Erythropoietin Therapy for Children With Cerebral Palsy: Phase 1

This study has been completed.
Sponsor:
Collaborator:
LG Life Sciences
Information provided by (Responsible Party):
MinYoung Kim, M.D., Bundang CHA Hospital
ClinicalTrials.gov Identifier:
NCT01586052
First received: April 23, 2012
Last updated: April 7, 2014
Last verified: April 2014
  Purpose

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


Condition Intervention Phase
Cerebral Palsy
Drug: Erythropoietin
Phase 1

Study Type: Interventional
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

Resource links provided by NLM:


Further study details as provided by Bundang CHA Hospital:

Primary Outcome Measures:
  • 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, 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:
  • 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


Enrollment: 11
Study Start Date: June 2012
Study Completion Date: October 2013
Primary Completion Date: October 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   6 Months to 3 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01586052

Locations
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
Investigators
Principal Investigator: Minyoung Kim, M.D., Ph.D. CHA University
  More Information

No publications provided

Responsible Party: MinYoung Kim, M.D., Associate Professor, Bundang CHA Hospital
ClinicalTrials.gov Identifier: NCT01586052     History of Changes
Other Study ID Numbers: CPEPOPhase1
Study First Received: April 23, 2012
Last Updated: April 7, 2014
Health Authority: Korea: Food and Drug Administration
Korea: Institutional Review Board

Keywords provided by Bundang CHA Hospital:
Cerebral Palsy
Erythropoietin
Rehabilitation

Additional relevant MeSH terms:
Paralysis
Cerebral Palsy
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Brain Damage, Chronic
Brain Diseases
Central Nervous System Diseases
Epoetin alfa
Hematinics
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on October 19, 2014