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Neurological DisorderPROMISE-MGNCT03490539

Disease-Modifying Treatments for Myasthenia Gravis

An observational study of Neurological Disorder and Autoimmune Diseases, run by Duke University at 19 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Neurological Disorder, Autoimmune Diseases
What is being tested?
Mycophenolate Mofetil and Azathioprine
Type of treatment
Pill or oral medicine
How many people
82 participants
Where
19 sitesin 2 countries
Study dates
7 May 2018to 31 Jan 2021
How long will I take part?
3 Years
Phase
Observational
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Study population: MG patients with autoimmune MG

Who may be able to join

  • 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)
Show all 9Show fewer
  • 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.)

Who cannot join

  • 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.

Trial locations and contacts

19 sites in 2 countries.

19 sites in 2 countries

United States17 sites

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Brigham and Women's Hospital

    Boston, Massachusetts, 02115

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts, 02215

  • St. Elizabeth's Medical Center

    Brighton, Massachusetts, 02135

  • University at Buffalo, SUNY

    Buffalo, New York, 14202

  • University of Vermont - Larner College of Medicine

    Burlington, Vermont, 05401

  • University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27599

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

  • Rush University Medical Center

    Chicago, Illinois, 60612

  • Cleveland Clinic

    Cleveland, Ohio, 44195

  • Duke University

    Durham, North Carolina, 27710

  • Unversity of Miami

    Miami, Florida, 33136

  • Ochsner Clinic Foundation

    New Orleans, Louisiana, 70121

  • Stanford University

    Palo Alto, California, 84303

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

  • University of Pittsburgh

    Pittsburgh, Pennsylvania, 15213

  • University of Rochester Medical Center

    Rochester, New York, 14642

Other trials in United States

Canada2 sites

  • University of Alberta Hospital, Faculty of Medicine

    Edmonton, Alberta, T6G2G3

  • London Health Sciences Centre

    London, Ontario, N6A5A5

Other trials in Canada

About this study

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

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

Read the 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.

Being tested
Mycophenolate Mofetil, Azathioprine
Keywords
observational, comparative effectiveness, myasthenia gravis
Medical subject headings
Nervous System Diseases, Autoimmune Diseases, Myasthenia Gravis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT03490539
Study groupRoleWhat they receive
azathioprine (AZT)Patients with MG who are receiving azathioprine as part of routine clinical care
Receives: Drug: Azathioprine
mycophenolate mofetil (MMF)Patients with MG who are receiving mycophenolate mofetil as part of routine clinical care
Receives: Drug: Mycophenolate Mofetil

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants With Improvement in Patient-Reported Myasthenia Gravis Quality of Life, 15, Revised ( MG-QOL15r)

    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

    Measured: Baseline, 24-36 months

  2. Number of Participants With Improvement in Composite Outcome of Clinical Improvement and Adverse Effects

    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".

    Measured: Baseline, 24-36 months

Secondary outcomes (4)

  1. Number of Participants With Improvement in Myasthenia Gravis Composite (MGC) Scores

    10 item scale of patient-reported functions and clinician-reported examination findings. Scores range from 0-50 (0- normal and 50- most severe)

    Measured: Baseline, 24-36 months

  2. Number of Participants With Improvement in Myasthenia Gravis Activities of Daily Living Scale (MG-ADL)

    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

    Measured: Baseline, 24-36 months

  3. Number of Participants With Improvement in Myasthenia Gravis Manual Muscle Test Scores (MG-MMT)

    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

    Measured: Baseline, 24-36 months

  4. Change in Number of Participants Who Were Hospitalized for Myasthenia Gravis (MG)

    Measured: Baseline, 24-36 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Apr 2018
  2. Study start7 May 2018
  3. Primary completion31 Jan 2021
  4. Study completion31 Jan 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
82 participants (actual)
Target duration
3 Years
IDs, dates and publications
ClinicalTrials.gov ID
NCT03490539
Sponsor's protocol ID
Pro00087883
Acronym
PROMISE-MG
Status last verified
May 2026
First submitted
21 Mar 2018
Last updated
18 Jun 2026
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Deenen JC, Horlings CG, Verschuuren JJ, Verbeek AL, van Engelen BG. The Epidemiology of Neuromuscular Disorders: A Comprehensive Overview of the Literature. J Neuromuscul Dis. 2015;2(1):73-85.

    PubMed 28198707

  • Sanders DB, Wolfe GI, Narayanaswami P. Author response: International consensus guidance for management of myasthenia gravis: Executive summary. Neurology. 2017 Jan 31;88(5):505-506. doi: 10.1212/WNL.0000000000003570. No abstract available.

