Developing standardized corticosteroid treatment for Duchenne muscular dystrophy

• Published in: Contemporary clinical trials Vol. 58; pp. 34 - 39
• Main Authors: Guglieri, Michela, Bushby, Kate, McDermott, Michael P, Hart, Kimberly A, Tawil, Rabi, Martens, William B, Herr, Barbara E, McColl, Elaine, Wilkinson, Jennifer, Kirschner, Janbernd, King, Wendy M, Eagle, Michele, Brown, Mary W, Willis, Tracey, Hirtz, Deborah, Shieh, Perry B, Straub, Volker, Childs, Anne-Marie, Ciafaloni, Emma, Butterfield, Russell J, Horrocks, Iain, Spinty, Stefan, Flanigan, Kevin M, Kuntz, Nancy L, Baranello, Giovanni, Roper, Helen, Morrison, Leslie, Mah, Jean K, Manzur, Adnan Y, McDonald, Craig M, Schara, Ulrike, von der Hagen, Maja, Barohn, Richard J, Campbell, Craig, Darras, Basil T, Finkel, Richard S, Vita, Giuseppe, Hughes, Imelda, Mongini, Tiziana, Pegoraro, Elena, Wicklund, Matthew, Wilichowski, Ekkehard, Bryan Burnette, W, Howard, James F, McMillan, Hugh J, Thangarajh, Mathula, Griggs, Robert C
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01.07.2017
• Subjects: Cardiovascular; Child; Child, Preschool; Deflazacort; Disability Evaluation; Double-Blind Method; Drug Administration Schedule; Duchenne muscular dystrophy; Heart Function Tests; Hematology, Oncology and Palliative Medicine; Humans; Immunosuppressive Agents - administration & dosage; Immunosuppressive Agents - adverse effects; Immunosuppressive Agents - therapeutic use; Male; Muscle Strength; Muscular Dystrophy, Duchenne - drug therapy; Patient Satisfaction; Prednisolone; Prednisone - administration & dosage; Prednisone - adverse effects; Prednisone - therapeutic use; Pregnenediones - administration & dosage; Pregnenediones - adverse effects; Pregnenediones - therapeutic use; Randomized; Range of Motion, Articular; Research Design; Standards of care; Vital Capacity; Standards of care; Deflazacort; Randomized; Duchenne muscular dystrophy; Prednisolone
• ISSN: 1551-7144; 1559-2030
• DOI: 10.1016/j.cct.2017.04.008

Abstract Despite corticosteroids being the only treatment documented to improve strength and function in boys with Duchenne muscular dystrophy (DMD) corticosteroid prescription is inconsistent and in some countries, corticosteroids are not prescribed. We are conducting a clinical trial that (1) compares the 3 most frequently prescribed corticosteroid regimes; (2) standardizes treatment of DMD complications; and (3) standardizes prevention of corticosteroid side effects. Investigators at 38 sites in 5 countries plan to recruit 300 boys aged 4–7 who are randomly assigned to one of three regimens: daily prednisone; daily deflazacort; or intermittent prednisone (10 days on/10 days off). Boys are followed for a minimum of 3 years to assess the relative effectiveness and adverse event profiles of the different regimens. The primary outcome is a 3-dimensional variable consisting of log-transformed time to rise from the floor, forced vital capacity, and subject/parent satisfaction with treatment, each averaged over all post-baseline visits. The study protocol includes evidence- and consensus-based treatment of DMD complications and of corticosteroid side effects. This study seeks to establish a standard corticosteroid regimen for DMD. Since all new interventions for DMD are being developed as add-on therapies to corticosteroids, defining the optimum regimen is of importance for all new treatments.