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

University of Rochester

Status: Begins enrollment this month

Conditions: Duchenne Muscular Dystrophy (DMD)

Study type: Observational

Funder types: Other; Other U.S. Federal agency

Identifiers

NCT07092540

STUDY00009939

US Department of Defense (Other Grant/Funding Number)

Details and patient eligibility

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.

Enrollment: 105 estimated patients

Sex: Male

Ages: Under 3 years old

Volunteers: No Healthy Volunteers

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 following a positive newborn screen.
• 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.

Trial design

105 participants in 1 patient group

Boys (0-3) of age diagnosed with DMD via new born screening