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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02473952
• Date of registration: 14/06/2015
• Prospective Registration: Yes
• Primary sponsor: Grifols Therapeutics LLC
• Public title: A Study to Evaluate the Efficacy and Safety of IGIV-C in Symptomatic Subjects With Generalized Myasthenia Gravis
• Scientific title: A Multi-center, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Immune Globulin (Human), 10% Caprylate/Chromatography Purified (IGIV-C) in Symptomatic Subjects With Generalized Myasthenia Gravis
• Date of first enrolment: August 2015
• Target sample size: 62
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02473952
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Belgium, Canada, Czech Republic, Czechia, Estonia, France, Germany, Hungary, Lithuania, Poland, United States

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 85 Years
Gender: All

Health Condition(s) or Problem(s) studied

Myasthenia Gravis, Generalized

Intervention(s)

Drug: IGIV-C

Drug: Placebo

Primary Outcome(s)

Improvement in Myasthenia Gravis (MG) Symptoms as Measured by the Mean Change in Quantitative Myasthenia Gravis (QMG) Total Score. [Time Frame: Baseline (Week 0) to Week 24]

Secondary ID(s)

GTI1408

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 05/03/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02473952