The purpose of this study is to determine whether the drug Leukine (GM-CFS) is safe and tolerated by patients with autoimmune myasthenia gravis (MG).
Twelve patients aged 18-80 with symptomatic generalized autoimmune MG that are not being treated with medication that suppresses their immune system, other than prednisone, will enter the study at UIC over a two year period. The study will involve a screening visit and visits at baseline and at days 5, 15, 30, 45, 60, 90, and 120. The study drug, Leukine (GM-CFS), is given by injection. Subjects will give themselves one dose of GM-CSF every day for 10 days. Study visits will include muscle testing, immunologic studies and quality-of-life studies.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Participants will receive one dose of GM-CSF (5 µg/kg) by subcutaneous injection for ten (10) consecutive days. The first dose of GM-CSF will be administered by the subject or caregiver under the observation and direction of the study staff during the baseline visit. The subject or caregiver will administer subsequent injections at home.
University of Illinois at Chicago, Department of Neurology
Chicago, Illinois, United States
Safety of GM-CSF in patients with myasthenia gravis
The number of subjects experiencing a Class II-V adverse event within 120 days of the start of treatment that is probably or definitely related to the study medication
Time frame: 150 days
The change from baseline in the quantitative and functional (suppressive capacity) characterization of circulating regulatory T cells at 30 days post-treatment
Time frame: 30 days
The change from baseline in the quantitative and functional (suppressive capacity) characterization of circulating regulatory T cells at 60 days post-treatment
Time frame: 60 days
The change from baseline in the quantitative and functional (suppressive capacity) characterization of circulating regulatory T cells at 120 days post-treatment
Time frame: 120 days
Change from baseline in the Quantitative Myasthenia Gravis (QMG) score at 60 days
Time frame: 60 days
Change from baseline in the Quantitative Myasthenia Gravis (QMG) score at day 120
Time frame: 120 days
Change from baseline in the Myasthenia Gravis Composite score at day 60
Time frame: 60 days
Change from baseline in the Myasthenia Gravis Composite score at day 120
Time frame: 120 days
Change from baseline in Manual Muscle Testing (MMT) score at day 60
Time frame: 60 days
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Change from baseline in Manual Muscle Testing (MMT) score at day 120
Time frame: 120 days
Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score at day 60
Time frame: 60 days
Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score at day 120
Time frame: 120 days
Change from baseline in Quality of Life Assessment (SF-36) at day 60
Time frame: 60 days
Change from baseline in Quality of Life Assessment (SF-36) at day 120
Time frame: 120 days
Change from baseline in acetylcholine receptor antibody titre level at day 60
Time frame: 60 days
Change from baseline in acetylcholine receptor antibody titre level at day 120
Time frame: 120 days
Change from baseline in prednisone dose at day 60
Time frame: 60 days
Change from baseline in prednisone dose at day 120 Days
Time frame: 120 days