Amyotrophic Lateral Sclerosis, Brain Injury, Cerebral Palsy, Parkinson's Disease
Conditions
Keywords
Umbilical cord blood, Granulocyte-colony stimulating factor, Brain injury, Neurodegenerative disorder, Efficacy, Safety
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.
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.
Interventions
PROCEDUREUmbilical cord blood therapy
BIOLOGICALFilgrastim
Sponsors
MinYoung Kim, M.D.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
19 Years to 75 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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) |
| 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 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.) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| Changes in Brain MRI | Baseline - 6 months | Analysis of Diffusion Tensor Image (DTI) for brain injury, cerebral palsy, ALS |
Countries
South Korea
Outcome results
None listed