SCOPUS 정보 검색 플랫폼

Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration

Volumn 15, Issue 1-2, 2014, Pages 1-8

Predicting success: Optimizing phase II ALS trials for the transition to phase III

(3) Berry, James D a Cudkowicz, Merit E a Shefner, Jeremy M b

a MASSACHUSETTS GENERAL HOSPITAL (United States)

b SUNY UPSTATE MEDICAL UNIVERSITY (United States)

Author keywords

Biomarkers; Clinical trials; Phase II; RCT

Indexed keywords

DEXPRAMIPEXOLE; MINOCYCLINE; PLACEBO; TALAMPANEL; TOPIRAMATE;

AMYOTROPHIC LATERAL SCLEROSIS; CEREBROSPINAL FLUID; COMPARATIVE STUDY; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; HUMAN; OUTCOME ASSESSMENT; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PREDICTION; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW;

AMYOTROPHIC LATERAL SCLEROSIS; CLINICAL TRIALS, PHASE II AS TOPIC; CLINICAL TRIALS, PHASE III AS TOPIC; HUMANS; OUTCOME ASSESSMENT (HEALTH CARE); PREDICTIVE VALUE OF TESTS; RANDOMIZED CONTROLLED TRIALS AS TOPIC; TREATMENT OUTCOME;

EID: 84895518815 PISSN: 21678421 EISSN: 21679223 Source Type: Journal
DOI: 10.3109/21678421.2013.838969 Document Type: Review

Times cited : (10)

References (30)

1
- 84895518217
- Pascuzzi R, editor. Arhus
- Pascuzzi R, editor. 10th International Symposium on ALS and Motor Neuron Disease; 2000; Arhus.
- (2000) 10th International Symposium on ALS and Motor Neuron Disease;

2
- 0034115767
- Topiramate blocks kainate-evoked cobalt influx into cultured neurons
- Skradski S, White HS. Topiramate blocks kainate-evoked cobalt influx into cultured neurons. Epilepsia. 2000;41 (Suppl 1):S45-7.
- (2000) Epilepsia. , vol.41 , Issue.1 SUPPL.
- Skradski, S.¹ White, H.S.²

3
- 0037468293
- Topiramate protects against motor neuron degeneration in organotypic spinal cord cultures but not in G93A SOD1 transgenic mice
- DOI 10.1016/S0304-3940(02)01386-1
- Maragakis NJ, Jackson M, Ganel R, Rothstein JD.Topiramate protects against motor neuron degeneration in organotypic spinal cord cultures but not in G93A-SOD1 transgenic mice.Neurosci Lett. 2003;338:107-10. (Pubitemid 36140149)
- (2003) Neuroscience Letters , vol.338 , Issue.2 , pp. 107-110
- Maragakis, N.J.¹ Jackson, M.² Ganel, R.³ Rothstein, J.D.⁴

4
- 10744222650
- A randomized, placebo-controlled trial of topiramate in amyotrophic lateral sclerosis
- Cudkowicz ME, Shefner JM, Schoenfeld DA, Brown RH Jr, Johnson H, Qureshi M, et al. A randomized, placebo-controlled trial of topiramate in amyotrophic lateral sclerosis. Neurology. 2003;61:456-64. (Pubitemid 37025360)
- (2003) Neurology , vol.61 , Issue.4 , pp. 456-464
- Cudkowicz, M.E.¹ Shefner, J.M.² Schoenfeld, D.A.³ Brown Jr., R.H.⁴ Johnson, H.⁵ Qureshi, M.⁶ Jacobs, M.⁷ Rothstein, J.D.⁸ Appel, S.H.⁹ Pascuzzi, R.M.¹⁰ Heiman-Patterson, T.D.¹¹ Donofrio, P.D.¹² David, W.S.¹³ Russell, J.A.¹⁴ Tandan, R.¹⁵ Pioro, E.P.¹⁶ Felice, K.J.¹⁷ Rosenfeld, J.¹⁸ Mandler, R.N.¹⁹ Sachs, G.M.²⁰ more..

