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

Plain-language summary

This study evaluates the drug Prednisolone (a corticosteroid) in young boys with Duchenne muscular dystrophy (a progressive muscle disease caused by mutations in the DMD gene). It included male infants aged 1-30 months with a genetically confirmed DMD diagnosis. It was a Phase 2 study and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 2

Status

Completed

Sponsor

Washington University School of Medicine

Intervention

Prednisolone

Start date

4/17/2014

Estimated completion

3/22/2017

Countries

United States

Related genes

Locations

• Sacramento, United States
• Orlando, United States
• Chicago, United States
• St Louis, United States
• Columbus, United States
• Dallas, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

1. Appropriate degree of weakness for age, creatine kinase greater than 20 times the upper limit of normal, and genetic mutation known to be causative for Duchenne muscular dystrophy .
2. Appropriate degree of weakness for age, creatine kinase greater than 20 times the upper limit of normal and genetic or biopsy confirmation of Duchenne muscular dystrophy in a primary relative (e.g. brother or maternal uncle).
3. De-identified, genetic studies will be reviewed by collaborator Kevin Flanigan, MD prior to enrollment of subjects.
4. Age at entry: one month through 30 months.

Exclusion Criteria:

* Prior treatment with corticosteroids