Once Weekly Infant Corticosteroid Trial for DMD

Purpose

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.

Condition

  • Duchenne Muscular Dystrophy

Eligibility

Eligible Ages
Between 1 Month and 30 Months
Eligible Sex
Male
Accepts Healthy Volunteers
No

Inclusion Criteria

  • 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.

Exclusion Criteria

  • Prior treatment with Glucocorticosteroids

Study Design

Phase
Phase 4
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Experimental 		This is a one-arm study and the group of subjects are all experimental and will receive drug.
  • Drug: Prednisolone
    Liquid, 5mg/kg per week, for one year
    Other names:
    • Corticosteroid

Recruiting Locations

Lurie Children's Hospital of Chicago
Chicago, Illinois 60611

University of Cincinnati
Cincinnati, Ohio 45220

Nationwide Children's Hospital
Columbus, Ohio 43205
Contact:
Kevin Warf, BS
(614) 355-2765
kevin.warf@nationwidechildrens.org

University of Texas Southwestern
Dallas, Texas 75235
Contact:
Shahera Ranjha, MS
214-456-5959
shahera.ranjha@utsouthwestern.edu

More Details

Status
Recruiting
Sponsor
Anne M. Connolly

Study Contact

Sara Marshall
614-355-3508
sara.adamczak@nationwidechildrens.org

Detailed Description

The investigators know that the muscle destruction from Duchenne muscular dystrophy (DMD) begins in infancy and we previously demonstrated that motor decline in infancy compared to typically developing infants reflects that destruction. Because of the known side effects of daily corticosteroids, most physicians do not begin treatment until age 3-5 years. Most side effects (Cushingoid faces, linear growth arrest, and bone density loss) did not happen in infants and in ambulatory boys in two separate studies. However, in infants and young boys taking (10mg/kg/week), 56% of infants and young boys did have an increase in weight percentile compared to baseline. This study will test this lower dose of prednisolone (5mg/kg/week) in an unblinded study in infants and young DMD boys (ages 1 through 30 months) to determine if equal efficacy can be achieved with fewer side effects. The primary outcome for this study will be gross motor function. The study team will enroll boys from age 1 month through 30 months and follow each for two years. We will assess gross motor function using three outcome measures: the Bayley-4 Scales of Infant and Toddler Development (Bayley-4) and the newly developed Neuromuscular Gross Motor Outcomes (GRO) and the NorthStar Ambulatory Assessment (NSAA). The one-year outcome will be the change in the Bayley-4 Scaled Score and the two-year outcome will be the change in the GRO score. This study will determine if a lower dose is equally effective and if that dose may lessen the weight gain seen in about half of the infants in the first study. Both the Bayley-4 and the GRO allow assessment of gross motor function and are feasible in all boys with DMD under the age of 42 months. In addition, the GRO allows continued assessment of motor function across a wide age span which will allow this cohort to be followed for two full years.