Clinical Trial: A Pilot Study of Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) in Generalized Myasthenia Gravis

Study Status: Terminated
Recruit Status: Unknown status
Study Type: Interventional

Official Title: A Pilot Study of Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) in Symptomatic Autoimmune Generalized Myasthenia Gravis

Brief Summary: 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).

Detailed Summary: 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.
Sponsor: Muscular Dystrophy Association

Current Primary Outcome:

  • Safety of GM-CSF in patients with myasthenia gravis [ Time Frame: 150 days ]
    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
  • 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 ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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 ]
  • 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 ]


Original Secondary Outcome: Same as current

Information By: Muscular Dystrophy Association

Dates:
Date Received: March 13, 2012
Date Started: April 2012
Date Completion: December 2013
Last Updated: March 14, 2012
Last Verified: March 2012