Random Control Trial to Evaluate the Safety and Efficacy of Erythropoietin Therapy for Children With Cerebral Palsy -Patient, Principle Investigator, Observer Blind
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Bundang CHA Hospital
- Enrollment
- 11
- Locations
- 1
- Primary Endpoint
- Changes in Quality of Movement
Study Overview
Brief Summary
This randomized placebo-controlled trial aims to investigate the efficacy and safety 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
- Anti-inflammation.
On the basis of many experimental studies, erythropoietin is suggested as a therapeutic method for cerebral palsy.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 4 Months to 4 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Cerebral Palsy
- •Abnormal Muscle Tone
- •Abnormal Brain MRI
- •Willing to Comply with All Study Procedure
Exclusion Criteria
- •Known Genetic Disorder
- •Other Etiologies Contributing Developmental Delay
- •Coagulopathy
- •Initial high Erythropoietin level in Serum
- •Previous Erythropoietin Treatment before 3 months
- •Presence of Drug Hypersensitivity Related to the Study Remedy
- •Intractable Seizure Disorder
- •Poor Cooperation of Guardian including Inactive Attitude for Rehabilitation
Arms & Interventions
Erythropoietin and Rehabilitation
recombinant human erythropoietin injection and active rehabilitation
Intervention: Erythropoietin (Drug)
Placebo and Rehabilitation
Placebo erythropoietin and rehabilitation
Intervention: Placebo erythropoietin (Drug)
Outcomes
Primary Outcomes
Changes in Quality of Movement
Time Frame: Baseline - 2 months
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).
Secondary Outcomes
- Changes in Motor Development(Baseline - 2 months)
- Changes in Spasticity(Baseline - 2 months)
- Changes in Gross Motor Function(Baseline - 2 months)
- Changes in Neurodevelopmental Outcomes(Baseline - 2 months)
Investigators
MinYoung Kim, M.D.
Associate Professor
Bundang CHA Hospital