Overview

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