5
- 0037067009
- Minocycline delays disease onset and mortality in a transgenic model of ALS
- van den Bosch L, Tilkin P, Lemmens G, Robberecht W. Minocycline delays disease onset and mortality in a transgenic model of ALS. Neuroreport. 2002;13:1067-70. (Pubitemid 34587742)
- (2002) NeuroReport , vol.13 , Issue.8 , pp. 1067-1070
- Van Den Bosch, L.¹ Tilkin, P.² Lemmens, G.³ Robberecht, W.⁴

6
- 2442715084
- Placebo-controlled phase I/II studies of minocycline in amyotrophic lateral sclerosis
- Gordon PH, Moore DH, Gelinas DF, Qualls C, Meister ME, Werner J, et al. Placebo-controlled phase I/II studies of minocycline in amyotrophic lateral sclerosis. Neurology. 2004;62:1845-7. (Pubitemid 38661748)
- (2004) Neurology , vol.62 , Issue.10 , pp. 1845-1847
- Gordon, P.H.¹ Moore, D.H.² Gelinas, D.F.³ Qualls, C.⁴ Meister, M.E.⁵ Werner, J.⁶ Mendoza, M.⁷ Mass, J.⁸ Kushner, G.⁹ Miller, R.G.¹⁰

7
- 26844550494
- Minocycline in amyotrophic lateral sclerosis: A pilot study
- DOI 10.1007/s10072-005-0474-x
- Pontieri FE, Ricci A, Pellicano C, Benincasa D, Buttarelli FR. Minocycline in amyotrophic lateral sclerosis: a pilot study. Neurol Sci. 2005;26:285-7. (Pubitemid 41447830)
- (2005) Neurological Sciences , vol.26 , Issue.4 , pp. 285-287
- Pontieri, F.E.¹ Ricci, A.² Pellicano, C.³ Benincasa, D.⁴ Buttarelli, F.R.⁵

8
- 36148960127
- Efficacy of minocycline in patients with amyotrophic lateral sclerosis: A phase III randomised trial
- DOI 10.1016/S1474-4422(07)70270-3, PII S1474442207702703
- Gordon PH, Moore DH, Miller RG, Florence JM, Verheijde JL, Doorish C, et al. Efficacy of minocycline in patients with amyotrophic lateral sclerosis: a phase III randomized trial . Lancet Neurol. 2007;6:1045-53. (Pubitemid 350116691)
- (2007) Lancet Neurology , vol.6 , Issue.12 , pp. 1045-1053
- Gordon, P.H.¹ Moore, D.H.² Miller, R.G.³ Florence, J.M.⁴ Verheijde, J.L.⁵ Doorish, C.⁶ Hilton, J.F.⁷ Spitalny, G.M.⁸ MacArthur, R.B.⁹ Mitsumoto, H.¹⁰ Neville, H.E.¹¹ Boylan, K.¹² Mozaffar, T.¹³ Belsh, J.M.¹⁴ Ravits, J.¹⁵ Bedlack, R.S.¹⁶ Graves, M.C.¹⁷ McCluskey, L.F.¹⁸ Barohn, R.J.¹⁹ Tandan, R.²⁰ more..

9
- 77952168830
- A phase II trial of talampanel in subjects with amyotrophic lateral sclerosis
- Pascuzzi RM, Shefner J, Chappell AS, Bjerke JS, Tamura R, Chaudhry V, et al. A phase II trial of talampanel in subjects with amyotrophic lateral sclerosis. Amyotroph Lateral Scler. 2009;11:266-71.
- (2009) Amyotroph Lateral Scler. , vol.11 , pp. 266-271
- Pascuzzi, R.M.¹ Shefner, J.² Chappell, A.S.³ Bjerke, J.S.⁴ Tamura, R.⁵ Chaudhry, V.⁶

10
- 84862793575
- Effects of dexpramipexole on brain mitochondrial conductances and cellular bioenergetic efficiency
- Alavian KN, Dworetzky SI, Bonanni L, Zhang P, Sacchetti S, Mariggio MA, et al. Effects of dexpramipexole on brain mitochondrial conductances and cellular bioenergetic efficiency. Brain Res.1446:1-11.
- Brain Res. , vol.1446 , pp. 1-11
- Alavian, K.N.¹ Dworetzky, S.I.² Bonanni, L.³ Zhang, P.⁴ Sacchetti, S.⁵ Mariggio, M.A.⁶

