Recruiting

Observational Study

Sponsor:

University of Rochester

Code:

NCT07092540

Conditions

Duchenne Muscular Dystrophy (DMD)

Eligibility Criteria

Sex: Male

Age: 0 - 3

Healthy Volunteers: Not accepted

Study Details

Brief summary:

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

Duchenne Muscular Dystrophy (DMD)

Study ID

NCT07092540

Start date

Jun 3, 2026

Status verified date

Aug, 2026

Completion date

Aug 31, 2029

Anticipated

Primary completion date

Aug 31, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Male

Age: 0 - 3

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

105 participants

Anticipated

Interventions and Outcome Measures

Arms

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

Primary outcome measure

  • Mean Change in Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4) Gross Motor Standard Score [ Time Frame: Baseline to 36 months ]

Central Contacts and Locations

Central contacts

Kimberly A Hart, MA

585-275-3767Kim_Hart@urmc.rochester.edu

Locations

University of Rochester

Recruiting

Rochester, New York, United States, 14618

Nationwide Children's Hospital

Recruiting

Columbus, Ohio, United States, 43205

Contacts

Smitha Poondla

614-722-8227Smitha.Poondla@nationwidechildrens.org

Principal Investigator:

Megan Waldrop, M.D.

More Information

Sponsor

University of Rochester

Last update posted

Aug 19, 2026

Last verified

Aug, 2026

Keywords

  • New born screening

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by University of Rochester on 2026-08-19.