Cerebral PalsyNCT01991145
Allogeneic UCB Therapy With EPO in Children With CP
A study testing Umbilical Cord Blood therapy, Erythropoietin alfa and Rehabilitation in people with Cerebral Palsy, run by MinYoung Kim, MD, PhD at 1 site in South Korea.
Trial summary
This randomized controlled study aims to evaluate the efficacy and safety of allogeneic umbilical cord blood therapy combined with erythropoietin for children with cerebral palsy. Read more about this study
- Who is it for?
- Ages 10 months to 6 years
- Conditions the study is for
- Cerebral Palsy
- What is being tested?
- Umbilical Cord Blood therapy, Erythropoietin alfa and Rehabilitation
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 92 participants
- Where
- 1 siteSouth Korea
- Study dates
- 26 Nov 2013to 24 Jun 2017
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosed with cerebral palsy
- Age of ≥10 months and ≤6 years
- Mismatch in HLA-A, B, and DR ≤2, and total nucleated cell count ≥3x107/kg. If the cell count is less than given values, more than 1 unit could be used.
- Hemoglobin ≤13.6 g/dL
- 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
Who cannot join
- Current aspiration pneumonia
- Known genetic disease
- History of hypersensitivity reaction to any study drugs pertinent to the study
- History of participation in any other study with stem cell
- Prior treatment with EPO within 3 months prior to study entry
- Known coagulopathy with family history of thrombosis or medical history of recurrent thrombosis
Show all 13Show fewer
- 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
- Absolute neutrophil count ≤500/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
Trial locations and contacts
1 site in 1 country.
South Korea1 site
CHA Bundang Medical Center, CHA University
Seongnam-si, Gyeonggi-do, 463-712
About this study
Official title: Safety and Efficacy of Allogeneic Umbilical Cord Blood Therapy Combined With Erythropoietin in Children With Cerebral Palsy: a Double-blind, Randomized, Placebo-controlled Clinical Trial
This randomized controlled study aims to evaluate the efficacy and safety of allogeneic umbilical cord blood therapy combined with erythropoietin for children with cerebral palsy.
Read the 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) and erythropoetin (EPO) 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. EPO is also known to have neuroprotective effects.
Based on animal studies and some clinical trials, UCB is suggested as a potential therapy for children with CP. EPO is combined to add synergistic effects to UCB therapy.
- Conditions
- Cerebral Palsy
- Being tested
- Umbilical Cord Blood therapy, Erythropoietin alfa, Rehabilitation, Placebo UCB, Placebo EPO
- Keywords
- Cerebral Palsy, Umbilical Cord Blood, Erythropoietin, Rehabilitation
- 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 therapyProcedure or surgery
HLA (Human Leukocyte Antigen) typing
Erythropoietin alfaBiological
Also called Espogen (LG Life Science Ltd.)
RehabilitationOther
Active rehabilitation
Placebo UCBProcedure or surgery
Placebo EPOBiological
| Study group | Role | What they receive |
|---|---|---|
| UCB and EPO | Experimental | UCB + EPO + Rehabilitation Receives: Procedure: Umbilical Cord Blood therapy; Biological: Erythropoietin alfa; Other: Rehabilitation |
| UCB and placebo EPO | Active comparator | UCB + placebo EPO + Rehabilitation Receives: Procedure: Umbilical Cord Blood therapy; Other: Rehabilitation; Biological: Placebo EPO |
| placebo UCB and EPO | Active comparator | placebo UCB + EPO + Rehabilitation Receives: Biological: Erythropoietin alfa; Other: Rehabilitation; Procedure: Placebo UCB |
| placebo UCB and placebo EPO | Placebo comparator | placebo UCB + placebo EPO + Rehabilitation Receives: Other: Rehabilitation; Procedure: Placebo UCB; Biological: Placebo EPO |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Baseline - 1 month - 3 months - 6 months - 12months
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).
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
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)
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
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)
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
Secondary outcomes (14)
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).
Measured: Baseline - 1 month - 3 months - 6 months - 12 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 score means more independent performance in daily activities.
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
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.
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
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.
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
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.
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
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).
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Spasticity
Muscle spasticity of biceps, hip adductors, hamstrings and heel cords is graded according to modified Ashworth scale (MAS).
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Dynamic Component of Spasticity
Dynamic component of spasticity in bilateral hamstrings is graded using modified Tardieu scale (MTS).
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
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.
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
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.
Measured: Baseline - 12 months
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.
Measured: Baseline - 12 months
Changes in EEG
Electroencephalography (EEG) will be performed twice prior to and 12 months after UCB therapy.
Measured: Baseline - 12 months
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.
Measured: Baseline - 12 months
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.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2013 (estimated)
- Study start26 Nov 2013
- Primary completion24 Jun 2017
- Study completion24 Jun 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- 92 participants (actual)
Sponsor and investigators
- Lead sponsor
- MinYoung Kim, MD, PhD
- Sponsor type
- University, hospital or other
- Collaborators
- Ministry of Health & Welfare, Korea, LG Life Sciences, Chong Kun Dang Pharmaceutical, CHA University
- Responsible party
- Sponsor-investigator, MinYoung Kim, MD, PhD, Bundang CHA Hospital
- Investigators
- MinYoung Kim, M.D., Ph.D., Principal investigator, CHA University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01991145
- Sponsor's protocol ID
- UCBnEPOinCP
- Status last verified
- Jan 2020
- First submitted
- 17 Nov 2013
- Last updated
- 18 Jan 2020
- Results posted
- No
- Data monitoring committee
- No
Publications
Min K, Suh MR, Cho KH, Park W, Kang MS, Jang SJ, Kim SH, Rhie S, Choi JI, Kim HJ, Cha KY, Kim M. Potentiation of cord blood cell therapy with erythropoietin for children with CP: a 2 x 2 factorial randomized placebo-controlled trial. Stem Cell Res Ther. 2020 Nov 27;11(1):509. doi: 10.1186/s13287-020-02020-y.
