UCB Therapy in Acquired Brain Injury

Study Overview
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Brief Summary
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This open label trial is conducted to investigate the efficacy and safety of allogeneic umbilical cord blood (UCB) therapy for patients with acquired brain injury.

Detailed Description
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Acquired brain injury (ABI) means brain damage caused by events after birth, rather than as part of a genetic or congenital disorders. Those with ABI suffer from cognitive, physical, or behavioral impairments limiting their activity and participation in society. ABI results from traumatic or non-traumatic brain injury due to internal or external source (e.g. infection, brain tumor, hypoxia). Preclinical studies regarding cell therapy in animal models of ABI showed improvements of neurological dysfunction. UCB possess various stem or progenitor cells and is known to secrete neurotrophic factors to repair injured brain. This clinical research aims to determine the safety and efficacy of allogeneic UCB for patients with ABI.

Official Title
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Umbilical Cord Blood Therapy for Patients With Acquired Brain Injury

Conditions
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Acquired Brain Injury

Intervention / Treatment
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* Procedure: Umbilical cord blood therapy


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Acquired brain injury Duration: over 12 months Willing to comply with all study procedure Exclusion Criteria: Medical instability including pneumonia or renal function at enrollment Uncontrolled persistent epilepsy Poor cooperation of guardian,including inactive attitude for rehabilitation and visits for follow-up Not eligible according to the principal investigator

Ages Eligible for Study
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Minimum Age: 5 Years
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: UCB therapy for TBI patient
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Umbilical cord blood therapy<br>Umbilical cord blood therapy | Procedure: Umbilical cord blood therapy<br>* Intravascular umbilical cord blood therapy<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Monitoring adverse events |  | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 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 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 | 
| 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 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 brain glucose metabolism | FDG (fludeoxyglucose)-PET (positron emission tomography) | Baseline - 12 months | 
| Changes in brain white matter integrity | diffusion tensor imaging | Baseline - 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Umbilical cord blood, Acquired brain injury