Trial Outcomes & Findings for Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy (NCT NCT02354352)
NCT ID: NCT02354352
Last Updated: 2019-10-07
Results Overview
a sensitive measure of heart muscle function
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
52 participants
Primary outcome timeframe
12 months
Results posted on
2019-10-07
Participant Flow
Participant milestones
| Measure |
Eplerenone
Eplerenone is an aldosterone antagonist used as an adjunct in the management of chronic heart failure. It is marketed under the trade name Inspra. Eplerenone is a potassium-sparing diuretic.
Eplerenone: 26 Subjects will take Eplerenone, one 50mg capsule by mouth once daily for 12 months.
|
Spironolactone
Spironolactone is an aldosterone antagonist used as an adjunct in the management of chronic heart failure. It is marketed under the trade name Aldactone. Spironolactone is a potassium-sparing diuretic.
Spironolactone: 26 Subjects will take Spironolactone, one 50mg capsule by mouth once daily for 12 months.
|
|---|---|---|
|
Overall Study
STARTED
|
26
|
26
|
|
Overall Study
2 Weeks
|
26
|
25
|
|
Overall Study
1 Month
|
24
|
23
|
|
Overall Study
2 Months
|
24
|
22
|
|
Overall Study
3 Months
|
25
|
23
|
|
Overall Study
6 Months
|
25
|
23
|
|
Overall Study
9 Months
|
25
|
23
|
|
Overall Study
COMPLETED
|
23
|
23
|
|
Overall Study
NOT COMPLETED
|
3
|
3
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
One patient did not receive a pulmonary function test at baseline.
Baseline characteristics by cohort
| Measure |
Eplerenone
n=26 Participants
Eplerenone is an aldosterone antagonist used as an adjunct in the management of chronic heart failure. It is marketed under the trade name Inspra. Eplerenone is a potassium-sparing diuretic.
Eplerenone: 26 Subjects will take Eplerenone, one 50mg capsule by mouth once daily for 12 months.
|
Spironolactone
n=26 Participants
Spironolactone is an aldosterone antagonist used as an adjunct in the management of chronic heart failure. It is marketed under the trade name Aldactone. Spironolactone is a potassium-sparing diuretic.
Spironolactone: 26 Subjects will take Spironolactone, one 50mg capsule by mouth once daily for 12 months.
|
Total
n=52 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
21 Participants
n=26 Participants
|
19 Participants
n=26 Participants
|
40 Participants
n=52 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
5 Participants
n=26 Participants
|
7 Participants
n=26 Participants
|
12 Participants
n=52 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=26 Participants
|
0 Participants
n=26 Participants
|
0 Participants
n=52 Participants
|
|
Age, Continuous
|
14 years
n=26 Participants
|
13 years
n=26 Participants
|
14 years
n=52 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=26 Participants
|
0 Participants
n=26 Participants
|
0 Participants
n=52 Participants
|
|
Sex: Female, Male
Male
|
26 Participants
n=26 Participants
|
26 Participants
n=26 Participants
|
52 Participants
n=52 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=26 Participants
|
6 Participants
n=26 Participants
|
8 Participants
n=52 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
24 Participants
n=26 Participants
|
20 Participants
n=26 Participants
|
44 Participants
n=52 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=26 Participants
|
0 Participants
n=26 Participants
|
0 Participants
n=52 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=26 Participants
|
0 Participants
n=26 Participants
|
0 Participants
n=52 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=26 Participants
|
0 Participants
n=26 Participants
|
0 Participants
n=52 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=26 Participants
|
0 Participants
n=26 Participants
|
0 Participants
n=52 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=26 Participants
|
1 Participants
n=26 Participants
|
2 Participants
n=52 Participants
|
|
Race (NIH/OMB)
White
|
24 Participants
n=26 Participants
|
25 Participants
n=26 Participants
|
49 Participants
n=52 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=26 Participants
|
0 Participants
n=26 Participants
|
1 Participants
n=52 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=26 Participants
|
0 Participants
n=26 Participants
|
0 Participants
n=52 Participants
|
|
Ambulatory
|
4 Participants
n=26 Participants
|
6 Participants
n=26 Participants
|
10 Participants
n=52 Participants
|
|
Nocturnal ventilatory support
|
7 Participants
n=26 Participants
|
6 Participants
n=26 Participants
|
13 Participants
n=52 Participants
|
|
Forced vital capacity
|
1.7 Liters
STANDARD_DEVIATION 0.8 • n=26 Participants • One patient did not receive a pulmonary function test at baseline.
|
1.9 Liters
STANDARD_DEVIATION 0.8 • n=25 Participants • One patient did not receive a pulmonary function test at baseline.
|
1.8 Liters
STANDARD_DEVIATION 0.8 • n=51 Participants • One patient did not receive a pulmonary function test at baseline.
