Actively Recruiting
The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy
Led by University of Rochester · Updated on 2026-05-04
105
Participants Needed
1
Research Sites
169 weeks
Total Duration
On this page
Sponsors
U
University of Rochester
Lead Sponsor
U
United States Department of Defense
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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 will not qualify if you...
- 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.
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
University of Rochester
Rochester, New York, United States, 14618
Actively Recruiting
Research Team
K
Kimberly A Hart, MA
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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