drugset / Trial / NCT01603407
Finding the Optimum Regimen for Duchenne Muscular Dystrophy
Phase 3
Completed
196 enrolled
University of Rochester
National Institute of Neurological Disorders and Stroke (NINDS) · collabNewcastle University · collabUniversity Medical Center Freiburg · collab
RandomizedParallel-groupQuadruple-blindTreatment
Summary
The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases muscle strength the most, and which causes the fewest side effects. Using the results of this study, the investigators aim to provide patients and families with clearer information about the best way to take these drugs.
Timeline
- Start
- 2013-01
- Primary completion
- 2019-11
- Completion
- 2019-11
Publications
- Background Angelini C, Pegoraro E, Turella E, Intino MT, Pini A, Costa C. Deflazacort in Duchenne dystrophy: study of long-term effect. Muscle Nerve. 1994 Apr;17(4):386-91. doi: 10.1002/mus.880170405.
- Background Atkinson MJ, Sinha A, Hass SL, Colman SS, Kumar RN, Brod M, Rowland CR. Validation of a general measure of treatment satisfaction, the Treatment Satisfaction Questionnaire for Medication (TSQM), using a national panel study of chronic disease. Health Qual Life Outcomes. 2004 Feb 26;2:12. doi: 10.1186/1477-7525-2-12.
- Background Backman E, Henriksson KG. Low-dose prednisolone treatment in Duchenne and Becker muscular dystrophy. Neuromuscul Disord. 1995 May;5(3):233-41. doi: 10.1016/0960-8966(94)00048-e.
- Background Bianchi ML. How to manage osteoporosis in children. Best Pract Res Clin Rheumatol. 2005 Dec;19(6):991-1005. doi: 10.1016/j.berh.2005.06.006.
- Background Biggar WD, Bachrach LK, Henderson RC, Kalkwarf H, Plotkin H, Wong BL. Bone health in Duchenne muscular dystrophy: a workshop report from the meeting in Cincinnati, Ohio, July 8, 2004. Neuromuscul Disord. 2005 Jan;15(1):80-5. doi: 10.1016/j.nmd.2004.09.010. No abstract available.
- Background Biggar WD, Gingras M, Fehlings DL, Harris VA, Steele CA. Deflazacort treatment of Duchenne muscular dystrophy. J Pediatr. 2001 Jan;138(1):45-50. doi: 10.1067/mpd.2001.109601.
- Background Biggar WD, Harris VA, Eliasoph L, Alman B. Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade. Neuromuscul Disord. 2006 Apr;16(4):249-55. doi: 10.1016/j.nmd.2006.01.010. Epub 2006 Mar 20.
- Background Biggar WD, Politano L, Harris VA, Passamano L, Vajsar J, Alman B, Palladino A, Comi LI, Nigro G. Deflazacort in Duchenne muscular dystrophy: a comparison of two different protocols. Neuromuscul Disord. 2004 Sep;14(8-9):476-82. doi: 10.1016/j.nmd.2004.05.001.
- Background Brooke MH, Fenichel GM, Griggs RC, Mendell JR, Moxley R, Miller JP, Province MA. Clinical investigation in Duchenne dystrophy: 2. Determination of the "power" of therapeutic trials based on the natural history. Muscle Nerve. 1983 Feb;6(2):91-103. doi: 10.1002/mus.880060204.
- Background Brooke MH, Fenichel GM, Griggs RC, Mendell JR, Moxley RT 3rd, Miller JP, Kaiser KK, Florence JM, Pandya S, Signore L, et al. Clinical investigation of Duchenne muscular dystrophy. Interesting results in a trial of prednisone. Arch Neurol. 1987 Aug;44(8):812-7. doi: 10.1001/archneur.1987.00520200016010.
- Background Brooke MH, Fenichel GM, Griggs RC, Mendell JR, Moxley R, Florence J, King WM, Pandya S, Robison J, Schierbecker J, et al. Duchenne muscular dystrophy: patterns of clinical progression and effects of supportive therapy. Neurology. 1989 Apr;39(4):475-81. doi: 10.1212/wnl.39.4.475.