|
|
Dystrophin mutation type
Exon deletion
|
16 Participants
n=26 Participants
|
15 Participants
n=26 Participants
|
31 Participants
n=52 Participants
|
|
Dystrophin mutation type
Exon duplication
|
4 Participants
n=26 Participants
|
3 Participants
n=26 Participants
|
7 Participants
n=52 Participants
|
|
Dystrophin mutation type
Other
|
6 Participants
n=26 Participants
|
8 Participants
n=26 Participants
|
14 Participants
n=52 Participants
|
|
Left ventricular ejection fraction
|
55 Percent of blood ejected from LV
STANDARD_DEVIATION 8.7 • n=26 Participants • One patient did not have adequate MRI images for analysis of this measure.
|
57 Percent of blood ejected from LV
STANDARD_DEVIATION 7.6 • n=25 Participants • One patient did not have adequate MRI images for analysis of this measure.
|
56 Percent of blood ejected from LV
STANDARD_DEVIATION 8.2 • n=51 Participants • One patient did not have adequate MRI images for analysis of this measure.
|
|
Left ventricular circumferential strain
|
-14.5 Percent change in circumference
STANDARD_DEVIATION 2.70 • n=25 Participants • One patient did not have adequate MRI images for analysis of this measure.
|
-14.2 Percent change in circumference
STANDARD_DEVIATION 2.16 • n=26 Participants • One patient did not have adequate MRI images for analysis of this measure.
|
-14.3 Percent change in circumference
STANDARD_DEVIATION 2.42 • n=51 Participants • One patient did not have adequate MRI images for analysis of this measure.
|
|
Late gadolinium enhancement
|
10.3 Percent of enhanced myocardium
STANDARD_DEVIATION 10.7 • n=22 Participants • Several patients did not have adequate MRI images for analysis of this measure.
|
14.5 Percent of enhanced myocardium
STANDARD_DEVIATION 14.7 • n=24 Participants • Several patients did not have adequate MRI images for analysis of this measure.
|
12.5 Percent of enhanced myocardium
STANDARD_DEVIATION 12.9 • n=46 Participants • Several patients did not have adequate MRI images for analysis of this measure.
|
|
Blood pressure
Systolic
|
112.6 mmHg
STANDARD_DEVIATION 14.1 • n=26 Participants
|
118.5 mmHg
STANDARD_DEVIATION 14.7 • n=26 Participants
|
115.6 mmHg
STANDARD_DEVIATION 14.6 • n=52 Participants
|
|
Blood pressure
Diastolic
|
69.4 mmHg
STANDARD_DEVIATION 15.7 • n=26 Participants
|
70.8 mmHg
STANDARD_DEVIATION 11.5 • n=26 Participants
|
70.1 mmHg
STANDARD_DEVIATION 13.7 • n=52 Participants
|
|
Heart rate
|
95.3 beats per minute
STANDARD_DEVIATION 14.3 • n=26 Participants • One patient did not get a heart rate taken at baseline.
|
99.9 beats per minute
STANDARD_DEVIATION 11.9 • n=25 Participants • One patient did not get a heart rate taken at baseline.
|
97.5 beats per minute
STANDARD_DEVIATION 13.2 • n=51 Participants • One patient did not get a heart rate taken at baseline.
|
|
Weight
|
55.6 kilograms
STANDARD_DEVIATION 15.1 • n=26 Participants
|
55.8 kilograms
STANDARD_DEVIATION 20.9 • n=26 Participants
|
55.7 kilograms
STANDARD_DEVIATION 18.1 • n=52 Participants
|
|
Serum potassium
|
4.1 mmol/L
STANDARD_DEVIATION 0.45 • n=26 Participants
|
4.1 mmol/L
STANDARD_DEVIATION 0.50 • n=26 Participants
|
4.1 mmol/L
STANDARD_DEVIATION 0.47 • n=52 Participants
|
|
Background medical therapy
ACEI
|
15 Participants
n=26 Participants
|
15 Participants
n=26 Participants
|
30 Participants
n=52 Participants
|
|
Background medical therapy
ARB
|
2 Participants
n=26 Participants
|
0 Participants
n=26 Participants
|
2 Participants
n=52 Participants
|
|
Background medical therapy
Beta-blocker
|
3 Participants
n=26 Participants
|
7 Participants
n=26 Participants
|
10 Participants
n=52 Participants
|
|
Background medical therapy
Any Steroid Use, Current or Previous
|
21 Participants
n=26 Participants
|
23 Participants
n=26 Participants
|
44 Participants
n=52 Participants
|
|
Background medical therapy
Prednisone
|
15 Participants
n=26 Participants
|
11 Participants
n=26 Participants
|
26 Participants
n=52 Participants
|
|
Background medical therapy
Deflazacort
|
6 Participants
n=26 Participants
|
12 Participants
n=26 Participants
|
18 Participants
n=52 Participants
|
PRIMARY outcome
Timeframe: 12 monthsPopulation: One patient in the spironolactone group did not receive a follow-up MRI.
a sensitive measure of heart muscle function
Outcome measures
| Measure |
Eplerenone
n=23 Participants
Eplerenone is an aldosterone antagonist used as an adjunct in the management of chronic heart failure. It is marketed under the trade name Inspra. Eplerenone is a potassium-sparing diuretic.
