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

NCT07092540

RecruitingN/A

Sponsor: University of Rochester

Conditions: Duchenne Muscular Dystrophy (DMD)

Countries: United States

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.

Eligibility overview

Sex: MALE

Age: 0 Days to 3 Years

Healthy volunteers: No

Study type: OBSERVATIONAL

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.

Locations (4)

Rochester, New York, United States
Stony Brook, New York, United States
Cincinnati, Ohio, United States
Columbus, Ohio, United States