Cerebral PalsyNCT01193660
Allogenic Umbilical Cord Blood and Erythropoietin Combination Therapy for Cerebral Palsy
A study testing Umbilical Cord Blood Infusion, Erythropoietin Injection and Active Rehabilitation in people with Cerebral Palsy, run by Sung Kwang Medical Foundation at 1 site in South Korea.
Trial summary
This randomized control study is aimed to determine efficacy of umbilical cord blood and erythropoietin combination therapy for children with cerebral palsy. Read more about this study
- Who is it for?
- Ages 10 months to 10 years
- Conditions the study is for
- Cerebral Palsy
- What is being tested?
- Umbilical Cord Blood Infusion, Erythropoietin Injection and Active Rehabilitation
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 105 participants
- Where
- 1 siteSouth Korea
- Study dates
- May 2010to Apr 2011
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 months to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Known cerebral palsy
- Willing to comply with all study procedure
Who cannot join
- High risk of pneumonia or renal function deterioration after using of immunosuppressant
- Presence of known genetic disease
- Possibility of drug hypersensitivity which is related to this study remedy
- History of previous cell therapy
- Poor cooperation of guardian,including inactive attitude for rehabilitation
- Intractable seizure disorder
- Autism
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Double-blind Randomized Controlled Trial to Evaluate the Efficacy and Safety of a Combination Therapy of Allogenic Umbilical Cord Blood and Erythropoietin for Children With Cerebral Palsy
This randomized control study is aimed to determine efficacy of umbilical cord blood and erythropoietin combination therapy for children with cerebral palsy.
Read the detailed description
Cerebral palsy is a disorder of movement and posture that result from a nonprogressive lesion or injury of the immature brain. It is a leading cause of childhood onset disability through one's life. Umbilical cord blood(UCB) is suggested as therapeutic method for cerebral palsy which resulted from animal studies. Stem cells included in UCB is expected to exert therapeutic efficacy for functional recovery.
It is also suggested that erythropoietin is useful to repair neurological injury in brain. The main mechanism of erythropoietin is supposed to be neuroprotection and neurogenesis which would reinforce the effect of stem cell as well.
Although autologous umbilical cord would be safe, the children who have problems at birth seldom have autologous umbilical cord blood. Allogenic umbilical cord blood might be useful for these children if its effect is approved.
- Conditions
- Cerebral Palsy
- Being tested
- Umbilical Cord Blood Infusion, Erythropoietin Injection, Active Rehabilitation, Placebo Umbilical Cord Blood, Placebo Erythropoietin
- Keywords
- umbilical cord blood, stem cell, cerebral palsy
- Medical subject headings
- Cerebral Palsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Umbilical Cord Blood InfusionBiological
Also called Donated Umbilical Cord Blood Units from Affiliated Cord Blood Bank
The subjects will be undertaken allogeneic umbilical cord blood infusion (total nucleated cells > 3x10^7/kg) intravenously under non-myeloablative immunosuppression (maintaining blood level of cyclosporine as 100-200ng/mL for 1 month).
Erythropoietin InjectionDrug
Also called Brand name of Erythropoietin: Espogen (made by LG Life Science)
Erythropoietin will be given as the schedule of twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times.
Active RehabilitationOther
All subjects should participate in active rehabilitation. They received two physical and occupational therapy sessions per day. Post discharge, each participant continued to receive rehabilitation therapy at least 3 days per week for additional 5 months.
Placebo Umbilical Cord BloodOther
Also called Placebo Umbilical Cord Blood that resembles cord blood in appearance was designed, : 1.5 to 3 ml of the subject's own blood was collected and mixed with 15 to 20 ml of albumin.
Placebo Umbilical Cord Blood will be given except the Experimental arm. Placebo Umbilical Cord Blood was made using peripheral blood. Participants and Investigators maintained as blind.
Placebo ErythropoietinOther
Placebo Erythropoietin containing Normal Saline
| Study group | Role | What they receive |
|---|---|---|
| Umbilical Cord Blood & Erythropoietin & Rehabilitation | Experimental | Allogenic umbilical cord blood infusion, erythropoietin injection \& active rehabilitation Receives: Biological: Umbilical Cord Blood Infusion; Drug: Erythropoietin Injection; Other: Active Rehabilitation |
| Erythropoietin & Rehabilitation | Active comparator | Erythropoietin injection, active rehabilitation Receives: Drug: Erythropoietin Injection; Other: Active Rehabilitation; Other: Placebo Umbilical Cord Blood |
| Only Rehabilitation | Placebo comparator | Active rehabilitation Receives: Other: Active Rehabilitation; Other: Placebo Umbilical Cord Blood; Other: Placebo Erythropoietin |
What is being measured
The primary outcome is the main question the study is designed to answer.
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). We reported changes of GMPM score between each assessment time points. Categories of outcome table are baseline and values of just subtracting the latter raw scores from the former ones.
