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Erythropoietin Therapy for Children With Cerebral Palsy: Phase 1

Primary Purpose

Cerebral Palsy

Status
Completed
Phase
Phase 1
Locations
Korea, Republic of
Study Type
Interventional
Intervention
Erythropoietin
Sponsored by
MinYoung Kim, M.D.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cerebral Palsy focused on measuring Cerebral Palsy, Erythropoietin, Rehabilitation

Eligibility Criteria

6 Months - 3 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • CHA Bundang Medical Center, CHA University

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Erythropoietin and Rehabilitation

Arm Description

recombinant human erythropoietin injection and active rehabilitation

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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).
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).
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).
Changes in Motor Development
AIMS (Alberta Infant Motor Scale) to assess motor development
Changes in Spasticity
MAS (modified Ashworth Scale) measured at biceps, hip adductor, hamstring, heel cord

Full Information

First Posted
April 23, 2012
Last Updated
April 7, 2014
Sponsor
MinYoung Kim, M.D.
Collaborators
LG Life Sciences
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1. Study Identification

Unique Protocol Identification Number
NCT01586052
Brief Title
Erythropoietin Therapy for Children With Cerebral Palsy: Phase 1
Official Title
A Phase 1 Clinical Research of Erythropoietin Therapy for Children With Cerebral Palsy: Safety and Efficacy
Study Type
Interventional

2. Study Status

Record Verification Date
April 2014
Overall Recruitment Status
Completed
Study Start Date
June 2012 (undefined)
Primary Completion Date
October 2013 (Actual)
Study Completion Date
October 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
MinYoung Kim, M.D.
Collaborators
LG Life Sciences

4. Oversight

Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cerebral Palsy
Keywords
Cerebral Palsy, Erythropoietin, Rehabilitation

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
11 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Erythropoietin and Rehabilitation
Arm Type
Experimental
Arm Description
recombinant human erythropoietin injection and active rehabilitation
Intervention Type
Drug
Intervention Name(s)
Erythropoietin
Other Intervention Name(s)
Espogen produced by LG Life Science
Intervention Description
250 IU/kg, Twice a week for 4 weeks
Primary Outcome Measure Information:
Title
Adverse Events
Description
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.
Time Frame
8 weeks
Secondary Outcome Measure Information:
Title
Changes in Quality of Movement
Description
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).
Time Frame
Baseline - 8 weeks
Title
Changes in Gross Motor Function
Description
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).
Time Frame
Baseline - 8 weeks
Title
Changes in Neurodevelopmental Outcomes
Description
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).
Time Frame
Baseline - 8 weeks
Title
Changes in Motor Development
Description
AIMS (Alberta Infant Motor Scale) to assess motor development
Time Frame
Baseline - 8 weeks
Title
Changes in Spasticity
Description
MAS (modified Ashworth Scale) measured at biceps, hip adductor, hamstring, heel cord
Time Frame
Baseline - 8 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Minyoung Kim, M.D., Ph.D.
Organizational Affiliation
CHA University
Official's Role
Principal Investigator
Facility Information:
Facility Name
CHA Bundang Medical Center, CHA University
City
Seongnam-si
State/Province
Gyeonggi-do
ZIP/Postal Code
463-712
Country
Korea, Republic of

12. IPD Sharing Statement

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Erythropoietin Therapy for Children With Cerebral Palsy: Phase 1

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