Open Label Study of Subcutaneous Immunoglobulin (SCIg) in Myasthenia Gravis

NCT ID: NCT02100969

Last Updated: 2021-06-02

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 23 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-05-31
• Study Completion Date: 2018-01-31

Brief Summary

The purpose of this study is to determine whether Hizentra is a safe and effective treatment for people with myasthenia gravis (MG).

Detailed Description

Myasthenia gravis (MG) is a rare autoimmune disorder which causes the muscles to become weak because the immune system attacks the connection between the nerves and the muscles.

Hizentra is a subcutaneous (under the skin) immunoglobin (SCIg). An immunoglobin is a blood protein. Hizentra is being studied for the treatment of patients with MG. Hizentra is administered by an injection into the skin through a portable infusion pump, which may be easier for patients to administer than the current treatments.

Participants will be asked to complete 9 clinic visits and 3 telephone calls. It could take up to 30 weeks to complete all study visits.

Conditions

Myasthenia Gravis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

HIZENTRA ®

Hizentra is a subcutaneous (under the skin) immunoglobin (SCIg). Participants will receive Hizentra in a minimum of one infusion per week and a maximum of 4 infusions per week. Dose and rate depend on the visit and how each participant tolerates the drug. Max cc per site is 50 cc per site per hour.

Group Type: EXPERIMENTAL

HIZENTRA ®

Intervention Type: DRUG

Patients must fulfill inclusion criteria and remain stable at week 0, which means QMG does not increase by 3 points, will enter receive Hizentra for 12 weeks.

Interventions

HIZENTRA ®

Patients must fulfill inclusion criteria and remain stable at week 0, which means QMG does not increase by 3 points, will enter receive Hizentra for 12 weeks.

Intervention Type: DRUG

Other Intervention Names

Immune Globulin Subcutaneous (Human), 20% Liquid

Eligibility Criteria

Inclusion Criteria

• Patients 18 and older
• Patients must have prior or current documentation of MGFA MG grades 2, 3, or 4 generalized MG, according to the MGFA classification system.48 These grades correspond to mild (2), moderate (3), and severe (4)
• Elevated AChR or MuSK Ab. These tests will have been performed at some time prior to entry into the study. Double seronegative MG patients with prior documentation of an abnormal decrement (>10%) on slow repetitive nerve stimulation or an abnormal single fiber EMG will also be allowed to participate
• Patient's signs and symptoms should not be better explained by another disease process.
• IVIg maintenance dose of 0.2 to 2 gm/kg/4 weeks or equivalent dose administered Q 2-4 weeks±3days
• Stable IVIg for at least 3 cycles (definition of stability: no change in prescribed dosage or frequency by the treating physician)
• Patient must be receiving no more than 200g/4weeks of IVIg.
• Patients must be willing to complete the study and return for follow-up visits.
• Patients must be willing to give written informed consent before participating in this study. A copy of the signed consent must be kept in the patient's medical record.
• Patients can be on the following drugs as long as there has been no dose change for 60 days: azathioprine, cyclosporine, cyclophosphamide, mycophenolate mofetil, tacrolimus, methotrexate or other immunosuppressive drugs.
• Patients can be on prednisone as long as there has been no dose change for 30 days.

Exclusion Criteria

• MGFA grade V within 6 months of screening.
• A history of chronic degenerative, psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue.
• Other major chronic or debilitating illnesses within six months prior to study entry.
• Female patients who are premenopausal and are: (a) pregnant on the basis of a serum pregnancy test, (b) breast-feeding, or (c) not using an effective method of double barrier (1 hormonal plus 1 barrier method or 2 simultaneous barrier methods) birth control (birth control pills, male condom, female condom, intrauterine device, Norplant, tubal ligation, or other sterilization procedures).
• Altered levels of consciousness, dementia, or abnormal mental status.
• Thymectomy in the previous three months.
• Evidence of renal insufficiency (Cr>1.5 x elevated) or liver disease (transaminases > 2.5 x elevation) at screening.
• Skin disease that would interfere with assessment of injection site reaction
• History of severe reactions to IVIg or SCIg.
• Participation in a research study within the last 3 months
• Treatment with rituximab or other biologics within 12 months of study entry
• Inability to provide informed consent.
• History of thrombotic episodes within the last year prior to enrollment
• Known allergic or other severe reactions to blood products including intolerability to previous normal human immunoglobulin for intravenous administration (IVIG) and/or subcutaneous immunoglobulin (SCIG), such as history of clinically relevant hemolysis after IVIG infusion, aseptic meningitis, recurrent severe headache, hypersensitivity, severe generalized or severe local skin reaction.
• History of IgA deficiency or evidence of IgA deficiency at screening.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

CSL Behring

INDUSTRY

Sponsor Role: collaborator

Mazen Dimachkie, MD

OTHER

Sponsor Role: lead

Responsible Party

Mazen Dimachkie, MD

Professor

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Mazen M Dimachkie, MD

Role: PRINCIPAL_INVESTIGATOR

University of Kansas Medical Center

Locations

Phoenix Neurological Associates

Phoenix, Arizona, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

University at Buffalo

Buffalo, New York, United States

University of Texas Southwestern Medical Center

Dallas, Texas, United States

University of Toronto

Toronto, Ontario, Canada

Countries

United States; Canada

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

STUDY00001041

Identifier Type: -

Identifier Source: org_study_id