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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

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
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


Participant Flow:   Overall Study
    Oral Prednisolone
STARTED   25 
COMPLETED   23 
NOT COMPLETED   2 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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.

Reporting Groups
  Description
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

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.


Baseline Measures
   Oral Prednisolone 
Overall Participants Analyzed 
[Units: Participants]
 25 
Age [1] 
[Units: Participants]
Count of Participants
 
Participants Analyzed   23 
<=18 years      23 100.0% 
Between 18 and 65 years      0   0.0% 
>=65 years      0   0.0% 
[1] The analysis population does not include the two who dropped out.
Age [1] 
[Units: Years]
Mean (Standard Deviation)
 
Participants Analyzed   23 
   1.5  (0.8) 
[1] The 23 analyzed does not include the two who dropped out.
Sex: Female, Male [1] 
[Units: Participants]
Count of Participants
 
Participants Analyzed   23 
Female      0   0.0% 
Male      23 100.0% 
[1] The number analyzed does not include the two who dropped out. See above.
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
 
Participants Analyzed   25 
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] 
[Units: Participants]
Count of Participants
 
United States   
Participants Analyzed   23 
United States   23 
[1] The number analyzed does not include the two who dropped out
Bayley III Gross Motor Scaled Score [1] [2] 
[Units: Units on a scale]
Mean (Standard Deviation)
 
Participants Analyzed   23 
   4.2  (2.5) 
[1] 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] The number analyzed does not include the two who dropped out.


  Outcome Measures

1.  Primary:   Bayley III Gross Motor Scaled Score (Change From Baseline to 12 Month)   [ Time Frame: One year ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

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
e-mail: connollya@wustl.edu


Publications:

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