Leukine (Sargramostim) for Parkinson's Disease
Parkinson's Disease
About this trial
This is an interventional treatment trial for Parkinson's Disease focused on measuring Parkinson's disease, motor function, immune activation, T cells, CD4, GMCSF, leukine, sargramostim, magnetoencephalography
Eligibility Criteria
Inclusion Criteria:
PD Patients
- Onset of bradykinesia and 1 or both of the following: rest tremor and/or rigidity
- Asymmetric onset of clinical signs
- Progressive motor symptoms
- Age at onset 35-85 years
- Duration of PD symptoms of at least 3 years
- Female subjects must be either:
Not pregnant, not breastfeeding, and not planning on becoming pregnant during the study; Not of childbearing potential, defined as one who has been postmenopausal for at least 1 year and with follicle stimulating hormone (FSH) levels in the laboratory defined postmenopausal range, or has been surgically sterilized, or has had a hysterectomy at least 3 months prior to the start of this trial; or If of childbearing potential, must agree to use an effective method of avoiding pregnancy to the end of the trial and must have a negative serum beta-human chorionic gonadotropin (β-HCG) test. Effective methods of avoiding pregnancy are contraceptive methods used consistently and correctly (including implantable contraceptives, injectable contraceptives, oral contraceptives, transdermal contraceptives, intrauterine devices, diaphragm with spermicide, male or female condoms with spermicide, or cervical cap), abstinence, or a sterile sexual partner
- Have the ability to comply with basic instructions and have the ability to sit still comfortably inside the MEG
- Must be stage 4 or less according to the Hoehn and Yahr scale
- Caregiver, spouse, friend, or relative must agree to participate in the research study
Control subjects:
- Age 35-85 years
- Caregiver, spouse, relative, or friend of eligible PD patient
- Female subjects must be either:
Not pregnant, not breastfeeding, and not planning on becoming pregnant during the study; Not of childbearing potential, defined as one who has been postmenopausal for at least 1 year and with follicle stimulating hormone (FSH) levels in the laboratory defined postmenopausal range, or has been surgically sterilized, or has had a hysterectomy at least 3 months prior to the start of this trial; or If of childbearing potential, must agree to use an effective method of avoiding pregnancy to the end of the trial and must have a negative serum beta-human chorionic gonadotropin (β-HCG) test. Effective methods of avoiding pregnancy are contraceptive methods used consistently and correctly (including implantable contraceptives, injectable contraceptives, oral contraceptives, transdermal contraceptives, intrauterine devices, diaphragm with spermicide, male or female condoms with spermicide, or cervical cap), abstinence, or a sterile sexual partner
- Have the ability to comply with basic instructions and have the ability to sit still comfortably inside the MEG
Exclusion Criteria:
PD Patients
- Atypical features indicative of a Parkinson-Plus disorder (Progressive Supranuclear Palsy (PSP), Multiple System Atrophy (MSA), Corticobasal Degeneration (CBD)) including cerebellar signs, supranuclear gaze palsy, apraxia and other cortical signs, or prominent autonomic failure
- Neuroleptic treatment at time of onset of parkinsonism
- Active treatment with a neuroleptic at time of study entry
- History of repeated strokes with stepwise progression of parkinsonism
- History of repeated head injury
- History of definite encephalitis
- More than one blood relative diagnosed with PD
- Prominent gait imbalance early in the course (< 5 years)
- Mini-mental state examination score <26
- Hematological malignancy or coagulopathy
- Abnormal blood analyses: hematocrit <30; WBC>11.5; clinically significant laboratory data (e.g. alanine aminotransferase [ALT] or aspartate aminotransferase [AST] 3x the upper limit of normal [ULN]), or any abnormal laboratory value that could interfere with the assessment of safety in the judgment of the investigator; significant abnormalities on the clinical examination, vital signs, and clinical chemistry or hematology results (excluding findings of Parkinson's disease), that may interfere with the study or present a safety risk for the subject as judged by the clinical investigator charged in the care of study participants
- Serious medical illness or co-morbidity that may interfere with participation in the study
- Brain surgery for parkinsonism (DBS, cell implantation, gene therapy)
- History of an autoimmune disorder or systemic inflammatory disorder
- Immunostimulatory or immunosuppressive treatment (including amphetamines or systemic corticosteroids) within 90 days
- Exclusively unilateral parkinsonism for longer than 3 years
- Known hypersensitivity to GM-CSF, yeast-derived products or benzyl alcohol
- Current lithium treatment
- Individuals who have ferrous metal implanted in their body other than fillings
- Individuals with current diagnoses of alcohol or substance abuse/dependence
- Anyone who is not appropriate for participation in this research protocol as deemed by the principal or co-investigator
Control subjects:
- Positive response to more than 3 items on the PD Screening Questionnaire
- More than one blood relative diagnosed with by PD
- Mini-mental state examination score <26
- Hematological malignancy or coagulopathy
- Abnormal blood analyses: hematocrit <30; WBC>11.5; clinically significant laboratory data (e.g. alanine aminotransferase [ALT] or aspartate aminotransferase [AST] 3x the upper limit of normal [ULN]), or any abnormal laboratory value that could interfere with the assessment of safety in the judgment of the investigator; significant abnormalities on the clinical examination, vital signs, and clinical chemistry or hematology results that may interfere with the study or present a safety risk for the subject as judged by the investigator
- Serious medical illness or comorbidity that may interfere with participation in the study
- History of an autoimmune disorder or systemic inflammatory disorder
- Immunostimulatory or immunosuppressive treatment (including amphetamines or systemic corticosteroids) within 90 days
- Individuals who have ferrous metal implanted in their body other than fillings
- Individuals with current diagnoses of alcohol or substance abuse/dependence
- Anyone who is not appropriate for participation in this research protocol as deemed by the principal or co-investigator
PD Screening Questionnaire
- Do you have trouble arising from a chair?
- Is your handwriting smaller than it once was?
- Do people tell you that your voice is softer than it once was?
- Is your balance poor?
- Do your feet ever seem to get stuck to the floor?
- Do people tell you that your face seems less expressive than it once did?
- Do your arms and legs shake?
- Do you have trouble buttoning buttons?
- Do you shuffle your feet and/or take tiny steps when you walk?
- Has anyone ever told you that you have Parkinson's disease?
- Have you ever taken levodopa or Sinemet?
Sites / Locations
- Neurology Consultants of Nebraska PC
- University of Nebraska Medical Center
Arms of the Study
Arm 1
Arm 2
Arm 3
Sham Comparator
Placebo Comparator
Experimental
Controls
PD Patients placebo
PD Patients sargramostim
Caregivers, spouse, friends, relatives of PD patients, have blood draws, MEG.
PD patients that receive placebo, have blood draw, physical exam and UPDRS part III assessment, MEG, Motion analysis.
PD patients that receive Leukine, have blood draw, physical exam and UPDRS part III assessment, MEG, Motion analysis.