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Allogeneic Umbilical Cord Blood Therapy in Children With CP

Primary Purpose

Cerebral Palsy

Status
Completed
Phase
Not Applicable
Locations
Korea, Republic of
Study Type
Interventional
Intervention
Allogeneic umbilical cord blood therapy
Sponsored by
MinYoung Kim, M.D.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cerebral Palsy focused on measuring Cerebral palsy, Umbilical cord blood, Rehabilitation, Cytokine, Outcome

Eligibility Criteria

undefined - 15 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Diagnosed with cerebral palsy
  • Age of ≤15 years
  • Mismatch in HLA-A, B, and DR ≤2, and total nucleated cell count ≥3x10^7/kg. If the cell count is less than given values, more than 1 unit could be used.
  • Decision of participation in the study by and acquisition of informed consent from the subject's representative
  • Willingness and ability to be hospitalized according to the schedule specified in the protocol and continue the study for 12 months after study entry

Exclusion Criteria:

  • Current aspiration pneumonia
  • Known genetic disease
  • History of hypersensitivity reaction to any study drugs pertinent to the study
  • Patient with severe seizure disease who has clinical convulsion despite combination therapy with 3 or more agents
  • Uncontrolled hypertension defined as systolic blood pressure >115 mmHg and/or diastolic blood pressure >70 mmHg
  • Hepatic impairment defined as asparate aminotransferase (AST) >55 IU/L and/or alanine aminotransferase (ALT) >45 IU/L
  • Renal impairment defined as creatinine (Cr) ≥1.2 mg/dL
  • Presence of diagnosed or suspected malignant tumor and/or hematologic malignancy
  • Non-compliance with study visits specified in the protocol or unwillingness of care-giver due to lack of understanding of the patient

Sites / Locations

  • CHA Bundang Medical Center, CHA University

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Allogeneic umbilical cord blood therapy

Arm Description

Allogeneic umbilical cord blood therapy

Outcomes

Primary Outcome Measures

Cytokine analysis
Cytokine analysis
Changes in Standardized Gross Motor Function
GMFM (Gross Motor Function Measure) is 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 Performance
GMPM (Gross Motor Performance Measure) is 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 Cognitive Neurodevelopmental Outcome
Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Mental Scale (range: 0~178; worst: 0, best: 178)
Changes in Motor Neurodevelopmental Outcome
Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Motor Scale (range: 0~112; worst: 0, best: 112)

Secondary Outcome Measures

Changes in Gross Motor Function Classification System
GMFCS (Gross Motor Function Classification System) is a five-level classification system based on self-initiated movement, with emphasis on sitting, transfers, and mobility (level I: walks without limitations, ll: walks with limitations, III: walks using a hand-held mobility device, IV: self-mobility with limitations, V: transported in a manual wheelchair).
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 score means more independent performance in daily activities.
Changes in Functional Performance in Daily Activities
Pediatric Evaluation of Disability Inventory (PEDI) is used to assess functional performance in daily activities in children (All values are adjusted and higher value means better functional performance, 0 - worst, 100 - best). PEDI consists of 2 scales such as Functional Skill Scale (FSS) and a Caregiver Assistance Scale (CAS) and each scale is composed of 3 domains including self care, mobility, and social function.
Changes in Upper Extremity Function
QUEST (Quality of Upper Extremity Skills Test) is a standardized measurement tool for assessing upper extremity function consisting of sub-scales; dissociated movement, grasps, weight bearing, and protective extension. QUEST ranges from 0 (or below 0 in grasp section) to 100 and higher values mean better upper extremity function.
Changes in Visual Perception Test
Visual perception function will be assessed with one of 3 tools such as DTVP (Developmental Test of Visual Perception), MVPT (Motor-free Visual Perception Test), and VMI (Visual-Motor Integration, Visual Perception and Motor Coordination). Higher value means better visual perception ability.
Changes in Selective Movement of Lower Extremity
SCALE (Selective Control Assessment of Lower Extremity) is a measurement tool of selective movement of hip, knee, ankle, subtalar joint and toes. Selective voluntary motor control is graded at each joint as normal (2 points), impaired (1 point) or unable (0 point).
Changes in Spasticity
Muscle spasticity of biceps, hip adductors, hamstrings and heel cords is graded according to modified Ashworth scale (MAS).
Changes in Dynamic Component of Spasticity
Dynamic component of spasticity in bilateral hamstrings is graded using modified Tardieu scale (MTS).
Changes in Muscle Strength
Muscle strength is measured using summated scores of manual muscle 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 score means stronger muscle power.
Changes in Brain MRI
Diffusion Tensor Image (DTI) of brain MRI (magnetic resonance imaging) provides quantitative information about the microscopic integrity of white matter. White matter normally possesses a high degree of diffusion anisotropy than gray matter. Fractional anisotropy (FA) will be measured and it ranges from 0 to 1. Higher FA value means more integrity of white matter.
Changes in Brain 18F-FDG PET
18F-FDG PET (Positron emission tomography with fluorine-18-fluorodeoxyglucose) imaging will be performed twice prior to and 12 months after UCB therapy.
Changes in EEG
Electroencephalography (EEG) will be performed twice prior to and 12 months after UCB therapy.
Changes in EP
Median, tibial somatosensory evoked potential (SEP), visual evoked potential (VEP), auditory evoked potential (AEP) will be performed twice prior to and 12 months after UCB therapy.
Number of adverse events and participants with those adverse events
The numbers of adverse events and subjects with those serious adverse events within each group; A serious adverse event is any untoward medical occurrence that at any dose: results in death or is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, or causes a congenital anomaly/birth defect.

