Trial Outcomes & Findings for Finding the Optimum Regimen for Duchenne Muscular Dystrophy (NCT NCT01603407)
NCT ID: NCT01603407
Last Updated: 2022-08-12
Results Overview
Forced vital capacity was measured during a spirometry test. Forced expiratory volume (FEV) measures how much air a person can exhale during a forced breath. Forced vital capacity (FVC) is the total amount of air exhaled during the FEV test.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
196 participants
Primary outcome timeframe
Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Results posted on
2022-08-12
Participant Flow
229 were screened for eligibility. 33 failed screening
Participant milestones
| Measure |
Daily Prednisone
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Overall Study
STARTED
|
65
|
65
|
66
|
|
Overall Study
COMPLETED
|
54
|
54
|
56
|
|
Overall Study
NOT COMPLETED
|
11
|
11
|
10
|
Reasons for withdrawal
| Measure |
Daily Prednisone
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
1
|
1
|
1
|
|
Overall Study
Protocol Violation
|
2
|
2
|
0
|
|
Overall Study
Withdrawal by Subject
|
8
|
5
|
7
|
|
Overall Study
Lost to Follow-up
|
0
|
3
|
2
|
Baseline Characteristics
Data was not collected for one participant in the intermittent prednisone arm.
Baseline characteristics by cohort
| Measure |
Daily Prednisone
n=65 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=65 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=66 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
Total
n=196 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
5.8 years
STANDARD_DEVIATION 1.0 • n=65 Participants
|
5.8 years
STANDARD_DEVIATION 1.0 • n=65 Participants
|
5.9 years
STANDARD_DEVIATION 1.1 • n=66 Participants
|
5.9 years
STANDARD_DEVIATION 1.0 • n=196 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=65 Participants
|
0 Participants
n=65 Participants
|
0 Participants
n=66 Participants
|
0 Participants
n=196 Participants
|
|
Sex: Female, Male
Male
|
65 Participants
n=65 Participants
|
65 Participants
n=65 Participants
|
66 Participants
n=66 Participants
|
196 Participants
n=196 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
9 Participants
n=65 Participants
|
9 Participants
n=65 Participants
|
13 Participants
n=66 Participants
|
31 Participants
n=196 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
56 Participants
n=65 Participants
|
56 Participants
n=65 Participants
|
53 Participants
n=66 Participants
|
165 Participants
n=196 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=65 Participants
|
0 Participants
n=65 Participants
|
0 Participants
n=66 Participants
|
0 Participants
n=196 Participants
|
|
Race/Ethnicity, Customized
Caucasian
|
59 Participants
n=65 Participants
|
58 Participants
n=65 Participants
|
49 Participants
n=66 Participants
|
166 Participants
n=196 Participants
|
|
Race/Ethnicity, Customized
race other than Caucasian
|
6 Participants
n=65 Participants
|
7 Participants
n=65 Participants
|
17 Participants
n=66 Participants
|
30 Participants
n=196 Participants
|
|
Region of Enrollment
Canada
|
5 participants
n=65 Participants
|
4 participants
n=65 Participants
|
4 participants
n=66 Participants
|
13 participants
n=196 Participants
|
|
Region of Enrollment
United States
|
27 participants
n=65 Participants
|
27 participants
n=65 Participants
|
28 participants
n=66 Participants
|
82 participants
n=196 Participants
|
|
Region of Enrollment
Italy
|
8 participants
n=65 Participants
|
7 participants
n=65 Participants
|
7 participants
n=66 Participants
|
22 participants
n=196 Participants
|
|
Region of Enrollment
United Kingdom
|
19 participants
n=65 Participants
|
20 participants
n=65 Participants
|
20 participants
n=66 Participants
|
59 participants
n=196 Participants
|
|
Region of Enrollment
Germany
|
6 participants
n=65 Participants
|
7 participants
n=65 Participants
|
7 participants
n=66 Participants
|
20 participants
n=196 Participants
|
|
Mean weight
|
20.0 kg
STANDARD_DEVIATION 3.0 • n=65 Participants
|
19.9 kg
STANDARD_DEVIATION 4.3 • n=65 Participants
|
20.2 kg
STANDARD_DEVIATION 3.5 • n=66 Participants
|
20.0 kg
STANDARD_DEVIATION 3.6 • n=196 Participants
|
|
Mean height
|
109.9 cm
STANDARD_DEVIATION 7.0 • n=65 Participants • Data was not collected for one participant in the intermittent prednisone arm.
