Subcutaneous Ig Maintenance Therapy for Myasthenia Gravis (SIMM)
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Subcutaneous Ig Maintenance Therapy for Myasthenia Gravis|
- To monitor number of participants completing the study for the six months period [ Time Frame: Baseline to 6 months ] [ Designated as safety issue: Yes ]To evaluate feasibility and tolerability of IGSC as a maintenance treatment of generalized MG as measured by treatment adherence and adverse events experienced by the patient. Patients will be asked if they have experienced any of the most commonly known side effects of Hizentra. A case report form detailing any adverse events will be included with every study visit and reviewed with the patient at each visit.
- To monitor number of adverse events in participants [ Time Frame: Baseline to 6 months ] [ Designated as safety issue: Yes ]•To evaluate safety IGSC for treatment of generalized MG as measured by adverse events experienced by each patient and reviewed monthly by the principal investigator.
- Number of participants able to decrease prednisone dose below 30 mgs [ Time Frame: Baseline to 6 months ] [ Designated as safety issue: No ]• To evaluate the potential steroid-sparing effect of IGSC treatment as measured by the total dose of prednisone required by each patient over the six month period.
- To monitor effect on manual muscle testing [ Time Frame: Baseline to 6 months ] [ Designated as safety issue: No ]• To evaluate the effect of IGSC treatment on symptoms associated with MG as measured by the QMG and MG-ADL.
- To measure changes on SF-36 quality of life measurement tool before and after completion of study [ Time Frame: Baseline to 6 months ] [ Designated as safety issue: No ]• To evaluate Quality of Life with IGSC treatment for MG as measured by the SF-36.
- To measure and correlate levels of serum IgG with clinical response of the participants [ Time Frame: Baseline to 6 months ] [ Designated as safety issue: No ]• To evaluate pharmacokinetics (IgG) levels associated with IGSC treatment for MG as measured by the monthly blood draws.
- To monitor minimal manifestation of Myasthenia Gravis [ Time Frame: Baseline to 6 months ] [ Designated as safety issue: No ]To evaluate the effect of IGSC treatment on symptoms associated with MG
- To monitor effect on Quantitative Myasthenia Gravis Score [ Time Frame: Baseline to 6 months ] [ Designated as safety issue: No ]To evaluate the effect of IGSC treatment on the Quantitative Myasthenia Score. Data collected will be qualitative and quantitative, and scores will be compared over time. Data will be captured on case report forms, and entered into an SPSS database. Dr. Gary Cutter, who is serving as a statistician for this study, will review the data monthly for safety, and a report will be sent to Dr. Hayat.
|Study Start Date:||October 2011|
|Estimated Study Completion Date:||December 2016|
|Estimated Primary Completion Date:||December 2016 (Final data collection date for primary outcome measure)|
Study population will include patients (18-80 years old) with non-thymomatous myasthenia gravis MGFA Class II-IV receiving a minimum of 30mg of Prednisone daily and no other immunosuppression and no more 240 mgs per day of Cholinesterase inhibitor. Patients will receive Subcutaneous immunoglobulins weekly for 6 months.
Drug: Subcutaneous immunoglobulins
Immunoglobulins used subcutaneously for maintenance of other immune mediated disorders.
Other Name: Hizentra
The study is a pilot study to ascertain the feasibility and tolerability of subcutaneous immunoglobulin (SCIG or IGSC) as a maintenance therapy for patients with non-thymomatous MG patients (MGFA class II-IV) at entry, aged 18-80 years, positive acetylcholine receptor antibody, receiving greater than 30mg of prednisone daily. Patients may or may not be receiving anticholinesterase agents.
The neurologist principal investigator at each site will have the overall responsibility for study performance is designated the medical coordinator (MC). The MC will assess patients from the sites clinic populations and identify potential subjects for inclusion and exclusion criteria. Once a subject is identified and provided informed consent to participate the Visit Schedule will be initiated.
At the initial visit the MC will perform the acetylcholine receptor antibody level, and record the prednisone and anticholinesterase doses. The MC will be responsible for assessment of adverse events. The research coordinator will arrange for the initial laboratory testing at the patient's local Quest, where the blood will be drawn. Baseline lab tests to be done will include IgA level to evaluate for deficiency, IgG level, CBC, AchR antibody, pregnancy tests in women, LFT's, PT/PTT and BUN/Creatinine. The patient will complete the SF-36 quality of life, MG, and MGFA ADL The research coordinator will be responsible for training subjects in performance of IGSC infusion. Subjects will have outpatient clinic assessments in one week and then monthly for the remainder of the study. Patients will receive 2gms/kg divided over 4 weeks initially and then will be given 250mgs/kg/wk for total of 6 months. This is similar to the standard IV treatment for patients which is 2 gm/kg given over 2-5 days for the initial dose. After the initial dose, a patient is started on monthly IV maintenance dose of 1 gm/kg each month given over 1-3 days.
The subject will be evaluated monthly for assessment of whether minimal manifestation (MM) status has been reached, which then allows reduction of corticosteroids by 5mg or more if clinically indicated. The MC will record adverse events and symptoms. The dose of anticholinesterase drugs will be decreased at the discretion of the MC. The prednisone dose will be decreased unless the MM status is lost; in that situation the prednisone dose will be increased 10mg every 2 weeks until the MM is again achieved. Titration of the prednisone and cholinesterase inhibitor medications will be at the discretion of the physician and will be based on the patient's symptoms as measured by symptoms and examination, leading to a determination of the MM. The patient will complete the SF-36 quality of life assessment.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01828294
|United States, District of Columbia|
|George Washington University|
|Washington, District of Columbia, United States, 20037|
|United States, Missouri|
|Saint Louis University|
|St. Louis, Missouri, United States, 63110|
|Principal Investigator:||Ghazala Hayat, M.D.||St. Louis University|
|Principal Investigator:||Jafar Kafaie, M.D.||St. Louis University|