Erythropoietin Therapy for Children With Cerebral Palsy: Phase 1

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01586052
Recruitment Status : Completed
First Posted : April 26, 2012
Last Update Posted : April 8, 2014
LG Life Sciences
Information provided by (Responsible Party):
MinYoung Kim, M.D., Bundang CHA Hospital

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

Condition or disease Intervention/treatment Phase
Cerebral Palsy Drug: Erythropoietin Phase 1

Detailed 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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 11 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1 Clinical Research of Erythropoietin Therapy for Children With Cerebral Palsy: Safety and Efficacy
Study Start Date : June 2012
Actual Primary Completion Date : October 2013
Actual Study Completion Date : October 2013

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Erythropoietin and Rehabilitation
recombinant human erythropoietin injection and active rehabilitation
Drug: Erythropoietin
250 IU/kg, Twice a week for 4 weeks
Other Name: Espogen produced by LG Life Science

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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).

  2. 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).

  3. 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).

  4. Changes in Motor Development [ Time Frame: Baseline - 8 weeks ]
    AIMS (Alberta Infant Motor Scale) to assess motor development

  5. Changes in Spasticity [ Time Frame: Baseline - 8 weeks ]
    MAS (modified Ashworth Scale) measured at biceps, hip adductor, hamstring, heel cord

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Ages Eligible for Study:   6 Months to 3 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01586052

Korea, Republic of
CHA Bundang Medical Center, CHA University
Seongnam-si, Gyeonggi-do, Korea, Republic of, 463-712
Sponsors and Collaborators
MinYoung Kim, M.D.
LG Life Sciences
Principal Investigator: Minyoung Kim, M.D., Ph.D. CHA University

Responsible Party: MinYoung Kim, M.D., Associate Professor, Bundang CHA Hospital Identifier: NCT01586052     History of Changes
Other Study ID Numbers: CPEPOPhase1
First Posted: April 26, 2012    Key Record Dates
Last Update Posted: April 8, 2014
Last Verified: April 2014

Keywords provided by MinYoung Kim, M.D., Bundang CHA Hospital:
Cerebral Palsy

Additional relevant MeSH terms:
Cerebral Palsy
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Brain Damage, Chronic
Brain Diseases
Central Nervous System Diseases
Epoetin Alfa