Lower dose corticosteroid treatment for infants with Duchenne Muscular Dystrophy
Phase-2 Trial of 5mg/kg/Week Prednisolone in Young Boys With DMD
This study is testing if a lower dose of corticosteroids can help infants and young boys with Duchenne Muscular Dystrophy improve their movement skills while causing fewer side effects than a higher dose.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 26 (estimated) |
| Ages | 1 Month to 30 Months |
| Sex | Male |
| Sponsor | Nationwide Children's Hospital Academic / other |
| Locations | 4 sites (Chicago, Illinois and 3 other locations) |
| Trial ID | NCT05412394 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the effectiveness of a lower dose of oral corticosteroids (5mg/kg/week) compared to a higher dose (10mg/kg/week) in infants and young boys diagnosed with Duchenne Muscular Dystrophy (DMD). The study aims to determine if the lower dose can achieve similar improvements in gross motor function while minimizing side effects commonly associated with corticosteroid use. Participants aged 1 month to 30 months will be monitored over two years, with assessments using various developmental scales. The goal is to provide a safer treatment option for infants affected by DMD.
Who should consider this trial
Good fit: Ideal candidates for this study are infants and young boys aged 1 month to 30 months who exhibit weakness consistent with Duchenne Muscular Dystrophy and have specific genetic mutations.
Not a fit: Patients who have previously been treated with glucocorticosteroids will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a safer corticosteroid treatment option for infants with Duchenne Muscular Dystrophy, potentially reducing side effects while maintaining efficacy.
How similar studies have performed: Previous studies have shown that lower doses of corticosteroids can be effective in managing DMD, suggesting potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Subjects ages 1 month through 30 months * Weakness consistent with Duchenne on exam, creatine kinase ≥ 20 times the upper limit of normal, and genetic mutation known to be causative for DMD. Exclusion Criteria: * Prior treatment with Glucocorticosteroids
Where this trial is running
Chicago, Illinois and 3 other locations
- Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (Recruiting)
- University of Cincinnati — Cincinnati, Ohio, United States (Recruiting)
- Nationwide Children's Hospital — Columbus, Ohio, United States (Recruiting)
- University of Texas Southwestern — Dallas, Texas, United States (Recruiting)
Study contacts
- Principal investigator: Anne Connolly, MD — Nationwide Children's Hospital
- Study coordinator: Sara Marshall
- Email: sara.adamczak@nationwidechildrens.org
- Phone: 614-355-3508
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.