Trial Outcomes & Findings for Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy (NCT NCT02167217)
NCT ID: NCT02167217
Last Updated: 2018-12-21
Results Overview
Bayley III Gross Motor Scaled Score measures motor development. This is normed for typically developing children and follow a bell shaped curve. The scale has mean of 10 +/-3 for children at all ages and is bell shaped. Therefore the two standard deviation range is 16 to 4 with higher values indicated better performance. Lower values have been shown to be common in boys with DMD and it this study the baseline average score was 4.2.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
25 participants
Primary outcome timeframe
One year
Results posted on
2018-12-21
Participant Flow
Participant milestones
| Measure |
Oral Prednisolone
Oral Prednisolone 5mg/kg/ day on two consecutive days, Friday and Saturday with breakfast
Prednisolone: Prednisolone (5mg per kg )will be taken on two consecutive days, Friday and Saturday mornings each week with breakfast
|
|---|---|
|
Overall Study
STARTED
|
25
|
|
Overall Study
COMPLETED
|
23
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
The analysis population does not include the two who dropped out.
Baseline characteristics by cohort
| Measure |
Oral Prednisolone
n=25 Participants
Oral Prednisolone 5mg/kg/ day on two consecutive days, Friday and Saturday with breakfast
Prednisolone: Prednisolone (5mg per kg )will be taken on two consecutive days, Friday and Saturday mornings each week with breakfast
Twenty-five steroid naïve boys four to 30 months of age with genetically confirmed Duchenne Muscular Dystrophy (DMD) were enrolled. 25 infants and boys were enrolled and 23 completed the study. One boy was lost to follow-up and one discontinued treatment after six months secondary to side effects.
|
|---|---|
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Age, Categorical
<=18 years
|
23 Participants
n=23 Participants • The analysis population does not include the two who dropped out.
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=23 Participants • The analysis population does not include the two who dropped out.
|
|
Age, Categorical
>=65 years
|
0 Participants
n=23 Participants • The analysis population does not include the two who dropped out.
|
|
Age, Continuous
|
1.5 years
STANDARD_DEVIATION 0.8 • n=23 Participants • The 23 analyzed does not include the two who dropped out.
|
|
Sex: Female, Male
Female
|
0 Participants
n=23 Participants • The number analyzed does not include the two who dropped out. See above.
|
|
Sex: Female, Male
Male
|
23 Participants
n=23 Participants • The number analyzed does not include the two who dropped out. See above.
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=25 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=25 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=25 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=25 Participants
|
|
Race (NIH/OMB)
White
|
22 Participants
n=25 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=25 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=25 Participants
|
|
Region of Enrollment
United States
|
23 Participants
n=23 Participants • The number analyzed does not include the two who dropped out
|
|
Bayley III Gross Motor Scaled Score
|
4.2 units on a scale
STANDARD_DEVIATION 2.5 • n=23 Participants • The number analyzed does not include the two who dropped out.
|
PRIMARY outcome
Timeframe: One yearPopulation: We analyzed the change in the gross motor score after one year. An increase indicates improvement compared to peers
Bayley III Gross Motor Scaled Score measures motor development. This is normed for typically developing children and follow a bell shaped curve. The scale has mean of 10 +/-3 for children at all ages and is bell shaped. Therefore the two standard deviation range is 16 to 4 with higher values indicated better performance. Lower values have been shown to be common in boys with DMD and it this study the baseline average score was 4.2.
Outcome measures
| Measure |
Oral Prednisolone
n=23 Participants
Oral Prednisolone 5mg/kg/ day on two consecutive days, Friday and Saturday with breakfast
Prednisolone: Prednisolone (5mg per kg )will be taken on two consecutive days, Friday and Saturday mornings each week with breakfast
|
|---|---|
|
Bayley III Gross Motor Scaled Score (Change From Baseline to 12 Month)
|
4.8 units on a scale
Standard Deviation 2.3
|
Adverse Events
Oral Prednisolone
Serious events: 0 serious events
Other events: 22 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Oral Prednisolone
n=25 participants at risk
Oral Prednisolone 5mg/kg/ day on two consecutive days, Friday and Saturday with breakfast
Prednisolone: Prednisolone (5mg per kg )will be taken on two consecutive days, Friday and Saturday mornings each week with breakfast
|
|---|---|
|
Nervous system disorders
irritability
|
16.0%
4/25 • Number of events 7 • Data for adverse event were collected over one year.
60 adverse events (AEs) were reported; 15 were possibly or probably related to medication. There were no severe AEs.
|
|
Respiratory, thoracic and mediastinal disorders
Upper Respiratory Track Infection
|
56.0%
14/25 • Number of events 17 • Data for adverse event were collected over one year.
60 adverse events (AEs) were reported; 15 were possibly or probably related to medication. There were no severe AEs.
|
|
Gastrointestinal disorders
Gastroenteritis
|
20.0%
5/25 • Number of events 5 • Data for adverse event were collected over one year.
60 adverse events (AEs) were reported; 15 were possibly or probably related to medication. There were no severe AEs.
|
|
Infections and infestations
Ear Infection
|
20.0%
5/25 • Number of events 5 • Data for adverse event were collected over one year.
60 adverse events (AEs) were reported; 15 were possibly or probably related to medication. There were no severe AEs.
|
|
Skin and subcutaneous tissue disorders
Rash
|
12.0%
3/25 • Number of events 3 • Data for adverse event were collected over one year.
60 adverse events (AEs) were reported; 15 were possibly or probably related to medication. There were no severe AEs.
|
|
General disorders
Fever
|
16.0%
4/25 • Number of events 4 • Data for adverse event were collected over one year.
60 adverse events (AEs) were reported; 15 were possibly or probably related to medication. There were no severe AEs.
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Additional Information
Anne M. Connolly, MD FAAN
Washington University School of Medicine
Phone: 314-362-6981
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place