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Therapeutic Trials in ALS — The Design of a Protocol

  • G. Küther
  • A. Struppler
  • H. G. Lipinski
Part of the Advances in Experimental Medicine and Biology book series (AEMB, volume 209)

Abstract

Therapeutic trials are an integral part of the development of all new medications, which are claimed to exert a beneficial effect on the course of a disease. Usually, the testing of a new agent in man is the last step towards an effective treatment, after basic research has provided more insight into the pathogenesis of the disorder and animal models had been established which allow a first evaluation of the effects in biological systems.

Keywords

Amyotrophic Lateral Sclerosis Forced Vital Capacity Duchenne Dystrophy Spinal Muscular Atrophy Therapeutic Trial 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.

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References

  1. 1.
    B. W. Festoff and N. J. Crigger, Therapeutic trials in amyotrophic lateral sclerosis: a review, in: “The Diagnosis and Treatment of Amyotrophic Lateral Sclerosis”, D. W. Mulder, ed., pp. 337–366, Houghton Mifflin Professional Publishers Medical Division, Boston (1980).Google Scholar
  2. 2.
    B. W. Festoff, Approaches to modern therapeutic clinical trails in ALS, in: “Research Progress in Motor Neuron Disease”, F. C. Rose, ed., pp. 432–442, Pitman, London (1984).Google Scholar
  3. 3.
    C. Angelini, S. P. Ringel, G. Micaglio, and C. Trevisan, Italian multicenter therapeutic trials in Duchenne dystrophy. I. Protocol, Ital.J.Neurol.Sci., Suppl. 3: 137–142 (1984).Google Scholar
  4. 4.
    W. G. Bradley, W. Hedlund, C. Cooper, G. J. Desousa, A. Gabbai, J. S. Mora, T. L. Munsat, and R. Scheife, A double-blind controlled trial of bovine brain gangliosides in amyotrophic lateral sclerosis, Neurology, 34: 1079–1082 (1984).PubMedCrossRefGoogle Scholar
  5. 5.
    M. H. Brooke, R. C. Griggs, J. R. Mendell, G. M. Fenichel, J. B. Shumate, and R. J. Pellegrino, Clinical trial in Duchenne dys-trophy. I. The design of the protocol, Muscle Nerve, 4: 186–197 (1981).PubMedCrossRefGoogle Scholar
  6. 6.
    M. H. Brooke, G. M. Fenichel, R. C. Griggs, J. R. Mendell, R. Moxley, P. Miller, M. A. Province, and C. Group, Clinical investigation in Duchenne dystrophy. II. Determination of the ‘power’ of therapeutic trials based on the natural history, Muscle Nerve, 6: 91–103 (1983).Google Scholar
  7. 7.
    C. Gonnella, G. Harmon, and M. Jacobs, The role of physical therapist in the gamma globulin poliomyelitis prevention study, Phys.Ther. Rev., 33: 337–345 (1953).PubMedGoogle Scholar
  8. 8.
    M. D. Iddings, L. K. Smith, and W. A. Spencer, Muscle testing. II. Reliability in clinical use, Phys.Ther., 41: 249–256 (1961).Google Scholar
  9. 9.
    A. M. Lilienfeld, M. Jacob, and M. Willis, Study of the reproducibility of muscle testing and certain other aspects of muscle scoring, Phys.Ther.Rev., 34: 279–289 (1954).PubMedGoogle Scholar
  10. 10.
    Medical Research Council, “Aids to the Examination of the Peripheral Nervous System”, Her Majesty’s Stationery Office, London, pp. 1–2 (1976).Google Scholar
  11. 11.