- Background Bushby K, Muntoni F, Urtizberea A, Hughes R, Griggs R. Report on the 124th ENMC International Workshop. Treatment of Duchenne muscular dystrophy; defining the gold standards of management in the use of corticosteroids. 2-4 April 2004, Naarden, The Netherlands. Neuromuscul Disord. 2004 Sep;14(8-9):526-34. doi: 10.1016/j.nmd.2004.05.006. No abstract available.
- Background Bushby K, Muntoni F, Bourke JP. 107th ENMC international workshop: the management of cardiac involvement in muscular dystrophy and myotonic dystrophy. 7th-9th June 2002, Naarden, the Netherlands. Neuromuscul Disord. 2003 Feb;13(2):166-72. doi: 10.1016/s0960-8966(02)00213-4. No abstract available.
- Background Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, Kaul A, Kinnett K, McDonald C, Pandya S, Poysky J, Shapiro F, Tomezsko J, Constantin C; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 2010 Jan;9(1):77-93. doi: 10.1016/S1474-4422(09)70271-6. Epub 2009 Nov 27.
- Background Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, Kaul A, Kinnett K, McDonald C, Pandya S, Poysky J, Shapiro F, Tomezsko J, Constantin C; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol. 2010 Feb;9(2):177-89. doi: 10.1016/S1474-4422(09)70272-8. Epub 2009 Nov 27.
- Background Carter GT, McDonald CM. Preserving function in Duchenne dystrophy with long-term pulse prednisone therapy. Am J Phys Med Rehabil. 2000 Sep-Oct;79(5):455-8. doi: 10.1097/00002060-200009000-00009.
- Background Collett BR, Ohan JL, Myers KM. Ten-year review of rating scales. V: scales assessing attention-deficit/hyperactivity disorder. J Am Acad Child Adolesc Psychiatry. 2003 Sep;42(9):1015-37. doi: 10.1097/01.CHI.0000070245.24125.B6.
- Background Collett BR, Ohan JL, Myers KM. Ten-year review of rating scales. VI: scales assessing externalizing behaviors. J Am Acad Child Adolesc Psychiatry. 2003 Oct;42(10):1143-70. doi: 10.1097/00004583-200310000-00006.
- Background 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.
- Background Duboc D, Meune C, Lerebours G, Devaux JY, Vaksmann G, Becane HM. Effect of perindopril on the onset and progression of left ventricular dysfunction in Duchenne muscular dystrophy. J Am Coll Cardiol. 2005 Mar 15;45(6):855-7. doi: 10.1016/j.jacc.2004.09.078.
- Background Dubowitz V, Kinali M, Main M, Mercuri E, Muntoni F. Remission of clinical signs in early duchenne muscular dystrophy on intermittent low-dosage prednisolone therapy. Eur J Paediatr Neurol. 2002;6(3):153-9. doi: 10.1053/ejpn.2002.0583.
- Background Eagle M, Baudouin SV, Chandler C, Giddings DR, Bullock R, Bushby K. Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation. Neuromuscul Disord. 2002 Dec;12(10):926-9. doi: 10.1016/s0960-8966(02)00140-2.
- Background Fenichel GM, Florence JM, Pestronk A, Mendell JR, Moxley RT 3rd, Griggs RC, Brooke MH, Miller JP, Robison J, King W, et al. Long-term benefit from prednisone therapy in Duchenne muscular dystrophy. Neurology. 1991 Dec;41(12):1874-7. doi: 10.1212/wnl.41.12.1874.
- Background Fenichel GM, Mendell JR, Moxley RT 3rd, Griggs RC, Brooke MH, Miller JP, Pestronk A, Robison J, King W, Signore L, et al. A comparison of daily and alternate-day prednisone therapy in the treatment of Duchenne muscular dystrophy. Arch Neurol. 1991 Jun;48(6):575-9. doi: 10.1001/archneur.1991.00530180027012.
