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

NCT02167217 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 25

Last updated 2018-12-21

Study results available
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Summary

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

Conditions

Interventions

DRUG

Prednisolone

Prednisolone (5mg per kg) will be taken on two consecutive days, Friday and Saturday mornings each week with breakfast

Sponsors & Collaborators

  • Nationwide Children's Hospital

    collaborator OTHER

  • Northwestern University Feinberg School of Medicine

    collaborator OTHER

  • University of Texas Southwestern Medical Center

    collaborator OTHER

  • University of California, Davis

    collaborator OTHER

  • Nemours Children's Hospital

    collaborator OTHER

  • Washington University School of Medicine

    lead OTHER

Principal Investigators

  • Anne M Connolly, MD · Washington University School of Medicine

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Month
Max Age
30 Months
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-04-17
Primary Completion
2017-02-22
Completion
2017-03-22

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02167217 on ClinicalTrials.gov