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Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT02167217
Recruitment Status : Completed
First Posted : June 19, 2014
Results First Posted : May 18, 2018
Last Update Posted : May 18, 2018
Sponsor:
Collaborators:
Nationwide Children's Hospital
Feinberg School of Medicine, Northwestern University
University of Texas Southwestern Medical Center
University of California, Davis
Nemours Hospital, Orlando, FL
Information provided by (Responsible Party):
Washington University School of Medicine

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Duchenne Muscular Dystrophy
Intervention Drug: Prednisolone
Enrollment 24

Recruitment Details  
Pre-assignment Details  
Arm/Group Title Oral Prednisolone
Hide Arm/Group Description

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

Period Title: Overall Study
Started 25
Completed 23
Not Completed 2
Arm/Group Title Oral Prednisolone
Hide Arm/Group Description

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.

Overall Number of Baseline Participants 25
Hide Baseline Analysis Population Description
Two of the 25 dropped out of study. One was lost to followup within a few months and the second discontinued the study after 6 months.
Age, Categorical   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 23 participants
<=18 years
23
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
[1]
Measure Analysis Population Description: The analysis population does not include the two who dropped out.
Age, Continuous   [1] 
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 23 participants
1.5  (0.8)
[1]
Measure Analysis Population Description: The 23 analyzed does not include the two who dropped out.
Sex: Female, Male   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 23 participants
Female
0
   0.0%
Male
23
 100.0%
[1]
Measure Analysis Population Description: The number analyzed does not include the two who dropped out. See above.
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 25 participants
American Indian or Alaska Native
0
   0.0%
Asian
1
   4.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
1
   4.0%
White
22
  88.0%
More than one race
0
   0.0%
Unknown or Not Reported
1
   4.0%
Region of Enrollment   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 23 participants
23
 100.0%
[1]
Measure Analysis Population Description: The number analyzed does not include the two who dropped out
Bayley III Gross Motor Scaled Score   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 23 participants
4.2  (2.5)
[1]
Measure Description: This scale is normed to typically developing children where at any age a child's expected score is 10 +/-3. This scale assesses gross motor function including sitting, standing and walking.
[2]
Measure Analysis Population Description: The number analyzed does not include the two who dropped out.
1.Primary Outcome
Title Bayley III Gross Motor Scaled Score (Change From Baseline to 12 Month)
Hide Description 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.
Time Frame One year
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
We analyzed the change in the gross motor score after one year. An increase indicates improvement compared to peers
Arm/Group Title Oral Prednisolone
Hide Arm/Group Description:

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 Number of Participants Analyzed 23
Mean (Standard Deviation)
Unit of Measure: units on a scale
4.8  (2.3)
Time Frame Data for adverse event were collected over one year.
Adverse Event Reporting Description 60 adverse events (AEs) were reported; 15 were possibly or probably related to medication. There were no severe AEs.
 
Arm/Group Title Oral Prednisolone
Hide Arm/Group Description

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

All-Cause Mortality
Oral Prednisolone
Affected / at Risk (%)
Total   0/25 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Oral Prednisolone
Affected / at Risk (%) # Events
Total   0/25 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Oral Prednisolone
Affected / at Risk (%) # Events
Total   22/25 (88.00%)    
Gastrointestinal disorders   
Gastroenteritis   5/25 (20.00%)  5
General disorders   
Fever   4/25 (16.00%)  4
Infections and infestations   
Ear Infection   5/25 (20.00%)  5
Nervous system disorders   
irritability  [1]  4/25 (16.00%)  7
Respiratory, thoracic and mediastinal disorders   
Upper Respiratory Track Infection   14/25 (56.00%)  17
Skin and subcutaneous tissue disorders   
Rash   3/25 (12.00%)  3
Indicates events were collected by systematic assessment
[1]
Irritability occurred 10 times in 4 children.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Anne M. Connolly, MD FAAN
Organization: Washington University School of Medicine
Phone: 314-362-6981
Responsible Party: Washington University School of Medicine
ClinicalTrials.gov Identifier: NCT02167217     History of Changes
Other Study ID Numbers: 201308062
First Submitted: February 3, 2014
First Posted: June 19, 2014
Results First Submitted: March 9, 2018
Results First Posted: May 18, 2018
Last Update Posted: May 18, 2018