Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy
NCT ID: NCT02167217
Last Updated: 2018-12-21
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
25 participants
INTERVENTIONAL
2014-04-17
2017-03-22
Brief Summary
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Detailed Description
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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.
Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Oral Prednisolone
Oral Prednisolone 5mg/kg/ day on two consecutive days, Friday and Saturday with breakfast
Prednisolone
Prednisolone (5mg per kg) will be taken on two consecutive days, Friday and Saturday mornings each week with breakfast
Interventions
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Prednisolone
Prednisolone (5mg per kg) will be taken on two consecutive days, Friday and Saturday mornings each week with breakfast
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
1 Month
30 Months
MALE
No
Sponsors
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Nationwide Children's Hospital
OTHER
Northwestern University Feinberg School of Medicine
OTHER
University of Texas Southwestern Medical Center
OTHER
University of California, Davis
OTHER
Nemours Children's Hospital
OTHER
Washington University School of Medicine
OTHER
Responsible Party
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Principal Investigators
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Anne M Connolly, MD
Role: PRINCIPAL_INVESTIGATOR
Washington University School of Medicine
Locations
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University of California Davis
Sacramento, California, United States
Nemours Hospital
Orlando, Florida, United States
Laurie Children's Hospital Of Chicago
Chicago, Illinois, United States
Washington University in Saint Louis
St Louis, Missouri, United States
Research Institute Center for Gene Therapy at Nationwide Children's Hospital
Columbus, Ohio, United States
University of Texas South Western Medical Center of Dallas
Dallas, Texas, United States
Countries
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References
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Connolly AM, Florence JM, Cradock MM, Malkus EC, Schierbecker JR, Siener CA, Wulf CO, Anand P, Golumbek PT, Zaidman CM, Philip Miller J, Lowes LP, Alfano LN, Viollet-Callendret L, Flanigan KM, Mendell JR, McDonald CM, Goude E, Johnson L, Nicorici A, Karachunski PI, Day JW, Dalton JC, Farber JM, Buser KK, Darras BT, Kang PB, Riley SO, Shriber E, Parad R, Bushby K, Eagle M; MDA DMD Clinical Research Network. Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network. Neuromuscul Disord. 2013 Jul;23(7):529-39. doi: 10.1016/j.nmd.2013.04.005. Epub 2013 May 28.
Connolly AM, Schierbecker J, Renna R, Florence J. High dose weekly oral prednisone improves strength in boys with Duchenne muscular dystrophy. Neuromuscul Disord. 2002 Dec;12(10):917-25. doi: 10.1016/s0960-8966(02)00180-3.
Escolar DM, Hache LP, Clemens PR, Cnaan A, McDonald CM, Viswanathan V, Kornberg AJ, Bertorini TE, Nevo Y, Lotze T, Pestronk A, Ryan MM, Monasterio E, Day JW, Zimmerman A, Arrieta A, Henricson E, Mayhew J, Florence J, Hu F, Connolly AM. Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy. Neurology. 2011 Aug 2;77(5):444-52. doi: 10.1212/WNL.0b013e318227b164. Epub 2011 Jul 13.
Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR; MDA DMD Clinical Research Network. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun;59(6):650-657. doi: 10.1002/mus.26441. Epub 2019 Feb 23.
Provided Documents
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Document Type: Statistical Analysis Plan
Document Type: Study Protocol
Other Identifiers
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201308062
Identifier Type: -
Identifier Source: org_study_id