Erythropoietin Therapy for Children With Cerebral Palsy

Study Overview
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Brief Summary
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This randomized placebo-controlled trial aims to investigate the efficacy and safety of erythropoetin for children with cerebral palsy.

Detailed Description
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Cerebral palsy is a disorder of movement and posture resulted from a nonprogressive lesion or injury of the immature brain. It is a leading cause of childhood onset disability. Many experimental animal studies have revealed that erythropoietin is useful to repair neurological injury in brain. The main mechanism of erythropoietin is supposed as follows; Neuroprotection effect Angiogenesis Anti-inflammation. On the basis of many experimental studies, erythropoietin is suggested as a therapeutic method for cerebral palsy.

Official Title
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Random Control Trial to Evaluate the Safety and Efficacy of Erythropoietin Therapy for Children With Cerebral Palsy -Patient, Principle Investigator, Observer Blind

Conditions
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Cerebral Palsy

Intervention / Treatment
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* Drug: Erythropoietin
* Drug: Placebo erythropoietin


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Cerebral Palsy Abnormal Muscle Tone Abnormal Brain MRI Willing to Comply with All Study Procedure Exclusion Criteria: Known Genetic Disorder Other Etiologies Contributing Developmental Delay Coagulopathy Initial high Erythropoietin level in Serum Previous Erythropoietin Treatment before 3 months Presence of Drug Hypersensitivity Related to the Study Remedy Intractable Seizure Disorder Poor Cooperation of Guardian including Inactive Attitude for Rehabilitation

Ages Eligible for Study
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Minimum Age: 4 Months
Maximum Age: 4 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Erythropoietin and Rehabilitation<br>recombinant human erythropoietin injection and active rehabilitation | Drug: Erythropoietin<br>* twice a week for 1 month<br>* Other names: Espogen;|
| Placebo Comparator: Placebo and Rehabilitation<br>Placebo erythropoietin and rehabilitation | Drug: Placebo erythropoietin<br>* twice a week for 1 month<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in Quality of Movement | 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. The interrater reliability of GMPM subscores and total scores was 0.758-0.886 (subject n=75, tester n=10). | Baseline - 2 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in Gross Motor Function | GMFM (Gross Motor Function Measure) as a standardized measurement tool for assessing Gross Motor Function consisting of 6 sub-scales; lying & rolling, sitting, crawling & kneeling, standing, walking, running & jumping. The measured interrater reliability of GMFM subscores and total scores was 0.974 - 0.997 (subject n=101, tester n=10) and intrarater reliability of GMFM subscores and total scores between one most experienced rater and another newly t rained rater was 0.994 - 1.000 (subject n=101, tester n=2). | Baseline - 2 months | 
| Changes in Neurodevelopmental Outcomes | K-BSID-II (Korean version of Bayley Scale of Infant Development-II) Motor and Mental Scales. The measured intrarater and interrater reliability of K-BSID-II motor and mental scales was 0.92 - 0.99 (subject n=55, tester n=10). | Baseline - 2 months | 
| Changes in Motor Development | AIMS (Alberta Infant Motor Scale)to measure the motor development | Baseline - 2 months | 
| Changes in Spasticity | MAS (modified Ashworth Scale)measured at biceps, hip adductor, hamstring, heel cord | Baseline - 2 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cerebral Palsy, Erythropoietin, Rehabilitation