Clinical Trial: Trial of Orencia in Patients With Myasthenia Gravis

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Pilot Trial of Orencia in Myasthenia Gravis Patients Inadequately Responsive to Conventional Immunotherapy

Brief Summary: This pilot research study is being done to see if the drug abatacept (Orencia ®) will be helpful in treating patients with myasthenia gravis (MG) who do not respond satisfactorily to other drugs that are used to suppress the immune system. Abatacept has been successful in treating experimental MG in laboratory animals, and this study is to determine its effectiveness in patients with MG.

Detailed Summary:
Sponsor: Johns Hopkins University

Current Primary Outcome:

  • Subjective Composite Score of Myasthenia Gravis (MG) Severity [ Time Frame: 12 months ]
    The "Subjective Score of MG Severity" is a composite score of subjective measurements of MG severity as designed by Dr. Daniel Drachman. The investigator will rate each subjective measure on a scale of 0 (no impairment) to 3 (severe impairment). The composite score of these measurements determined at baseline and at two-month increments through 12 months. The domains that make up the subjective score are: Diplopia, Ptosis, Arm strength, Leg strength, Speech, Voice, Chewing, Swallowing, Respiration, and General Health Status. The change in the composite subjective score at 12 months as compared to baseline is one of the primary outcomes. Patients that show an improvement in MG symptoms should have a lower score at 12 months as compared to baseline.
  • Objective Composite Score of Myasthenia Gravis (MG) Severity [ Time Frame: 12 months ]
    The "Objective Score of MG Severity" is a composite score of subjective measurements of MG severity as designed by Dr. Daniel Drachman. The investigator will score each objective measure on a scale of 0 (no impairment) to 3 (severe impairment). The composite score of these measurements determined at baseline and at two-month increments through 12 months. The domains that make up the objective score are: Diplopia, Ptosis, Arm abduction, Gait, Orbicularis Oculi, Orbicularis Oris, Tongue, Slurp Test. The change in the composite objective score at 12 months as compared to baseline is one of the primary outcomes. Patients that show an improvement in MG symptoms should have a lower score at 12 months as compared to baseline.
  • Muscle Strength (Dynamometry) [ 

    Original Primary Outcome: Same as current

    Current Secondary Outcome:

    • Myasthenia Gravis Composite Score (MGC) [ Time Frame: 12 months ]
      The Myasthenia Gravis Composite (MGC) scale consists of test items that measure symptoms and signs of MG, with weighted response options. The domains included in the MGC are: Ptosis, Double vision, Eye closure, Talking, Chewing, Swallowing, Breathing, Neck strength, Shoulder abduction and Hip flexion. Each domain is rated by the Investigator on a weighted scale between 0 (no impairment) and 6 (severe impairment). Patients that show an improvement in MG symptoms should have a lower score at 12 months as compared to baseline.
    • Myasthenia Gravis Quality of Life-15 (MG QOL15) Score [ Time Frame: 12 months ]
      The MG-QOL15 is a brief survey, completed by the patient, that is designed to assess some aspects of "quality of life" related to myasthenia gravis. Patients are given 15 statements that relate to their quality of life and are asked to chose how often that statement applies to them (Not At All, A Little Bit, Some-what, Quite A Bit, Very Much). Their answers are assigned a score from 0 (not at all) to 4 (Very Much). Patients that show an improvement in quality of life as related to their MG symptoms should have a lower score at 12 months as compared to baseline.
    • Acetylcholine Receptor (AChR) or Muscle-Specific Kinase (MuSK) Antibody Concentrations [ Time Frame: 12 months ]
      For patients with positive AChR or MuSK antibody tests, the change in antibody concentration will be a secondary outcome. Patients with MG will only be positive for one of these antibodies. Improvement is often seen with reduction in titer of > 50%, therefore patients that show an improvement in MG symptoms should show a reduction in antibody levels at 12 months as compared to baseline.


    Original Secondary Outcome: Same as current

    Information By: Johns Hopkins University

    Dates:
    Date Received: February 17, 2017
    Date Started: March 1, 2017
    Date Completion: January 2019
    Last Updated: February 17, 2017
    Last Verified: February 2017