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Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT02167217
Recruitment Status : Completed
First Posted : June 19, 2014
Results First Posted : May 18, 2018
Last Update Posted : May 18, 2018
Sponsor:
Collaborators:
Nationwide Children's Hospital
Feinberg School of Medicine, Northwestern University
University of Texas Southwestern Medical Center
University of California, Davis
Nemours Hospital, Orlando, FL
Information provided by (Responsible Party):
Washington University School of Medicine

Brief 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

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Prednisolone Phase 2

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.


Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 2 Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy
Actual Study Start Date : April 17, 2014
Actual Primary Completion Date : February 22, 2017
Actual Study Completion Date : March 22, 2017


Arm Intervention/treatment
Experimental: Oral Prednisolone
Oral Prednisolone 5mg/kg/ day on two consecutive days, Friday and Saturday with breakfast
Drug: Prednisolone
Prednisolone (5mg per kg) will be taken on two consecutive days, Friday and Saturday mornings each week with breakfast
Other Name: prednisolone (Morton Grove Pharm.) National Drug Code is 60432-212-08




Primary Outcome Measures :
  1. Bayley III Gross Motor Scaled Score (Change From Baseline to 12 Month) [ Time Frame: One year ]
    Bayley III Gross Motor Scaled Score measures motor development. This is normed for typically developing children and follow a bell shaped curve. The scale has mean of 10 +/-3 for children at all ages and is bell shaped. Therefore the two standard deviation range is 16 to 4 with higher values indicated better performance. Lower values have been shown to be common in boys with DMD and it this study the baseline average score was 4.2.



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Ages Eligible for Study:   1 Month to 30 Months   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Appropriate degree of weakness for age, creatine kinase greater than 20 times the upper limit of normal, and genetic mutation known to be causative for Duchenne muscular dystrophy .
  2. Appropriate degree of weakness for age, creatine kinase greater than 20 times the upper limit of normal and genetic or biopsy confirmation of Duchenne muscular dystrophy in a primary relative (e.g. brother or maternal uncle).
  3. De-identified, genetic studies will be reviewed by collaborator Kevin Flanigan, MD prior to enrollment of subjects.
  4. Age at entry: one month through 30 months.

Exclusion Criteria:

  • Prior treatment with corticosteroids

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02167217


Locations
United States, California
University of California Davis
Sacramento, California, United States, 95817
United States, Florida
Nemours Hospital
Orlando, Florida, United States, 32827
United States, Illinois
Laurie Children's Hospital Of Chicago
Chicago, Illinois, United States, 60611-2605
United States, Missouri
Washington University in Saint Louis
Saint Louis, Missouri, United States, 63109
United States, Ohio
Research Institute Center for Gene Therapy at Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
United States, Texas
University of Texas South Western Medical Center of Dallas
Dallas, Texas, United States, 75390
Sponsors and Collaborators
Washington University School of Medicine
Nationwide Children's Hospital
Feinberg School of Medicine, Northwestern University
University of Texas Southwestern Medical Center
University of California, Davis
Nemours Hospital, Orlando, FL
Investigators
Principal Investigator: Anne M Connolly, MD Washington University School of Medicine
  Study Documents (Full-Text)

Documents provided by Washington University School of Medicine:
Statistical Analysis Plan  [PDF] March 8, 2018
Study Protocol  [PDF] October 29, 2013


Publications:
Responsible Party: Washington University School of Medicine
ClinicalTrials.gov Identifier: NCT02167217     History of Changes
Other Study ID Numbers: 201308062
First Posted: June 19, 2014    Key Record Dates
Results First Posted: May 18, 2018
Last Update Posted: May 18, 2018
Last Verified: April 2018

Keywords provided by Washington University School of Medicine:
Duchenne Muscular Dystrophy
Corticosteroids
Infants
Young Children
Toddlers

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Prednisolone acetate
Methylprednisolone acetate
Prednisolone
Methylprednisolone
Methylprednisolone Hemisuccinate
Prednisolone hemisuccinate
Prednisolone phosphate
Anti-Inflammatory Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents
Gastrointestinal Agents
Neuroprotective Agents
Protective Agents