    PubMed 28138082

  • Burns TM, Sadjadi R, Utsugisawa K, Gwathmey KG, Joshi A, Jones S, Bril V, Barnett C, Guptill JT, Sanders DB, Hobson-Webb L, Juel VC, Massey J, Gable KL, Silvestri NJ, Wolfe G, Cutter G, Nagane Y, Murai H, Masuda M, Farrugia ME, Carmichael C, Birnbaum S, Hogrel JY, Nafissi S, Fatehi F, Ou C, Liu W, Conaway M. International clinimetric evaluation of the MG-QOL15, resulting in slight revision and subsequent validation of the MG-QOL15r. Muscle Nerve. 2016 Dec;54(6):1015-1022. doi: 10.1002/mus.25198. Epub 2016 Nov 7.

    PubMed 27220659

  • Mullins LL, Carpentier MY, Paul RH, Sanders DB; Muscle Study Group. Disease-specific measure of quality of life for myasthenia gravis. Muscle Nerve. 2008 Aug;38(2):947-56. doi: 10.1002/mus.21016.

    PubMed 18697209

  • Burns TM, Grouse CK, Wolfe GI, Conaway MR, Sanders DB; MG Composite and MG-OL15 Study Group. The MG-QOL15 for following the health-related quality of life of patients with myasthenia gravis. Muscle Nerve. 2011 Jan;43(1):14-8. doi: 10.1002/mus.21883.

    PubMed 21082698

  • Jaretzki A 3rd, Barohn RJ, Ernstoff RM, Kaminski HJ, Keesey JC, Penn AS, Sanders DB. Myasthenia gravis: recommendations for clinical research standards. Task Force of the Medical Scientific Advisory Board of the Myasthenia Gravis Foundation of America. Neurology. 2000 Jul 12;55(1):16-23. doi: 10.1212/wnl.55.1.16. No abstract available.

    PubMed 10891897

  • Burns TM, Conaway M, Sanders DB; MG Composite and MG-QOL15 Study Group. The MG Composite: A valid and reliable outcome measure for myasthenia gravis. Neurology. 2010 May 4;74(18):1434-40. doi: 10.1212/WNL.0b013e3181dc1b1e.

    PubMed 20439845

  • Sadjadi R, Conaway M, Cutter G, Sanders DB, Burns TM; MG Composite MG-QOL15 Study Group. Psychometric evaluation of the myasthenia gravis composite using Rasch analysis. Muscle Nerve. 2012 Jun;45(6):820-5. doi: 10.1002/mus.23260.

    PubMed 22581534

  • Burns TM, Conaway MR, Cutter GR, Sanders DB; Muscle Study Group. Construction of an efficient evaluative instrument for myasthenia gravis: the MG composite. Muscle Nerve. 2008 Dec;38(6):1553-62. doi: 10.1002/mus.21185.

    PubMed 19016543

  • Wolfe GI, Herbelin L, Nations SP, Foster B, Bryan WW, Barohn RJ. Myasthenia gravis activities of daily living profile. Neurology. 1999 Apr 22;52(7):1487-9. doi: 10.1212/wnl.52.7.1487.

    PubMed 10227640

  • Sanders DB, Tucker-Lipscomb B, Massey JM. A simple manual muscle test for myasthenia gravis: validation and comparison with the QMG score. Ann N Y Acad Sci. 2003 Sep;998:440-4. doi: 10.1196/annals.1254.057. No abstract available.

    PubMed 14592912

  • Gilhus NE, Verschuuren JJ. Myasthenia gravis: subgroup classification and therapeutic strategies. Lancet Neurol. 2015 Oct;14(10):1023-36. doi: 10.1016/S1474-4422(15)00145-3.

    PubMed 26376969

  • Bang H, Robins JM. Doubly robust estimation in missing data and causal inference models. Biometrics. 2005 Dec;61(4):962-73. doi: 10.1111/j.1541-0420.2005.00377.x.

    PubMed 16401269

  • Li F, Morgan KL, Zaslavsky AM. Balancing Covariates via Propensity Score Weighting. Journal of the American Statistical Association 2016;(In press).

  • Crump RK. Dealing with limited overlap in estimation of average treatment effects. Biometrika 2009;96(1):187-199.

Questions about this trial

What is this clinical trial studying?

An observational study of Neurological Disorder and Autoimmune Diseases, run by Duke University at 19 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 18 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 19 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Yes. Results are posted in the registry (first posted 18 Jun 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Improvement in Patient-Reported Myasthenia Gravis Quality of Life, 15, Revised ( MG-QOL15r) (time frame: Baseline, 24-36 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03490539, which also lists 17 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jun 2026.

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