Issues in SMA clinical trial design. The International Coordinating Committee (ICC) for SMA Subcommittee on SMA Clinical Trial Design

Neuromuscular Disorders

Volumn 17, Issue 6, 2007, Pages 499-505

  Kaufmann, P ^a   Muntoni, F ^b  

^a Och Spine at New York Presbyterian Hospitals   (United States)

^b HAMMERSMITH HOSPITAL   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

SURVIVAL MOTOR NEURON PROTEIN;

AMYOTROPHIC LATERAL SCLEROSIS; ARTICLE; CLINICAL RESEARCH; DATA BASE; DISEASE MARKER; DRUG APPROVAL; DRUG SCREENING; FOLLOW UP; GENETIC ASSOCIATION; HUMAN; LIFE EXPECTANCY; MEDICAL CARE; METHODOLOGY; MOTOR PERFORMANCE; OUTCOME ASSESSMENT; PATIENT CARE; PRIORITY JOURNAL; QUALITY OF LIFE; SAMPLE SIZE; SPINAL MUSCULAR ATROPHY; TREATMENT INDICATION; TREATMENT PLANNING;

CLINICAL TRIALS; DISTRICT OF COLUMBIA; HUMANS; INTERNATIONAL COOPERATION; MUSCULAR ATROPHY, SPINAL; OUTCOME ASSESSMENT (HEALTH CARE); QUALITY OF LIFE;

EID: 34249076977     PISSN: 09608966     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.nmd.2006.12.001     Document Type: Article

References (35)

1. Hirtz D., Iannaccone S., Heemskerk J., Gwinn-Hardy K., Moxley III R., and Rowland L.P. Challenges opportunities in clinical trials for spinal muscular atrophy. Neurology 65 (2005) 1352-1357
2. Traynor B.J., Bruijn L., Conwit R., Beal F., et al. Neuroproctective agents for clinical trials in ALS: a systematic assessment. Neurology 67 (2006) 20-27
3. .
4. ctep.cancer.gov/forms/CTCAEv3.pdf.
5. Friedman L.M., Furberg C.D., and DeMets D.L. Fundamentals of Clinical Trials (1998), Springer, New York
6. Bensimon G., Lacomblez L., and Meininger V. A controlled trial of riluzole in amyotrophic lateral sclerosis. ALS/Riluzole Study Group. N Engl J Med 330 (1994) 585-591
7. Lacomblez L., Bensimon G., Leigh P.N., Guillet P., and Meininger V. Dose-ranging study of riluzole in amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis/Riluzole Study Group II. Lancet 347 (1996) 1425-1431
8. Lacomblez L., Bensimon G., Leigh P.N., et al. A confirmatory dose-ranging study of riluzole in ALS. ALS/Riluzole Study Group-II. Neurology 47 (1996) S242-S250
9. Cudkowicz M.E., Shefner J.M., Schoenfeld D.A., et al. A randomized, placebo-controlled trial of topiramate in amyotrophic lateral sclerosis. Neurology 61 (2003) 456-464
10. Kaufmann P., Engelstad K., Wei Y., et al. Dichloroacetate causes toxic neuropathy in MELAS: a randomized, controlled clinical trial. Neurology 66 (2006) 324-330
11. Hall CT. Dying doctor's noble choice. Stricken S.F. neurologist enters own 'placebo' trial. San Francisco Chronicle 2005.
12. Cheung Y.K. On the use of nonparametric curves in phase I trials with low toxicity tolerance. Biometrics 58 (2002) 237-240
13. Cheung Y.K., and Chappell R. Sequential designs for phase I clinical trials with late-onset toxicities. Biometrics 56 (2000) 1177-1182
14. Cheung Y.K., and Chappell R. A simple technique to evaluate model sensitivity in the continual reassessment method. Biometrics 58 (2002) 671-674
15. Levy G., Kaufmann P., Buchsbaum R., et al. A two-stage design for a phase II clinical trial of coenzyme Q10 in ALS. Neurology 66 (2006) 660-663
16. Levin B. The utility of futility. Stroke 36 (2005) 2331-2332
17. Ensign L.G., Gehan E.A., Kamen D.S., and Thall P.F. An optimal three-stage design for phase II clinical trials. Statistics in medicine 13 (1994) 1727-1736
18. Simon R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials 10 (1989) 1-10
19. .
20. Groeneveld G.J., van der Tweel I., Wokke J.H., and van den Berg L.H. Sequential designs for clinical trials in amyotrophic lateral sclerosis. Amyotroph Lateral Scler Other Motor Neuron Disord 5 (2004) 202-207
21. Groeneveld G.J., Veldink J.H., van der Tweel I., et al. A randomized sequential trial of creatine in amyotrophic lateral sclerosis. Ann Neurol 53 (2003) 437-445
22. Dubowitz V. Chaos in the classification of SMA: a possible resolution. Neuromuscul Disord 5 (1995) 3-5
23. Mellies U., Dohna-Schwake C., Stehling F., and Voit T. Sleep disordered breathing in spinal muscular atrophy. Neuromuscul Disord 14 (2004) 797-803
24. Bromberg M.B., and Swoboda K.J. Motor unit number estimation in infants and children with spinal muscular atrophy. Muscle Nerve 25 (2002) 445-447
25. Bromberg M.B., and Swoboda K.J. Motor unit number estimation in infants and children with spinal muscular atrophy. Muscle Nerve 25 (2002) 445-447
26. Swoboda K.J., Prior T.W., Scott C.B., et al. Natural history of denervation in SMA: relation to age, SMN2 copy number, and function. Ann Neurol 57 (2005) 704-712
27. Crawford T.O. Concerns about the design of clinical trials for spinal muscular atrophy. Neuromuscul Disord 14 (2004) 456-460
28. Barois A., Mayer M., Desguerre I., et al. Spinal muscular atrophy. A 4-year prospective, multicenter, longitudinal study (168 cases). Bull Acad Natl Med 189 (2005) 1181-1198
29. Nelson L., Owens H., Hynan L.S., Iannaccone S.T., and Amsmart G. The gross motor function measuretrade mark is a valid and sensitive outcome measure for spinal muscular atrophy. Neuromuscul Disord (2006)
30. Iannaccone S.T. Outcome measures for pediatric spinal muscular atrophy. Arch Neurol 59 (2002) 1445-1450
31. Mercuri E., Messina S., Battini R., et al. Reliability of the Hammersmith functional motor scale for spinal muscular atrophy in a multicentric study. Neuromuscul Disord 16 (2006) 93-98
32. Main M., Kairon H., Mercuri E., and Muntoni F. The Hammersmith functional motor scale for children with spinal muscular atrophy: a scale to test ability and monitor progress in children with limited ambulation. Eur J Paediatr Neurol 7 (2003) 155-159
33. Berard C., Payan C., Fermanian J., and Girardot F. A motor function measurement scale for neuromuscular diseases - description and validation study. Rev Neurol (Paris) 162 (2006) 485-493
34. Berard C., Payan C., Hodgkinson I., and Fermanian J. A motor function measure for neuromuscular diseases. Construction and validation study. Neuromuscul Disord 15 (2005) 463-470
35. .