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

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

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

Conditions
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Brain Injury, Cerebral Palsy, Amyotrophic Lateral Sclerosis, Parkinson's Disease

Intervention / Treatment
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* Procedure: Umbilical cord blood therapy
* Biological: Filgrastim


Participation Criteria
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Eligibility Criteria
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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 Exclusion Criteria: 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

Ages Eligible for Study
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Minimum Age: 19 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: UCB + G-CSF<br>UCB + G-CSF | Procedure: Umbilical cord blood therapy<br> <br> Biological: Filgrastim<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in Brain MRI | Analysis of Diffusion Tensor Image (DTI) for brain injury, cerebral palsy, ALS | Baseline - 6 months | 
| Changes in Brain PET | for parkinson's disease | Baseline - 6 months | 
| Number of adverse events and participants with those adverse events | 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. | 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Safety, Umbilical cord blood, Granulocyte-colony stimulating factor, Brain injury, Neurodegenerative disorder, Efficacy