Umbilical Cord Blood Therapy for Global Developmental Delay

Umbilical Cord Blood Therapy for Children With Global Developmental Delay

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.

Study Overview

• Status: Completed
• Conditions: Global Developmental Delay
• Intervention / Treatment: Procedure: Umbilical cord blood transplantation

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 Type: Interventional
• Enrollment (Actual): 14
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Korea, Republic of
  • Gyeonggi-do
    • Seongnam-si, Gyeonggi-do, Korea, Republic of, 463-712
      • Cha Bundang Medical Center, Cha University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 15 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: 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.	Procedure: Umbilical cord blood transplantation; Other Names: Autologous or Allogeneic Umbilical Cord Blood

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: MinYoung Kim, M.D.
• Collaborators: CHA University

Study record dates

Study Major Dates

• Study Start: December 1, 2012
• Primary Completion (ACTUAL): December 1, 2017
• Study Completion (ACTUAL): December 1, 2017

Study Registration Dates

• First Submitted: January 15, 2013
• First Submitted That Met QC Criteria: January 16, 2013
• First Posted (ESTIMATE): January 17, 2013

Study Record Updates

• Last Update Posted (ACTUAL): January 23, 2018
• Last Update Submitted That Met QC Criteria: January 22, 2018
• Last Verified: January 1, 2018

More Information

Terms related to this study

• Keywords: Rehabilitation; Umbilical cord blood; Global developmental delay
• Other Study ID Numbers: UCBGDD