Efficacy and Safety of IGIV-C in Corticosteroid Dependent Patients With Generalized Myasthenia Gravis

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 classification
of Class II, III, IV, or V historically

- At Screening, subjects may have symptoms controlled by CS. 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.

- At least 1 previous attempt to taper CS in order to minimize CS dose

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

- Evidence of malignancy or bulky thymoma potentially requiring surgical intervention
during the course of the trial

- Thymectomy within the preceding 3 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, ticlodipine)

- 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 levels <9 g/dL

Principal Investigator

Mazen Dimachkie, MD

Study Contact

Tina Liu 913-945-9932

Estimated Completion Date

Sunday, July 1, 2018

ClinicalTrials.gov #

NCT02473965
06/16/2015