NCT03490539

Disease-Modifying Treatments for Myasthenia Gravis

Prospective Multicenter Observational Cohort Study of Comparative Effectiveness of Disease-modifying Treatments for Myasthenia Gravis

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 167 (actual)
• Sponsor: Duke University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Detailed description

Design \& procedures - This is an observational study in the real world clinical setting to evaluate immunosuppressive treatment (IS) of myasthenia gravis (MG). Patients with acquired autoimmune MG ≥ 18 years of age who are not on IS agents, and have not been on corticosteroids for at least 30 days will be enrolled at 20 sites in the US and Canada. These patients will be treated according to the physician's judgment and patient preferences as in routine clinical practice. Patients will be followed prospectively, with the frequency of clinical visits and laboratory monitoring determined by the treating physician, which may differ among patients. Standard outcome measures measuring efficacy and adverse effects that are used in clinical practice will be collected, with emphasis on patient reported outcomes. Informed consent will be obtained using an approved consent form. Patient identifiable / clinical information from the medical record, including the study outcome measures will be uploaded to a centralized REDCap database. The investigators plan to recruit 220 patients, adjusting for a 10% drop out rate, with a final sample of 200 patients for analysis.

Conditions

• Neurological Disorder
• Autoimmune Diseases

Interventions

Type	Name	Description
DRUG	Mycophenolate Mofetil	oral tablets
DRUG	Azathioprine	oral tablet

Timeline

• Start date: 2018-05-07
• Primary completion: 2021-01-31
• Completion: 2021-01-31
• First posted: 2018-04-06
• Last updated: 2021-02-03

Locations

19 sites across 2 countries: United States, Canada