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Clinical trialNCT02167217CompletedPhase 2Interventional

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

SponsorWashington University School of MedicineEnrollment25Duchenne Muscular Dystrophy
View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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

Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

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Sites

6 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States
  • University of California Davis

    Sacramento, California, United States

  • Nemours Hospital

    Orlando, Florida, United States

  • Laurie Children'S Hospital of Chicago

    Chicago, Illinois, United States

  • Washington University in Saint Louis

    St Louis, Missouri, United States

  • Research Institute Center for Gene Therapy at Nationwide Children'S Hospital

    Columbus, Ohio, United States

  • University of Texas South Western Medical Center of Dallas

    Dallas, Texas, United States

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start
17 Apr 2014
Primary completion
22 Feb 2017
Completion
22 Mar 2017
Last update posted
21 Dec 2018
Registered enrollment
25
Registry id
NCT02167217

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.