Eplerenone: 26 Subjects will take Eplerenone, one 50mg capsule by mouth once daily for 12 months.
|
Spironolactone
n=22 Participants
Spironolactone is an aldosterone antagonist used as an adjunct in the management of chronic heart failure. It is marketed under the trade name Aldactone. Spironolactone is a potassium-sparing diuretic.
Spironolactone: 26 Subjects will take Spironolactone, one 50mg capsule by mouth once daily for 12 months.
|
|---|---|---|
|
Left Ventricular Strain
|
0.2 Percent change in circumference
Interval -0.2 to 0.7
|
0.4 Percent change in circumference
Interval -0.4 to 1.0
|
Adverse Events
Eplerenone
Serious events: 3 serious events
Other events: 2 other events
Deaths: 2 deaths
Spironolactone
Serious events: 2 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Eplerenone
n=26 participants at risk
Eplerenone is an aldosterone antagonist used as an adjunct in the management of chronic heart failure. It is marketed under the trade name Inspra. Eplerenone is a potassium-sparing diuretic.
Eplerenone: 26 Subjects will take Eplerenone, one 50mg capsule by mouth once daily for 12 months.
|
Spironolactone
n=26 participants at risk
Spironolactone is an aldosterone antagonist used as an adjunct in the management of chronic heart failure. It is marketed under the trade name Aldactone. Spironolactone is a potassium-sparing diuretic.
Spironolactone: 26 Subjects will take Spironolactone, one 50mg capsule by mouth once daily for 12 months.
|
|---|---|---|
|
Injury, poisoning and procedural complications
Accidental Drowning
|
3.8%
1/26 • Number of events 1 • 1 year
The definitions used to collect adverse event information did not differ from the clinicaltrials.gov definitions.
|
0.00%
0/26 • 1 year
The definitions used to collect adverse event information did not differ from the clinicaltrials.gov definitions.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia Hospitalization
|
3.8%
1/26 • Number of events 1 • 1 year
The definitions used to collect adverse event information did not differ from the clinicaltrials.gov definitions.
|
0.00%
0/26 • 1 year
The definitions used to collect adverse event information did not differ from the clinicaltrials.gov definitions.
|
|
Injury, poisoning and procedural complications
Fracture
|
3.8%
1/26 • Number of events 1 • 1 year
The definitions used to collect adverse event information did not differ from the clinicaltrials.gov definitions.
|
7.7%
2/26 • Number of events 2 • 1 year
The definitions used to collect adverse event information did not differ from the clinicaltrials.gov definitions.
|
Other adverse events
| Measure |
Eplerenone
n=26 participants at risk
Eplerenone is an aldosterone antagonist used as an adjunct in the management of chronic heart failure. It is marketed under the trade name Inspra. Eplerenone is a potassium-sparing diuretic.
Eplerenone: 26 Subjects will take Eplerenone, one 50mg capsule by mouth once daily for 12 months.
|
Spironolactone
n=26 participants at risk
Spironolactone is an aldosterone antagonist used as an adjunct in the management of chronic heart failure. It is marketed under the trade name Aldactone. Spironolactone is a potassium-sparing diuretic.
Spironolactone: 26 Subjects will take Spironolactone, one 50mg capsule by mouth once daily for 12 months.
|
|---|---|---|
|
Skin and subcutaneous tissue disorders
Rash
|
3.8%
1/26 • Number of events 1 • 1 year
The definitions used to collect adverse event information did not differ from the clinicaltrials.gov definitions.
|
0.00%
0/26 • 1 year
The definitions used to collect adverse event information did not differ from the clinicaltrials.gov definitions.
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/26 • 1 year
The definitions used to collect adverse event information did not differ from the clinicaltrials.gov definitions.
|
7.7%
2/26 • Number of events 2 • 1 year
The definitions used to collect adverse event information did not differ from the clinicaltrials.gov definitions.
|
|
Endocrine disorders
Gynecomastia
|
0.00%
0/26 • 1 year
The definitions used to collect adverse event information did not differ from the clinicaltrials.gov definitions.
|
3.8%
1/26 • Number of events 1 • 1 year
The definitions used to collect adverse event information did not differ from the clinicaltrials.gov definitions.
|
|
Renal and urinary disorders
Nephrolithiasis
|
3.8%
1/26 • Number of events 1 • 1 year
The definitions used to collect adverse event information did not differ from the clinicaltrials.gov definitions.
|
0.00%
0/26 • 1 year
The definitions used to collect adverse event information did not differ from the clinicaltrials.gov definitions.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place