Clinical Trial: Efficacy of Methotrexate in Myasthenia Gravis

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Phase II Trial of Methotrexate in Myasthenia Gravis

Brief Summary:

Myasthenia gravis is a rare neuromuscular disorder characterized by weakness and fatigability of ocular, bulbar, and extremity musculature. The specific aim of this study is to determine if oral methotrexate is an effective therapy for myasthenia gravis (MG) patients who are prednisone dependent. Patients will be randomized to receive either methotrexate or placebo and those who are entered onto this trial will have symptoms and signs of the disease while on prednisone therapy. The hypothesis is that adding methotrexate therapy in these patients will improve the MG manifestations so that the prednisone dose can be reduced and clinical measures of MG severity will improve.

Funding Source - FDA OOPD

Detailed Summary:
Sponsor: University of Kansas Medical Center

Current Primary Outcome: Total Prednisone Dose Area Under the Curve [ Time Frame: 9 months ]

Original Primary Outcome: Total prednisone dose [ Time Frame: 9 months ]

Current Secondary Outcome:

• Average Prednisone Daily Dose (mg/Day) [ Time Frame: Total length of time daily dose information was collected, i.e. 9 months. ]
  Participants were asked to fill out the amount of prednisone they took every day on a paper diary.
• Quantitative Myasthenia Gravis (QMG) Score [ Time Frame: Change from Baseline to Month 12 ]
  The QMG is a 13 item ordinal scale which measures ocular, bulbar, extremity fatigue and strength, along with respiratory function. The scale is from 0 - 3 for each item, with 0 meaning normal and 3 is severe. Total score can range from 0 to 39.
• Manual Muscle Testing 12 Month Change [ Time Frame: Change from Baseline to Month 12 ]
  This measurement was developed to measure the strength of muscle groups in the face, neck, arms and legs. Measurement is made by grading the amount of weakness. Participants are graded as having normal, mild (25%) weakness, moderate (50%) weakness or severe (75%) weakness and 4 = paralyzed/unable to do. Normal would receive a score of 0, mild would receive a score of 1, moderate would receive a score of 2, severe would receive a score of 3 and unable to perform would receive a score of 4. Range would be from 0 (no weakness) to 76 (complete paralysis).
• MGQOL 12 Month Change [ Time Frame: Change from Baseline to Month 12 ]
  This test is a 15 item patient-reported scale indicating how myasthenia gravis affects the quality of life. Each item is graded as how true each statement has been over the past 7 days. The scale is 0=Not at all, 1= a little bit, 2= somewhat, 3= quite a bit and 4= very much. The numbers are then added to produce a total score. The MGQOL score would range from 0 (no MG symptoms that affected their quality of life) to a score of 60 (MG symptoms affected they quality of life very much).
• MG-ADL 12 Month Change [ Time Frame: Change from Baseline to Month 12 ]
  The MG-ADL is an 8 item scale developed to assess myasthenia gravis symptoms. Score will range from 0 (normal - no MG symptoms) to 24 (severe MG symptoms)
• MG Composite Change Over 12 Months [ Time Frame: Change from Baseline to Month 12 ]
  This scale is composed of components of the QMG, MG-ADL and the MMT. These components have been shown to be the most responsive in previous clinical trials. Each item in the QMG, MG-ADL and the MMT was weighed (Rasch analysis performed) and then assigned a score. Score would range from 0 (no effects from the myasthenia gravis) to a score of 50. A participant with a score of 50 wwould be in the hospital on a ventilator.

Original Secondary Outcome: Quantitative Myasthenia Gravis score [ Time Frame: baseline, monthly for 12 months ]

Information By: University of Kansas Medical Center

Dates:
Date Received: December 23, 2008
Date Started: April 2009
Date Completion:
Last Updated: April 26, 2016
Last Verified: April 2016