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

Recruiting now

Conditions studied: Duchenne Muscular Dystrophy (DMD)

In brief

The aim of the BABY DUCHENNE study is to evaluate the natural history and characterize the early clinical outcomes in very young children (0-3 years) with Duchenne muscular dystrophy (DMD) identified by newborn screening programs.

Key facts

Study ID

NCT07092540

Run by

University of Rochester

People needed

105

Starts

2026-05-30

Expected to finish

2029-08-31

Last updated by the study team

2026-05-04

Who can join

Age: any, up to 3. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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 is running

• University of Rochester — Rochester, New York, United States (enrolling)

Full record on ClinicalTrials.gov

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