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- Background Griggs RC, Moxley RT 3rd, Mendell JR, Fenichel GM, Brooke MH, Pestronk A, Miller JP, Cwik VA, Pandya S, Robison J, et al. Duchenne dystrophy: randomized, controlled trial of prednisone (18 months) and azathioprine (12 months). Neurology. 1993 Mar;43(3 Pt 1):520-7. doi: 10.1212/wnl.43.3_part_1.520.
- Background Griggs RC, Moxley RT 3rd, Mendell JR, Fenichel GM, Brooke MH, Pestronk A, Miller JP. Prednisone in Duchenne dystrophy. A randomized, controlled trial defining the time course and dose response. Clinical Investigation of Duchenne Dystrophy Group. Arch Neurol. 1991 Apr;48(4):383-8. doi: 10.1001/archneur.1991.00530160047012.
- Background Hinton VJ, Nereo NE, Fee RJ, Cyrulnik SE. Social behavior problems in boys with Duchenne muscular dystrophy. J Dev Behav Pediatr. 2006 Dec;27(6):470-6. doi: 10.1097/00004703-200612000-00003.
- Background Hopke PK, Liu C, Rubin DB. Multiple imputation for multivariate data with missing and below-threshold measurements: time-series concentrations of pollutants in the Arctic. Biometrics. 2001 Mar;57(1):22-33. doi: 10.1111/j.0006-341x.2001.00022.x.
- Background Kinali M, Mercuri E, Main M, Muntoni F, Dubowitz V. An effective, low-dosage, intermittent schedule of prednisolone in the long-term treatment of early cases of Duchenne dystrophy. Neuromuscul Disord. 2002 Oct;12 Suppl 1:S169-74. doi: 10.1016/s0960-8966(02)00097-4.
- Background Lippuner K, Casez JP, Horber FF, Jaeger P. Effects of deflazacort versus prednisone on bone mass, body composition, and lipid profile: a randomized, double blind study in kidney transplant patients. J Clin Endocrinol Metab. 1998 Nov;83(11):3795-802. doi: 10.1210/jcem.83.11.5235.
- Background Little R, Yau L. Intent-to-treat analysis for longitudinal studies with drop-outs. Biometrics. 1996 Dec;52(4):1324-33.
- Background Loftus J, Allen R, Hesp R, David J, Reid DM, Wright DJ, Green JR, Reeve J, Ansell BM, Woo PM. Randomized, double-blind trial of deflazacort versus prednisone in juvenile chronic (or rheumatoid) arthritis: a relatively bone-sparing effect of deflazacort. Pediatrics. 1991 Sep;88(3):428-36.
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- Background Manzur AY, Kuntzer T, Pike M, Swan A. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev. 2004;(2):CD003725. doi: 10.1002/14651858.CD003725.pub2.
- Background Markham A, Bryson HM. Deflazacort. A review of its pharmacological properties and therapeutic efficacy. Drugs. 1995 Aug;50(2):317-33. doi: 10.2165/00003495-199550020-00008.
- Background Markham LW, Spicer RL, Khoury PR, Wong BL, Mathews KD, Cripe LH. Steroid therapy and cardiac function in Duchenne muscular dystrophy. Pediatr Cardiol. 2005 Nov-Dec;26(6):768-71. doi: 10.1007/s00246-005-0909-4.
- Background Mayhew A, Cano S, Scott E, Eagle M, Bushby K, Muntoni F; North Star Clinical Network for Paediatric Neuromuscular Disease. Moving towards meaningful measurement: Rasch analysis of the North Star Ambulatory Assessment in Duchenne muscular dystrophy. Dev Med Child Neurol. 2011 Jun;53(6):535-42. doi: 10.1111/j.1469-8749.2011.03939.x. Epub 2011 Mar 17.
- Background Mendell JR, Moxley RT, Griggs RC, Brooke MH, Fenichel GM, Miller JP, King W, Signore L, Pandya S, Florence J, et al. Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy. N Engl J Med. 1989 Jun 15;320(24):1592-7. doi: 10.1056/NEJM198906153202405.
