Trial Outcomes & Findings for Twice Weekly Steroids and Exercise as Therapy for DMD (NCT NCT04322357)
NCT ID: NCT04322357
Last Updated: 2026-03-13
Results Overview
Participant body mass index change (weight and height will be combined to report BMI in kg/m\^2) over the course of one year
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
21 participants
Primary outcome timeframe
Baseline up to 12 months
Results posted on
2026-03-13
Participant Flow
21 patients were enrolled in the study. Of these, 17 steroid-naive boys with DMD were enrolled into the 12 month twice weekly steroid arm and 2 of the 17 underwent cycle exercise training from month 6 to 12. Of the 21 enrolled patients, 4 boys on daily steroids were enrolled into a 6-month exercise training arm.
Participant milestones
| Measure |
Twice Weekly Glucocorticoid Without Exercise
Steroid naive patients were enrolled and administered low-dose prednisone (dose of 0.75 mg/kg per day) twice weekly for 12 months.
|
Daily Glucocorticoid With Exercise
Patients on daily glucocorticoids for at least 3 months will undergo 6 months of structured, supervised and home-based exercise training.
In-home Exercise Training: For boys on current standard of care (daily glucocorticoid use), 6-months in-home, remotely supervised exercise training program involving a combination of aerobic and isometric leg strength exercises.
|
Twice Weekly Glucocorticoid With Exercise
Steroid naive boys with DMD were enrolled and underwent the twice weekly steroid protocol for 6 months; exercise was initiated at 6 months while continuing twice-weekly steroids.
|
|---|---|---|---|
|
Overall Study
STARTED
|
15
|
4
|
2
|
|
Overall Study
COMPLETED
|
10
|
4
|
2
|
|
Overall Study
NOT COMPLETED
|
5
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Twice Weekly Steroids and Exercise as Therapy for DMD
Baseline characteristics by cohort
| Measure |
Twice Weekly Glucocorticoid Without Exercise
n=15 Participants
Steroid naive patients will be enrolled and administered low-dose prednisone (dose of 0.75 mg/kg per day) twice weekly.
|
Daily Glucocorticoid With Exercise
n=4 Participants
Patients on daily glucocorticoids will undergo 6 months of structured, supervised and home-based exercise training (aerobic and isometric leg strength).
|
Twice Weekly Glucocorticoid With Exercise
n=2 Participants
Steroid naive patients will be enrolled and administered low-dose prednisone (dose of 0.75 mg/kg per day) twice weekly. At 6-months months, they will also undergo 6 months of structured supervised home based exercise training.
|
Total
n=21 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
15 Participants
n=41 Participants
|
4 Participants
n=39 Participants
|
2 Participants
n=80 Participants
|
21 Participants
n=296 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=41 Participants
|
0 Participants
n=39 Participants
|
0 Participants
n=80 Participants
|
0 Participants
n=296 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=41 Participants
|
0 Participants
n=39 Participants
|
0 Participants
n=80 Participants
|
0 Participants
n=296 Participants
|
|
Age, Continuous
|
6.2 years
STANDARD_DEVIATION 1.7 • n=41 Participants
|
8.1 years
STANDARD_DEVIATION 1.0 • n=39 Participants
|
7.8 years
STANDARD_DEVIATION 0.85 • n=80 Participants
|
6.5 years
STANDARD_DEVIATION 1.7 • n=296 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=41 Participants
|
0 Participants
n=39 Participants
|
0 Participants
n=80 Participants
|
0 Participants
n=296 Participants
|
|
Sex: Female, Male
Male
|
15 Participants
n=41 Participants
|
4 Participants
n=39 Participants
|
2 Participants
n=80 Participants
|
21 Participants
n=296 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=41 Participants
|
0 Participants
n=39 Participants
|
0 Participants
n=80 Participants
|
3 Participants
n=296 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
12 Participants
n=41 Participants
|
4 Participants
n=39 Participants
|
2 Participants
n=80 Participants
|
18 Participants
n=296 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=41 Participants
|
0 Participants
n=39 Participants
|
0 Participants
n=80 Participants
|
0 Participants
n=296 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=41 Participants
|
0 Participants
n=39 Participants
|
0 Participants
n=80 Participants
|
0 Participants
n=296 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=41 Participants
|
0 Participants
n=39 Participants
|
0 Participants
n=80 Participants
|
3 Participants
n=296 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=41 Participants
|
0 Participants
n=39 Participants
|
0 Participants
n=80 Participants
|
0 Participants
n=296 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=41 Participants
|
0 Participants
n=39 Participants
|
1 Participants
n=80 Participants
|
3 Participants
n=296 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=41 Participants
|
4 Participants
n=39 Participants
|
1 Participants
n=80 Participants
|
12 Participants
n=296 Participants
|
|
Race (NIH/OMB)
More than one race
|
2 Participants
n=41 Participants
|
0 Participants
n=39 Participants
|
0 Participants
n=80 Participants
|
2 Participants
n=296 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=41 Participants
|
0 Participants
n=39 Participants
|
0 Participants
n=80 Participants
|
1 Participants
n=296 Participants
|
|
Region of Enrollment
United States
|
12 participants
n=41 Participants
|
4 participants
n=39 Participants
|
2 participants
n=80 Participants
|
18 participants
n=296 Participants
|
|
Region of Enrollment
Canada
|
2 participants
n=41 Participants
|
0 participants
n=39 Participants
|
0 participants
n=80 Participants
|
2 participants
n=296 Participants
|
|
Region of Enrollment
Finland
|
1 participants
n=41 Participants
|
0 participants
n=39 Participants
|
0 participants
n=80 Participants
|
1 participants
n=296 Participants
|
PRIMARY outcome
Timeframe: Baseline up to 12 monthsPopulation: Change in BMI was a primary outcome for only 1 arm (Twice weekly glucocorticoid).