Measured: Baseline -1 month - 3 months - 6 months
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). We reported changes of GMFM between each assessment time points. Categories of outcome table are baseline and values of just subtracting the latter raw scores from the former ones.
Measured: Baseline - 1 month - 3 months - 6 months
Secondary outcomes (9)
Changes in Cognitive Neurodevelopmental Outcome
Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Mental Scales (higher value means better mental function: 0 - worst, 178 - best). We reported changes of BSID-II Mental Scale raw score between each assessment time points. Categories of outcome data are values of subtracting the latter scores from the former ones.
Measured: Baseline -1 month - 3 months - 6 months
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). We reported changes of BSID-II Motor Scale raw score between each assessment time points. Categories of outcome data are values of subtracting the latter scores from the former ones.
Measured: Baseline - 1 month - 3 months - 6 months
Changes in Brain MRI
Changes on brain Diffusion Tensor Image (DTI); DTI provides quantitative information about the microscopic integrity of white matter. White matter normally possesses a high degree of diffusion anisotropy than gray matter. We can measure fractional anisotropy (FA) value in DTI imaging and it ranges from 0 to 1. Higher FA value of a certain region of interest means the area has more integrity of white matter.
Measured: Baseline - 6 months
Comparison of Changes in Brain Glucose Metabolism Using by Brain 18F-FDG PET: Increased and Decreased Areas of Brain Glucose Metabolism
18F-FDG PET imaging was performed twice prior to and then 2 weeks post-treatment. Ninety slices of each emission image were obtained, and all scans were reviewed by a nuclear physician. Spatial pre-processing and statistical analyses were performed using SPM8 implanted in Matlab to compare differences in regional brain glucose metabolism between groups and differences between pre- and post-therapy imaging data. We reported increased areas and decreased areas of glucose metabolism in three groups. We defined that "1" refers to INCREASED areas, "-1", DECREASED areas and "0", just NO CHANGE.
Measured: Baseline - 2 weeks
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 reported here 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 are each domain scores measured at each assessment time point.
Measured: Baseline -1 month - 3 months - 6 months
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. Categories of outcome table are total WeeFIM scores measured at each assessment time point.
Measured: Baseline - 1 month - 3 months - 6 months
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 score means better muscle strength. Categories of outcome table are summation of MMT scores measured at each assessment time point.
Measured: Baseline - 1 month - 3 months - 6 months
Changes in Hand Function
QUEST (Quality of Upper Extremity Skills Test) as a standardized measurement tool for assessing hand function consisting of sub-scales; dissociated movement, grasps, weight bearing, and protective extension. These are standardized to range from zero (or below zero in grasp section) to 100 and higher values mean better hand function. We reported QUEST differences between each assessment times.
Measured: Baseline - 1 month - 3 months - 6 months
Number of Participants With Serious Adverse Events as a Measure of Safety,Which Are Related to Umbilical Cord Blood, Erythropoietin, or Immunosuppressant
The number of patients with serious adverse events within each group; Serious adverse events were defined as any event that resulted in death, was life-threatening, required hospitalization or prolonged the hospital stay, or was otherwise serious in the judgment of the investigator.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Sep 2010 (estimated)
- Study startMay 2010
- Primary completionApr 2011
- Study completionApr 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cerebral PalsyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 105 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sung Kwang Medical Foundation
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Minyoung Kim, MD, PhD, Study chair, CHA University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01193660
- Sponsor's protocol ID
- RCTUBSC
- Other IDs
- PBC09-095
- Status last verified
- Nov 2020
- First submitted
- 29 Aug 2010
- Last updated
- 24 Nov 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double-blind Randomized Controlled Trial to Evaluate the Efficacy and Safety of a Combination Therapy of Allogenic Umbilical Cord Blood and Erythropoietin for Children With Cerebral Palsy
- ClinicalTrials.gov ID
- NCT01193660
- Acronym / sponsor ID
- RCTUBSC
- Conditions
- Cerebral Palsy
- Keywords
- umbilical cord blood, stem cell, cerebral palsy
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 105 participants (actual)
- Lead sponsor
- Sung Kwang Medical FoundationUniversity, hospital or other
- Study start
- May 2010
- Primary completion
- Apr 2011 (actual)
- Study completion
- Apr 2011 (actual)
- First posted
- 2 Sep 2010 (estimated)
- Results
- Posted 1 Mar 2012
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This randomized control study is aimed to determine efficacy of umbilical cord blood and erythropoietin combination therapy for children with cerebral palsy.
Detailed description
Cerebral palsy is a disorder of movement and posture that result from a nonprogressive lesion or injury of the immature brain. It is a leading cause of childhood onset disability through one's life. Umbilical cord blood(UCB) is suggested as therapeutic method for cerebral palsy which resulted from animal studies. Stem cells included in UCB is expected to exert therapeutic efficacy for functional recovery.
It is also suggested that erythropoietin is useful to repair neurological injury in brain. The main mechanism of erythropoietin is supposed to be neuroprotection and neurogenesis which would reinforce the effect of stem cell as well.
