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Completed Phase 2 Interventional Results available

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

ClinicalTrials.gov ID: NCT02167217

Public ClinicalTrials.gov record NCT02167217. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 2, 2026, 11:03 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

Phase 2 Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

While it has been known for many years that corticosteroid use benefits boys with Duchenne Muscular dystrophy (DMD), most clinicians do not consider treating until after age 3 or 4 years of age. The primary reason for the delay is that daily corticosteroid use has many side effects including short stature, obesity, and osteoporosis. A recent randomized blinded study of weekend oral corticosteroid use over one year showed equal improvement in strength with fewer side effects, particularly as related to growth and cushingoid changes. The investigators will test the efficacy of oral weekend corticosteroid use in infants and young boys with DMD who are under age 30 months. The investigators have demonstrated that the Bayley-III Scales of Infant development shows that infants and young boys in this age group who are untreated decline in abilities when compared to their peers. Here, in this Phase 2 historically controlled trial, the investigators will use these two measures and treat boys at five Muscular Dystrophy Association-DMD centers

Study identification

NCT ID
NCT02167217
Recruitment status
Completed
Study type
Interventional
Phase
Phase 2
Enrollment
25 participants

Conditions and interventions

Interventions

Drug

Eligibility (public fields only)

Age range
1 Month to 30 Months
Sex
Male
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Apr 16, 2014
Primary completion
Feb 21, 2017
Completion
Mar 21, 2017
Last update posted
Dec 20, 2018

2014 – 2017

United States locations

U.S. sites
6
U.S. states
6
U.S. cities
6
Facility City State ZIP Site status
University of California Davis Sacramento California 95817
Nemours Hospital Orlando Florida 32827
Laurie Children's Hospital Of Chicago Chicago Illinois 60611-2605
Washington University in Saint Louis St Louis Missouri 63109
Research Institute Center for Gene Therapy at Nationwide Children's Hospital Columbus Ohio 43205
University of Texas South Western Medical Center of Dallas Dallas Texas 75390

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT02167217, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Dec 20, 2018 · Synced Sep 2, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT02167217 live on ClinicalTrials.gov.

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