Efficacy and Safety of IGIV-C in Corticosteroid Dependent Patients With Generalized Myasthenia Gravis
Condition: Myasthenia Gravis
MeSH terms: Myasthenia Gravis; Muscle Weakness
Intervention: IGIV-C; Placebo
Intervention description: Run-Phase: 1 loading dose of 2 g/kg IGIV-C and 2 maintenance doses of 1 g/kg IGIV-C
Corticosteroid Tapering/IGIV-C Maintenance Phase: 1 g/kg IGIV-C every 3 weeks for up to 36 weeks
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 60.0
Sex: All
Minimum age: 18 Years
Maximum age: 85 Years
Healthy volunteers: No

Summary:

      This is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the
      efficacy and safety of Immune Globulin (Human), 10% Caprylate/Chromatography Purified
      (IGIV-C) as a corticosteroid (CS)-sparing agent in subjects with CS-dependent Myasthenia
      Gravis (MG).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Anti-acetylcholine receptor antibody positive

          -  Confirmed diagnosis of generalized MG historically meeting the clinical criteria for
             diagnosis of MG defined by the Myasthenia Gravis Foundation of America (MGFA)
             classification of Class II, III, IV, or V historically

          -  At Screening, subjects may have symptoms controlled by CS or were MGFA Class II-IVa
             inclusive (Class IVb and Class V excluded). Subjects who only have a history of ocular
             MG may not enroll.

          -  On systemic CS for a minimum period of at least 3 months and on a stable CS dose of
             >=15 mg/day and <=60 mg/day (prednisone equivalent) for the month prior to Screening.

          -  Had a tapering CS dose that the study investigator considered to be appropriate.

          -  At least 1 previous completed attempt to taper CS in order to minimize CS dose (lowest
             feasible dose based on observed MG signs and symptoms)

        Exclusion Criteria:

          -  Any dose change in concomitant immunosuppressant therapy, other than CS, in the prior
             6 months

          -  Any change in CS dose or acetylcholinesterase inhibitor (e.g., pyridostigmine) dose in
             the 1 month prior to Screening

          -  A 3-point change in Quantitative Myasthenia Gravis score, increased or decreased,
             between the Screening/Week -3 (Visit 0) and Baseline (Week 0 [Visit 1])

          -  Any episode of myasthenic crisis (MC) in the 1 month prior to Screening, or (at any
             time in the past) MC or hospitalization for MG exacerbation associated with a previous
             CS taper attempt

          -  Evidence of malignancy within the past 5 years (non-melanoma skin cancer, carcinoma in
             situ of cervix is allowed) or thymoma potentially requiring surgical intervention
             during the course of the trial (intent to perform thymectomy)

          -  Thymectomy within the preceding 6 months prior to Screening

          -  Rituximab, belimumab, eculizumab or any monoclonal antibody used for immunomodulation
             within the past 12 months prior to Screening

          -  Have received immune globulin treatment given by IV, subcutaneous, or intramuscular
             route within the last 3 months prior to Screening

          -  Received plasma exchange performed within the last 3 months prior to Screening

          -  History of anaphylactic reactions or severe reactions to any blood-derived product

          -  History of recent (within the last year) myocardial infarction or stroke

          -  Uncontrolled congestive heart failure; embolism; or historically documented (within
             the last year) electrocardiogram changes indicative of myocardial ischemia or atrial
             fibrillation

          -  Current known hyperviscosity or hypercoagulable state

          -  Currently receiving anti-coagulation therapy. Oral anti-platelet agents are allowed
             (e.g., aspirin, clopidogrel, ticlopidine)

          -  Females of child-bearing potential who are pregnant, have a positive serum pregnancy
             test, breastfeeding, or are unwilling to practice a highly effective method of
             contraception throughout the study.

          -  Renal impairment

          -  Aspartate aminotransferase or alanine aminotransferase levels exceeding more than 2.5
             times the upper limit of normal for the expected normal range for the testing
             laboratory.

          -  Hemoglobin (Hb) levels <9 g/dL