Advances in clinical trials for amyotrophic lateral sclerosis

Current Neurology and Neuroscience Reports

Volumn 5, Issue 1, 2005, Pages 48-54

  Gordon, Paul H ^a  

^a NEUROLOGICAL INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA TOCOPHEROL; BRAIN DERIVED NEUROTROPHIC FACTOR; CEFTRIAXONE; CELECOXIB; CILIARY NEUROTROPHIC FACTOR; CREATINE; CYCLOPHOSPHAMIDE; DEXTROMETHORPHAN; GABAPENTIN; IMMUNOGLOBULIN; LAMOTRIGINE; MINOCYCLINE; PENTOXIFYLLINE; QUINIDINE; RILUZOLE; SELEGILINE; SOMATOMEDIN C; SOMATOMEDIN C RECEPTOR; TCH 346; TOPIRAMATE; UBIQUINONE; UNCLASSIFIED DRUG; XALIPRODEN;

AMYOTROPHIC LATERAL SCLEROSIS; BLOOD CLOTTING DISORDER; BODY WEIGHT DISORDER; CLINICAL TRIAL; DIAGNOSTIC ACCURACY; DISEASE COURSE; DOSE RESPONSE; DRUG EFFICACY; DRUG MECHANISM; DRUG SAFETY; EXPERIMENTAL DESIGN; HUMAN; HYPOTHESIS; LUNG EMBOLISM; MENTAL INSTABILITY; METHODOLOGY; NEPHROLITHIASIS; NEUROPROTECTION; NONHUMAN; OPEN STUDY; OUTCOMES RESEARCH; PATIENT CARE; PATIENT MONITORING; QUALITY OF LIFE; RATING SCALE; RESPIRATORY FUNCTION; REVIEW; RISK BENEFIT ANALYSIS; STEM CELL TRANSPLANTATION; TRANSGENIC MOUSE;

EID: 16344364681     PISSN: 15284042     EISSN: None     Source Type: Journal    
DOI: 10.1007/s11910-005-0023-2     Document Type: Review

References (39)

