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Once Weekly Infant Corticosteroid Trial for Duchenne Muscular Dystrophy

This study is looking at a lower weekly dose of prednisolone (a type of corticosteroid) for infants and young boys with Duchenne Muscular Dystrophy (DMD). Researchers want to see if giving 5mg/kg of prednisolone once a week is as effective as a higher dose (10mg/kg/week) but with fewer side effects, like weight gain. You might be able to join if you are a boy between 1 month and 30 months old, have weakness consistent with DMD, very high creatine kinase levels (a marker of muscle damage), and a confirmed genetic mutation causing DMD. The study will measure changes in gross motor skills (like sitting, crawling, and walking) over 24 months to see if the treatment is successful. The current recruitment status is unclear.

Study design
This is an unblinded interventional study, meaning both participants and researchers will know who is receiving the prednisolone. It plans to enroll 26 male participants.
What's involved
Participants will receive liquid prednisolone once a week for one year. Gross motor function will be assessed at baseline and then again at the 24-month visit.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for two years after starting the study treatment.

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NCT05412394

Once Weekly Infant Corticosteroid Trial for DMD

Recruiting
PHASE4Ages 1–30InterventionalTreatment
Anne M. Connolly
~26 participants
Updated 2026-08-14 on ClinicalTrials.gov
What's tested:Prednisolone

At a glance

Recruiting sites
4 of 4 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
The change from baseline to 24 months for the Gross Motor Scaled Score.
Measured over Baseline visit to 24 month visit

Conditions

Duchenne Muscular Dystrophy

Explore related trials

Duchenne Muscular DystrophyCreatine KinaseDMD Gene

Where it's being run

4 sites across 3 states
Ohio2
Illinois1
Texas1

Study leadership

  • Anne Connolly, MD · PRINCIPAL_INVESTIGATOR · Nationwide Children's Hospital

Who to contact

Sara Marshall
[email protected]
614-355-3508
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

Prior treatment with Glucocorticosteroids

What this trial measures

  • The change from baseline to 24 months for the Gross Motor Scaled Score.Baseline visit to 24 month visit

    Neuromuscular Gross Motor Outcome (GRO): The Neuromuscular GRO is a gross motor outcome measure developed to assess whole body strength, motor development, and function for all levels of ability across the lifespan in those diagnosed with neuromuscular disease. Items are administered following the developmental sequence, as appropriate for age and ability. Maximum score is 100 points.