11
- 84856101199
- The effects of dexpramipexole (KNS-760704) in individuals with amyotrophic lateral sclerosis
- Cudkowicz M, Bozik ME, Ingersoll EW, Miller R, Mitsumoto H, Shefner J, et al. The effects of dexpramipexole (KNS-760704) in individuals with amyotrophic lateral sclerosis. Nat Med. 2011;17:1652-6.
- (2011) Nat Med. , vol.17 , pp. 1652-1656
- Cudkowicz, M.¹ Bozik, M.E.² Ingersoll, E.W.³ Miller, R.⁴ Mitsumoto, H.⁵ Shefner, J.⁶

12
- 70249116820
- Phase II trial of CoQ10 for ALS finds insufficient evidence to justify phase III
- Kaufmann P, Thompson JL, Levy G, Buchsbaum R, Shefner J, Krivickas LS, et al. Phase II trial of CoQ10 for ALS finds insufficient evidence to justify phase III. Ann Neurol. 2009;66:235-44.
- (2009) Ann Neurol. , vol.66 , pp. 235-244
- Kaufmann, P.¹ Thompson, J.L.² Levy, G.³ Buchsbaum, R.⁴ Shefner, J.⁵ Krivickas, L.S.⁶

13
- 27644532415
- The utility of futility
- DOI 10.1161/01.STR.0000185722.99167.56
- Levin B. The utility of futility. Stroke. 2005;36:2331-2. (Pubitemid 41566945)
- (2005) Stroke , vol.36 , Issue.11 , pp. 2331-2332
- Levin, B.¹

14
- 84877621591
- A Bayesian decision-theoretic sequential response-adaptive randomization design
- Jiang F, Jack Lee J, Muller P. A Bayesian decision-theoretic sequential response-adaptive randomization design. Stat Med. 2013.
- (2013) Stat Med.
- Jiang, F.¹ Jack Lee, J.² Muller, P.³

15
- 0037379650
- A randomized sequential trial of creatine in amyotrophic lateral sclerosis
- DOI 10.1002/ana.10554
- Groeneveld GJ, Veldink JH, van der Tweel I, Kalmijn S, Beijer C, de Visser M, et al. A randomized sequential trial of creatine in amyotrophic lateral sclerosis. Ann Neurol.2003;53:437-45. (Pubitemid 36373255)
- (2003) Annals of Neurology , vol.53 , Issue.4 , pp. 437-445
- Groeneveld, G.J.¹ Veldink, J.H.² Van Der Tweel, I.³ Kalmijn, S.⁴ Beijer, C.⁵ De Visser, M.⁶ Wokke, J.H.J.⁷ Franssen, H.⁸ Van Den Berg, L.H.⁹

16
- 70149086057
- Randomized sequential trial of valproic acid in amyotrophic lateral sclerosis
- Piepers S, Veldink JH, de Jong SW, van der Tweel I, van der Pol WL, Uijtendaal EV, et al. Randomized sequential trial of valproic acid in amyotrophic lateral sclerosis. Ann Neurol.2009;66:227-34.
- (2009) Ann Neurol. , vol.66 , pp. 227-234
- Piepers, S.¹ Veldink, J.H.² De Jong, S.W.³ Van Der Tweel, I.⁴ Van Der Pol, W.L.⁵ Uijtendaal, E.V.⁶

17
- 33750586051
- Predictors of delay in the diagnosis and clinical trial entry of amyotrophic lateral sclerosis patients: A population-based study
- DOI 10.1016/j.jns.2006.06.027, PII S0022510X0600308X
- Zoccolella S, Beghi E, Palagano G, Fraddosio A, Samarelli V, Lamberti P, et al. Predictors of delay in the diagnosis and clinical trial entry of amyotrophic lateral sclerosis patients: a population based study. J Neurol Sci. 2006;250:45-9. (Pubitemid 44679704)
- (2006) Journal of the Neurological Sciences , vol.250 , Issue.1-2 , pp. 45-49
- Zoccolella, S.¹ Beghi, E.² Palagano, G.³ Fraddosio, A.⁴ Samarelli, V.⁵ Lamberti, P.⁶ Lepore, V.⁷ Serlenga, L.⁸ Logroscino, G.⁹

