Erythropoietin Therapy for Children With Cerebral Palsy: Phase 1

Study Overview
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Brief Summary
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This purpose of this phase 1 study is to investigate the safety and efficacy 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, and anti-inflammation. On the basis of many experimental studies, erythropoietin is suggested as a potential therapy for cerebral palsy.

Official Title
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A Phase 1 Clinical Research of Erythropoietin Therapy for Children With Cerebral Palsy: Safety and Efficacy

Conditions
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Cerebral Palsy

Intervention / Treatment
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* Drug: Erythropoietin


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Cerebral Palsy Abnormal Muscle Tone GMFCS (Gross Motor Functional Classification System): II to IV Age: 6 months   3 years Abnormal Brain MRI compatible to clinical features and non-progressive Willing to Comply with All Study Procedure Exclusion Criteria: Known Genetic Disorder Baseline Erythropoietin level > 45 mU/mL Presence of Drug Hypersensitivity Related to the Study Remedy Previous Erythropoietin Treatment before 3 months Coagulopathy: Family History, Unknown Cerebral Infarction, Thromboembolic Events History Intractable Seizure Disorder Poor Cooperation of Guardian including Inactive Attitude for Rehabilitation Uncontrolled Hypertension Liver Dysfunction Renal Dysfunction Absolute Neutrophil Count < 500/dL Intracerebral or Intraventricular Hemorrhage Malignancy

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 3 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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Erythropoietin and Rehabilitation<br>recombinant human erythropoietin injection and active rehabilitation | Drug: Erythropoietin<br>* 250 IU/kg, Twice a week for 4 weeks<br>* Other names: Espogen produced by LG Life Science;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse Events | Monitoring of all adverse events during 8 weeks, the study period We selected specially considered adverse events: encephalopathy, intracranial hemorrhage, seizure, hypertension, thromboembolic event, hypoxia, and acute kidney injury. Other adverse events will be recorded. The list was determined by the clinical experience and all adverse reactions reported by the pharmaceutical company. | 8 weeks | 

Secondary 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 - 8 weeks | 
| 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 - 8 weeks | 
| 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 - 8 weeks | 
| Changes in Motor Development | AIMS (Alberta Infant Motor Scale) to assess motor development | Baseline - 8 weeks | 
| Changes in Spasticity | MAS (modified Ashworth Scale) measured at biceps, hip adductor, hamstring, heel cord | Baseline - 8 weeks | 

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