- Background Merlini L, Cicognani A, Malaspina E, Gennari M, Gnudi S, Talim B, Franzoni E. Early prednisone treatment in Duchenne muscular dystrophy. Muscle Nerve. 2003 Feb;27(2):222-7. doi: 10.1002/mus.10319.
- Background Moxley RT 3rd, Ashwal S, Pandya S, Connolly A, Florence J, Mathews K, Baumbach L, McDonald C, Sussman M, Wade C; Quality Standards Subcommittee of the American Academy of Neurology; Practice Committee of the Child Neurology Society. Practice parameter: corticosteroid treatment of Duchenne dystrophy [RETIRED]: report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society. Neurology. 2005 Jan 11;64(1):13-20. doi: 10.1212/01.WNL.0000148485.00049.B7.
- Background O'Brien PC. Procedures for comparing samples with multiple endpoints. Biometrics. 1984 Dec;40(4):1079-87.
- Background Phillips MF, Quinlivan RC, Edwards RH, Calverley PM. Changes in spirometry over time as a prognostic marker in patients with Duchenne muscular dystrophy. Am J Respir Crit Care Med. 2001 Dec 15;164(12):2191-4. doi: 10.1164/ajrccm.164.12.2103052.
- Background Pocock SJ, Geller NL, Tsiatis AA. The analysis of multiple endpoints in clinical trials. Biometrics. 1987 Sep;43(3):487-98.
- Background Quinlivan R, Roper H, Davie M, Shaw NJ, McDonagh J, Bushby K. Report of a Muscular Dystrophy Campaign funded workshop Birmingham, UK, January 16th 2004. Osteoporosis in Duchenne muscular dystrophy; its prevalence, treatment and prevention. Neuromuscul Disord. 2005 Jan;15(1):72-9. doi: 10.1016/j.nmd.2004.09.009. Epub 2004 Dec 10. No abstract available.
- Background Reitter B. Deflazacort vs. prednisone in Duchenne muscular dystrophy: trends of an ongoing study. Brain Dev. 1995;17 Suppl:39-43.
- Background Sansome A, Royston P, Dubowitz V. Steroids in Duchenne muscular dystrophy; pilot study of a new low-dosage schedule. Neuromuscul Disord. 1993 Sep-Nov;3(5-6):567-9. doi: 10.1016/0960-8966(93)90117-3.
- Background Silversides CK, Webb GD, Harris VA, Biggar DW. Effects of deflazacort on left ventricular function in patients with Duchenne muscular dystrophy. Am J Cardiol. 2003 Mar 15;91(6):769-72. doi: 10.1016/s0002-9149(02)03429-x. No abstract available.
- Background Simonds AK, Muntoni F, Heather S, Fielding S. Impact of nasal ventilation on survival in hypercapnic Duchenne muscular dystrophy. Thorax. 1998 Nov;53(11):949-52. doi: 10.1136/thx.53.11.949.
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- Background Varni JW, Seid M, Knight TS, Uzark K, Szer IS. The PedsQL 4.0 Generic Core Scales: sensitivity, responsiveness, and impact on clinical decision-making. J Behav Med. 2002 Apr;25(2):175-93. doi: 10.1023/a:1014836921812.
- Background Varni JW, Seid M, Kurtin PS. PedsQL 4.0: reliability and validity of the Pediatric Quality of Life Inventory version 4.0 generic core scales in healthy and patient populations. Med Care. 2001 Aug;39(8):800-12. doi: 10.1097/00005650-200108000-00006.
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- Background Witt WP, Kasper JD, Riley AW. Mental health services use among school-aged children with disabilities: the role of sociodemographics, functional limitations, family burdens, and care coordination. Health Serv Res. 2003 Dec;38(6 Pt 1):1441-66. doi: 10.1111/j.1475-6773.2003.00187.x.
- Background Xie H, Heitjan DF. Sensitivity analysis of causal inference in a clinical trial subject to crossover. Clin Trials. 2004 Feb;1(1):21-30. doi: 10.1191/1740774504cn005oa.
