Umbilical Cord Blood Therapy for Children With Global Developmental Delay

Global Developmental Delay Clinical Trial

Official title:

Safety and Efficacy of Allogeneic Umbilical Cord Blood Therapy for Children With Global Developmental Delay

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01601158
• Other study ID #: GDDUCB
• Status: Completed
• Phase: N/A
• First received: May 16, 2012
• Last updated: January 8, 2014
• Start date: May 2012
• Est. completion date: August 2013

Study information

• Verified date: January 2014
• Source: Bundang CHA Hospital
• Contact: n/a
• Is FDA regulated: No
• Health authority: Korea: Institutional Review Board
• Study type: Interventional

Clinical Trial Summary

This open-label study aims to evaluate the safety and efficacy of umbilical cord blood therapy for children with global developmental delay.

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: August 2013
• Est. primary completion date: August 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Months to 15 Years

Eligibility

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

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Global Developmental Delay

Intervention

Biological:
• Umbilical Cord Blood administration
The subjects will be undertaken allogeneic umbilical cord blood infusion intravenously or intraarterially under non-myeloablative immunosuppression.

Other:
• Active Rehabilitation
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.

Locations

Country	Name	City	State
Korea, Republic of	CHA Bundang Medical Center, CHA University	Seongnam-si	Gyeonggi-do

Sponsors (1)

Lead Sponsor	Collaborator
MinYoung Kim, M.D.

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No
Primary	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	No
Secondary	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	No
Secondary	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	No
Secondary	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	No
Secondary	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	No
Secondary	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	No
Secondary	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	No
Secondary	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	No
Secondary	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	No