NCT02983708

Brief Summary

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.

Trial Health

100
On Track

Trial Health Score

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

Enrollment
57

participants targeted

Target at P50-P75 for phase_1

Timeline
Completed

Started Aug 2011

Typical duration for phase_1

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

Study Start

First participant enrolled

August 1, 2011

Completed
3.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2014

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2014

Completed
2.3 years until next milestone

First Submitted

Initial submission to the registry

December 2, 2016

Completed
4 days until next milestone

First Posted

Study publicly available on registry

December 6, 2016

Completed
Last Updated

December 7, 2016

Status Verified

December 1, 2016

Enrollment Period

3.1 years

First QC Date

December 2, 2016

Last Update Submit

December 5, 2016

Conditions

NeurodegenerationG-CSFPeripheral Blood Mononuclear CellsCerebral Palsy

Keywords

NeuroregenerationG-CSFPeripheral blood mononuclear cellsCerebral palsy

Outcome Measures

Primary Outcomes (1)

  • Overall improvement as a score changes in GMFM > 4 points

    6 months

Study Arms (2)

mPBMC group

EXPERIMENTAL

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.

Biological: Peripheral blood mononuclear cells (mPBMC)Drug: G-CSF

Placebo group

PLACEBO COMPARATOR

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.

Drug: G-CSFDrug: Placebo

Interventions

Peripheral blood mononuclear cells (mPBMC)BIOLOGICAL
mPBMC group
G-CSFDRUG
Placebo groupmPBMC group
PlaceboDRUG
Placebo group

Eligibility Criteria

Age2 Years - 10 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • Previous trials of autologous cord blood infusion or erythropoietin/G-CSF
  • Chromosomal abnormalities

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Related Publications (1)

  • 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.

    PMID: 28109298DERIVED

MeSH Terms

Conditions

Nerve DegenerationCerebral Palsy

Interventions

Granulocyte Colony-Stimulating Factor

Condition Hierarchy (Ancestors)

Pathologic ProcessesPathological Conditions, Signs and SymptomsBrain Damage, ChronicBrain DiseasesCentral Nervous System DiseasesNervous System Diseases

Intervention Hierarchy (Ancestors)

Colony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological Factors

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

December 2, 2016

First Posted

December 6, 2016

Study Start

August 1, 2011

Primary Completion

September 1, 2014

Study Completion

September 1, 2014

Last Updated

December 7, 2016

Record last verified: 2016-12