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

RecruitingObservational study

Who can join

0 Days – 3 Years · Men

Full eligibility criteria
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.

About the study

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.

Sponsor: University of Rochester · Participants: 105 · Started: Jun 3, 2026

Contact the study team

Official record on ClinicalTrials.gov — NCT07092540

Locations in the U.S.

New YorkUniversity of Rochester, Rochester
Stony Brook Medicine, Stony Brook (Not yet recruiting)
OhioCincinnati Children's Hospital Medical Center, Cincinnati (Not yet recruiting)
Nationwide Children's Hospital, Columbus

Conditions

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.