NCT02236065

Brief Summary

This open label trial is conducted to investigate the efficacy and safety of the combination therapy of allogeneic umbilical cord blood (UCB) and granulocyte-colony stimulating factor (G-CSF) for patients with brain injury or neurodegenerative disorders.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
10

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Aug 2014

Typical duration 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

Study Start

First participant enrolled

August 1, 2014

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

September 8, 2014

Completed
2 days until next milestone

First Posted

Study publicly available on registry

September 10, 2014

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2016

Completed
Last Updated

October 12, 2017

Status Verified

October 1, 2017

Enrollment Period

1.9 years

First QC Date

September 8, 2014

Last Update Submit

October 10, 2017

Conditions

Brain InjuryCerebral PalsyAmyotrophic Lateral SclerosisParkinson's Disease

Keywords

Umbilical cord bloodGranulocyte-colony stimulating factorBrain injuryNeurodegenerative disorderEfficacySafety

Outcome Measures

Primary Outcomes (6)

  • Changes in Berg Balance Scale

    Berg Balance Scale for brain injury and parkinson's disease (range: 0 to 56, Higher scores indicates better balance function.)

    Baseline - 1 month - 3 months - 6 months

  • Changes in the Level of Disability

    FIM (Functional Independence Measure) for brain injury and cerebral palsy (range: 18 to 126, Higher scores indicate more independence in activities of daily living.)

    Baseline - 1 month - 3 months - 6 months

  • Changes in Standardized Gross Motor Function

    GMFM (Gross Motor Function Measure) for cerebral palsy (range: 0 to 100, Higher scores indicate better gross motor function.)

    Baseline - 1 month - 3 months - 6 months

  • Changes in Motor Performance

    GMPM (Gross Motor Performance Measure) for cerebral palsy (range: 0 to 100, Higher scores indicate better motor quality.)

    Baseline - 1 month - 3 months - 6 months

  • Changes in ALSFRS-R

    ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-revised) for ALS (range: 0 to 48, Higher scores indicate better physical function.)

    Baseline - 1 month - 3 months - 6 months

  • Changes in UPDRS

    UPDRS (Unified Parkinson's Disease Rating Scale) for parkinson's disease (Part 1: mentation, behavior and mood; Part 2: activities of daily living; Part 3: motor examination; Part 4: complications of therapy; Part 5: Schwab and England activities of daily living scale)

    Baseline - 1 month - 3 months - 6 months

Secondary Outcomes (3)

  • Changes in Brain MRI

    Baseline - 6 months

  • Changes in Brain PET

    Baseline - 6 months

  • Number of adverse events and participants with those adverse events

    6 months

Study Arms (1)

UCB + G-CSF

EXPERIMENTAL

UCB + G-CSF

Procedure: Umbilical cord blood therapyBiological: Filgrastim

Interventions

Umbilical cord blood therapyPROCEDURE
UCB + G-CSF
FilgrastimBIOLOGICAL
UCB + G-CSF

Eligibility Criteria

Age19 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Brain injury: onset duration over 12 months, Age: 19 years or over
  • Cerebral palsy: spastic, dyskinetic or ataxic, Age: 19 years or over
  • Parkinson's disease: modified Hoehn and Yahr stage 2.5, 3, 4, Age: 30 to 75 years
  • ALS: definite of possible ALS, progression during the past 6 months, Age: 19 to 65

You may not qualify if:

  • Uncontrolled pulmonary, renal dysfunction at enrollment
  • Uncontrolled seizure
  • Malignant cancer
  • Possibility of hypersensitivity to drugs used in this study
  • Contraindication to the study intervention or assessment
  • Pregnant or breast feeding women
  • Non-compliance with the study visits specified in the protocol

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

MeSH Terms

Conditions

Brain InjuriesCerebral PalsyAmyotrophic Lateral SclerosisParkinson DiseaseNeurodegenerative Diseases

Interventions

Filgrastim

Condition Hierarchy (Ancestors)

Brain DiseasesCentral Nervous System DiseasesNervous System DiseasesCraniocerebral TraumaTrauma, Nervous SystemWounds and InjuriesBrain Damage, ChronicSpinal Cord DiseasesMotor Neuron DiseaseTDP-43 ProteinopathiesNeuromuscular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic DiseasesParkinsonian DisordersBasal Ganglia DiseasesMovement DisordersSynucleinopathies

Intervention Hierarchy (Ancestors)

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

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

September 8, 2014

First Posted

September 10, 2014

Study Start

August 1, 2014

Primary Completion

July 1, 2016

Study Completion

July 1, 2016

Last Updated

October 12, 2017

Record last verified: 2017-10

Locations

KR(1)