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August 1987

Clinical Investigation of Duchenne Muscular Dystrophy: A Methodology for Therapeutic Trials Based on Natural History Controls

Author Affiliations

From the Department of Neurology, The Ohio State University College of Medicine, Columbus (Dr Mendell, and Mss Signore and King); University of Rochester (NY), Strong Memorial Hospital (Drs Griggs and Moxley, and Ms Pandya); Irene Walter Johnson Institute of Rehabilitation, Department of Neurology and Neurosurgery, Division of Biostatistics, Washington University School of Medicine, St Louis (Drs Brooke, Province, and Seyfried, Ms Florence, and Messrs Miller, Kaiser, and Mandel); and the Department of Neurology, Vanderbilt University, Nashville, Tenn (Dr Fenichel and Ms Robison).

Arch Neurol. 1987;44(8):808-811. doi:10.1001/archneur.1987.00520200012009

• Between 1979 and 1987 we documented the natural history of Duchenne muscular dystrophy in 170 patients, aged from 3 to 23 years, by making serial measurements in over 5000 individual evaluations. This database makes it possible to design and conduct therapeutic trials using natural history controls. Such trials do not replace the need for randomized placebo-controlled trials of promising agents but they do require fewer patients, are cost-effective, and permit the use of high-risk therapy where toxicity monitoring may be important. Natural history-controlled trials, therefore, may serve as a screening method for new therapeutic agents. Drugs showing a significant benefit can then be evaluated in a randomized controlled trial.

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