|
109.5 cm
STANDARD_DEVIATION 7.6 • n=65 Participants • Data was not collected for one participant in the intermittent prednisone arm.
|
110.9 cm
STANDARD_DEVIATION 7.1 • n=65 Participants • Data was not collected for one participant in the intermittent prednisone arm.
|
110.1 cm
STANDARD_DEVIATION 7.2 • n=195 Participants • Data was not collected for one participant in the intermittent prednisone arm.
|
|
Mean body mass index
|
16.5 kg/m^2
STANDARD_DEVIATION 1.3 • n=65 Participants • Data was not collected for one participant in the intermittent prednisone arm.
|
16.5 kg/m^2
STANDARD_DEVIATION 2.0 • n=65 Participants • Data was not collected for one participant in the intermittent prednisone arm.
|
16.4 kg/m^2
STANDARD_DEVIATION 1.6 • n=65 Participants • Data was not collected for one participant in the intermittent prednisone arm.
|
16.4 kg/m^2
STANDARD_DEVIATION 1.6 • n=195 Participants • Data was not collected for one participant in the intermittent prednisone arm.
|
|
Mean rise from floor velocity
|
0.19 rise/sec
STANDARD_DEVIATION 0.09 • n=65 Participants
|
0.19 rise/sec
STANDARD_DEVIATION 0.07 • n=65 Participants
|
0.18 rise/sec
STANDARD_DEVIATION 0.08 • n=66 Participants
|
0.18 rise/sec
STANDARD_DEVIATION 0.08 • n=196 Participants
|
|
Mean 10 meter walk/run velocity
|
0.17 10 m/s
STANDARD_DEVIATION 0.04 • n=65 Participants
|
0.17 10 m/s
STANDARD_DEVIATION 0.04 • n=65 Participants
|
0.19 10 m/s
STANDARD_DEVIATION 0.04 • n=66 Participants
|
0.17 10 m/s
STANDARD_DEVIATION 0.04 • n=196 Participants
|
|
Mean NSAA total score
|
22.0 units on a scale
STANDARD_DEVIATION 5.7 • n=65 Participants
|
21.1 units on a scale
STANDARD_DEVIATION 5.7 • n=65 Participants
|
21.1 units on a scale
STANDARD_DEVIATION 4.9 • n=66 Participants
|
21.4 units on a scale
STANDARD_DEVIATION 5.4 • n=196 Participants
|
|
Mean Six-minute walk test distance
|
342.4 meters
STANDARD_DEVIATION 58.0 • n=63 Participants • Data was not collected for two participants in the daily prednisone arm and one participant in the intermittent prednisone arm.
|
330.4 meters
STANDARD_DEVIATION 65.1 • n=65 Participants • Data was not collected for two participants in the daily prednisone arm and one participant in the intermittent prednisone arm.
|
331.6 meters
STANDARD_DEVIATION 63.6 • n=65 Participants • Data was not collected for two participants in the daily prednisone arm and one participant in the intermittent prednisone arm.
|
334.7 meters
STANDARD_DEVIATION 62.2 • n=193 Participants • Data was not collected for two participants in the daily prednisone arm and one participant in the intermittent prednisone arm.
|
|
Mean forced vital capacity
|
1.0 liters
STANDARD_DEVIATION 0.4 • n=65 Participants
|
1.1 liters
STANDARD_DEVIATION 0.4 • n=65 Participants
|
1.1 liters
STANDARD_DEVIATION 0.4 • n=66 Participants
|
1.1 liters
STANDARD_DEVIATION 0.4 • n=196 Participants
|
|
Mean percentage of predicted normal forced vital capacity
|
85.4 percentage predicted normal
STANDARD_DEVIATION 29.8 • n=65 Participants • Data was not collected for one participant in the intermittent prednisone arm.