Full Information

First Posted
December 29, 2013
Last Updated
October 10, 2017
Sponsor
MinYoung Kim, M.D.
Collaborators
CHA University
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1. Study Identification

Unique Protocol Identification Number
NCT02025972
Brief Title
Allogeneic Umbilical Cord Blood Therapy in Children With CP
Official Title
Changes in Cytokines and Functional Outcomes of Allogeneic Cord Blood Therapy in Children With Cerebral Palsy
Study Type
Interventional

2. Study Status

Record Verification Date
October 2017
Overall Recruitment Status
Completed
Study Start Date
December 2013 (undefined)
Primary Completion Date
November 15, 2015 (Actual)
Study Completion Date
November 15, 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
MinYoung Kim, M.D.
Collaborators
CHA University

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
This open-label study aims to analyze cytokines related to clinical outcomes of allogeneic umbilical cord blood therapy for children with cerebral palsy.
Detailed Description
Cerebral palsy (CP) is a group of neurodevelopmental conditions with abnormal movement and posture resulted from a non-progressive cerebral disturbance. It is the most common cause of motor disability in childhood. Most therapies are palliative rather than restorative. Umbilical cord blood (UCB) may be used as restorative approach for children with CP. Many experimental animal studies have revealed that UCB is beneficial to improve and repair neurological injuries. Based on animal studies and some clinical trials, UCB is suggested as a potential therapy for children with CP. This study was designed to find cytokines relevant to UCB therapy.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cerebral Palsy
Keywords
Cerebral palsy, Umbilical cord blood, Rehabilitation, Cytokine, Outcome

