Erythropoietin Therapy for Children With Cerebral Palsy: Phase 1

MinYoung Kim, M.D.11 enrolled

Overview

This purpose of this phase 1 study is to investigate the safety and efficacy of erythropoetin for children with cerebral palsy.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Cerebral Palsy

Interventions

ErythropoietinDRUG

250 IU/kg, Twice a week for 4 weeks

Eligibility

Sex: ALLMin age: 6 MonthsMax age: 3 Years

Medical Language ↔ Plain English

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

Locations (1)

CHA Bundang Medical Center, CHA University

Seongnam-si, Gyeonggi-do, South Korea

Outcomes

Primary Outcomes

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.

Time frame: 8 weeks

Secondary Outcomes

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).

Time frame: 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).

Time frame: 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).

Time frame: Baseline - 8 weeks

Changes in Motor Development

AIMS (Alberta Infant Motor Scale) to assess motor development

Data from ClinicalTrials.gov

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