Health TrialsbyHealthPanel

Once Weekly Infant Corticosteroid Trial for DMD

ConditionDuchenne Muscular Dystrophy
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexMale
Age1-30
SponsorAnne M. Connolly

About this trial

The hypothesis tested here is that a lower dose of intermittent oral corticosteroids (5mg/kg/week) will be equally effective to the 10mg/kg/week dose.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Subjects ages 1 month through 30 months

Weakness consistent with Duchenne on exam, creatine kinase ≥ 20 times the upper limit of normal, and genetic mutation known to be causative for DMD.

Disqualifiers

Prior treatment with Glucocorticosteroids

Trial design

Design model

Single group

Treatments tested in this trial

  • Prednisolone

    Drug

    Liquid, 5mg/kg per week, for one year

Treatment groups

26 Participants
are divided into 1 treatment group
Group A: ExperimentalExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

The change from baseline to 24 months for the Gross Motor Scaled Score.

Neuromuscular Gross Motor Outcome (GRO): The Neuromuscular GRO is a gross motor outcome measure developed to assess whole body strength, motor development, and function for all levels of ability across the lifespan in those diagnosed with neuromuscular disease. Items are administered following the developmental sequence, as appropriate for age and ability. Maximum score is 100 points.

Time frame
Baseline visit to 24 month visit

Secondary outcomes

1

Language (expressive and receptive), Social and Fine Motor skills at 24 months as assessed by the Bayley-4 Scales of Infant and Toddler Development

The Bayley Scales of Infant and Toddler Development (Bayley-4) are recognized internationally as a comprehensive tool to assess children from as young as 15 days old. With Bayley-4, it is possible to obtain detailed information from non-verbal children as to their functioning. Children are assessed in the five key developmental domains of cognition, language (receptive and expressive), \& motor (fine and gross).

Time frame
Baseline visit to 24 month visit
2

Linear growth

We have previously shown that all infants and young children treated with 10mg/kg/week did maintain their linear growth. This protocol is designed to determine also if the lower dose will still maintain benefit.

Time frame
Baseline visit to 24 month visit

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Anne M. Connolly

Lead sponsor

Nationwide Children's Hospital

Sponsor institution

Children's Hospital Medical Center, Cincinnati

Collaborator

Muscular Dystrophy Association

Collaborator

University of Texas

Collaborator

Virginia Commonwealth University

Collaborator

Ann & Robert H Lurie Children's Hospital of Chicago

Collaborator

Source ClinicalTrials.gov
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