Allogeneic UCB Therapy With EPO in Children With CP

N/ACompletedNCT01991145

MinYoung Kim, MD, PhD92 enrolled

Overview

This randomized controlled study aims to evaluate the efficacy and safety of allogeneic umbilical cord blood therapy combined with erythropoietin for children with cerebral palsy.

Cerebral palsy (CP) is a group of neurodevelopmental conditions with abnormal movement and posture resulted from a non-progressive cerebral disturbance. It is the most common cause of motor disability in childhood. Most therapies are palliative rather than restorative. Umbilical cord blood (UCB) and erythropoetin (EPO) may be used as restorative approach for children with CP. Many experimental animal studies have revealed that UCB is beneficial to improve and repair neurological injuries. EPO is also known to have neuroprotective effects. Based on animal studies and some clinical trials, UCB is suggested as a potential therapy for children with CP. EPO is combined to add synergistic effects to UCB therapy.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Cerebral Palsy

Interventions

Umbilical Cord Blood therapyPROCEDURE

HLA (Human Leukocyte Antigen) typing

Erythropoietin alfaBIOLOGICAL

RehabilitationOTHER

Active rehabilitation

Placebo UCB

Eligibility

Sex: ALLMin age: 10 MonthsMax age: 6 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Diagnosed with cerebral palsy * Age of ≥10 months and ≤6 years * Mismatch in HLA-A, B, and DR ≤2, and total nucleated cell count ≥3x107/kg. If the cell count is less than given values, more than 1 unit could be used. * Hemoglobin ≤13.6 g/dL * Decision of participation in the study by and acquisition of informed consent from the subject's representative * Willingness and ability to be hospitalized according to the schedule specified in the protocol and continue the study for 12 months after study entry Exclusion Criteria: * Current aspiration pneumonia * Known genetic disease * History of hypersensitivity reaction to any study drugs pertinent to the study * History of participation in any other study with stem cell * Prior treatment with EPO within 3 months prior to study entry * Known coagulopathy with family history of thrombosis or medical history of recurrent thrombosis * Patient with severe seizure disease who has clinical convulsion despite combination therapy with 3 or more agents * Uncontrolled hypertension defined as systolic blood pressure \>115 mmHg and/or diastolic blood pressure \>70 mmHg * Hepatic impairment defined as asparate aminotransferase (AST) \>55 IU/L and/or alanine aminotransferase (ALT) \>45 IU/L * Renal impairment defined as creatinine (Cr) ≥1.2 mg/dL * Absolute neutrophil count ≤500/dL * Presence of diagnosed or suspected malignant tumor and/or hematologic malignancy * Non-compliance with study visits specified in the protocol or unwillingness of care-giver due to lack of understanding of the patient

Locations (1)

CHA Bundang Medical Center, CHA University

Seongnam-si, Gyeonggi-do, South Korea

Outcomes

Primary Outcomes

Changes in Standardized Gross Motor Function

GMFM (Gross Motor Function Measure) is a standardized measurement tool for assessing gross motor function consisting of sub-scales; lying \& rolling, sitting, crawling \& kneeling, standing, walking, running \& jumping (range: 0\~100, higher value means better gross motor function).

Time frame: Baseline - 1 month - 3 months - 6 months - 12months

Changes in Motor Performance

GMPM (Gross Motor Performance Measure) is a standardized measurement tool for assessing quality of movement regarding 3 properties of 5 ones; alignment, coordination, dissociated movement, stability, and weight shift (range: 0\~100, higher value means better motor quality).