Trial Outcomes & Findings for Efficacy of Mifepristone in Males With Type 2 Diabetes Mellitus (NCT NCT03052400)
NCT ID: NCT03052400
Last Updated: 2023-05-18
Results Overview
Glycemic lowering
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
8 participants
Primary outcome timeframe
Baseline to 3 months
Results posted on
2023-05-18
Participant Flow
Participant milestones
| Measure |
Mifepristone 600 mg Daily
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
|---|---|---|
|
Overall Study
STARTED
|
4
|
4
|
|
Overall Study
COMPLETED
|
3
|
3
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
| Measure |
Mifepristone 600 mg Daily
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
|---|---|---|
|
Overall Study
Lost to Follow-up
|
1
|
0
|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
Efficacy of Mifepristone in Males With Type 2 Diabetes Mellitus
Baseline characteristics by cohort
| Measure |
Mifepristone 600 mg Daily
n=3 Participants
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
n=3 Participants
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
Total
n=6 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Continuous
|
49.8 years
STANDARD_DEVIATION 6.6 • n=99 Participants
|
55.4 years
STANDARD_DEVIATION 1.2 • n=107 Participants
|
52.6 years
STANDARD_DEVIATION 5.2 • n=206 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=99 Participants
|
3 participants
n=107 Participants
|
6 participants
n=206 Participants
|
|
Hemoglobin A1c
|
9.8 Percentage of hemoglobin
STANDARD_DEVIATION 0.8 • n=99 Participants
|
9.8 Percentage of hemoglobin
STANDARD_DEVIATION 0.9 • n=107 Participants
|
9.8 Percentage of hemoglobin
STANDARD_DEVIATION 0.8 • n=206 Participants
|
|
Diabetes duration
|
11.7 Years
STANDARD_DEVIATION 8.8 • n=99 Participants
|
14.0 Years
STANDARD_DEVIATION 2.0 • n=107 Participants
|
12.9 Years
STANDARD_DEVIATION 5.8 • n=206 Participants
|
|
Basal insulin daily dose
|
73.3 Units per day
STANDARD_DEVIATION 19.4 • n=99 Participants
|
65.0 Units per day
STANDARD_DEVIATION 39.5 • n=107 Participants
|
69.2 Units per day
STANDARD_DEVIATION 28.2 • n=206 Participants
|
|
Body weight
|
107.6 kg
STANDARD_DEVIATION 46.5 • n=99 Participants
|
99.8 kg
STANDARD_DEVIATION 4.5 • n=107 Participants
|
103.7 kg
STANDARD_DEVIATION 29.9 • n=206 Participants
|
|
Body mass index
|
39.3 kg/m^2
STANDARD_DEVIATION 15.0 • n=99 Participants
|
36.3 kg/m^2
STANDARD_DEVIATION 6.6 • n=107 Participants
|
37.8 kg/m^2
STANDARD_DEVIATION 10.5 • n=206 Participants
|
PRIMARY outcome
Timeframe: Baseline to 3 monthsGlycemic lowering
Outcome measures
| Measure |
Mifepristone 600 mg Daily
n=3 Participants
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
n=3 Participants
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
|---|---|---|
|
Hemoglobin A1c
|
-2.4 Percentage of hemoglobin
Standard Deviation 1.2
|
-1.5 Percentage of hemoglobin
Standard Deviation 1.4
|
SECONDARY outcome
Timeframe: Baseline to 3 monthsWeight in kg
Outcome measures
| Measure |
Mifepristone 600 mg Daily
n=3 Participants
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
n=3 Participants
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
|---|---|---|
|
Weight
|
1.4 kg, change from baseline
Standard Deviation 1.4
|
0.5 kg, change from baseline
Standard Deviation 3.3
|
SECONDARY outcome
Timeframe: Baseline to 3 monthsBody mass index in kg/m\^2
Outcome measures
| Measure |
Mifepristone 600 mg Daily
n=3 Participants
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
n=3 Participants
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
|---|---|---|
|
Body Mass Index
|
0.6 kg/m^2, change from baseline
Standard Deviation 0.5
|
0.0 kg/m^2, change from baseline
Standard Deviation 1.3
|
SECONDARY outcome
Timeframe: Baseline to 3 monthsSystolic blood pressure
Outcome measures
| Measure |
Mifepristone 600 mg Daily
n=3 Participants
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
n=3 Participants
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
|---|---|---|
|
Systolic BP
|
3.0 mm Hg, change from baseline
Standard Deviation 8.7
|
-3.7 mm Hg, change from baseline
Standard Deviation 1.2
|
SECONDARY outcome
Timeframe: Baseline to 3 monthsDiastolic blood pressure
Outcome measures
| Measure |
Mifepristone 600 mg Daily
n=3 Participants
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
n=3 Participants
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
|---|---|---|
|
Diastolic BP
|
-4.3 mm Hg, change from baseline
Standard Deviation 8.5
|
-3.7 mm Hg, change from baseline
Standard Deviation 5.5
|
SECONDARY outcome
Timeframe: Baseline to 3 monthsLow-density lipoprotein cholesterol
Outcome measures
| Measure |
Mifepristone 600 mg Daily
n=3 Participants
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
n=3 Participants
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
|---|---|---|
|
LDL-cholesterol
|
-1.3 mg/dL, change from baseline
Standard Deviation 18.2
|
-9.7 mg/dL, change from baseline
Standard Deviation 19.3
|
