Trial Outcomes & Findings for Disease-Modifying Treatments for Myasthenia Gravis (NCT NCT03490539)
NCT ID: NCT03490539
Last Updated: 2026-06-18
Results Overview
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
COMPLETED
82 participants
Baseline, 24-36 months
2026-06-18
Participant Flow
Participant milestones
| Measure |
Azathioprine (AZT)
Patients with MG who are receiving azathioprine as part of routine clinical care
Azathioprine: oral tablet
|
Mycophenolate Mofetil (MMF)
Patients with MG who are receiving mycophenolate mofetil as part of routine clinical care
Mycophenolate Mofetil: oral tablets
|
|---|---|---|
|
Overall Study
STARTED
|
34
|
48
|
|
Overall Study
COMPLETED
|
34
|
48
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Disease-Modifying Treatments for Myasthenia Gravis
Baseline characteristics by cohort
| Measure |
Azathioprine (AZT)
n=34 Participants
Patients with MG who are receiving azathioprine as part of routine clinical care
Azathioprine: oral tablet
|
Mycophenolate Mofetil (MMF)
n=48 Participants
Patients with MG who are receiving mycophenolate mofetil as part of routine clinical care
Mycophenolate Mofetil: oral tablets
|
Total
n=82 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=20 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
20 Participants
n=20 Participants
|
24 Participants
n=20 Participants
|
44 Participants
n=40 Participants
|
|
Age, Categorical
>=65 years
|
14 Participants
n=20 Participants
|
24 Participants
n=20 Participants
|
38 Participants
n=40 Participants
|
|
Age, Continuous
|
67 years
n=20 Participants
|
67 years
n=20 Participants
|
67 years
n=40 Participants
|
|
Sex: Female, Male
Female
|
16 Participants
n=20 Participants
|
12 Participants
n=20 Participants
|
28 Participants
n=40 Participants
|
|
Sex: Female, Male
Male
|
18 Participants
n=20 Participants
|
36 Participants
n=20 Participants
|
54 Participants
n=40 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=20 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
0 Participants
n=20 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
34 Participants
n=20 Participants
|
48 Participants
n=20 Participants
|
82 Participants
n=40 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=20 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=20 Participants
|
1 Participants
n=20 Participants
|
2 Participants
n=40 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=20 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=20 Participants
|
1 Participants
n=20 Participants
|
1 Participants
n=40 Participants
|
|
Race (NIH/OMB)
White
|
32 Participants
n=20 Participants
|
46 Participants
n=20 Participants
|
78 Participants
n=40 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=20 Participants
|
0 Participants
n=20 Participants
|
0 Participants
n=40 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=20 Participants
|
0 Participants
n=20 Participants
|
1 Participants
n=40 Participants
|
|
Region of Enrollment
Canada
|
6 participants
n=20 Participants
|
8 participants
n=20 Participants
|
14 participants
n=40 Participants
|
|
Region of Enrollment
United States
|
28 participants
n=20 Participants
|
40 participants
n=20 Participants
|
68 participants
n=40 Participants
|
|
Number of Participants with Hypertension
|
15 Participants
n=20 Participants
|
25 Participants
n=20 Participants
|
40 Participants
n=40 Participants
|
|
Number of Participants with Diabetes Mellitus
|
4 Participants
n=20 Participants
|
6 Participants
n=20 Participants
|
10 Participants
n=40 Participants
|
|
Number of Participants with Psychiatric Illness
|
1 Participants
n=20 Participants
|
3 Participants
n=20 Participants
|
4 Participants
n=40 Participants
|
|
Number of Participants with Cancer
|
2 Participants
n=20 Participants
|
8 Participants
n=20 Participants
|
10 Participants
n=40 Participants
|
|
Number of Participants with Elevated AChR Binding Antibodies
|
1 Participants
n=20 Participants
|
1 Participants
n=20 Participants
|
2 Participants
n=40 Participants
|
|
Number of Participants with Elevated Thymoma
|
2 Participants
n=20 Participants
|
3 Participants
n=20 Participants
|
5 Participants
n=40 Participants
|
|
Number of Participants with Late Onset MG (> age 50)
|
29 Participants
n=20 Participants
|
43 Participants
n=20 Participants
|
72 Participants
n=40 Participants
|
PRIMARY outcome
Timeframe: Baseline, 24-36 monthsPopulation: Participants who are taking adequate dose of either medication
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
Outcome measures
| Measure |
Azathioprine (AZT)
n=34 Participants
Patients with MG who are receiving azathioprine as part of routine clinical care
Azathioprine: oral tablet
|
Mycophenolate Mofetil (MMF)
n=48 Participants
Patients with MG who are receiving mycophenolate mofetil as part of routine clinical care
Mycophenolate Mofetil: oral tablets
|
|---|---|---|
|
Number of Participants With Improvement in Patient-Reported Myasthenia Gravis Quality of Life, 15, Revised ( MG-QOL15r)
|
22 Participants
|
36 Participants
|
PRIMARY outcome
Timeframe: Baseline, 24-36 monthsMeasured 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".
