NCT01991145

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
92

participants targeted

Target at P50-P75 for not_applicable

Timeline
Completed

Started Nov 2013

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 17, 2013

Completed
8 days until next milestone

First Posted

Study publicly available on registry

November 25, 2013

Completed
1 day until next milestone

Study Start

First participant enrolled

November 26, 2013

Completed
3.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 24, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 24, 2017

Completed
Last Updated

January 18, 2020

Status Verified

January 1, 2020

Enrollment Period

3.6 years

First QC Date

November 17, 2013

Last Update Submit

January 15, 2020

Conditions

Cerebral Palsy

Keywords

Cerebral PalsyUmbilical Cord BloodErythropoietinRehabilitation

Outcome Measures

Primary Outcomes (4)

  • 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).

    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).

    Baseline - 1 month - 3 months - 6 months - 12 months

  • Changes in Cognitive Neurodevelopmental Outcome

    Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Mental Scale (range: 0\~178; worst: 0, best: 178)

    Baseline - 1 month - 3 months - 6 months - 12 months

  • Changes in Motor Neurodevelopmental Outcome

    Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Motor Scale (range: 0\~112; worst: 0, best: 112)

    Baseline - 1 month - 3 months - 6 months - 12 months

Secondary Outcomes (14)

  • Changes in Gross Motor Function Classification System

    Baseline - 1 month - 3 months - 6 months - 12 months

  • Changes in Functional Independence in Daily Activities

    Baseline - 1 month - 3 months - 6 months - 12 months

  • Changes in Functional Performance in Daily Activities

    Baseline - 1 month - 3 months - 6 months - 12 months

  • Changes in Upper Extremity Function

    Baseline - 1 month - 3 months - 6 months - 12 months

  • Changes in Visual Perception Test

    Baseline - 1 month - 3 months - 6 months - 12 months

  • +9 more secondary outcomes

Study Arms (4)

UCB and EPO

EXPERIMENTAL

UCB + EPO + Rehabilitation

Procedure: Umbilical Cord Blood therapyBiological: Erythropoietin alfaOther: Rehabilitation

UCB and placebo EPO

ACTIVE COMPARATOR

UCB + placebo EPO + Rehabilitation

Procedure: Umbilical Cord Blood therapyOther: RehabilitationBiological: Placebo EPO

placebo UCB and EPO

ACTIVE COMPARATOR

placebo UCB + EPO + Rehabilitation

Biological: Erythropoietin alfaOther: RehabilitationProcedure: Placebo UCB

placebo UCB and placebo EPO

PLACEBO COMPARATOR

placebo UCB + placebo EPO + Rehabilitation

Other: RehabilitationProcedure: Placebo UCBBiological: Placebo EPO

Interventions

Umbilical Cord Blood therapyPROCEDURE

HLA (Human Leukocyte Antigen) typing

UCB and EPOUCB and placebo EPO
Erythropoietin alfaBIOLOGICAL
Also known as: Espogen (LG Life Science Ltd.)
UCB and EPOplacebo UCB and EPO
RehabilitationOTHER

Active rehabilitation

UCB and EPOUCB and placebo EPOplacebo UCB and EPOplacebo UCB and placebo EPO
Placebo UCBPROCEDURE
placebo UCB and EPOplacebo UCB and placebo EPO
Placebo EPOBIOLOGICAL
UCB and placebo EPOplacebo UCB and placebo EPO

Eligibility Criteria

Age10 Months - 6 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

CHA Bundang Medical Center, CHA University

Seongnam-si, Gyeonggi-do, 463-712, South Korea

Location

Related Publications (1)

  • Min K, Suh MR, Cho KH, Park W, Kang MS, Jang SJ, Kim SH, Rhie S, Choi JI, Kim HJ, Cha KY, Kim M. Potentiation of cord blood cell therapy with erythropoietin for children with CP: a 2 x 2 factorial randomized placebo-controlled trial. Stem Cell Res Ther. 2020 Nov 27;11(1):509. doi: 10.1186/s13287-020-02020-y.

    PMID: 33246489DERIVED

MeSH Terms

Conditions

Cerebral Palsy

Interventions

Rehabilitation

Condition Hierarchy (Ancestors)

Brain Damage, ChronicBrain DiseasesCentral Nervous System DiseasesNervous System Diseases

Intervention Hierarchy (Ancestors)

AftercareContinuity of Patient CarePatient CareTherapeuticsHealth ServicesHealth Care Facilities Workforce and Services

Study Officials

  • MinYoung Kim, M.D., Ph.D.

    CHA University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Professor of CHA University, M.D., Ph.D.

Study Record Dates

First Submitted

November 17, 2013

First Posted

November 25, 2013

Study Start

November 26, 2013

Primary Completion

June 24, 2017

Study Completion

June 24, 2017

Last Updated

January 18, 2020

Record last verified: 2020-01

Locations

KR(1)