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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02473965
• Date of registration: 14/06/2015
• Prospective Registration: No
• Primary sponsor: Grifols Therapeutics LLC
• Public title: Efficacy and Safety of IGIV-C in Corticosteroid Dependent Patients With Generalized Myasthenia Gravis
• Scientific title: A Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of lGIV-C as a Corticosteroid Sparing Agent in Corticosteroid Dependent Patients With Generalized Myasthenia Gravis
• Date of first enrolment: June 2015
• Target sample size: 60
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02473965
• 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 antibody positive

- Confirmed diagnosis of generalized MG historically meeting the clinical criteria for
diagnosis of MG defined by the Myasthenia Gravis Foundation of America (MGFA)
classification of Class II, III, IV, or V historically

- At Screening, subjects may have symptoms controlled by CS or were MGFA Class II-IVa
inclusive (Class IVb and Class V excluded). 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.

- Had a tapering CS dose that the study investigator considered to be appropriate.

- At least 1 previous completed attempt to taper CS in order to minimize CS dose (lowest
feasible dose based on observed MG signs and symptoms)

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, or (at any
time in the past) MC or hospitalization for MG exacerbation associated with a previous
CS taper attempt

- 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 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, ticlopidine)

- 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 (Hb) levels <9 g/dL



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

Health Condition(s) or Problem(s) studied

Myasthenia Gravis

Intervention(s)

Drug: IGIV-C

Drug: Placebo

Primary Outcome(s)

Percent of Subjects Achieving a 50% or Greater Reduction in CS Dose (Prednisone or Equivalent) From Baseline to Week 39 [Time Frame: Baseline/Week 0 (Visit 1) and Week 39 (Visit 14).]

Secondary Outcome(s)

Mean Percent Change in Daily CS Dose (Prednisone or Equivalent) From Baseline to Week 39 [Time Frame: Baseline/Week 0 (Visit 1) and Week 39 (Visit 14).]

Median Time to First Episode of MG Worsening [Time Frame: From Baseline/Week 0 (Visit 1) to Week 39 (Visit 14).]

Secondary ID(s)

GTI1306

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 26/02/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02473965