- Degan C, Tobin RA, de Vries SI, Jimenez-Requena A, Peco A, Guglieri M, Diaz-Manera J, van der Burgt YEM, van Vlijmen BJM; FOR-DMD investigators of the Muscle Study Group; Hathout Y, Szigyarto CA, Dang UJ, Tsonaka R, Spitali P. Evaluation of a serum protein signature as monitoring biomarker for Duchenne muscular dystrophy in a long-term clinical trial with corticosteroids. Skelet Muscle. 2026 Jul 13. doi: 10.1186/s13395-026-00437-2. Online ahead of print.
- Naveed A, Recinos E, Chow D, Degan C, Tsonaka R, Guglieri M, Al-Khalili Szigyarto C, Spitali P, van der Burgt YEM, Diaz-Manera J, Dang UJ; FOR-DMD investigators of the Muscle Study Group; Hathout Y. Quantitative tandem mass tag-based serum proteomics for longitudinal biomarker monitoring in Duchenne muscular dystrophy. Clin Proteomics. 2026 Jun 6. doi: 10.1186/s12014-026-09613-4. Online ahead of print.
- Clemens PR, Berglund A, Schiava M, James MK, McDermott MP, Bushby K, Lampa E, Rochford ETJ, Ward LM, Griggs RC, Hoffman EP, Guglieri M. Vamorolone for Duchenne Muscular Dystrophy: A Cross-Trial Efficacy Comparison With Classic Corticosteroids From the FOR-DMD Trial. Neurology. 2026 Jul 14;107(1):e214756. doi: 10.1212/WNL.0000000000214756. Epub 2026 May 27.
- Gadaleta G, Geagan C, Schiava M, Riguzzi P, Hendriksen J, Campbell C, McDermott MP, Martens WB, Gregory SJ, Mongini TE, Griggs RC, Guglieri M; FOR-DMD Investigators of the Muscle Study Group. Neurobehavioral Profiles in Young Steroid-Naive Boys With Duchenne Muscular Dystrophy: A Baseline Data Analysis From the FOR-DMD Trial. Neurology. 2026 Jun 9;106(11):e218094. doi: 10.1212/WNL.0000000000218094. Epub 2026 May 15.
- Guglieri M, Bushby K, McDermott MP, Hart KA, Tawil R, Martens WB, Herr BE, McColl E, Speed C, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Willis T, Griggs RC; FOR-DMD Investigators of the Muscle Study Group; Straub V, van Ruiten H, Childs AM, Ciafaloni E, Shieh PB, Spinty S, Maggi L, Baranello G, Butterfield RJ, Horrocks IA, Roper H, Alhaswani Z, Flanigan KM, Kuntz NL, Manzur A, Darras BT, Kang PB, Morrison L, Krzesniak-Swinarska M, Mah JK, Mongini TE, Ricci F, von der Hagen M, Finkel RS, O'Reardon K, Wicklund M, Kumar A, McDonald CM, Han JJ, Joyce N, Henricson EK, Schara-Schmidt U, Gangfuss A, Wilichowski E, Barohn RJ, Statland JM, Campbell C, Vita G, Vita GL, Howard JF Jr, Hughes I, McMillan HJ, Pegoraro E, Bello L, Burnette WB, Thangarajh M, Chang T. Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial. JAMA. 2022 Apr 19;327(15):1456-1468. doi: 10.1001/jama.2022.4315.
- Schiava M, Amos R, VanRuiten H, McDermott MP, Martens WB, Gregory S, Mayhew A, McColl E, Tawil R, Willis T, Bushby K, Griggs RC, Guglieri M; FOR-DMD Investigators of the Muscle Study Group. Clinical and Genetic Characteristics in Young, Glucocorticoid-Naive Boys With Duchenne Muscular Dystrophy. Neurology. 2022 Jan 24;98(4):e390-e401. doi: 10.1212/WNL.0000000000013122.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Deflazacort | Other / unclassified | 0.9 mg/kg | Oral |
| Subject | Prednisone | Other / unclassified | 0.75 mg/kg | Oral |