PubMed 33246489
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Allogeneic Umbilical Cord Blood Therapy Combined With Erythropoietin in Children With Cerebral Palsy: a Double-blind, Randomized, Placebo-controlled Clinical Trial
- ClinicalTrials.gov ID
- NCT01991145
- Acronym / sponsor ID
- UCBnEPOinCP
- Conditions
- Cerebral Palsy
- Keywords
- Cerebral Palsy, Umbilical Cord Blood, Erythropoietin, Rehabilitation
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 92 participants (actual)
- Lead sponsor
- MinYoung Kim, MD, PhDUniversity, hospital or other
- Collaborators
- Ministry of Health & Welfare, Korea, LG Life Sciences, Chong Kun Dang Pharmaceutical, CHA University
- Study start
- 26 Nov 2013 (actual)
- Primary completion
- 24 Jun 2017 (actual)
- Study completion
- 24 Jun 2017 (actual)
- First posted
- 25 Nov 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This randomized controlled study aims to evaluate the efficacy and safety of allogeneic umbilical cord blood therapy combined with erythropoietin 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) and erythropoetin (EPO) 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. EPO is also known to have neuroprotective effects.
Based on animal studies and some clinical trials, UCB is suggested as a potential therapy for children with CP. EPO is combined to add synergistic effects to UCB therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| UCB and EPO | Experimental | Procedure: Umbilical Cord Blood therapy, Biological: Erythropoietin alfa, Other: RehabilitationUCB + EPO + Rehabilitation |
| UCB and placebo EPO | Active comparator | Procedure: Umbilical Cord Blood therapy, Other: Rehabilitation, Biological: Placebo EPOUCB + placebo EPO + Rehabilitation |
| placebo UCB and EPO | Active comparator | Biological: Erythropoietin alfa, Other: Rehabilitation, Procedure: Placebo UCBplacebo UCB + EPO + Rehabilitation |
| placebo UCB and placebo EPO | Placebo comparator | Other: Rehabilitation, Procedure: Placebo UCB, Biological: Placebo EPOplacebo UCB + placebo EPO + Rehabilitation |
Who may qualify
- Ages
- 10 months to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Diagnosed with cerebral palsy
- Age of ≥10 months and ≤6 years
- Mismatch in HLA-A, B, and DR ≤2, and total nucleated cell count ≥3x107/kg. If the cell count is less than given values, more than 1 unit could be used.
- Hemoglobin ≤13.6 g/dL
- Decision of participation in the study by and acquisition of informed consent from the subject's representative
Key exclusion criteria (13 listed)
- Current aspiration pneumonia
- Known genetic disease
- History of hypersensitivity reaction to any study drugs pertinent to the study
- History of participation in any other study with stem cell
- Prior treatment with EPO within 3 months prior to study entry
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 therapyHLA (Human Leukocyte Antigen) typing | Procedure or surgery | UCB and EPO, UCB and placebo EPO |
| Erythropoietin alfaAlso: Espogen (LG Life Science Ltd.) | Biological | UCB and EPO, placebo UCB and EPO |
| RehabilitationActive rehabilitation | Other | UCB and EPO, UCB and placebo EPO, placebo UCB and EPO, placebo UCB and placebo EPO |
| Placebo UCB | Procedure or surgery | placebo UCB and EPO, placebo UCB and placebo EPO |
| Placebo EPO | Biological | UCB and placebo EPO, placebo UCB and placebo EPO |
Primary outcome
Changes in Standardized Gross Motor Function
Time frame: Baseline - 1 month - 3 months - 6 months - 12months
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
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
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
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
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
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Motor Scale (range: 0~112; worst: 0, best: 112)
14 secondary outcomes
Changes in Gross Motor Function Classification System
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Functional Independence in Daily Activities
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Functional Performance in Daily Activities
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Upper Extremity Function
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Visual Perception Test
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Selective Movement of Lower Extremity
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Spasticity
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Dynamic Component of Spasticity
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Muscle Strength
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Brain MRI
Time frame: Baseline - 12 months
Changes in Brain 18F-FDG PET
Time frame: Baseline - 12 months
Changes in EEG
Time frame: Baseline - 12 months
Changes in EP
Time frame: Baseline - 12 months
Number of adverse events and participants with those adverse events
Time frame: 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jan 2020.
- Derived from this study: PubMed 33246489Min K, Suh MR, Cho KH, Park W, Kang MS, Jang SJ, Kim SH, Rhie S, Choi JI, Kim HJ, Cha KY, Kim M. Potentiation of cord blood cell therapy with erythropoietin for children with CP: a 2 x 2 factorial randomized placebo-controlled trial. Stem Cell Res Ther. 2020 Nov 27;11(1):509. doi: 10.1186/s13287-020-02020-y.
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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, M.D., Ph.D. principal investigator
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 therapy, Erythropoietin alfa and Rehabilitation in people with Cerebral Palsy, run by MinYoung Kim, MD, PhD 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 6 inclusion and 13 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 18 Jan 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 Standardized Gross Motor Function (time frame: Baseline - 1 month - 3 months - 6 months - 12months). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01991145, which also lists 1 linked publication.
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 18 Jan 2020.