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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 : December 21, 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

Tracking Information
First Submitted Date  ICMJE February 3, 2014
First Posted Date  ICMJE June 19, 2014
Results First Submitted Date  ICMJE March 9, 2018
Results First Posted Date  ICMJE May 18, 2018
Last Update Posted Date December 21, 2018
Actual Study Start Date  ICMJE April 17, 2014
Actual Primary Completion Date February 22, 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 17, 2018)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: June 18, 2014)
Gross Motor Scaled Score [ Time Frame: One year ]
Gross Motor Scaled Score measures motor development
Change History Complete list of historical versions of study NCT02167217 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE
 (submitted: June 18, 2014)
North Star Ambulatory Assessment (NSAA) [ Time Frame: One Year ]
The NSAA measures the quality of ambulation in young boys with Duchenne Muscular Dystrophy
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy
Official Title  ICMJE Phase 2 Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy
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
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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE 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
Study Arms  ICMJE Experimental: Oral Prednisolone
Oral Prednisolone 5mg/kg/ day on two consecutive days, Friday and Saturday with breakfast
Intervention: Drug: Prednisolone
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 19, 2018)
25
Original Estimated Enrollment  ICMJE
 (submitted: June 18, 2014)
24
Actual Study Completion Date  ICMJE March 22, 2017
Actual Primary Completion Date February 22, 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 1 Month to 30 Months   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02167217
Other Study ID Numbers  ICMJE 201308062
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Washington University School of Medicine
Study Sponsor  ICMJE Washington University School of Medicine
Collaborators  ICMJE
  • 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  ICMJE
Principal Investigator: Anne M Connolly, MD Washington University School of Medicine
PRS Account Washington University School of Medicine
Verification Date December 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP