Disease-Modifying Treatments for Myasthenia Gravis (PROMISE-MG)
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|ClinicalTrials.gov Identifier: NCT03490539|
Recruitment Status : Completed
First Posted : April 6, 2018
Last Update Posted : February 3, 2021
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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
|Condition or disease||Intervention/treatment|
|Neurological Disorder Autoimmune Diseases||Drug: Mycophenolate Mofetil Drug: Azathioprine|
|Study Type :||Observational [Patient Registry]|
|Actual Enrollment :||167 participants|
|Target Follow-Up Duration:||3 Years|
|Official Title:||Prospective Multicenter Observational Cohort Study of Comparative Effectiveness of Disease-modifying Treatments for Myasthenia Gravis|
|Actual Study Start Date :||May 7, 2018|
|Actual Primary Completion Date :||January 31, 2021|
|Actual Study Completion Date :||January 31, 2021|
Patients with MG who are receiving azathioprine as part of routine clinical care
Other Name: Imuran
mycophenolate mofetil (MMF)
Patients with MG who are receiving mycophenolate mofetil as part of routine clinical care
Drug: Mycophenolate Mofetil
Other Name: Cellcept
- Change in Patient-Reported Myasthenia Gravis Quality of Life, 15, revised ( MG-QOL15r) [ Time Frame: Baseline, 24-36 months ]Measures MG symptoms, physical, social and emotional functioning related to MG, with 15 items, 3 response option, 0-2 for each item, Total score range 0-30, higher scores indicating worse function
- Change in composite outcome of clinical improvement and adverse effects [ Time Frame: Baseline, 24-36 months ]
measured by a composite of clinical improvement and adverse effects of treatments. Clinical improvement: achievement of MGFA Post-Intervention Status (PIS) Minimal Manifestation Status (MM) or better, defined below.
Adverse effects end point: no more than Grade 1 CTCAE (Common Terminology Criteria for Adverse Events) medication side-effects, defined below.
MGFA PIS- MM: the patient has no symptoms or functional limitations from MG but has some weakness on examination of some muscles
CTCAE: list of adverse event (AE) terms commonly encountered in oncology but is useful to monitor the side effects of any intervention Each AE term is defined and graded on a 1 to 5 scale indicating the severity of the AE, 1 representing the mildest side effect and 5 representing death Grade 1 CTCAE side-effects: "asymptomatic or only mild symptoms; intervention not indicated"
- Change in Myasthenia gravis composite (MGC) scores [ Time Frame: Baseline, 24-36 months ]
10 item scale of patient-reported functions and clinician-reported examination findings.
Scores range from 0-50 (0- normal and 50- most severe)
- Change in Myasthenia Gravis Activities of Daily Living Scale (MG-ADL) [ Time Frame: Baseline, 24-36 months ]Patient-reported 8- item questionnaire evaluating commonly reported symptoms in MG on a 4 response scale from 0-3 (0 - normal, 3- highest disability) Range 0-24, higher score is worse
- Change in Myasthenia Gravis Manual Muscle Test scores (MG-MMT) [ Time Frame: Baseline, 24-36 months ]Clinician-assessed scale of 18 muscle functions commonly affected by MG, each graded from 0 (normal) to 4 (paralyzed/unable to perform), Range 0-120, higher score reflects worse function
- Change in Visual Analogue Scale (VAS) for Disease Severity [ Time Frame: Baseline, 24-36 months ]Patient perception of disease severity measured in millimeters on a 100 mm line. Higher number indicates more severe disease
- Change in Visual Analogue Scale (VAS) for Treatment Side effects [ Time Frame: Baseline, 24-36 months ]patient perception of side effects of treatment measured in millimeters on a 100 mm line. Higher number indicates worse side effects
- Change in number of hospitalizations for MG [ Time Frame: Baseline, 24-36 months ]counts of hospitalizations for MG- higher count is worse
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
|Sampling Method:||Non-Probability Sample|
Participants eligible for inclusion in this study must fulfill all of the following 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.
- 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.)
Patients fulfilling any of the following criteria are not eligible for inclusion in this study. No additional exclusions may be applied by the investigator, in order to ensure that the study population will be representative of all eligible participants.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03490539
|Principal Investigator:||Jeffery Guptill, MD||Duke University|
|Principal Investigator:||Donald Sanders, MD||Duke University|
|Principal Investigator:||Pushpa Narayanaswami, MD||Beth Israel Deaconess Medical Center|
|Responsible Party:||Duke University|
|Other Study ID Numbers:||
|First Posted:||April 6, 2018 Key Record Dates|
|Last Update Posted:||February 3, 2021|
|Last Verified:||February 2021|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
Nervous System Diseases
Immune System Diseases
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
Autoimmune Diseases of the Nervous System
Neuromuscular Junction Diseases
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs