Umbilical Cord Blood Therapy for Children With Global Developmental Delay

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
MinYoung Kim, M.D., Bundang CHA Hospital
ClinicalTrials.gov Identifier:
NCT01601158
First received: May 16, 2012
Last updated: January 8, 2014
Last verified: January 2014
  Purpose

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


Condition Intervention
Global Developmental Delay
Biological: Umbilical Cord Blood administration
Other: Active Rehabilitation

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Safety and Efficacy of Allogeneic Umbilical Cord Blood Therapy for Children With Global Developmental Delay

Resource links provided by NLM:


Further study details as provided by Bundang CHA Hospital:

Primary Outcome Measures:
  • Changes in Cognition [ Time Frame: Baseline - 6 months - 12 months ] [ Designated as safety issue: No ]
    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.

  • Changes in Cognitive Neurodevelopmental Outcome [ Time Frame: Baseline - 1 month - 3 months - 6 months - 12 months ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • Changes in Motor Performance [ Time Frame: Baseline - 1 month - 3 months - 6 months - 12 months ] [ Designated as safety issue: No ]
    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.

  • Changes in Standardized Gross Motor Function [ Time Frame: Baseline - 1 month - 3 months - 6 months - 12 months ] [ Designated as safety issue: No ]
    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.

  • Changes in Motor Neurodevelopmental Outcome [ Time Frame: Baseline - 1 month - 3 months - 6 months - 12 months ] [ Designated as safety issue: No ]
    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.

  • Changes in Functional Independence in Daily Activities [ Time Frame: Baseline - 1 month - 3 months - 6 months - 12 months ] [ Designated as safety issue: No ]
    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.

  • Changes in Visual Perception Test [ Time Frame: Baseline - 1 month - 3 months - 6 months - 12 months ] [ Designated as safety issue: No ]
    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

  • Changes in Muscle Strength [ Time Frame: Baseline - 1 month - 3 months - 6 months - 12 months ] [ Designated as safety issue: No ]
    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.

  • Changes in Functional Performance in Daily Activities [ Time Frame: Baseline - 1 month - 3 months - 6 months - 12 months ] [ Designated as safety issue: No ]
    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.

  • Changes in Language Evaluation [ Time Frame: Baseline - 6 months - 12 months ] [ Designated as safety issue: No ]
    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.


Enrollment: 10
Study Start Date: May 2012
Study Completion Date: August 2013
Primary Completion Date: August 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Umbilical Cord Blood and Rehabilitation
Allogeneic Umbilical Cord Blood infusion and Active Rehabilitation
Biological: Umbilical Cord Blood administration
The subjects will be undertaken allogeneic umbilical cord blood infusion intravenously or intraarterially under non-myeloablative immunosuppression.
Other Name: Donated Umbilical Cord Blood Units from Affiliated Cord Blood Bank
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.

Detailed 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.

  Eligibility

Ages Eligible for Study:   6 Months to 15 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01601158

Locations
Korea, Republic of
CHA Bundang Medical Center, CHA University
Seongnam-si, Gyeonggi-do, Korea, Republic of, 463-712
Sponsors and Collaborators
MinYoung Kim, M.D.
Investigators
Principal Investigator: Minyoung Kim, M.D., Ph.D. CHA University
  More Information

No publications provided

Responsible Party: MinYoung Kim, M.D., Associate Professor, Bundang CHA Hospital
ClinicalTrials.gov Identifier: NCT01601158     History of Changes
Other Study ID Numbers: GDDUCB
Study First Received: May 16, 2012
Last Updated: January 8, 2014
Health Authority: Korea: Institutional Review Board

Keywords provided by Bundang CHA Hospital:
Global developmental delay
Umbilical cord blood
Rehabilitation

ClinicalTrials.gov processed this record on July 29, 2014