|
87.2 percentage predicted normal
STANDARD_DEVIATION 36.2 • n=65 Participants • Data was not collected for one participant in the intermittent prednisone arm.
|
88.1 percentage predicted normal
STANDARD_DEVIATION 34.0 • n=65 Participants • Data was not collected for one participant in the intermittent prednisone arm.
|
86.9 percentage predicted normal
STANDARD_DEVIATION 33.3 • n=195 Participants • Data was not collected for one participant in the intermittent prednisone arm.
|
PRIMARY outcome
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visitsPopulation: Data was not collected on 4 participants in the daily prednisone arm, 3 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm.
Forced vital capacity was measured during a spirometry test. Forced expiratory volume (FEV) measures how much air a person can exhale during a forced breath. Forced vital capacity (FVC) is the total amount of air exhaled during the FEV test.
Outcome measures
| Measure |
Daily Prednisone
n=61 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=62 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=65 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Forced Vital Capacity
|
1.4 liters
Standard Error 0.03
|
1.4 liters
Standard Error 0.03
|
1.5 liters
Standard Error 0.03
|
PRIMARY outcome
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visitsPopulation: Data was not collected on 3 participants in the daily prednisone arm, 2 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm.
Reciprocal of time to rise from the floor
Outcome measures
| Measure |
Daily Prednisone
n=62 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=63 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=65 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Rise From the Floor Velocity
|
0.24 rise/sec
Standard Error 0.01
|
0.24 rise/sec
Standard Error 0.01
|
0.18 rise/sec
Standard Error 0.01
|
PRIMARY outcome
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visitsPopulation: Data was not collected on 3 participants in the daily prednisone arm, 3 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm.
The TSQM Global Satisfaction with Treatment is a 14-item questionnaire that ranges from 0 - 100 with higher scores indicating better outcomes.
Outcome measures
| Measure |
Daily Prednisone
n=62 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=62 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=65 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score
|
71.2 score on a scale
Standard Error 2.3
|
67.8 score on a scale
Standard Error 2.3
|
65.1 score on a scale
Standard Error 2.3
|
SECONDARY outcome
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visitsPopulation: Data was not collected on 3 participants in the daily prednisone arm, 2 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm.
The North Star Ambulatory Assessment (NSAA) is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with Duchenne Muscular Dystrophy (DMD). It is usually used to monitor the progression of the disease and treatment effects. The activities are graded as follows: 2 - "Normal" - no obvious modification of activity 1 - Modified method but achieves goal independent of physical assistance from another 0 - Unable to achieve independently This scale is ordinal with 34 as the maximum score indicating fully-independent function.
Outcome measures
| Measure |
Daily Prednisone
n=62 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=63 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=65 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
North Star Ambulatory Assessment (NSAA) Score
|
23.7 score on a scale
Standard Error 0.6
|
24.0 score on a scale
Standard Error 0.6
|
20.7 score on a scale
Standard Error 0.6
|
SECONDARY outcome
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visitsPopulation: Data was not collected on 5 participants in the daily prednisone arm, 2 participants in the deflazacort arm and 2 participant in the intermittent prednisone arm.
Measures the total distance walked in 6 minutes averaged over all post-baseline follow-up visits through Month 36.
Outcome measures
| Measure |
Daily Prednisone
n=60 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=63 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=64 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
6 Minute Walk Test
|
384.95 meters
Standard Error 9.6
|
384.17 meters
Standard Error 9.7
|
346.81 meters
Standard Error 9.4
|
SECONDARY outcome
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visitsPopulation: Data was not collected on 3 participants in the daily prednisone arm, 3 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm.
Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits.
Outcome measures
| Measure |
Daily Prednisone
n=62 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=62 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=65 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Range of Motion (Goniometry) of Left Ankle
|
4.39 degrees
Standard Deviation 0.83
|
3.29 degrees
Standard Deviation 0.85
|
2.67 degrees
Standard Deviation 0.82
|
SECONDARY outcome
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visitsPopulation: Data was not collected on 3 participants in the daily prednisone arm, 3 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm.
Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits.
Outcome measures
| Measure |
Daily Prednisone
n=62 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=62 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=65 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Range of Motion (Goniometry) of Right Ankle
|
4.05 degrees
Standard Deviation 0.89
|
2.81 degrees
Standard Deviation 0.92
|
2.29 degrees
Standard Deviation 0.89
|
SECONDARY outcome
Timeframe: 3 yearsThe number of participants who completed 36 months of follow-up on the originally assigned dosage (for weight) of study medication.
Outcome measures
| Measure |
Daily Prednisone
n=65 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=65 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=66 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Number of Participants Who Tolerated the Regimen
|
36 Participants
|
36 Participants
|
37 Participants
|
SECONDARY outcome
Timeframe: 36 monthsPopulation: Data was not collected on 1 participant in the daily prednisone arm, and 2 participants in the intermittent prednisone arm.
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Outcome measures
| Measure |
Daily Prednisone
n=64 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=65 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=64 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Heart Rate
|
94.10 bpm
Standard Error 1.96
|
93.52 bpm
Standard Error 2.09
|
91.65 bpm
Standard Error 2.10
|
SECONDARY outcome
Timeframe: Average of Months 12, 24, and 36 visitsPopulation: Data was not collected on 3 participants in the daily prednisone arm, 1 participants in the deflazacort arm and 2 participant in the intermittent prednisone arm.
Quality of life was measured by parent/guardian self-report for all children utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life for the child.
Outcome measures
| Measure |
Daily Prednisone
n=62 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=64 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=64 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Quality of Life - Parent
|
64.88 score on a scale
Standard Error 1.67
|
63.71 score on a scale
Standard Error 1.69
|
61.33 score on a scale
Standard Error 1.69
|
SECONDARY outcome
Timeframe: Average of Months 12, 24, and 36 visitsPopulation: Only children over the age of 5 self-reported quality of life.
Quality of life was measured by child self-report in children age 5 and older utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life.
Outcome measures
| Measure |
Daily Prednisone
n=54 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=52 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=47 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Quality of Life- Child
|
67.39 score on a scale
Standard Error 2.16
|
64.96 score on a scale
Standard Error 2.17
|
65.07 score on a scale
Standard Error 2.17
|
SECONDARY outcome
Timeframe: 36 monthsPopulation: Data was not collected on 2 participants in the daily prednisone arm, 3 participants in the deflazacort arm and 5 participants in the intermittent prednisone arm.
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Outcome measures
| Measure |
Daily Prednisone
n=63 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=62 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=61 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Left Ventricular Ejection Fraction Percent
|
61.88 percentage of ejection fraction
Standard Error 1.09
|
62.65 percentage of ejection fraction
Standard Error 1.17
|
62.45 percentage of ejection fraction
Standard Error 1.23
|
SECONDARY outcome
Timeframe: 36 monthsPopulation: Data was not collected on 4 participants in the daily prednisone arm, 4 participants in the deflazacort arm and 8 participants in the intermittent prednisone arm.
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Outcome measures
| Measure |
Daily Prednisone
n=61 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=61 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=58 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Fractional Shortening Percent
|
33.74 percentage of fractional shortening
Standard Error 0.81
|
34.01 percentage of fractional shortening
Standard Error 0.87
|
34.33 percentage of fractional shortening
Standard Error 0.87
|
SECONDARY outcome
Timeframe: 36 monthsPopulation: Data was not collected on 3 participants in the daily prednisone arm, and 3 participants in the intermittent prednisone arm.
