Lower dose corticosteroid treatment for infants with Duchenne Muscular Dystrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• VM100: a nutritional supplement to see if it improves quality of life for boys with Duchenne

  This Phase 2 study tests VM100, a nutritional drink, in boys aged 8 and up with Duchenne muscular dystrophy to see if it can improve quality of life and reduce fatigue.

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• KER-065 for Duchenne Muscular Dystrophy: safety and potential benefit in boys and men

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• GNR-097 gene therapy for Duchenne muscular dystrophy in boys

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  Recruiting at 6 sites Phase 1/2 Ao Generium · Minsk and 5 other locations · NCT07673809

• ELEVIDYS safety in Duchenne patients treated after market approval

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  Recruiting Phase 4 Sarepta Therapeutics, Inc. · Houston, Texas · NCT07542314

• SGT-003 gene therapy for ambulant boys and young men with Duchenne muscular dystrophy (IMPACT DUCHENNE)

  This trial will test whether a single intravenous infusion of SGT-003 can help ambulatory boys and young men with Duchenne muscular dystrophy maintain or improve walking and motor function.

  Recruiting at 4 sites Phase 3 Solid Biosciences Inc. · Little Rock, Arkansas and 6 other locations · NCT07160634

• Evaluating Givinostat for Young Children with Duchenne Muscular Dystrophy

  This study is testing a new drug called Givinostat to see if it's safe and how it works in young children aged 2 to 6 with Duchenne Muscular Dystrophy.

  Recruiting at 7 sites Phase 2 Italfarmaco · Brussels and 8 other locations · NCT06769633

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.