SECONDARY outcome
Timeframe: Baseline to 3 monthsSerum cortisol level (AM)
Outcome measures
| Measure |
Mifepristone 600 mg Daily
n=3 Participants
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
n=3 Participants
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
|---|---|---|
|
Cortisol
|
18.8 mg/dL, change from baseline
Standard Deviation 9.0
|
-0.9 mg/dL, change from baseline
Standard Deviation 0.9
|
SECONDARY outcome
Timeframe: Baseline to 3 monthsSerum adrenocorticotrophic hormone level (AM)
Outcome measures
| Measure |
Mifepristone 600 mg Daily
n=3 Participants
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
n=3 Participants
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
|---|---|---|
|
ACTH
|
56.0 pg/mL, change from baseline
Standard Deviation 39.8
|
-8.3 pg/mL, change from baseline
Standard Deviation 11.2
|
SECONDARY outcome
Timeframe: Baseline to 3 monthsSerum uric acid level
Outcome measures
| Measure |
Mifepristone 600 mg Daily
n=3 Participants
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
n=3 Participants
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
|---|---|---|
|
Uric Acid
|
-0.8 mg/dL, change from baseline
Standard Deviation 0.6
|
1.1 mg/dL, change from baseline
Standard Deviation 0.4
|
SECONDARY outcome
Timeframe: Baseline to 3 monthsProstate-specific antigen level
Outcome measures
| Measure |
Mifepristone 600 mg Daily
n=3 Participants
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
n=3 Participants
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
|---|---|---|
|
PSA
|
-0.3 ng/mL, change from baseline
Standard Deviation 0.2
|
0.1 ng/mL, change from baseline
Standard Deviation 0.2
|
SECONDARY outcome
Timeframe: Baseline to 3 monthsSymptomatic mild and severe hypoglycemic events
Outcome measures
| Measure |
Mifepristone 600 mg Daily
n=3 Participants
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
n=3 Participants
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
|---|---|---|
|
Hypoglycemic Events
|
22 number of events
|
31 number of events
|
SECONDARY outcome
Timeframe: Baseline to 3 monthsNon-hypoglycemia-related adverse events
Outcome measures
| Measure |
Mifepristone 600 mg Daily
n=3 Participants
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
n=3 Participants
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
|---|---|---|
|
Adverse Events
|
6 number of events
|
2 number of events
|
SECONDARY outcome
Timeframe: Baseline to 3 monthsTotal daily basal insulin dosage
Outcome measures
| Measure |
Mifepristone 600 mg Daily
n=3 Participants
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
n=3 Participants
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
|---|---|---|
|
Basal Insulin Dose
|
-1.3 Units of insulin per day
Standard Deviation 16.0
|
0.7 Units of insulin per day
Standard Deviation 17.9
|
Adverse Events
Mifepristone 600 mg Daily
Serious events: 1 serious events
Other events: 3 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Mifepristone 600 mg Daily
n=3 participants at risk
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
n=3 participants at risk
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
|---|---|---|
|
Cardiac disorders
Worsening of heart failure
|
33.3%
1/3 • Number of events 1 • 3 months
Adverse events not including hypoglycemia
|
0.00%
0/3 • 3 months
Adverse events not including hypoglycemia
|
Other adverse events
| Measure |
Mifepristone 600 mg Daily
n=3 participants at risk
Mifepristone 300 mg po daily x 2 weeks, followed by mifepristone 600 mg po daily x 10 weeks
Mifepristone 600 mg daily: Glucocorticoid receptor antagonist
|
Placebo
n=3 participants at risk
Matching, blinded placebo 1 tablet po daily x 2 weeks, followed by matching, blinded placebo 2 tablets po daily x 10 weeks
Placebo: Matching placebo
|
|---|---|---|
|
Social circumstances
Work-related injury
|
33.3%
1/3 • Number of events 1 • 3 months
Adverse events not including hypoglycemia
|
66.7%
2/3 • Number of events 2 • 3 months
Adverse events not including hypoglycemia
|
|
Musculoskeletal and connective tissue disorders
Myalgias
|
33.3%
1/3 • Number of events 1 • 3 months
Adverse events not including hypoglycemia
|
0.00%
0/3 • 3 months
Adverse events not including hypoglycemia
|
|
Gastrointestinal disorders
Constipation
|
33.3%
1/3 • Number of events 1 • 3 months
Adverse events not including hypoglycemia
|
0.00%
0/3 • 3 months
Adverse events not including hypoglycemia
|
|
Cardiac disorders
Prolonged QT Interval
|
33.3%
1/3 • Number of events 1 • 3 months
Adverse events not including hypoglycemia
|
0.00%
0/3 • 3 months
Adverse events not including hypoglycemia
|
|
Cardiac disorders
Peripheral edema
|
33.3%
1/3 • Number of events 1 • 3 months
Adverse events not including hypoglycemia
|
0.00%
0/3 • 3 months
Adverse events not including hypoglycemia
|
Additional Information
Stanley H. Hsia, MD
Charles R. Drew University of Medicine and Science
Phone: 323-357-3633
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place