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Outcome measures
| Measure |
Daily Prednisone
n=62 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=65 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=63 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
PR Interval
|
115.59 milliseconds
Standard Error 2.30
|
116.87 milliseconds
Standard Error 2.33
|
117.90 milliseconds
Standard Error 2.22
|
SECONDARY outcome
Timeframe: 36 monthsOutcome measures
| Measure |
Daily Prednisone
n=62 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=62 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=65 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Participant Weight
|
26.3 kilograms
Standard Error 0.36
|
24.9 kilograms
Standard Error 0.36
|
26.3 kilograms
Standard Error 0.35
|
SECONDARY outcome
Timeframe: 36 monthsOutcome measures
| Measure |
Daily Prednisone
n=62 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=62 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=65 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Participant Height
|
116.8 centimeters
Standard Error 0.26
|
115.3 centimeters
Standard Error 0.27
|
119.9 centimeters
Standard Error 0.26
|
SECONDARY outcome
Timeframe: 36 monthsOutcome measures
| Measure |
Daily Prednisone
n=62 Participants
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=62 Participants
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=65 Participants
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Participant Body Mass Index
|
18.9 kilograms/square meter
Standard Error 0.27
|
18.3 kilograms/square meter
Standard Error 0.26
|
18.1 kilograms/square meter
Standard Error 0.25
|
Adverse Events
Daily Prednisone
Serious events: 5 serious events
Other events: 61 other events
Deaths: 0 deaths
Daily Deflazacort
Serious events: 10 serious events
Other events: 58 other events
Deaths: 0 deaths
Intermittent Prednisone
Serious events: 5 serious events
Other events: 55 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Daily Prednisone
n=65 participants at risk
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=65 participants at risk
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=66 participants at risk
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
1.5%
1/65 • 36 months
|
0.00%
0/65 • 36 months
|
1.5%
1/66 • 36 months
|
|
General disorders
adenotonsillectomy
|
1.5%
1/65 • 36 months
|
0.00%
0/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Gastrointestinal disorders
Constipation
|
1.5%
1/65 • 36 months
|
0.00%
0/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Renal and urinary disorders
Fetal Incontinence
|
1.5%
1/65 • 36 months
|
0.00%
0/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Renal and urinary disorders
mildly increase urine calcium/creatinine ratio
|
1.5%
1/65 • 36 months
|
0.00%
0/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Musculoskeletal and connective tissue disorders
Intervertebral disc compression
|
1.5%
1/65 • 36 months
|
0.00%
0/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Infections and infestations
lower respiratory tract infection
|
1.5%
1/65 • 36 months
|
0.00%
0/65 • 36 months
|
1.5%
1/66 • 36 months
|
|
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
|
1.5%
1/65 • 36 months
|
1.5%
1/65 • 36 months
|
1.5%
1/66 • 36 months
|
|
Musculoskeletal and connective tissue disorders
Tentomy
|
1.5%
1/65 • 36 months
|
1.5%
1/65 • 36 months
|
1.5%
1/66 • 36 months
|
|
Gastrointestinal disorders
Appendicitis
|
0.00%
0/65 • 36 months
|
1.5%
1/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Musculoskeletal and connective tissue disorders
Bone fracture
|
0.00%
0/65 • 36 months
|
1.5%
1/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Renal and urinary disorders
Chromaturia
|
0.00%
0/65 • 36 months
