Erythropoietin Therapy for Children With Cerebral Palsy: Phase 1

Cerebral Palsy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01586052
• Other study ID #: CPEPOPhase1
• Status: Completed
• Phase: Phase 1
• First received: April 23, 2012
• Last updated: April 7, 2014
• Start date: June 2012
• Est. completion date: October 2013

Study information

• Verified date: April 2014
• Source: Bundang CHA Hospital
• Contact: n/a
• Is FDA regulated: No
• Health authority: Korea: Food and Drug AdministrationKorea: Institutional Review Board
• Study type: Interventional

Clinical Trial Summary

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

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 11
• Est. completion date: October 2013
• Est. primary completion date: October 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Months to 3 Years

Eligibility

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cerebral Palsy

Intervention

Drug:
• Erythropoietin
250 IU/kg, Twice a week for 4 weeks

Locations

Country	Name	City	State
Korea, Republic of	CHA Bundang Medical Center, CHA University	Seongnam-si	Gyeonggi-do

Sponsors (2)

Lead Sponsor	Collaborator
MinYoung Kim, M.D.	LG Life Sciences

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Yes
Secondary	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	No
Secondary	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	No
Secondary	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	No
Secondary	Changes in Motor Development	AIMS (Alberta Infant Motor Scale) to assess motor development	Baseline - 8 weeks	No
Secondary	Changes in Spasticity	MAS (modified Ashworth Scale) measured at biceps, hip adductor, hamstring, heel cord	Baseline - 8 weeks	No