Umbilical Cord Blood Therapy for Global Developmental Delay

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2014 by Bundang CHA Hospital
Sponsor:
Collaborator:
CHA University
Information provided by (Responsible Party):
MinYoung Kim, M.D., Bundang CHA Hospital
ClinicalTrials.gov Identifier:
NCT01769716
First received: January 15, 2013
Last updated: March 31, 2014
Last verified: March 2014
  Purpose

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.


Condition Intervention Phase
Global Developmental Delay
Procedure: Umbilical Cord Blood administration
Phase 1

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

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 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 [ 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 Mental Scales (Higher value means better cognitive function: raw score 0 - worst, 178 - best.)


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.)

  • 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.)

  • 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 Motor Scales (Higher value means better motor function: raw score 0 - worst, 112 - best)

  • 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.

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

  • 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 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 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.)

  • 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)

  • Changes in Brain glucose metabolism [ Time Frame: Baseline - 12 months ] [ Designated as safety issue: No ]
    FDG-PET


Estimated Enrollment: 12
Study Start Date: December 2012
Estimated Study Completion Date: December 2014
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Umbilical cord blood therapy
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 administration
Other Name: Autologous or Allogeneic Umbilical Cord Blood

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.

  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: NCT01769716

Contacts
Contact: MinYoung Kim, M.D., Ph.D. 82-31-780-6281 kmin@cha.ac.kr
Contact: Kyunghoon Min, M.D. 82-31-780-6281 minkh@chamc.co.kr

Locations
Korea, Republic of
CHA Bundang Medical Center, CHA University Recruiting
Seongnam-si, Gyeonggi-do, Korea, Republic of, 463-712
Contact: MinYoung Kim, M.D., Ph.D.    82-31-780-6281    kmin@cha.ac.kr   
Contact: Kyunghoon Min, M.D.    82-31-780-6281    minkh@chamc.co.kr   
Principal Investigator: MinYoung Kim, M.D., Ph.D.         
Sub-Investigator: Myung Seo Kang, M.D., Ph.D.         
Sub-Investigator: Sang Heum Kim, M.D.         
Sub-Investigator: Su Jin Jang, M.D.         
Sub-Investigator: Kyunghoon Min, M.D.         
Sub-Investigator: Su Jin Yoo, M.D.         
Sponsors and Collaborators
MinYoung Kim, M.D.
CHA University
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: NCT01769716     History of Changes
Other Study ID Numbers: UCBGDD
Study First Received: January 15, 2013
Last Updated: March 31, 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 24, 2014