18
- 84862014062
- Factors affecting the diagnostic delay in amyotrophic lateral sclerosis
- Cellura E, Spataro R, Taiello AC, La Bella V. Factors affecting the diagnostic delay in amyotrophic lateral sclerosis. Clin Neurol Neurosurg. 2012;114:550-4.
- (2012) Clin Neurol Neurosurg. , vol.114 , pp. 550-554
- Cellura, E.¹ Spataro, R.² Taiello, A.C.³ La Bella, V.⁴

19
- 40849083833
- Predictors of long survival in amyotrophic lateral sclerosis: A population-based study
- DOI 10.1016/j.jns.2007.10.023, PII S0022510X07007228
- Zoccolella S, Beghi E, Palagano G, Fraddosio A, Guerra V, Samarelli V, et al. Predictors of long survival in amyotrophic lateral sclerosis: a population based study. J Neurol Sci. 2008;268:28-32. (Pubitemid 351395319)
- (2008) Journal of the Neurological Sciences , vol.268 , Issue.1-2 , pp. 28-32
- Zoccolella, S.¹ Beghi, E.² Palagano, G.³ Fraddosio, A.⁴ Guerra, V.⁵ Samarelli, V.⁶ Lepore, V.⁷ Simone, I.L.⁸ Lamberti, P.⁹ Serlenga, L.¹⁰ Logroscino, G.¹¹

20
- 37749045848
- Analysis of survival and prognostic factors in amyotrophic lateral sclerosis: A population based study
- Zoccolella S, Beghi E, Palagano G, Fraddosio A, Guerra V, Samarelli V, et al. Analysis of survival and prognostic factors in amyotrophic lateral sclerosis: a population based study. J Neurol Neurosurg Psychiatry. 2008;79:33-7.
- (2008) J Neurol Neurosurg Psychiatry. , vol.79 , pp. 33-37
- Zoccolella, S.¹ Beghi, E.² Palagano, G.³ Fraddosio, A.⁴ Guerra, V.⁵ Samarelli, V.⁶

21
- 70350111330
- Latent cluster analysis of ALS phenotypes identifies prognostically differing groups
- Ganesalingam J, Stahl D, Wijesekera L, Galtrey C, Shaw CE, Leigh PN, et al. Latent cluster analysis of ALS phenotypes identifies prognostically differing groups. PLoS One. 2009;4:e7107.
- (2009) PLoS One. , vol.4
- Ganesalingam, J.¹ Stahl, D.² Wijesekera, L.³ Galtrey, C.⁴ Shaw, C.E.⁵ Leigh, P.N.⁶

22
- 33750396937
- Amyotrophic lateral sclerosis: Early predictors of prolonged survival
- DOI 10.1007/s00415-006-0226-8
- Czaplinski A, Yen AA, Appel SH. Amyotrophic lateral sclerosis: early predictors of prolonged survival. J Neurol. 2006;253:1428-36. (Pubitemid 44963428)
- (2006) Journal of Neurology , vol.253 , Issue.11 , pp. 1428-1436
- Czaplinski, A.¹ Yen, A.A.² Appel, S.H.³

23
- 84876466100
- An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: A phase I, randomized, first-in-man study
- Miller TM, Pestronk A, David W, Rothstein J, Simpson E, Appel SH, et al. An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase I, randomized, first-in-man study. Lancet Neurol. 2013;12:435-42.
- (2013) Lancet Neurol. , vol.12 , pp. 435-442
- Miller, T.M.¹ Pestronk, A.² David, W.³ Rothstein, J.⁴ Simpson, E.⁵ Appel, S.H.⁶

24
- 0032692481
- The ALSFRS-R: A revised ALS functional rating scale that incorporates assessments of respiratory function
- DOI 10.1016/S0022-510X(99)00210-5, PII S0022510X99002105
- Cedarbaum JM, Stambler N, Malta E, Fuller C, Hilt D, Thurmond B, et al.The ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function. BDNF ALS Study Group (Phase III). J Neurol Sci. 1999;169:13-21. (Pubitemid 29502501)
- (1999) Journal of the Neurological Sciences , vol.169 , Issue.1-2 , pp. 13-21
- Cedarbaum, J.M.¹ Stambler, N.² Malta, E.³ Fuller, C.⁴ Hilt, D.⁵ Thurmond, B.⁶ Nakanishi, A.⁷