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Allogeneic umbilical cord blood therapy
Arm Type
Experimental
Arm Description
Allogeneic umbilical cord blood therapy
Intervention Type
Procedure
Intervention Name(s)
Allogeneic umbilical cord blood therapy
Primary Outcome Measure Information:
Title
Cytokine analysis
Description
Cytokine analysis
Time Frame
12 months
Title
Changes in Standardized Gross Motor Function
Description
GMFM (Gross Motor Function Measure) is 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).
Time Frame
Baseline - 3 months - 6 months - 12months
Title
Changes in Motor Performance
Description
GMPM (Gross Motor Performance Measure) is 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).
Time Frame
Baseline - 3 months - 6 months - 12 months
Title
Changes in Cognitive Neurodevelopmental Outcome
Description
Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Mental Scale (range: 0~178; worst: 0, best: 178)
Time Frame
Baseline - 3 months - 6 months - 12 months
Title
Changes in Motor Neurodevelopmental Outcome
Description
Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Motor Scale (range: 0~112; worst: 0, best: 112)
Time Frame
Baseline - 3 months - 6 months - 12 months
Secondary Outcome Measure Information:
Title
Changes in Gross Motor Function Classification System
Description
GMFCS (Gross Motor Function Classification System) is a five-level classification system based on self-initiated movement, with emphasis on sitting, transfers, and mobility (level I: walks without limitations, ll: walks with limitations, III: walks using a hand-held mobility device, IV: self-mobility with limitations, V: transported in a manual wheelchair).
Time Frame
Baseline - 3 months - 6 months - 12 months
Title
Changes in Functional Independence in Daily Activities
Description
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 score means more independent performance in daily activities.
Time Frame
Baseline - 3 months - 6 months - 12 months
Title
Changes in Functional Performance in Daily Activities
Description
Pediatric Evaluation of Disability Inventory (PEDI) is used to assess functional performance in daily activities in children (All values are adjusted and higher value means better functional performance, 0 - worst, 100 - best). PEDI consists of 2 scales such as Functional Skill Scale (FSS) and a Caregiver Assistance Scale (CAS) and each scale is composed of 3 domains including self care, mobility, and social function.
Time Frame
Baseline - 3 months - 6 months - 12 months
Title
Changes in Upper Extremity Function
Description
QUEST (Quality of Upper Extremity Skills Test) is a standardized measurement tool for assessing upper extremity function consisting of sub-scales; dissociated movement, grasps, weight bearing, and protective extension. QUEST ranges from 0 (or below 0 in grasp section) to 100 and higher values mean better upper extremity function.
Time Frame
Baseline - 3 months - 6 months - 12 months
Title
Changes in Visual Perception Test
Description
Visual perception function will be assessed with one of 3 tools such as DTVP (Developmental Test of Visual Perception), MVPT (Motor-free Visual Perception Test), and VMI (Visual-Motor Integration, Visual Perception and Motor Coordination). Higher value means better visual perception ability.
Time Frame
Baseline - 3 months - 6 months - 12 months
Title
Changes in Selective Movement of Lower Extremity
Description
SCALE (Selective Control Assessment of Lower Extremity) is a measurement tool of selective movement of hip, knee, ankle, subtalar joint and toes. Selective voluntary motor control is graded at each joint as normal (2 points), impaired (1 point) or unable (0 point).
Time Frame
Baseline - 3 months - 6 months - 12 months
Title
Changes in Spasticity
Description
Muscle spasticity of biceps, hip adductors, hamstrings and heel cords is graded according to modified Ashworth scale (MAS).
Time Frame
Baseline - 3 months - 6 months - 12 months
Title
Changes in Dynamic Component of Spasticity
Description
Dynamic component of spasticity in bilateral hamstrings is graded using modified Tardieu scale (MTS).
Time Frame
Baseline - 3 months - 6 months - 12 months
Title
Changes in Muscle Strength
Description
Muscle strength is measured using summated scores of manual muscle 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 score means stronger muscle power.
Time Frame
Baseline - 3 months - 6 months - 12 months
Title
Changes in Brain MRI
Description
Diffusion Tensor Image (DTI) of brain MRI (magnetic resonance imaging) provides quantitative information about the microscopic integrity of white matter. White matter normally possesses a high degree of diffusion anisotropy than gray matter. Fractional anisotropy (FA) will be measured and it ranges from 0 to 1. Higher FA value means more integrity of white matter.
Time Frame
Baseline - 12 months
Title
Changes in Brain 18F-FDG PET
Description
18F-FDG PET (Positron emission tomography with fluorine-18-fluorodeoxyglucose) imaging will be performed twice prior to and 12 months after UCB therapy.
Time Frame
Baseline - 12 months
Title
Changes in EEG
Description
Electroencephalography (EEG) will be performed twice prior to and 12 months after UCB therapy.
Time Frame
Baseline - 12 months
Title
Changes in EP
Description
Median, tibial somatosensory evoked potential (SEP), visual evoked potential (VEP), auditory evoked potential (AEP) will be performed twice prior to and 12 months after UCB therapy.
Time Frame
Baseline - 12 months
Title
Number of adverse events and participants with those adverse events
Description
The numbers of adverse events and subjects with those serious adverse events within each group; A serious adverse event is any untoward medical occurrence that at any dose: results in death or is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, or causes a congenital anomaly/birth defect.
Time Frame
12 months

10. Eligibility

Sex
All
Maximum Age & Unit of Time
15 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Diagnosed with cerebral palsy Age of ≤15 years Mismatch in HLA-A, B, and DR ≤2, and total nucleated cell count ≥3x10^7/kg. If the cell count is less than given values, more than 1 unit could be used. Decision of participation in the study by and acquisition of informed consent from the subject's representative Willingness and ability to be hospitalized according to the schedule specified in the protocol and continue the study for 12 months after study entry Exclusion Criteria: Current aspiration pneumonia Known genetic disease History of hypersensitivity reaction to any study drugs pertinent to the study Patient with severe seizure disease who has clinical convulsion despite combination therapy with 3 or more agents Uncontrolled hypertension defined as systolic blood pressure >115 mmHg and/or diastolic blood pressure >70 mmHg Hepatic impairment defined as asparate aminotransferase (AST) >55 IU/L and/or alanine aminotransferase (ALT) >45 IU/L Renal impairment defined as creatinine (Cr) ≥1.2 mg/dL Presence of diagnosed or suspected malignant tumor and/or hematologic malignancy Non-compliance with study visits specified in the protocol or unwillingness of care-giver due to lack of understanding of the patient
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
MinYoung Kim, M.D., Ph.D.
Organizational Affiliation
CHA University
Official's Role
Principal Investigator
Facility Information:
Facility Name
CHA Bundang Medical Center, CHA University
City
Seongnam-si
State/Province
Gyeonggi-do
ZIP/Postal Code
463-712
Country
Korea, Republic of

12. IPD Sharing Statement

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Allogeneic Umbilical Cord Blood Therapy in Children With CP

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