Parkinson Disease
Conditions
Brief summary
The purpose of this study is to evaluate the effectiveness of neuroprotection from granulocyte colony-stimulating factor on Parkinson disease
Detailed description
Parkinson's disease (PD) is the second most common neurodegenerative disease and the severity of PD still progress during the ensuing years. Currently, there is no promising medical or surgical treatment to abrogate the disease deterioration. Granulocyte colony-stimulating factor (G-CSF), one of hematopoietic growth factors, has been routinely used for hematologic disorders and stem cell harvest from normal subject. G-CSF also demonstrated neuroprotection for rodents PD model. We hypothesize G-CSF will exert the effectiveness of neuroprotection for PD patients.
Interventions
DRUGG-CSF
G-CSF(granulocyte-colony stimulating factor) is planned injected subcutaneously
DRUGPlacebo
Sodium Chloride (NaCl) 0.9 %
Sponsors
Buddhist Tzu Chi General Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
40 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* Hoehn & Yahr stage I\ III * Patients with idiopathic Parkinson's disease(PD), who meet the United Kingdom Parkinson's Disease Society Brain Bank diagnostic criteria, with a good response to levodopa * The onset of PD symptoms must be occurred \> = 40 years of age (to exclude YOPD), and the patient must have been diagnosed with idiopathic PD \> =40 years of age * Patients may have symptoms of wearing OFF and/or levodopa induced dyskinesia * Patients must rate between Hoehn & Yahr stage I to III, when in an OFF medication state * Patients must in their optimal medication treatment state, and will not changing their medications within 3 months before and after enrollment
Exclusion criteria
* Patients who are proved to be Young Onset Parkinson's Disease (YOPD) and/or genetically related (e.g. Parkin). * Women of child-bearing potential, pregnant or lactating. * Patients who are proved to have tumor growth and/or malignancy. * Patients with a past (within one year) or present history of psychotic symptoms requiring anti- psychotic treatment. * Patients with active symptoms of major depression with suicidal ideation or suicide attempt. * Patients with previous brain surgery (including pallidotomy and deep brain stimulation). * Patients with significant cognitive impairment ( Mini-Mental State Examination, MMSE \< 24). * Patients who do not sign the inform consent,
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Motor Performance of Unified Parkinson's Disease Rating Scale | 2 years | To assess Unified Parkinson's Disease Rating Scale part III (motor function) scores from baseline Medication-off status to Medication-off status after G-CSF injection one year. Scores of UPDRS Part III ranges from 0 to 108 and higher values indicate worse outcome. |
Countries
Taiwan
Participant flow
Recruitment details
Parkinson's disease research center, Tzu Chi Hospital
Pre-assignment details
No participants were randomized to receive 1.65ug/kg/day of G-CSF and placebo
Participants by arm
| Arm | Count |
|---|---|
| High Dose G-CSF Injection for Parkinson's Disease 3.3 ug/kg/day for consecutive 5 days of each 60 day cycle | 4 |
| Low Dose G-CSF Injection for Parkinson's Disease 1.65 ug/kg/day for consecutive 5 days of each 60 day cycle | 0 |
| Placebo normal saline 0.9% injection | 0 |
| Total | 4 |
Baseline characteristics
| Characteristic | High Dose G-CSF Injection for Parkinson's Disease | Total |
|---|---|---|
| Age, Categorical <=18 years | 0 participants | 0 participants |
| Age, Categorical >=65 years | 0 participants | 0 participants |
| Age, Categorical Between 18 and 65 years | 4 participants | 4 participants |
| Age, Continuous | 49.25 years | 49.25 years |
| Gender Female | 0 participants | 0 participants |
| Gender Male | 4 participants | 4 participants |
| Region of Enrollment Taiwan | 4 participants | 4 participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 3 / 4 |
| serious Total, serious adverse events | 0 / 4 |
Outcome results
Primary
Motor Performance of Unified Parkinson's Disease Rating Scale
To assess Unified Parkinson's Disease Rating Scale part III (motor function) scores from baseline Medication-off status to Medication-off status after G-CSF injection one year. Scores of UPDRS Part III ranges from 0 to 108 and higher values indicate worse outcome.
Time frame: 2 years
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| G-CSF Injection for Parkinson's Disease | Motor Performance of Unified Parkinson's Disease Rating Scale | Baseline | 23 scores on a scale |
| G-CSF Injection for Parkinson's Disease | Motor Performance of Unified Parkinson's Disease Rating Scale | post G-CSF one year | 22 scores on a scale |
Comparison: Null hypothesis: there is significant deterioration of UPDRS part III scores from baseline to post G-CSF injection one yearp-value: 0.64t-test, 2 sided