Erythropoietin Therapy for Children With Cerebral Palsy: Phase 1
This purpose of this phase 1 study is to investigate the safety and efficacy of erythropoetin for children with cerebral palsy.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase 1 Clinical Research of Erythropoietin Therapy for Children With Cerebral Palsy: Safety and Efficacy|
- Adverse Events [ Time Frame: 8 weeks ] [ Designated as safety issue: Yes ]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.
- Changes in Quality of Movement [ Time Frame: Baseline - 8 weeks ] [ Designated as safety issue: No ]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 ] [ Designated as safety issue: No ]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 ] [ Designated as safety issue: No ]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 ] [ Designated as safety issue: No ]AIMS (Alberta Infant Motor Scale) to assess motor development
- Changes in Spasticity [ Time Frame: Baseline - 8 weeks ] [ Designated as safety issue: No ]MAS (modified Ashworth Scale) measured at biceps, hip adductor, hamstring, heel cord
|Study Start Date:||June 2012|
|Estimated Study Completion Date:||October 2013|
|Estimated Primary Completion Date:||October 2013 (Final data collection date for primary outcome measure)|
Experimental: Erythropoietin and Rehabilitation
recombinant human erythropoietin injection and active rehabilitation
250 IU/kg, Twice a week for 4 weeks
Other Name: Espogen produced by LG Life Science
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.
|Korea, Republic of|
|CHA Bundang Medical Center, CHA University|
|Seongnam-si, Gyeonggi-do, Korea, Republic of, 463-712|
|Principal Investigator:||Minyoung Kim, M.D., Ph.D.||CHA Bundang Medical center, CHA university|