Outcome measures
| Measure |
Azathioprine (AZT)
n=34 Participants
Patients with MG who are receiving azathioprine as part of routine clinical care
Azathioprine: oral tablet
|
Mycophenolate Mofetil (MMF)
n=48 Participants
Patients with MG who are receiving mycophenolate mofetil as part of routine clinical care
Mycophenolate Mofetil: oral tablets
|
|---|---|---|
|
Number of Participants With Improvement in Composite Outcome of Clinical Improvement and Adverse Effects
|
22 Participants
|
36 Participants
|
SECONDARY outcome
Timeframe: Baseline, 24-36 months10 item scale of patient-reported functions and clinician-reported examination findings. Scores range from 0-50 (0- normal and 50- most severe)
Outcome measures
| Measure |
Azathioprine (AZT)
n=34 Participants
Patients with MG who are receiving azathioprine as part of routine clinical care
Azathioprine: oral tablet
|
Mycophenolate Mofetil (MMF)
n=48 Participants
Patients with MG who are receiving mycophenolate mofetil as part of routine clinical care
Mycophenolate Mofetil: oral tablets
|
|---|---|---|
|
Number of Participants With Improvement in Myasthenia Gravis Composite (MGC) Scores
|
27 Participants
|
44 Participants
|
SECONDARY outcome
Timeframe: Baseline, 24-36 monthsPatient-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
Outcome measures
| Measure |
Azathioprine (AZT)
n=34 Participants
Patients with MG who are receiving azathioprine as part of routine clinical care
Azathioprine: oral tablet
|
Mycophenolate Mofetil (MMF)
n=48 Participants
Patients with MG who are receiving mycophenolate mofetil as part of routine clinical care
Mycophenolate Mofetil: oral tablets
|
|---|---|---|
|
Number of Participants With Improvement in Myasthenia Gravis Activities of Daily Living Scale (MG-ADL)
|
28 Participants
|
43 Participants
|
SECONDARY outcome
Timeframe: Baseline, 24-36 monthsClinician-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
Outcome measures
| Measure |
Azathioprine (AZT)
n=34 Participants
Patients with MG who are receiving azathioprine as part of routine clinical care
Azathioprine: oral tablet
|
Mycophenolate Mofetil (MMF)
n=48 Participants
Patients with MG who are receiving mycophenolate mofetil as part of routine clinical care
Mycophenolate Mofetil: oral tablets
|
|---|---|---|
|
Number of Participants With Improvement in Myasthenia Gravis Manual Muscle Test Scores (MG-MMT)
|
25 Participants
|
45 Participants
|
SECONDARY outcome
Timeframe: Baseline, 24-36 monthsOutcome measures
| Measure |
Azathioprine (AZT)
n=34 Participants
Patients with MG who are receiving azathioprine as part of routine clinical care
Azathioprine: oral tablet
|
Mycophenolate Mofetil (MMF)
n=48 Participants
Patients with MG who are receiving mycophenolate mofetil as part of routine clinical care
Mycophenolate Mofetil: oral tablets
|
|---|---|---|
|
Change in Number of Participants Who Were Hospitalized for Myasthenia Gravis (MG)
|
4 Participants
|
5 Participants
|
Adverse Events
Azathioprine (AZT)
Mycophenolate Mofetil (MMF)
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place