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Neuroregenerative Potential of Intravenous G-CSF and Autologous Peripheral Blood Stem Cells

NCT ID: NCT02983708

Last Updated: 2016-12-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

57 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-08-31

Study Completion Date

2014-09-30

Brief Summary

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The current study describes a randomized, double-blind, cross-over study of intravenous G-CSF followed by infusion with autologous mobilized peripheral blood mononuclear cells (mPBMCs) in children with cerebral palsy (CP) to determine the safety and feasibility of the procedure, as well as the potential efficacy for improving neurological impairment.

Detailed Description

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We hypothesized that mobilized peripheral blood mononuclear cells (mPBMCs) would be a better source of cell therapy for children with CP, if these cells had a similar neuroregenerative potential to bone marrow/cord blood mononuclear cells (MNCs). Multipotent precursor cells exist in peripheral blood, and a fraction of elutriated blood cells from normal individuals contains MNCs that have the potential to be MSCs. There are several advantages to using mPBMCs for cell therapy in children with CP: the G-CSF that is used to mPBMCs has neuroregenerative potential; the collection and fractionation of stem cells can be repeated; and, the therapy is suitable for most children with CP.

Conditions

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Neurodegeneration G-CSF Peripheral Blood Mononuclear Cells Cerebral Palsy

Keywords

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Neuroregeneration G-CSF Peripheral blood mononuclear cells Cerebral palsy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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mPBMC group

G-CSF would be administered for 5 days and then mobilized peripheral blood mononuclear cells (mPBMCs) would be collected in all included patients. One month after cryopreservation of the mPBMCs (M1), patients will be randomized to receive either mPBMCs or placebo. Six months after randomization (M7), cross-over infusion of mPBMCs or placebo will be performed and the patients are observed for another 6 months. mPBMCs group would be included all patients who received mPBMCs at M1 or M7.

Group Type EXPERIMENTAL

Peripheral blood mononuclear cells (mPBMC)

Intervention Type BIOLOGICAL

G-CSF

Intervention Type DRUG

Placebo group

G-CSF would be administered for 5 days and then mobilized peripheral blood mononuclear cells (mPBMCs) would be collected in all included patients. One month after cryopreservation of the mPBMCs (M1), patients will be randomized to receive either mPBMCs or placebo. Six months after randomization (M7), cross-over infusion of mPBMCs or placebo will be performed and the patients are observed for another 6 months. Placebo group would be included all patients who received placebo at M1 or M7.

Group Type PLACEBO_COMPARATOR

G-CSF

Intervention Type DRUG

Placebo

Intervention Type DRUG

Interventions

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Peripheral blood mononuclear cells (mPBMC)

Intervention Type BIOLOGICAL

G-CSF

Intervention Type DRUG

Placebo

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Non severe type of cerebral palsy
* Evidences of abnormal MRI findings such as periventricular leukomalacia
* Collected mobilized peripheral blood mononuclear cell counts \> 1×10\^8/kg or CD34+ cell counts \> 1×10\^6/kg
* Consent form

Exclusion Criteria

* Previous trials of autologous cord blood infusion or erythropoietin/G-CSF
* Chromosomal abnormalities
Minimum Eligible Age

2 Years

Maximum Eligible Age

10 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ministry of Health & Welfare, Korea

OTHER_GOV

Sponsor Role collaborator

Hanyang University Seoul Hospital

OTHER

Sponsor Role lead

Responsible Party

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Young-Ho Lee

Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

References

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Rah WJ, Lee YH, Moon JH, Jun HJ, Kang HR, Koh H, Eom HJ, Lee JY, Lee YJ, Kim JY, Choi YY, Park K, Kim MJ, Kim SH. Neuroregenerative potential of intravenous G-CSF and autologous peripheral blood stem cells in children with cerebral palsy: a randomized, double-blind, cross-over study. J Transl Med. 2017 Jan 21;15(1):16. doi: 10.1186/s12967-017-1120-0.

Reference Type DERIVED
PMID: 28109298 (View on PubMed)

Other Identifiers

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CP-PB-2011

Identifier Type: -

Identifier Source: org_study_id