For Boys Aged 1-30 Months With Duchenne Muscular Dystrophy Taking Oral Liquid Prednisolone Once Weekly
Phase: Recruiting
First Posted: March
Condition(s): Duchenne Muscular Dystrophy (DMD)
Other Study ID Number(s): Infant Steroid Phase II
What Is the Purpose of This Study?
To determine if a lower dose of intermittent oral corticosteroids (5mg/kg/week) will be equally effective to the 10mg/kg/week dose.
Who Can Take Part in This Study?
- Male patients 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
For a full list of eligibility requirements, visit https://clinicaltrials.gov/study/NCT05412394
What Will Happen During This Study?
- Patients will take oral prednisone once a week at a dose of 5mg/kg
- Gross Motor Outcome is the primary outcome and will be tested at visits every six months over 2 years.
Principal Investigator
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Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is a rare condition defined by worsening muscle weakness and damage. It is the most common inherited neuromuscular disorder that affects all races and ethnicities. DMD only affects males and children with DMD may lose the ability to walk as early as 7 years of age.
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