Subcutaneous Immunoglobulin for Myasthenia Gravis
Subcutaneous Immunoglobulin for Myasthenia Gravis
This is a prospective open-label, randomized, parallel arm clinical trial.

 The primary objective of the study is to evaluate the safety and efficacy of Cuvitru 20%
 subcutaneous immunoglobulin in patients with myasthenia gravis (MG). The secondary objective
 is to evaluate patient preferences and effects on quality of life when treating MG patients
 with SCIG. Exploratory objectives are to compare de novo administration starting SCIG
 directly with those starting with a loading dose of IVIG followed by SCIG administration.

 Patients over age 18 with moderate to severe MG with MGFA Class II-IV without
 contraindications to immunoglobulin will be considered for the study.

 All patients will be eligible to enter either arm of the study, Arm 1: 10% Gammagard IVIG
 followed by 20% Cuvitry SCIG and Arm 2: Cuvitru 20% SCIG alone.
Myasthenia gravis (MG) is an autoimmune neuromuscular condition which can cause fatigable
 weakness of skeletal muscles including bulbar, ocular, limb, axial and respiratory muscles.
 Symptoms range from mild, transient double vision and ptosis to severe, life threatening
 diffuse weakness, and treatments which are immunosuppressive or immunomodulatory have been
 shown to improve outcome in MG. This includes 10% intravenous immunoglobulin (IVIG), which
 has previous been shown by our group to improve strength at 2 and 4 weeks in patients with
 MG. Due to delay in the onset of many immunosuppressives and potential side effects from
 steroids and other immunosuppressive medications, IVIG is also used in clinical practice as
 bridging of maintenance therapy until other therapies take effect or the patient stabilizes.
 In addition, there have been other studies suggesting that 20% subcutaneous immunoglobuin
 (SCIG) can be helpful to improve strength in patients with MG over a relatively short period
 of time (6 weeks). Some of these studies are ongoing and are further evaluating the ability
 of IVIG and SCIG to stabilize or improve strength IVIG in patients with MG, however, there is
 no current published study evaluating different administration routes of immunoglobulin (IVIG
 and SCIG) in patients with MG over a longer 6 month follow-up period.

 The current study aims to be the first to evaluate 20% Cuvitru SCIG formulation in patients
 with neuromuscular conditions including MG. The study aim is to evaluate the safety and
 efficacy of 6 months of 20% SCIG Cuvitru treatment vs three months of 10% IVIG
 "pre-treatment" followed by 3 months of 20% SCIG Cuvitru treatment. The study will also be
 the first to use the myasthenia gravis impairment index (MGII) as primary outcome in
 conjunction with standard MG outcomes.
Inclusion Criteria:

 1. Patients over age 18

 2. Patients with a confirmed diagnosis of myasthenia gravis based on clinical criteria
 including fatiguable weakness and supported by either serological (acetylcholine
 receptor, muscle specific kinase or anti-low-density lipoprotein receptor- related
 protein 4 antibodies or electrophysiological testing (repetitive nerve stimulation of
 single fiber electromyography)

 3. Myasthenia Gravis Federation of America class II-IV

 4. Moderate to severe myasthenia gravis as defined by a quantitative myasthenia gravis
 score >10 or generalized myasthenia gravis impairment index score > 11

 5. Patient able to give consent and is able and willing to complete all study procedures
 and activities

 Exclusion Criteria:

 1. Patients who are pregnant or breastfeeding

 2. Patients not able to complete the study procedures or with an alternate diagnosis

 3. Patients with recent thymectomy in the past 6 months

 4. Patients receiving another biologic agent such as rituximab, belimubab,
 cyclophosphamide and eculizumab in the past 6 months prior to study entry

 5. No IVIG or subcutaneous immunoglobulin within the past month

 6. Patients on prednisone who have had alterations in prednisone dose over the past month
 prior to study entry

 7. Patients will previous known allergy or severe adverse reaction to intravenous or
 subcutaneous immunoglobulin

 8. Evidence of renal insufficiency (Cr>1.5 x elevated) or liver disease (transaminases >
 2.5 x elevation) at screening.