NCT07092540

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
105

participants targeted

Target at P50-P75 for all trials

Timeline
40mo left

Started May 2026

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 22, 2025

Completed
8 days until next milestone

First Posted

Study publicly available on registry

July 30, 2025

Completed
10 months until next milestone

Study Start

First participant enrolled

May 30, 2026

Expected
3.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 31, 2029

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 31, 2029

Last Updated

May 4, 2026

Status Verified

April 1, 2026

Enrollment Period

3.3 years

First QC Date

July 22, 2025

Last Update Submit

April 27, 2026

Conditions

Duchenne Muscular Dystrophy (DMD)

Keywords

New born screening

Outcome Measures

Primary Outcomes (1)

  • Mean Change in Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4) Gross Motor Standard Score

    The Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4), Gross Motor domain measures age-appropriate motor abilities such as crawling, standing, and walking in children aged 1 to 42 months. The Gross Motor Standard Score is derived by converting raw scores to age-normed standard scores (mean = 100; standard deviation = 15). Higher scores indicate better gross motor development.

    Baseline to 36 months

Secondary Outcomes (13)

  • Mean Change in Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4) Cognitive Standard Score

    Baseline to 36 months

  • Mean Change in Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4) Fine Motor Standard Score

    Baseline to 36 months

  • Mean Change in Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4) Expressive Language Standard Score

    Baseline to 36 months

  • Mean Change in Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4) Receptive Language Standard Score

    Baseline to 36 months

  • Mean Change in World Health Organization Motor Milestone (WHOMM) Checklist Score

    Baseline to 36 months

  • +8 more secondary outcomes

Study Arms (1)

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

Eligibility Criteria

Age0 Days - 3 Years
Sexmale(Gender-based eligibility)
Healthy VolunteersNo
Age GroupsChild (0-17)
Sampling MethodNon-Probability Sample
Study Population

Males with genetically confirmed diagnosis of DMD (Duchenne Muscular Dystrophy)

You may qualify if:

  • 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 may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Rochester

Rochester, New York, 14618, United States

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Kimberly A Hart, MA

CONTACT

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

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Assistant professor of neurology

Study Record Dates

First Submitted

July 22, 2025

First Posted

July 30, 2025

Study Start (Estimated)

May 30, 2026

Primary Completion (Estimated)

August 31, 2029

Study Completion (Estimated)

August 31, 2029

Last Updated

May 4, 2026

Record last verified: 2026-04

Locations

US(1)