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
|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Drug: Prednisolone||Phase 2|
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 :||25 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
Experimental: Oral Prednisolone
Oral Prednisolone 5mg/kg/ day on two consecutive days, Friday and Saturday with breakfast
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
- 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.
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
|United States, California|
|University of California Davis|
|Sacramento, California, United States, 95817|
|United States, Florida|
|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|
|Principal Investigator:||Anne M Connolly, MD||Washington University School of Medicine|