Combination Therapy of Cord Blood and G-CSF for Patients With Brain Injury or Neurodegenerative Disorders

Sponsor

MinYoung Kim, M.D. (Other)

Overall Status

Completed

CT.gov ID

NCT02236065

Collaborator

(none)

Enrollment

Location

Arm

Duration (Months)

0.4

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Brain Injury Cerebral Palsy Amyotrophic Lateral Sclerosis Parkinson's Disease	Procedure: Umbilical cord blood therapy Biological: Filgrastim	N/A

Detailed Description

Current treatments for brain injury or neurodegenerative disorders are palliative rather than curative. Preclinical and some clinical studies suggest that UCB and G-CSF can be used as restorative approach for such disorders.

Study Design

Study Type:

Interventional

Actual Enrollment :

10 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A Pilot Study of Combination Therapy of Allogeneic Umbilical Cord Blood and Granulocyte-colony Stimulating Factor for Patients With Brain Injury or Neurodegenerative Disorders

Study Start Date :

Aug 1, 2014

Actual Primary Completion Date :

Jul 1, 2016

Actual Study Completion Date :

Jul 1, 2016

Arms and Interventions

Arm	Intervention/Treatment
Experimental: UCB + G-CSF UCB + G-CSF	Procedure: Umbilical cord blood therapy Biological: Filgrastim

Arm

Intervention/Treatment

Experimental: UCB + G-CSF

UCB + G-CSF

Procedure: Umbilical cord blood therapy

Biological: Filgrastim

Outcome Measures

Primary Outcome Measures

Changes in Berg Balance Scale [Baseline - 1 month - 3 months - 6 months]
Berg Balance Scale for brain injury and parkinson's disease (range: 0 to 56, Higher scores indicates better balance function.)
Changes in the Level of Disability [Baseline - 1 month - 3 months - 6 months]
FIM (Functional Independence Measure) for brain injury and cerebral palsy (range: 18 to 126, Higher scores indicate more independence in activities of daily living.)
Changes in Standardized Gross Motor Function [Baseline - 1 month - 3 months - 6 months]
GMFM (Gross Motor Function Measure) for cerebral palsy (range: 0 to 100, Higher scores indicate better gross motor function.)
Changes in Motor Performance [Baseline - 1 month - 3 months - 6 months]
GMPM (Gross Motor Performance Measure) for cerebral palsy (range: 0 to 100, Higher scores indicate better motor quality.)
Changes in ALSFRS-R [Baseline - 1 month - 3 months - 6 months]
ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-revised) for ALS (range: 0 to 48, Higher scores indicate better physical function.)
Changes in UPDRS [Baseline - 1 month - 3 months - 6 months]
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)

Secondary Outcome Measures

Changes in Brain MRI [Baseline - 6 months]
Analysis of Diffusion Tensor Image (DTI) for brain injury, cerebral palsy, ALS
Changes in Brain PET [Baseline - 6 months]
for parkinson's disease
Number of adverse events and participants with those adverse events [6 months]
The numbers of adverse events and subjects with those serious adverse events within each group; A serious adverse event is any untoward medical occurrence that at any dose: results in death or is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, or causes a congenital anomaly/birth defect.

Eligibility Criteria

Criteria

Ages Eligible for Study:

19 Years to 75 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria: should be included one of the 4 disorders

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