Disease-Modifying Treatments for Myasthenia Gravis (NCT03490539) | Clinical Trial Compass
CompletedNot Applicable
Disease-Modifying Treatments for Myasthenia Gravis
United States, Canada82 participantsStarted 2018-05-07
Plain-language summary
This study is designed to address the evidence gaps in a real-world setting and help patients with MG choose treatments that are best suited to them. It is a prospective, multicenter observational cohort study of comparative effectiveness of MG treatments, with a patient-centered primary outcome measure, to guide clinicians, patients and payers regarding the choice of treatment options for this chronic and serious disease.
Primary: To compare the effectiveness of azathioprine (AZT) and mycophenolate mofetil (MMF).
Secondary: To compare the outcomes in patients receiving an adequate dose and duration of AZT or MMF over the 2-3 year study period, vs. patients not receiving adequate doses and duration of these agents
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Age ≥ 18 years of age
. Acquired autoimmune MG, with weakness and confirmed by one or more of the following:
. Elevated AChR or MuSK antibodies
. Unequivocal response to cholinesterase inhibitors
. Abnormal RNS or increased jitter (without nerve or muscle disease sufficient to produce a decrement or increased jitter)
. Patients seen initially at the participating center after January 1, 2017.
. Patients on pyridostigmine at the first evaluation at the participating center ("baseline visit") may be included if pyridostigmine was started ≤3 months before the baseline visit.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Participants With Improvement in Patient-Reported Myasthenia Gravis Quality of Life, 15, Revised ( MG-QOL15r)
Timeframe: Baseline, 24-36 months
2
Number of Participants With Improvement in Composite Outcome of Clinical Improvement and Adverse Effects
. Patients who received corticosteroids \>90 days prior to baseline visit for a non-MG indication may be included. (Patients who have received corticosteroids for a non-MG indication between 31 and 90 days before baseline visit will be evaluated by the primary investigators on a case by case basis to determine if the extent and dose of corticosteroid could have impacted the course of MG or symptoms of MG.)
Exclusion criteria
. Patients with non-autoimmune MG (congenital myasthenic syndromes, drug-induced MG)
. Patients on immunosuppressive agents at the baseline visit.
. Patients who have previously received steroids for the treatment of MG.
. Patients with steroid use for a non-MG indication \< 30 days prior to the baseline visit.
. Patients with previous thymectomy, IVIg or plasma exchange, or treatment with a non-steroidal immunosuppressive agent (azathioprine, mycophenolate mofetil cyclosporine, methotrexate, cyclophosphamide, tacrolimus, rituximab, or any investigational immunosuppressive agent). Patients who have outcomes measured within 24 hours after initiation of IVIg or PLEX are acceptable.