Umbilical Cord Blood Therapy for Children With Cerebral Palsy

• ClinicalTrials.gov Identifier: NCT01639404
• Recruitment Status: Completed
• First Posted: July 12, 2012
• Last Update Posted: August 12, 2013
• Sponsor: MinYoung Kim, M.D.
• Information provided by (Responsible Party): MinYoung Kim, M.D., Bundang CHA Hospital

Tracking Information

• First Submitted Date: July 10, 2012
• First Posted Date: July 12, 2012
• Last Update Posted Date: August 12, 2013
• Study Start Date: July 2012
• Actual Primary Completion Date: March 2013 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: July 10, 2012)

• Changes in Motor Performance [ Time Frame: Baseline - 1 month - 3 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). GMPM scores at each assessment time points will be reported.
• Changes in Standardized Gross Motor Function [ Time Frame: Baseline - 1 month - 3 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). GMFM scores at each assessment time points will be reported.

• Original Primary Outcome Measures: Same as current
• Change History: Complete list of historical versions of study NCT01639404 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures (submitted: July 10, 2012)

• Changes in Cognitive Neurodevelopmental Outcome [ Time Frame: Baseline - 1 month - 3 months ]
  Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Mental Scales (higher value means better mental function: 0 - worst, 178 - best). K-BSID-II Mental Scale raw scores at each assessment time points will be reported.
• Changes in Motor Neurodevelopmental Outcome [ Time Frame: Baseline - 1 month - 3 months ]
  Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Motor Scales (higher value means better motor function: 0 - worst, 112 - best). K-BSID-II Motor Scale raw scores at each assessment time points will be reported.
• Changes in Functional Independence in Daily Activities [ Time Frame: Baseline - 1 month - 3 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. Total WeeFIM scores measured at each assessment time points will be reported.
• Changes in Visual Perception Test [ Time Frame: Baseline - 1 month - 3 months ]
  Visual perception function will be evaluated 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 ]
  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 ]
  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.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Umbilical Cord Blood Therapy for Children With Cerebral Palsy
• Official Title: Allogeneic Umbilical Cord Blood Therapy for Children With Cerebral Palsy
• Brief Summary: This study aims to evaluate the efficacy of umbilical cord blood therapy for children with cerebral palsy.

Detailed Description

Cerebral palsy is a disorder of movement and posture resulted from a non-progressive lesion or injury of the immature brain. It is a leading cause of childhood onset disability.

Many experimental animal studies have revealed that umbilical cord blood is useful to repair neurological injury in brain.

On the basis of many experimental studies, umbilical cord blood is suggested as a potential therapy for cerebral palsy.

• Study Type: Interventional
• Study Phase: Not Applicable
• Study Design: Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Cerebral Palsy

Intervention

• Procedure: 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.

Study Arms

Experimental: Umbilical Cord Blood and Rehabilitation

Allogeneic Umbilical Cord Blood Administration and Active Rehabilitation

Interventions:

• Procedure: Umbilical Cord Blood Administration
• Other: Active Rehabilitation

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: August 8, 2013): 17
• Original Estimated Enrollment (submitted: July 10, 2012): 18
• Actual Study Completion Date: March 2013
• Actual Primary Completion Date: March 2013 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Cerebral Palsy with abnormal muscle tone
• Gross Motor Function Classification System (GMFCS): I, II, III, IV, V
• Willing to comply with all study procedure

Exclusion Criteria:

• Medical illnesses including pneumonia or renal function at enrollment
• Presence of known genetic disease
• Presence of drug hypersensitivity which is related to this study remedy
• Poor cooperation of guardian,including inactive attitude for rehabilitation and visits for follow-up
• Decision by the principal investigator when there are unexpected events that may affect the outcomes

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 6 Months to 20 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Korea, Republic of

Administrative Information

• NCT Number: NCT01639404
• Other Study ID Numbers: CPUCB
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: MinYoung Kim, M.D., Bundang CHA Hospital
• Study Sponsor: MinYoung Kim, M.D.
• Collaborators: Not Provided

Investigators

• Principal Investigator: MinYoung Kim, M.D., Ph.D.; CHA University

• PRS Account: Bundang CHA Hospital
• Verification Date: August 2013