Participant body mass index change (weight and height will be combined to report BMI in kg/m\^2) over the course of one year
Outcome measures
| Measure |
Twice Weekly Glucocorticoid Without Exercise
n=15 Participants
Steroid-naive boys with DMD were administered twice-weekly, low dose steroids (prednisone 0.75 mg/kg) for 12 months.
|
Daily Glucocorticoid With Exercise
n=4 Participants
Patients on daily glucocorticoids will undergo 6 months of structured, supervised and home-based exercise training involving a combination of aerobic and isometric leg strength exercises.
|
Twice Weekly Glucocorticoids With Exercise
n=2 Participants
At 6 months, boys were randomized to 6 months of exercise training while continuing twice weekly steroids, or no exercise.
|
|---|---|---|---|
|
Change in BMI
|
0.67 kg/m^2
Standard Deviation 1.4
|
1.34 kg/m^2
Standard Deviation 1.7
|
1.8 kg/m^2
Standard Deviation 1.9
|
Adverse Events
Twice Weekly Glucocorticoid Without Exercise
Serious events: 1 serious events
Other events: 3 other events
Deaths: 0 deaths
Daily Glucocorticoid With Exercise
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Twice Weekly Glucocorticoids With Exercise
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Twice Weekly Glucocorticoid Without Exercise
n=17 participants at risk；n=15 participants at risk
Steroid-naive boys with DMD were administered twice-weekly, low dose steroids (prednisone 0.75 mg/kg) for 12 months. At 6 months, boys were randomized to 6 months of exercise training while continuing twice weekly steroids, or no exercise.
|
Daily Glucocorticoid With Exercise
n=4 participants at risk
Patients on daily glucocorticoids will undergo 6 months of structured, supervised and home-based exercise training.
involving a combination of aerobic and isometric leg strength exercises.
|
Twice Weekly Glucocorticoids With Exercise
n=2 participants at risk
Steroid-naive boys with DMD were administered twice-weekly, low dose steroids (prednisone 0.75 mg/kg) for 12 months. At 6 months, boys were randomized to 6 months of exercise training while continuing twice weekly steroids, or no exercise.
|
|---|---|---|---|
|
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
|
6.7%
1/15 • Through study completion, an average of 12 months for twice-weekly steroids and an average of 6 months for daily steroids and exercise; and an average for 6 months for twice weekly glucocorticoids and exercise
|
0.00%
0/4 • Through study completion, an average of 12 months for twice-weekly steroids and an average of 6 months for daily steroids and exercise; and an average for 6 months for twice weekly glucocorticoids and exercise
|
0.00%
0/2 • Through study completion, an average of 12 months for twice-weekly steroids and an average of 6 months for daily steroids and exercise; and an average for 6 months for twice weekly glucocorticoids and exercise
|
Other adverse events
| Measure |
Twice Weekly Glucocorticoid Without Exercise
n=17 participants at risk；n=15 participants at risk
Steroid-naive boys with DMD were administered twice-weekly, low dose steroids (prednisone 0.75 mg/kg) for 12 months. At 6 months, boys were randomized to 6 months of exercise training while continuing twice weekly steroids, or no exercise.
|
Daily Glucocorticoid With Exercise
n=4 participants at risk
Patients on daily glucocorticoids will undergo 6 months of structured, supervised and home-based exercise training.
involving a combination of aerobic and isometric leg strength exercises.
|
Twice Weekly Glucocorticoids With Exercise
n=2 participants at risk
Steroid-naive boys with DMD were administered twice-weekly, low dose steroids (prednisone 0.75 mg/kg) for 12 months. At 6 months, boys were randomized to 6 months of exercise training while continuing twice weekly steroids, or no exercise.
|
|---|---|---|---|
|
Renal and urinary disorders
bed wetting
|
17.6%
3/17 • Through study completion, an average of 12 months for twice-weekly steroids and an average of 6 months for daily steroids and exercise; and an average for 6 months for twice weekly glucocorticoids and exercise
|
0.00%
0/4 • Through study completion, an average of 12 months for twice-weekly steroids and an average of 6 months for daily steroids and exercise; and an average for 6 months for twice weekly glucocorticoids and exercise
|
0.00%
0/2 • Through study completion, an average of 12 months for twice-weekly steroids and an average of 6 months for daily steroids and exercise; and an average for 6 months for twice weekly glucocorticoids and exercise
|
|
Gastrointestinal disorders
Vomit
|
5.9%
1/17 • Through study completion, an average of 12 months for twice-weekly steroids and an average of 6 months for daily steroids and exercise; and an average for 6 months for twice weekly glucocorticoids and exercise
|
0.00%
0/4 • Through study completion, an average of 12 months for twice-weekly steroids and an average of 6 months for daily steroids and exercise; and an average for 6 months for twice weekly glucocorticoids and exercise
|
0.00%
0/2 • Through study completion, an average of 12 months for twice-weekly steroids and an average of 6 months for daily steroids and exercise; and an average for 6 months for twice weekly glucocorticoids and exercise
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place