    T. L. Munsat, W. Hedlund, M. Felmus, C. Cooper, and A. Gabbai, Clinical clues to the cause of amyotrophic lateral sclerosis, in: “Neuromuscular Diseases”, G. Serratrice, D. Cros, C. Desnuelle, J. Gastaut, J. Pellisier, J. Pouget, and A. Schiano, eds., pp. 329–333, Raven Press, New York (1984).Google Scholar
  12. 12.
    F. H. Norris, P. R. Calanchini, R. J. Fallat, S. Panchari, and B. Jewett, The administration of guanidine in amyotrophic lateral sclerosis, Neurology, 24: 721–728 (1971).CrossRefGoogle Scholar
  13. 13.
    O. M. Scott, S. A. Hyde, C. Goddard, and V. Dubowitz, Quantitation of muscle function in children: a prospective study in Duchenne muscular dystrophy, Muscle Nerve, 5: 291–301 (1982).PubMedCrossRefGoogle Scholar
  14. 14.
    W. G. Bradley, P. Good, C. G. Rasool, and L. S. Adelman, Morphometric and biochemical studies of peripheral nerves in amyotrophic lateral sclerosis, Ann.Neurol., 14: 267–277 (1983).PubMedCrossRefGoogle Scholar
  15. 15.
    R. J. Fallat and F. H. Norris, Respiratory problems, in: “The Diagnosis and Treatment of Amyotrophic Lateral Sclerosis:”, D. W. Mulder, ed., pp. 301–315, Houghton Mifflin Professional Publishers Medical Division, Boston (1980).Google Scholar
  16. 16.
    B. E. Tulio, Clinical trials in Duchenne muscular dystrophy: the experinece in Memphis, Tennessee, Ital.J.Neurol.Sci., Suppl. 3: 153–158 (1984).Google Scholar
  17. 17.
    P. J. Vignos, G. E. Spencer, and K. C. Archibald, Management of progressive muscular dystrophy of childhood, J.Am.Med.Assoc., 184: 89–96 (1963).CrossRefGoogle Scholar
  18. 18.
    J. Cohen, “Statistical Power Analysis for the Behavioral Sciences”, Acad. Press, New York (1977).Google Scholar
  19. 19.
    H. Harrington, M. Hallet, and H. R. Tyler, Ganglioside therapy for amyotrophic lateral sclerosis: a double-blind controlled trial, Neurology, 34: 1083–1085 (1984).PubMedCrossRefGoogle Scholar
  20. 20.
    M. R. Olarte, Therapeutic trials in amyotrophic lateral sclerosis, in: “Human Motor Neuron Disease”, L. P. Rowland, ed., pp. 555–557, Raven Press, New York (1982).Google Scholar
  21. 21.
    B. Schoenberg, Controlled therapeutic trials in motor neuron disease: methodological considerations, in: “Human Motor Neuron Disease”, L. P. Rowland, ed., pp. 547–553, Raven Press, New York (1982).Google Scholar
  22. 22.
    S. Q. Shafer and M. R. Olarte, Methodological considerations for clinical trials in motor neuron disease, in: “Human Motor Neuron Disease”, L. P. Rowland, ed., pp. 559–565, Raven Press, New York (1982).Google Scholar
  23. 23.
    H. R. Tyler, Comment, in: “Human Motor Neuron Disease”, L. P. Rowland, ed., pp.546,566, Raven Press, New York (1982).Google Scholar
  24. 24.
    C. M. Wiles and Y. Kami, The measurement of muscle strength in patients with peripheral neuromuscular disorders, J.Neurol. Neurosurg.Psych., 46: 1006–1013 (1983).CrossRefGoogle Scholar
  25. 25.
    P. L. Andres, W. Hedlund, L. Finison, T. Conlon, M. Felmus, and T. Munsat, Quantitative motor assessment in amyotrophic lateral sclerosis, Neurology, 36: 937–941 (1986).PubMedCrossRefGoogle Scholar

Copyright information

© Plenum Press, New York 1987

Authors and Affiliations

  • G. Küther
    • 1
  • A. Struppler
    • 1
  • H. G. Lipinski
    • 1
  1. 1.Neurologischen Klinik der Technischen UniversitätMünchenWest Germany

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