CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy
Corresponding Author
Diana M. Escolar MD
Children's National Medical Center, George Washington University, Washington, DC
Associate Professor of Neurology and Pediatrics, Children's National Medical Center, Research Center for Genetic Medicine, 111 Michigan Ave, NW, Washington, DC 20010Search for more papers by this authorGunnar Buyse MD, PhD
University Hospitals, KU Leuven, Leuven, Belgium
Search for more papers by this authorErik Henricson MPH
Children's National Medical Center, George Washington University, Washington, DC
Search for more papers by this authorRobert Leshner MD
Virginia Commonwealth University, Richmond, VA
Search for more papers by this authorCarolina Tesi-Rocha MD
Children's National Medical Center, George Washington University, Washington, DC
Search for more papers by this authorKsenija Gorni MD
Children's National Medical Center, George Washington University, Washington, DC
Search for more papers by this authorLivia Pasquali MD
Children's National Medical Center, George Washington University, Washington, DC
Search for more papers by this authorKantilal M. Patel PhD
Children's National Medical Center, George Washington University, Washington, DC
Search for more papers by this authorRobert McCarter ScD
Children's National Medical Center, George Washington University, Washington, DC
Search for more papers by this authorJennifer Huang PhD, MPH
Children's National Medical Center, George Washington University, Washington, DC
Search for more papers by this authorThomas Mayhew PT, PhD
Virginia Commonwealth University, Richmond, VA
Search for more papers by this authorPaula R. Clemens MD
Neurology Service, Department of Veterans Affairs Medical Center, Pittsburgh, PA
Children's Hospital of Pittsburgh, Pittsburgh, PA
Search for more papers by this authorNathalie Goemans MD
University Hospitals, KU Leuven, Leuven, Belgium
Search for more papers by this authorSusan T. Iannaccone MD
Texas Scottish Rite Hospital for Children, Dallas, TX
Search for more papers by this authorHenry Wessel MD
Children's Hospital of Pittsburgh, Pittsburgh, PA
Search for more papers by this authorCINRG Goup
Members of the CINRG Study Group are listed in the Appendix on page 153.
Search for more papers by this authorCorresponding Author
Diana M. Escolar MD
Children's National Medical Center, George Washington University, Washington, DC
Associate Professor of Neurology and Pediatrics, Children's National Medical Center, Research Center for Genetic Medicine, 111 Michigan Ave, NW, Washington, DC 20010Search for more papers by this authorGunnar Buyse MD, PhD
University Hospitals, KU Leuven, Leuven, Belgium
Search for more papers by this authorErik Henricson MPH
Children's National Medical Center, George Washington University, Washington, DC
Search for more papers by this authorRobert Leshner MD
Virginia Commonwealth University, Richmond, VA
Search for more papers by this authorCarolina Tesi-Rocha MD
Children's National Medical Center, George Washington University, Washington, DC
Search for more papers by this authorKsenija Gorni MD
Children's National Medical Center, George Washington University, Washington, DC
Search for more papers by this authorLivia Pasquali MD
Children's National Medical Center, George Washington University, Washington, DC
Search for more papers by this authorKantilal M. Patel PhD
Children's National Medical Center, George Washington University, Washington, DC
Search for more papers by this authorRobert McCarter ScD
Children's National Medical Center, George Washington University, Washington, DC
Search for more papers by this authorJennifer Huang PhD, MPH
Children's National Medical Center, George Washington University, Washington, DC
Search for more papers by this authorThomas Mayhew PT, PhD
Virginia Commonwealth University, Richmond, VA
Search for more papers by this authorPaula R. Clemens MD
Neurology Service, Department of Veterans Affairs Medical Center, Pittsburgh, PA
Children's Hospital of Pittsburgh, Pittsburgh, PA
Search for more papers by this authorNathalie Goemans MD
University Hospitals, KU Leuven, Leuven, Belgium
Search for more papers by this authorSusan T. Iannaccone MD
Texas Scottish Rite Hospital for Children, Dallas, TX
Search for more papers by this authorHenry Wessel MD
Children's Hospital of Pittsburgh, Pittsburgh, PA
Search for more papers by this authorCINRG Goup
Members of the CINRG Study Group are listed in the Appendix on page 153.
Search for more papers by this authorAbstract
We tested the efficacy and safety of glutamine (0.6gm/kg/day) and creatine (5gm/day) in 50 ambulant boys with Duchenne muscular dystrophy in a 6-month, double-blind, placebo-controlled clinical trial. Drug efficacy was tested by measuring muscle strength manually (34 muscle groups) and quantitatively (10 muscle groups). Timed functional tests, functional parameters, and pulmonary function tests were secondary outcome measures. Although there was no statistically significant effect of either therapy based on manual and quantitative measurements of muscle strength, a disease-modifying effect of creatine in older Duchenne muscular dystrophy and creatine and glutamine in younger Duchenne muscular dystrophy cannot be excluded. Creatine and glutamine were well tolerated. Ann Neurol 2005;58:151–155
References
- 1 Granchelli JA, Pollina C, Hudecki MS. Pre-clinical screening of drugs using the mdx mouse. Neuromuscul Disord 2000; 10: 235–239.
- 2 Pulido SM, Passaquin AC, Leijendekker WJ, et al. Creatine supplementation improves intracellular Ca2+ handling and survival in mdx skeletal muscle cells. FEBS Lett 1998; 439: 357–362.
- 3 Brooke MH, Griggs RC, Mendell JR, et al. Clinical trial in Duchenne dystrophy. I. The design of the protocol. Muscle Nerve 1981; 4: 186–197.
- 4 Escolar DM, Henricson EK, Mayhew J, et al. Clinical evaluator reliability for quantitative and manual muscle testing measures of strength in children. Muscle Nerve 2001; 24: 787–793.
- 5 Mendell JR, Moxley RT, Griggs RC, et al. Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy. N Engl J Med 1989; 320: 1592–1597.
- 6 Fenichel GM, Griggs RC, Kissel J, et al. A randomized efficacy and safety trial of oxandrolone in the treatment of Duchenne dystrophy. Neurology 2001; 56: 1075–1079.
- 7 Mendell JR, Province MA, Moxley RTd, et al. Clinical investigation of Duchenne muscular dystrophy. A methodology for therapeutic trials based on natural history controls. Arch Neurol 1987; 44: 808–811.
- 8 Brooke MH, Fenichel GM, Griggs RC, et al. Clinical investigation in Duchenne dystrophy: 2. Determination of the “power” of therapeutic trials based on the natural history. Muscle Nerve 1983; 6: 91–103.