Baby Duchenne: Tracking development in boys with Duchenne muscular dystrophy during their first three years

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

The Baby Duchenne study follows male infants and toddlers with a confirmed pathogenic or likely pathogenic DMD gene variant, identified through newborn screening, to document early clinical and developmental outcomes from birth to age three. Parents provide consent and bring the child to scheduled study visits where clinicians collect developmental measures, medical history, physical exams, and other relevant clinical data. The protocol is observational with no experimental treatments, aiming to build a detailed natural-history dataset that can inform clinical care and the design of future trials. Collected data will help define early milestones, onset timing of symptoms, and potential early markers of disease progression.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* 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 Criteria:

* 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.

Where it's running

• University of Rochester
  Rochester, New York, United States Recruiting
• Nationwide Children's Hospital
  Columbus, Ohio, United States Recruiting
• Stony Brook Medicine
  Stony Brook, New York, United States Not yet recruiting
• Cincinnati Children's Hospital Medical Center
  Cincinnati, Ohio, United States Not yet recruiting

Study contacts

• Study coordinator: Kimberly A Hart, MA
• Email: Kim_Hart@urmc.rochester.edu
• Phone: 585-275-3767

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

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• Zeleciment Rostudirsen (DYNE-251) for Duchenne Muscular Dystrophy in Ambulatory Boys

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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.