Clinical Trial: Erythropoietin Therapy for Children With Cerebral Palsy: Phase 1

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

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

Detailed Summary:

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.


Sponsor: MinYoung Kim, M.D.

Current Primary Outcome: Adverse Events [ Time Frame: 8 weeks ]

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.


Original Primary Outcome: Adverse Events [ Time Frame: 8 weeks ]

Monitoring of all adverse events during 8 weeks, the study period We selected specially considered adverse events: encephalopathy, intracranial hemorrhage, seizure, hypertension, thromboemboilc 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.


Current Secondary Outcome:

  • Changes in Quality of Movement [ Time Frame: Baseline - 8 weeks ]
    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).
  • Changes in Gross Motor Function [ Time Frame: Baseline - 8 weeks ]
    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).
  • Changes in Neurodevelopmental Outcomes [ Time Frame: Baseline - 8 weeks ]
    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).
  • Changes in Motor Development [ Time Frame: Baseline - 8 weeks ]
    AIMS (Alberta Infant Motor Scale) to assess motor development
  • Changes in Spasticity [ Time Frame: Baseline - 8 weeks ]
    MAS (modified Ashworth Scale) measured at biceps, hip adductor, hamstring, heel cord


Original Secondary Outcome:

  • Changes in Quality of Movement [ Time Frame: Baseline - 8 weeks ]
    GMPM (Gross Motor Perfomance 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).
  • Changes in Gross Motor Function [ Time Frame: Baseline - 8 weeks ]
    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).
  • Changes in Neurodevelopmental Outcomes [ Time Frame: Baseline - 8 weeks ]
    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).
  • Changes in Motor Development [ Time Frame: Baseline - 8 weeks ]
    AIMS (Alberta Infant Motor Scale) to assess motor development
  • Changes in Spasticity [ Time Frame: Baseline - 8 weeks ]
    MAS (modified Ashworth Scale) measured at biceps, hip adductor, hamstring, heel cord


Information By: Bundang CHA Hospital

Dates:
Date Received: April 23, 2012
Date Started: June 2012
Date Completion:
Last Updated: April 7, 2014
Last Verified: April 2014