NCT01555580

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

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

• Status: Unknown
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Muscular Dystrophy Association · Academic / Other
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

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 description

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.

Conditions

• Myasthenia Gravis

Interventions

Type	Name	Description
DRUG	Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF)	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.

Timeline

• Start date: 2012-04-01
• Primary completion: 2013-12-01
• Completion: 2013-12-01
• First posted: 2012-03-15
• Last updated: 2012-03-15

Locations

1 site across 1 country: United States