visual Perception Test), and VMI (Visual-Motor Integration, Visual Perception and Motor Coordination). 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 strength test score): 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. Categories of outcome table will be summation of MMT scores measured at each assessment time point. | 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). We will report 2 scales and 3 domains of each scale: a Functional Skill Scale (FSS) and a Caregiver Assistance Scale (CAS) which are divided respectively into 3 domains: self care, mobility, and social function. Categories of outcome table will be each domain scores measured at each assessment time point. | 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) will be used depending on age and function. | Baseline - 6 months - 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