Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Dystrophy
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne
Study type: Interventional
Phase: Phase 2

Summary: 
      While it has been known for many years that corticosteroid use benefits boys with Duchenne
      Muscular dystrophy (DMD), most clinicians do not consider treating until after age 3 or 4
      years of age. The primary reason for the delay is that daily corticosteroid use has many side
      effects including short stature, obesity, and osteoporosis. A recent randomized blinded study
      of weekend oral corticosteroid use over one year showed equal improvement in strength with
      fewer side effects, particularly as related to growth and cushingoid changes. The
      investigators will test the efficacy of oral weekend corticosteroid use in infants and young
      boys with DMD who are under age 30 months. The investigators have demonstrated that the
      Bayley-III Scales of Infant development shows that infants and young boys in this age group
      who are untreated decline in abilities when compared to their peers. Here, in this Phase 2
      historically controlled trial, the investigators will use these two measures and treat boys
      at five Muscular Dystrophy Association-DMD centers
    

Detailed description: 
      Objective. Determine if twice-weekly high dose oral prednisone improves gross motor
      development in infants and young boys with DMD. The investigators will perform a phase 2
      historically controlled trial of oral twice-weekly prednisone (5mg/kg/dose on two consecutive
      days) in infants and young boys with DMD. Here the investigators propose to study the effect
      of this therapy in a multicenter trial of boys with DMD who are less than 30 months old at
      the baseline visit. Each boy will be followed for one year.

      Aim 1. Determine if treatment improves gross motor function in infants with DMD over a
      6-12-month period as measured by the Bayley-III. The Bayley-III infant score is the primary
      motor clinical endpoint of this therapeutic trial. Secondary outcomes include fine motor
      function, speech and language, and social function.

      Aim 2. Determine if treatment improves the Adaptive Behavior Subtest of the Bayley-III (ABS)
      as scored by the infants' primary caregiver. In the study of untreated boys, the primary
      caregiver noted clear deficits, predominantly related to areas relevant to gross motor
      function. The ABS Aim 3. Determine if treatment improves performance on the North Star
      Ambulatory Assessment (NSAA) for those boys who are ambulatory.

      Aim 4. Determine if treatment with weekly corticosteroids is tolerated and is safe in boys
      with DMD who are less than 30 months of age.

      Objective 2. Determine if ultrasound of biceps and quadriceps using calibrated backscatter
      improves in infants and young boys with DMD who are treated with oral high dose weekly
      corticosteroids. Preliminary data of ultrasound imaging in infants and young boys with DMD
      demonstrate progressive structural damage as measured by calibrated backscatter. The
      ultrasound studies will be limited to the infants and boys who will enroll at the primary
      site (Washington University) where Dr. Craig Zaidman has the equipment and expertise to
      accomplish this aim.

      Objective 3. Determine if caregiver burden changes with treatment of infants and young boys
      with DMD. Preliminary data from questionnaires suggests the caregiver burden for the primary
      caregiver of untreated infant and young boys with DMD is minimal. Assessment of this with in
      this trial will allow us to discern if this changes with a therapeutic trial.
    

Intervention name(s): Prednisolone
Intervention type(s): Drug
Intervention description(s): Prednisolone (5mg per kg) will be taken on two consecutive days, Friday and Saturday mornings each week with breakfast
Other intervention name(s): prednisolone (Morton Grove Pharm.) National Drug Code is 60432-212-08
Intervention MeSH terms: Prednisolone; Methylprednisolone Acetate; Methylprednisolone; Methylprednisolone Hemisuccinate; Prednisolone acetate; Prednisolone hemisuccinate; Prednisolone phosphate
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Duchenne Muscular Dystrophy; Corticosteroids; Infants; Young Children; Toddlers