|
1.5%
1/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/65 • 36 months
|
1.5%
1/65 • 36 months
|
1.5%
1/66 • 36 months
|
|
Infections and infestations
Ear infection
|
0.00%
0/65 • 36 months
|
1.5%
1/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
General disorders
Fall
|
0.00%
0/65 • 36 months
|
1.5%
1/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Infections and infestations
Gastrointestinal infection
|
0.00%
0/65 • 36 months
|
1.5%
1/65 • 36 months
|
1.5%
1/66 • 36 months
|
|
Metabolism and nutrition disorders
Joint pain
|
0.00%
0/65 • 36 months
|
1.5%
1/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
0.00%
0/65 • 36 months
|
1.5%
1/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Renal and urinary disorders
Myoglobinuria
|
0.00%
0/65 • 36 months
|
1.5%
1/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Blood and lymphatic system disorders
Tonsillitis
|
0.00%
0/65 • 36 months
|
1.5%
1/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Infections and infestations
Oral Infection
|
0.00%
0/65 • 36 months
|
1.5%
1/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/65 • 36 months
|
1.5%
1/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/65 • 36 months
|
1.5%
1/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Reproductive system and breast disorders
Circumsion
|
0.00%
0/65 • 36 months
|
0.00%
0/65 • 36 months
|
1.5%
1/66 • 36 months
|
|
General disorders
Epistaxis
|
0.00%
0/65 • 36 months
|
0.00%
0/65 • 36 months
|
1.5%
1/66 • 36 months
|
|
Nervous system disorders
Headache
|
0.00%
0/65 • 36 months
|
0.00%
0/65 • 36 months
|
1.5%
1/66 • 36 months
|
|
Injury, poisoning and procedural complications
Head injury
|
0.00%
0/65 • 36 months
|
0.00%
0/65 • 36 months
|
1.5%
1/66 • 36 months
|
|
Reproductive system and breast disorders
Orchidopexy
|
0.00%
0/65 • 36 months
|
0.00%
0/65 • 36 months
|
1.5%
1/66 • 36 months
|
|
General disorders
Pyrexia
|
0.00%
0/65 • 36 months
|
0.00%
0/65 • 36 months
|
1.5%
1/66 • 36 months
|
|
Surgical and medical procedures
Umbilical hernia repair
|
0.00%
0/65 • 36 months
|
0.00%
0/65 • 36 months
|
1.5%
1/66 • 36 months
|
Other adverse events
| Measure |
Daily Prednisone
n=65 participants at risk
daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
|
Daily Deflazacort
n=65 participants at risk
daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
|
Intermittent Prednisone
n=66 participants at risk
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
|
|---|---|---|---|
|
Gastrointestinal disorders
Abdominal discomfort
|
6.2%
4/65 • 36 months
|
1.5%
1/65 • 36 months
|
3.0%
2/66 • 36 months
|
|
Metabolism and nutrition disorders
Cushingoid
|
9.2%
6/65 • 36 months
|
9.2%
6/65 • 36 months
|
4.5%
3/66 • 36 months
|
|
Eye disorders
Cataracts
|
1.5%
1/65 • 36 months
|
7.7%
5/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Gastrointestinal disorders
Vomiting
|
29.2%
19/65 • 36 months
|
24.6%
16/65 • 36 months
|
22.7%
15/66 • 36 months
|
|
Gastrointestinal disorders
Diarrhea
|
13.8%
9/65 • 36 months
|
10.8%
7/65 • 36 months
|
15.2%
10/66 • 36 months
|
|
Gastrointestinal disorders
Abdominal pain
|
12.3%
8/65 • 36 months
|
12.3%
8/65 • 36 months
|
12.1%
8/66 • 36 months
|
|
Gastrointestinal disorders
Abdominal pain upper
|
15.4%
10/65 • 36 months
|
6.2%
4/65 • 36 months
|
15.2%
10/66 • 36 months
|
|
Gastrointestinal disorders
Constipation
|
10.8%
7/65 • 36 months
|
13.8%
9/65 • 36 months
|
10.6%
7/66 • 36 months
|
|
Gastrointestinal disorders
Nausea
|
7.7%
5/65 • 36 months
|
6.2%
4/65 • 36 months
|
1.5%
1/66 • 36 months
|
|
Gastrointestinal disorders
Gastroenteritis
|
6.2%
4/65 • 36 months
|
4.6%
3/65 • 36 months
|
1.5%
1/66 • 36 months
|
|
Gastrointestinal disorders
Gastrointestinal disorders
|
6.2%
4/65 • 36 months
|
3.1%
2/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
