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Recruiting NCT07092540

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

— Observational Duchenne Muscular Dystrophy (DMD)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
This is an observational study: the protocol does not assign a study treatment.
Who it may be relevant to
Registry conditions: Duchenne Muscular Dystrophy (DMD). Basic parameters: 0 Days — 3 years · Male.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Overview

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.

Primary outcome measures

  • Mean Change in Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4) Gross Motor Standard Score [Time frame: Baseline to 36 months]
Secondary outcome measures (12)
  • Mean Change in Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4) Cognitive Standard Score [Time frame: Baseline to 36 months]
  • Mean Change in Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4) Fine Motor Standard Score [Time frame: Baseline to 36 months]
  • Mean Change in Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4) Expressive Language Standard Score [Time frame: Baseline to 36 months]
  • Mean Change in Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4) Receptive Language Standard Score [Time frame: Baseline to 36 months]
  • Mean Change in World Health Organization Motor Milestone (WHOMM) Checklist Score [Time frame: Baseline to 36 months]
  • Percentage of Participants Receiving Early Intervention Services [Time frame: Baseline to 36 months]
  • Age at Initiation of Early Intervention Services [Time frame: Baseline to 36 months]
  • Percentage of Participants Initiating Duchenne Muscular Dystrophy-Targeted Pharmacologic Treatment [Time frame: Baseline to 36 months]
  • Age at Initiation of Duchenne Muscular Dystrophy-Targeted Pharmacologic Treatment [Time frame: Baseline to 36 months]
  • Percentage of Participants With a Neurobehavioral Diagnosis [Time frame: Baseline to 36 months]
  • Age at First Neurobehavioral Diagnosis [Time frame: Baseline to 36 months]
  • Mean Change in Duchenne Muscular Dystrophy Caregiver Reported Health Index Short Form (DMD-HI-SF) Score [Time frame: Baseline to 36 months]

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Other

Study locations

United States · 1 center
  • University of Rochester — Rochester

Identifiers

NCT: NCT07092540 · STUDY00009939 · US Department of Defense

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