Umbilical Cord Blood Therapy for Global Developmental Delay

Not Applicable

Completed

• Conditions: Global Developmental Delay
• Interventions: Procedure: Umbilical cord blood transplantation

• Registration Number: NCT01769716
• Lead Sponsor: MinYoung Kim, M.D.

Brief Summary

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

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.

Study Timeline

• Study Start: 2012-12
• Study First Submitted: 2013-01-15
• Study First Submitted QC: 2013-01-16
• Study First Posted: 2013-01-17
• Primary Completion: 2017-12
• Study Completion: 2017-12
• Status Verified: 2018-01
• Last Update Submitted: 2018-01-22
• Last Update Posted: 2018-01-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 14

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Umbilical cord blood transplantation	Umbilical cord blood transplantation	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.

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Changes in Standardized Gross Motor Function	Baseline - 1 month - 3 months - 6 months - 12 months	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.)
Changes in Motor Performance	Baseline - 1 month - 3 months - 6 months - 12 months	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.)
Changes in Functional Performance in Daily Activities	Baseline - 1 month - 3 months - 6 months - 12 months	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.)
Changes in Motor Neurodevelopmental Outcome	Baseline - 1 month - 3 months - 6 months - 12 months	Korean version of Bayley Scale of Infant Development-II Motor Scales (Higher value means better motor function: raw score 0 - worst, 112 - best)
Changes in Functional Independence in Daily Activities	Baseline - 1 month - 3 months - 6 months - 12 months	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.
Changes in Muscle Strength	Baseline - 1 month - 3 months - 6 months - 12 months	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.
Changes in Language Evaluation	Baseline - 6 months - 12 months	Sequenced Language Scale for Infant (SELSI), Preschool Receptive-Expressive Language Scale (PRES) or Korean Western Aphasia Battery (K-WAB)
Changes in Brain glucose metabolism	Baseline - 12 months	FDG-PET
Changes in Visual Perception Test	Baseline - 1 month - 3 months - 6 months - 12 months	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.

Trial Locations

Locations (1)

CHA Bundang Medical Center, CHA University; 🇰🇷 Seongnam-si, Gyeonggi-do, Korea, Republic of