1. Pascuzzi RM: Blinded and seeing the light, (John Noseworthy), Lou Gehrig and other tales of enlightenment. Semin Neurol 1998, 18:415-418.
2. Rowland LP, Shneider NA: Amyotrophic lateral sclerosis. N Engl J Med 2001, 344:1688-1700.
3. Young FE, Norris JA, Levitt JA, Nightingale SL: The FDA's new procedures for the use of investigational drugs in treatment. JAMA 1988, 25:2267-2270.
4. Cedarbaum JM, Stambler N: Disease status and use of ventilatory support by ALS patients. BDNF Study Group. Amyotroph Lateral Scler Other Motor Neuron Disord 2001, 2:19-22.
5. Miller RG, Mitchell JD, Lyon M, Moore DH: Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND). Cochrane Neuromuscular Disease Group. Cochrane Database of Systematic Reviews 2004, 2.
6. ALS CNTF Treatment Study (ACTS) Study Group: A double-blind placebo-controlled clinical trial of subcutaneous recombinant human ciliary neurotrophic factor (rHCNTF) in amyotrophic lateral sclerosis. Neurology 1996, 46:1244-1249.
7. Cudkowicz ME, Shefner JM, Schoenfeld DA, et al.: Northeast ALS Consortium: A randomized, placebo-controlled trial of topiramate in amyotrophic lateral sclerosis. Neurology 2003, 61:456-464.
8. Miller RG, Moore DH 2nd, Gelinas DF, et al.; Western ALS Study Group: Phase III randomized trial of gabapentin in patients with amyotrophic lateral sclerosis. Neurology 2001, 56: 43-848.
9. Sorenson: A Comparison of musle strength testing techniques in amyotrophic lateral sclerosis. Paper presented at the 12th International Symposium on ALS/MND, Oakland, CA, October, 2001.
10. A controlled trial of recombinant methionyl human BDNF in ALS: the BDNF study group (phase III). Neurology 1999, 52: 427-1433.
11. Meininger V, Bensimon G, Bradley WR, et al.: Efficacy and safety of xaliproden in amyotrophic lateral sclerosis: results of two phase III trials. Amyotroph Lateral Scler Other Motor Neuron Disord 2004, 5:107-117.
12. The ALS CNTF Treatment Study (ACTS) Phase I-II Study Group: The amyotrophic lateral sclerosis functional rating scale. Assessment of activities of daily living in patients with amyotrophic lateral sclerosis. Arch Neurol 1996, 53:141-147.
13. Cedarbaum JM, Stambler N: Performance of the amyotrophic lateral sclerosis functional rating scale (ALSFRS) in multicenter clinical trials. J Neurol Sci 1997, 152 (Suppl 1):S1-S9.
14. Cedarbaum JM, Stambler N, Malta E, et al.: The ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function. BDNF ALS Study Group (Phase II /III). J Neurol Sci 1999, 169:13-21.
15. Kaufmann P, Levy G, Thompson JL, et al.: The ALSFRSr predicts mortality in an ALS clinic population. Neurology 2004, In press.
16. Kaufmann P, Pullman SL, Shungu DC, Chan S: Objective tests for upper motor neuron involvement in amyotrophic lateral sclerosis. Neurology 2004, 62:1753-1757.
17. Bogdanov M, Brown RH, Matson W, et al.: Increased oxidative damage to DNA in ALS patients. Free Radic Biol Med 2000, 29:652-658.
18. Beal MF, Ferrante RJ, Browne SE, et al.: Increased 3-nitrotyrosine in both sporadic and familial amyotrophic lateral sclerosis. Ann Neurol 1997, 42:644-654.
19. Simpson EP, Henry YK, Henkel JS, et al.: Increased lipid peroxidation in sera of ALS patients. A potential biomarker of disease burden. Neurology 2004, 62:1758-1765.
20. Beuche W, Yushchenko M, Mader M, et al.: Matrix metalloproteinase-9 is elevated in serum of patients with amyotrophic lateral sclerosis. Neuroreport 2000, 11:3419-3422.
21. Houi K, Kobayashi T, Kato S, et al.: Increased plasma TGF-beta1 in patients with ALS. Acta Neurol Scand 2002, 106:299-301.
22. McComas AJ, Fawcett PR, Campbell MJ, Sica RE: Electrophysiological estimation of the number of motor units within a human muscle. J Neurol Neurosurg Psychiatry 1971, 34: 21-131.
23. Brooks BR, Miller RG, Swash M, Munsat TL, for the World Federation of Neurology Research Group on Motor Neuron Diseases: El Escorial revisited: revised criteria for the diagnosis of amyotrophic lateral sclerosis. Amyotroph Lateral Scler Other Motor Neuron Disord 2000, 1:293-299.
24. Gordon PH, Moore DH, Gelinas DF, et al.: Placebo controlled phase I/II studies of minocycline in amyotrophic lateral sclerosis. Neurology 2004, 62:1845-1847.
25. Mitsumoto H, Chad DA, Pioro EP: Treatment trials. In Amyotrophic Lateral Sclerosis. Edited by Mitsumoto H, Chad DA, Pioro EP. Philadelphia: FA Davis; 1998:329-354.
26. Palesch YY, Tilley BC, Broaderick JP: Value of a single-arm futility design for Phase II clinical trial of acute stroke therapy. Neurology 2003, 60(Suppl 1):A339.
27. Bradley WG: The need for double-blind controlled trials in amyotrophic lateral sclerosis. In ALS - From Charcot to the Present and Into the Future. Edited by Rose FC. London; Smith-Gordon; 1994:263-265.
28. Bensimon G, Lacomblez L, Meininger V: A controlled trial of riluzole in amyotrophic lateral sclerosis. ALS/Riluzole Study Group. N Engl J Med 1994, 330:585-591.
29. Lacomblez L, Bensimon G, Leigh PN, et al.: Dose-ranging study of riluzole in amyotrophic lateral sclerosis. Amyotrophic lateral sclerosis/riluzole study group II. Lancet 1996, 347:1425-1431.
30. Groeneveld GJ, Veldink JH, van der Tweel I, et al.: A randomized sequential trial of creatine in amyotrophic lateral sclerosis. Ann Neurol 2003, 53:437-445.
31. Smith RA, Berg JE, Pope LE, Thisted RA: Measuring pseudobulbar affect in amyotrophic lateral sclerosis. Amyotroph Lateral Scler Other Motor Neuron Disord 2004, In press.
32. Mitsumoto H, Davidson M, Moore D, et al.; ALS CARE Study Group: Percutaneous endoscopic gastrostomy (PEG) in patients with ALS and bulbar dysfunction. Amyotroph Lateral Scler Other Motor Neuron Disord 2003, 4:177-185.
33. Miller RG, Rosenberg JA, Gelinas DF, et al.: Practice parameter: the care of the patient with amyotrophic lateral sclerosis (an evidence-based review): report of the Quality Standards Subcommittee of the American Academy of Neurology: ALS Practice parameters Task Force. Neurology 1999, 52:1311-1323.
34. Janson CG, Ramesh TM, During MJ, et al.: Human intrathecal transplantation of peripheral blood stem cells in amyotrophic lateral sclerosis. J Hematother Stem Cell Res 2001, 10: 13-915.
35. Mazzini L, Fagioli F, Boccaletti R, et al.: Stem cell therapy in amyotrophic lateral sclerosis: a methodological approach in humans. Amyotroph Lateral Scler Other Motor Neuron Disord 2003, 4:158-161.
36. Kriz J, Nguyen MD, Julien JP: Minocycline slows disease progression in a mouse model of amyotrophic lateral sclerosis. Neurobiol Dis 2002, 10:268-278.
37. Van Den Bosch L, Tilkin P, Lemmens G, Robberecht W: Minocycline delays disease onset and mortality in a transgenic model of ALS. Neuroreport 2002, 13:1067-1070.
38. Zhu S, Stravrovskaya IG, Drozda M, et al.: Minocycline inhibits cytochrome c release and delays progression of amyotrophic lateral sclerosis in mice. Nautre 2002, 417: 4-78.
39. Haynes RB, Dames R: Patient compliance and the conduct and interpretation of therapeutic trials. Control Clin Trial 1987, 8:12-19.