A Study to Evaluate the Efficacy and Safety of IGIV-C in Symptomatic Subjects With Generalized Myasthenia Gravis

• Status: Completed
• Trial end date: 2018-01-01
• Target enrollment: 0
• Participant gender: All

Summary

The primary objective is to evaluate whether IGIV-C improves MG symptoms as compared to placebo in subjects with MG.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Grifols Therapeutics Inc.; Grifols Therapeutics LLC
• Treatments: Antibodies; gamma-Globulins; Immunoglobulins; Immunoglobulins, Intravenous; Rho(D) Immune Globulin

Criteria

Inclusion Criteria:

• Anti-acetylcholine receptor (AChR) antibody positive

• Confirmed diagnosis of generalized myasthenia gravis (MG).

• Myasthenia Gravis Foundation of America (MGFA) classification of Class II, III, or IVa inclusive at Screening.

• QMG >= 10 at Screening. Note: Subjects who only have a history of ocular MG may not enroll.

• Receiving standard of care MG treatment at a stable dose consisting of any one of the following for the time intervals delineated below (time intervals apply to medications and maintenance of stable dose level):

1. Cholinesterase inhibitor (pyridostigmine or equivalent) for at least 2 weeks prior to Screening and no immunosuppressants

2. Cholinesterase inhibitor (pyridostigmine or equivalent) for at least 2 weeks prior to Screening AND/OR only one of the following:

1. Prednisone (up to 60 mg/day or equivalent) for at least 2 months prior to Screening, OR

2. Azathioprine for at least 6 months prior to Screening, OR

3. Mycophenolate mofetil for at least 6 months prior to Screening, OR

4. Methotrexate for at least 6 months prior to Screening, OR

5. Cyclosporine or tacrolimus for at least 3 months prior to Screening

3. Cholinesterase inhibitor (pyridostigmine or equivalent) for at least 2 weeks prior to Screening AND/OR prednisone (up to 60 mg/day or equivalent) for at least one month prior to Screening and only one of the following:

1. Azathioprine for at least 6 months prior to Screening, OR

2. Mycophenolate mofetil for at least 6 months prior to Screening, OR

3. Methotrexate for at least 6 months prior to Screening, OR

4. Cyclosporine or tacrolimus for at least 3 months prior to Screening

Exclusion Criteria:

• Have received cyclophosphamide or any other immunosuppressive agent apart from the ones allowed per inclusion criteria within the past 6 months

• Any change in MG treatment regimen between Screening (Week -3, Visit 0) and Baseline (Week 0, Visit 1)

• Greater than two point change in QMG score, increased or decreased, between Screening (Week -3, Visit 0) and Baseline (Week 0, Visit 1)

• Any episode of myasthenic crisis in the one month prior to Screening

• 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

• Rituximab, belimumab, eculizumab or any monoclonal antibody used for immunomodulation within the past 12 months

• Have received immune globulin (Ig) treatment given by intravenous (IV), subcutaneous, or intramuscular route within the last 3 months

• Current known hyperviscosity or hypercoagulable state

• Currently receiving anti-coagulation therapy (vitamin K antagonists, nonvitamin K antagonist oral anticoagulants [e.g., dabigatran etexilate, rivaroxaban, edoxaban, and apixaban], parenteral anticoagulants [e.g., fondaparinux]). Note that oral anti-platelet agents are allowed (e.g., aspirin, clopidogrel, ticlodipine)

• Documented diagnosis of thrombotic complications to polyclonal intravenous immunoglobulin (IVIg) therapy in the past

• History of recent (within the last year) myocardial infarction or stroke

• Uncontrolled congestive heart failure; embolism; or historically documented (within the last year) electrocardiogram (ECG) changes indicative of myocardial ischemia or atrial fibrillation

• History of chronic alcoholism or illicit drug abuse (addiction) in the 12 months preceding the Screening/Week -3 (Visit 0)

• Plasma exchange (PLEX) performed within the last 3 months

• Renal impairment (i.e., serum creatinine exceeds more than 1.5 times the upper limit of normal [ULN] for the expected normal range for the testing laboratory).

• Hemoglobin levels less than 9 g per dL