General disorders
Pyrexia
|
18.5%
12/65 • 36 months
|
16.9%
11/65 • 36 months
|
15.2%
10/66 • 36 months
|
|
General disorders
Malaise
|
3.1%
2/65 • 36 months
|
6.2%
4/65 • 36 months
|
3.0%
2/66 • 36 months
|
|
Infections and infestations
Ear infection
|
6.2%
4/65 • 36 months
|
10.8%
7/65 • 36 months
|
12.1%
8/66 • 36 months
|
|
Infections and infestations
Influenza
|
12.3%
8/65 • 36 months
|
1.5%
1/65 • 36 months
|
3.0%
2/66 • 36 months
|
|
Infections and infestations
Nasopharyngitis
|
6.2%
4/65 • 36 months
|
3.1%
2/65 • 36 months
|
3.0%
2/66 • 36 months
|
|
Injury, poisoning and procedural complications
Fall
|
15.4%
10/65 • 36 months
|
10.8%
7/65 • 36 months
|
18.2%
12/66 • 36 months
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
1.5%
1/65 • 36 months
|
6.2%
4/65 • 36 months
|
1.5%
1/66 • 36 months
|
|
Metabolism and nutrition disorders
Abnormal weight gain
|
13.8%
9/65 • 36 months
|
6.2%
4/65 • 36 months
|
7.6%
5/66 • 36 months
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
10.8%
7/65 • 36 months
|
9.2%
6/65 • 36 months
|
7.6%
5/66 • 36 months
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
12.3%
8/65 • 36 months
|
15.4%
10/65 • 36 months
|
7.6%
5/66 • 36 months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
7.7%
5/65 • 36 months
|
9.2%
6/65 • 36 months
|
12.1%
8/66 • 36 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
4.6%
3/65 • 36 months
|
9.2%
6/65 • 36 months
|
3.0%
2/66 • 36 months
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
1.5%
1/65 • 36 months
|
7.7%
5/65 • 36 months
|
4.5%
3/66 • 36 months
|
|
Musculoskeletal and connective tissue disorders
Intervertebral disc space narrowing
|
6.2%
4/65 • 36 months
|
0.00%
0/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin papilloma
|
7.7%
5/65 • 36 months
|
1.5%
1/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Nervous system disorders
Headache
|
13.8%
9/65 • 36 months
|
13.8%
9/65 • 36 months
|
9.1%
6/66 • 36 months
|
|
Psychiatric disorders
Abnormal behavior
|
18.5%
12/65 • 36 months
|
26.2%
17/65 • 36 months
|
24.2%
16/66 • 36 months
|
|
Psychiatric disorders
Mood altered
|
6.2%
4/65 • 36 months
|
10.8%
7/65 • 36 months
|
3.0%
2/66 • 36 months
|
|
Psychiatric disorders
Aggression
|
6.2%
4/65 • 36 months
|
6.2%
4/65 • 36 months
|
3.0%
2/66 • 36 months
|
|
Psychiatric disorders
Insomnia
|
1.5%
1/65 • 36 months
|
6.2%
4/65 • 36 months
|
3.0%
2/66 • 36 months
|
|
Psychiatric disorders
Psychomotor hyperactivity
|
3.1%
2/65 • 36 months
|
1.5%
1/65 • 36 months
|
6.1%
4/66 • 36 months
|
|
Renal and urinary disorders
Pollakiuria
|
6.2%
4/65 • 36 months
|
3.1%
2/65 • 36 months
|
7.6%
5/66 • 36 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
20.0%
13/65 • 36 months
|
18.5%
12/65 • 36 months
|
16.7%
11/66 • 36 months
|
|
Respiratory, thoracic and mediastinal disorders
Naspharyngitis
|
13.8%
9/65 • 36 months
|
13.8%
9/65 • 36 months
|
16.7%
11/66 • 36 months
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract infection
|
16.9%
11/65 • 36 months
|
16.9%
11/65 • 36 months
|
10.6%
7/66 • 36 months
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis
|
6.2%
4/65 • 36 months
|
7.7%
5/65 • 36 months
|
9.1%
6/66 • 36 months
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
3.1%
2/65 • 36 months
|
7.7%
5/65 • 36 months
|
3.0%
2/66 • 36 months
|
|
Skin and subcutaneous tissue disorders
Hypertrichosis
|
7.7%
5/65 • 36 months
|
10.8%
7/65 • 36 months
|
0.00%
0/66 • 36 months
|
|
Skin and subcutaneous tissue disorders
Rash
|
4.6%
3/65 • 36 months
|
6.2%
4/65 • 36 months
|
3.0%
2/66 • 36 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The PI cannot publish any manuscript without review and written consent of the FOR-DMD study steering committee.
- Publication restrictions are in place
Restriction type: OTHER