25
- 84875744026
- The Combined Assessment of Function and Survival (CAFS): A new endpoint for ALS clinical trials
- Berry JD, Miller R, Moore DH, Cudkowicz ME, van den Berg LH, Kerr DA, et al. The Combined Assessment of Function and Survival (CAFS): a new endpoint for ALS clinical trials. Amyotroph Lateral Scler Frontotemporal Degener. 2012;14:162-8.
- (2012) Amyotroph Lateral Scler Frontotemporal Degener , vol.14 , pp. 162-168
- Berry, J.D.¹ Miller, R.² Moore, D.H.³ Cudkowicz, M.E.⁴ Van Den Berg, L.H.⁵ Kerr, D.A.⁶

26
- 77956623868
- Statistical motor unit number estimation and ALS trials: The effect of motor unit instability
- Shefner JM. Statistical motor unit number estimation and ALS trials: the effect of motor unit instability. Suppl Clin Neurophysiol. 2009;60:135-41.
- (2009) Suppl Clin Neurophysiol. , vol.60 , pp. 135-141
- Shefner, J.M.¹

27
- 35148849247
- Electrical impedance myography to assess outcome in amyotrophic lateral sclerosis clinical trials
- DOI 10.1016/j.clinph.2007.08.004, PII S1388245707004099
- Rutkove SB, Zhang H, Schoenfeld DA, Raynor EM, Shefner JM, Cudkowicz ME, et al. Electrical impedance myography to assess outcome in amyotrophic lateral sclerosis clinical trials. Clin Neurophysiol. 2007;118: 2413-8. (Pubitemid 47539264)
- (2007) Clinical Neurophysiology , vol.118 , Issue.11 , pp. 2413-2418
- Rutkove, S.B.¹ Zhang, H.² Schoenfeld, D.A.³ Raynor, E.M.⁴ Shefner, J.M.⁵ Cudkowicz, M.E.⁶ Chin, A.B.⁷ Aaron, R.⁸ Shiffman, C.A.⁹

28
- 84857537860
- A proposed staging system for amyotrophic lateral sclerosis
- Roche JC, Rojas-Garcia R, Scott KM, Scotton W, Ellis CE, Burman R, et al. A proposed staging system for amyotrophic lateral sclerosis. Brain. 2012;135:847-52.
- (2012) Brain. , vol.135 , pp. 847-852
- Roche, J.C.¹ Rojas-Garcia, R.² Scott, K.M.³ Scotton, W.⁴ Ellis, C.E.⁵ Burman, R.⁶

29
- 84919719348
- Development of a Clinical Staging System for Amyotrophic Lateral Sclerosis. American Academy of Neurology; February 12, 2013; San Diego
- Chio A, Hammond E, Mora G, Bonito V, Filippini G. Development of a Clinical Staging System for Amyotrophic Lateral Sclerosis. American Academy of Neurology; February 12, 2013; San Diego: Neurology; 2013. p. P07.071.
- (2013) Neurology
- Chio, A.¹ Hammond, E.² Mora, G.³ Bonito, V.⁴ Filippini, G.⁵

30
- 84895541637
- Evaluation of a Health State Staging System Defined by Loss of Independence in Amyotrophic Lateral Sclerosis: Assessment in a Second Dataset. American Academy of Neurology; February 12, 2013; San Diego
- Hammond E, Young C, Chio A, van den Berg L, Al-Chalabi A, Berry J, et al. Evaluation of a Health State Staging System Defined by Loss of Independence in Amyotrophic Lateral Sclerosis: Assessment in a Second Dataset. American Academy of Neurology; February 12, 2013; San Diego: Neurology; 2013. p. P07.066.
- (2013) Neurology
- Hammond, E.¹ Young, C.² Chio, A.³ Van Den Berg, L.⁴ Al-Chalabi, A.⁵ Berry, J.⁶

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