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The Baby Duchenne Study: Understanding Early Development in Duchenne Muscular Dystrophy

This study, called The Baby Duchenne Study, aims to understand how Duchenne Muscular Dystrophy (DMD) affects very young children, specifically from birth to three years old. Researchers want to learn about the natural progression of the condition and early developmental outcomes. This is an observational study, meaning no specific treatments are being tested. To join, your child must be a male between birth and 3 years old with a confirmed genetic change (pathogenic or likely pathogenic variant) in the DMD gene. The study will measure changes in gross motor skills using a special test called the Bayley Scales of Infant and Toddler Development over 36 months. The study is currently unclear on its recruitment status.

Study design
This is an observational study planning to enroll 105 male participants aged 0 days to 3 years.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for 36 months to measure changes in gross motor skills.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07092540

The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy

Recruiting
Not specifiedAges 0–3Observational
University of Rochester
~105 participants
Updated 2026-08-19 on ClinicalTrials.gov

At a glance

Recruiting sites
2 of 4 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Mean Change in Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4) Gross Motor Standard Score
Measured over Baseline to 36 months

Conditions

Duchenne Muscular Dystrophy (DMD)

Explore related trials

Duchenne Muscular DystrophyDMD Gene

Where it's being run

4 sites across 2 states
New York2
Ohio2

Who to contact

Kimberly A Hart, MA
[email protected]
585-275-3767
Email the study team

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Eligibility criteria

Inclusion

Male child between birth and 3.0 years of age at time of enrollment.
A confirmed and documented pathogenic or likely pathogenic variant in the DMD gene.
Ability of parent/guardian to understand and provide written informed consent (signing Parental Permission and Consent Form).
Willingness of parent/guardian to comply with the protocol Schedule of Activities, including all study site visits.

Exclusion

Female
Presence of any confirmed genetic disease, other than DMD, that could impact early development, which, in the opinion of the PI, may confound interpretation of developmental progress.
Presence of any significant medical condition (i.e., extreme prematurity, hypoxic ischemic encephalopathy) which, in the opinion of the PI, may confound interpretation of the clinical course of DMD.
Inability/unwillingness of parent/guardian to provide written permission (sign PPF) or to comply with the protocol Schedule of Activities.

What this trial measures

  • Mean Change in Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4) Gross Motor Standard ScoreBaseline to 36 months

    The Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4), Gross Motor domain measures age-appropriate motor abilities such as crawling, standing, and walking in children aged 1 to 42 months. The Gross Motor Standard Score is derived by converting raw scores to age-normed standard scores (mean = 100; standard deviation = 15). Higher scores indicate better gross motor development.