A Multicenter, Prospective, Open-label, Non-controlled Clinical Trial to Assess the Efficacy and Safety of Immune Globulin (Human), 10% Caprylate/Chromatography Purified (IGIV-C) in Patients With Myasthenia Gravis Exacerbations

Grifols Therapeutics LLC31 sites in 12 countries49 target enrollmentMarch 2015

ConditionsMyasthenia Gravis Exacerbations

Overview

Phase: Phase 3
Intervention: Not specified
Conditions: Myasthenia Gravis Exacerbations
Sponsor: Grifols Therapeutics LLC
Enrollment: 49
Locations: 31
Primary Endpoint: Change in Quantitative Myasthenia Gravis (QMG) Scale Score
Status: Completed
Last Updated: 6 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

This was a multicenter, prospective, open-label, non-controlled study to assess the efficacy and safety of an IV dose of 2 g/kg of IGIV-C in subjects with MG exacerbations.

Detailed Description

The study consisted of a single dose course of IGIV-C treatment followed by 28-days of post-infusion assessments. The total duration of study participation for each subject was up to 28 ± 2 days. Approximately 50 subjects, ages 18 or greater, were planned to be enrolled in the study and receive a single, total dose of 2 g/kg of IGIV-C over 2 consecutive days (dose of 1 g/kg per day) across multiple centers in Argentina, Canada, Europe, and South Africa.

Registry

clinicaltrials.gov

Start Date

March 2015

End Date

April 2018

Last Updated

6 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Grifols Therapeutics LLC

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Was male or female aged ≥18 years.
•Subjects must be willing and able to provide written informed consent (if applicable, a legally authorized representative may provide informed consent on behalf of the subject).
•Subjects who met the clinical criteria for diagnosis of MG with an exacerbation defined as worsening of MG symptoms as defined by an Myasthenia Gravis Foundation of America (MGFA) classification IVb or V.
•Subjects on long-term (8 weeks) corticosteroid treatment for MG.
•Female subjects of child-bearing potential must have a negative test for pregnancy (human chorionic gonadotropin \[HCG\]-based assay).
•Subjects must be willing to comply with all aspects of the clinical trial protocol, including blood sampling and long-term storage of extra samples, for the entire duration of the study.

Exclusion Criteria

•Subjects who had received immune globulin treatment given by IV, subcutaneous or intramuscular route within the last 30 days.
•Subjects with documentation of a lack of clinical response to intravenous immunoglobulin (IVIg) therapy for MG.
•Subjects documented positive for antibodies directed against Muscle specific kinase (MuSK).
•Subjects with corticosteroid (CS) treatment initiated within the last 8 weeks or modified within the last 2 weeks.
•Subjects with plasma exchange (PLEX) within the last 30 days.
•Subjects with MG exacerbation attributable to change in medication or infection or evident infection as defined by, but not limited to, the presence of at least one of the following diagnostic features: 1) axillary temperature ≥38°C, 2) positive blood culture of infective microorganism, 3) white blood cell count \>12×10\^9/L and differential white blood cell count of \>10% band neutrophils (\>1.2×10\^9/L), and 4) pulmonary infiltrate with consolidation on chest X-ray. Alternatively, other signs and symptoms may be considered for the diagnosis of evident infection according to the Investigator's judgement.
•Subjects with inadequate venous access.
•Subjects with a history of anaphylactic reactions or severe reactions to any blood-derived product.
•Subjects with a history of intolerance to any component of the investigational products.
•Subjects with a documented diagnosis of thrombotic complications to polyclonal IVIG therapy in the past.

Outcomes

Primary Outcomes

Change in Quantitative Myasthenia Gravis (QMG) Scale Score

Time Frame: From Baseline (Day 0) to Day 14

Mean Change in Quantitative Myasthenia Gravis (QMG) Scale Score from Baseline (Day 0) to Day 14. The minimum and maximum scores of the QMG Scale are 0 and 39, respectively, and a higher score means a worse outcome.

Secondary Outcomes

Percentage of Subjects With Clinical Improvement Assessed by QMG(Baseline (Day 0) to Day 14)
Percentage of Subjects With Clinical Improvement Assessed by MG-Activities of Daily Living (MG-ADL) Scale(Baseline (Day 0) to Day 14)
Percentage of Subjects With Clinical Improvement Assessed by the MG Composite(Baseline (Day 0) to Day 14)