Although autologous umbilical cord would be safe, the children who have problems at birth seldom have autologous umbilical cord blood. Allogenic umbilical cord blood might be useful for these children if its effect is approved.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Umbilical Cord Blood & Erythropoietin & Rehabilitation | Experimental | Biological: Umbilical Cord Blood Infusion, Drug: Erythropoietin Injection, Other: Active RehabilitationAllogenic umbilical cord blood infusion, erythropoietin injection \& active rehabilitation |
| Erythropoietin & Rehabilitation | Active comparator | Drug: Erythropoietin Injection, Other: Active Rehabilitation, Other: Placebo Umbilical Cord BloodErythropoietin injection, active rehabilitation |
| Only Rehabilitation | Placebo comparator | Other: Active Rehabilitation, Other: Placebo Umbilical Cord Blood, Other: Placebo ErythropoietinActive rehabilitation |
Who may qualify
- Ages
- 10 months to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Known cerebral palsy
- Willing to comply with all study procedure
Key exclusion criteria (7 listed)
- High risk of pneumonia or renal function deterioration after using of immunosuppressant
- Presence of known genetic disease
- Possibility of drug hypersensitivity which is related to this study remedy
- History of previous cell therapy
- Poor cooperation of guardian,including inactive attitude for rehabilitation
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Umbilical Cord Blood InfusionAlso: Donated Umbilical Cord Blood Units from Affiliated Cord Blood BankThe subjects will be undertaken allogeneic umbilical cord blood infusion (total nucleated cells > 3x10^7/kg) intravenously under non-myeloablative immunosuppression (maintaining blood level of cyclosporine as 100-200ng/mL for 1 month). | Biological | Umbilical Cord Blood & Erythropoietin & Rehabilitation |
| Erythropoietin InjectionAlso: Brand name of Erythropoietin: Espogen (made by LG Life Science)Erythropoietin will be given as the schedule of twice a week for 4 weeks with the dosage of 500 IU/kg for 2 times intravenously and 250 IU/kg subcutaneously for 6 times. | Drug | Erythropoietin & Rehabilitation, Umbilical Cord Blood & Erythropoietin & Rehabilitation |
| Active RehabilitationAll subjects should participate in active rehabilitation. They received two physical and occupational therapy sessions per day. Post discharge, each participant continued to receive rehabilitation therapy at least 3 days per week for additional 5 months. | Other | Erythropoietin & Rehabilitation, Only Rehabilitation, Umbilical Cord Blood & Erythropoietin & Rehabilitation |
| Placebo Umbilical Cord BloodAlso: Placebo Umbilical Cord Blood that resembles cord blood in appearance was designed, : 1.5 to 3 ml of the subject's own blood was collected and mixed with 15 to 20 ml of albumin.Placebo Umbilical Cord Blood will be given except the Experimental arm. Placebo Umbilical Cord Blood was made using peripheral blood. Participants and Investigators maintained as blind. | Other | Erythropoietin & Rehabilitation, Only Rehabilitation |
| Placebo ErythropoietinPlacebo Erythropoietin containing Normal Saline | Other | Only Rehabilitation |
Primary outcome
Changes in Motor Performance
Time frame: Baseline -1 month - 3 months - 6 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). We reported changes of GMPM score between each assessment time points. Categories of outcome table are baseline and values of just subtracting the latter raw scores from the former ones.
Changes in Standardized Gross Motor Function
Time frame: Baseline - 1 month - 3 months - 6 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). We reported changes of GMFM between each assessment time points. Categories of outcome table are baseline and values of just subtracting the latter raw scores from the former ones.
9 secondary outcomes
Changes in Cognitive Neurodevelopmental Outcome
Time frame: Baseline -1 month - 3 months - 6 months
Changes in Motor Neurodevelopmental Outcome
Time frame: Baseline - 1 month - 3 months - 6 months
Changes in Brain MRI
Time frame: Baseline - 6 months
Comparison of Changes in Brain Glucose Metabolism Using by Brain 18F-FDG PET: Increased and Decreased Areas of Brain Glucose Metabolism
Time frame: Baseline - 2 weeks
Changes in Functional Performance in Daily Activities
Time frame: Baseline -1 month - 3 months - 6 months
Changes in Functional Independence in Daily Activities
Time frame: Baseline - 1 month - 3 months - 6 months
Changes in Muscle Strength
Time frame: Baseline - 1 month - 3 months - 6 months
Changes in Hand Function
Time frame: Baseline - 1 month - 3 months - 6 months
Number of Participants With Serious Adverse Events as a Measure of Safety,Which Are Related to Umbilical Cord Blood, Erythropoietin, or Immunosuppressant
Time frame: 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Nov 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Minyoung Kim, MD, PhD study chair
CHA University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Umbilical Cord Blood Infusion, Erythropoietin Injection and Active Rehabilitation in people with Cerebral Palsy, run by Sung Kwang Medical Foundation at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 Mar 2012). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes in Motor Performance (time frame: Baseline -1 month - 3 months - 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Nov 2020.