Design innovations and baseline findings in a long-term parkinson’s trial

The national institute of neurological disorders and stroke exploratory trials in parkinson’s disease long-term study-1

Jordan J. Elm, Robert Hauser, Barbara C. Tilley, Karl Kieburtz, Michael J. Aminoff, Erika Augustine, Susan Bennett, Ivan G. Bodis-Wollner, Franca Cambi, Julie H. Carter, Kelvin Chou, Chadwick W. Christine, Rohit Dhall, Richard B. Dewey, Rodger J. Elble, John Fang, Andrew Feigin, Wendy Galpern, Irenita Gardiner, Jennifer Harman & 30 others John Goudreau, Jorge L. Juncos, Maureen Leehey, Cornelia Kamp, Mark F. Lew, Grace S. Lin Liang, Zoltan Mari, Wayne Martin, Martha Nance, Sotirios Parashos, Rajesh Pahwa, Kelly E. Lyons, Helen Petrovitch, Brad A. Racette, Bernard Ravina, G. Webster Ross, Jacob I. Sage, Lisa Shulman, David K. Simon, Tanya Simuni, Carlos Singer, John T. Slevin, Oksana Suchowersky, Caroline M. Tanner, Aleksandar Videnovic, Tiffini S. Voss, Harrison Walker, Anne Marie Wills, Richard Zweig, The NINDS NET-PD Investigators

Research output: Contribution to journalArticle

Abstract

Based on the preclinical data and the results of a phase II futility study, creatine was Based on the preclinical data and the results of a phase II futility study, creatine was selected for an efficacy trial in Parkinson’s disease (PD). We present the design rationale and a description of the study cohort at baseline. A randomized, multicenter, double-blind, parallel-group, placebo-controlled phase III study of creatine (10 g daily) in participants with early, treated PD, the Long-term Study-1 (LS-1), is being conducted by the National Institute of Neurological Disorders and Stroke Exploratory Trials in Parkinson’s Disease network. The study utilizes a global statistical test (GST) encompassing five clinical rating scales to provide a multidimensional assessment of disease progression. A total of 1,741 PD participants from 45 sites in the United States and Canada were randomized 1:1 to either 10 g of creatine/day or matching placebo. Participants are being evaluated for a minimum of 5 years. The LS-1 baseline cohort includes participants treated with dopaminergic therapy and generally mild PD. LS-1 represents the largest cohort of patients with early treated PD ever enrolled in a clinical trial. The GST approach should provide high power to test the hypothesis that daily administration of creatine (10 g/day) is more effective than placebo in slowing clinical decline in PD between baseline and the 5-year follow-up visit against the background of dopaminergic therapy and best PD care.

Original languageEnglish (US)
Pages (from-to)1513-1521
Number of pages9
JournalMovement Disorders
Volume27
Issue number12
DOIs
StatePublished - 2012

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National Institute of Neurological Disorders and Stroke
Parkinson Disease
Creatine
Medical Futility
Placebos
Canada
Disease Progression
Cohort Studies
Clinical Trials

Keywords

  • Clinical trial
  • Creatine
  • Global statistical test
  • Neuroprotection
  • Parkinson’s disease

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

Cite this

Design innovations and baseline findings in a long-term parkinson’s trial : The national institute of neurological disorders and stroke exploratory trials in parkinson’s disease long-term study-1. / Elm, Jordan J.; Hauser, Robert; Tilley, Barbara C.; Kieburtz, Karl; Aminoff, Michael J.; Augustine, Erika; Bennett, Susan; Bodis-Wollner, Ivan G.; Cambi, Franca; Carter, Julie H.; Chou, Kelvin; Christine, Chadwick W.; Dhall, Rohit; Dewey, Richard B.; Elble, Rodger J.; Fang, John; Feigin, Andrew; Galpern, Wendy; Gardiner, Irenita; Harman, Jennifer; Goudreau, John; Juncos, Jorge L.; Leehey, Maureen; Kamp, Cornelia; Lew, Mark F.; Lin Liang, Grace S.; Mari, Zoltan; Martin, Wayne; Nance, Martha; Parashos, Sotirios; Pahwa, Rajesh; Lyons, Kelly E.; Petrovitch, Helen; Racette, Brad A.; Ravina, Bernard; Webster Ross, G.; Sage, Jacob I.; Shulman, Lisa; Simon, David K.; Simuni, Tanya; Singer, Carlos; Slevin, John T.; Suchowersky, Oksana; Tanner, Caroline M.; Videnovic, Aleksandar; Voss, Tiffini S.; Walker, Harrison; Wills, Anne Marie; Zweig, Richard; The NINDS NET-PD Investigators.

In: Movement Disorders, Vol. 27, No. 12, 2012, p. 1513-1521.

Research output: Contribution to journalArticle

Elm, JJ, Hauser, R, Tilley, BC, Kieburtz, K, Aminoff, MJ, Augustine, E, Bennett, S, Bodis-Wollner, IG, Cambi, F, Carter, JH, Chou, K, Christine, CW, Dhall, R, Dewey, RB, Elble, RJ, Fang, J, Feigin, A, Galpern, W, Gardiner, I, Harman, J, Goudreau, J, Juncos, JL, Leehey, M, Kamp, C, Lew, MF, Lin Liang, GS, Mari, Z, Martin, W, Nance, M, Parashos, S, Pahwa, R, Lyons, KE, Petrovitch, H, Racette, BA, Ravina, B, Webster Ross, G, Sage, JI, Shulman, L, Simon, DK, Simuni, T, Singer, C, Slevin, JT, Suchowersky, O, Tanner, CM, Videnovic, A, Voss, TS, Walker, H, Wills, AM, Zweig, R & The NINDS NET-PD Investigators 2012, 'Design innovations and baseline findings in a long-term parkinson’s trial: The national institute of neurological disorders and stroke exploratory trials in parkinson’s disease long-term study-1', Movement Disorders, vol. 27, no. 12, pp. 1513-1521. https://doi.org/10.1002/mds.25175
Elm, Jordan J. ; Hauser, Robert ; Tilley, Barbara C. ; Kieburtz, Karl ; Aminoff, Michael J. ; Augustine, Erika ; Bennett, Susan ; Bodis-Wollner, Ivan G. ; Cambi, Franca ; Carter, Julie H. ; Chou, Kelvin ; Christine, Chadwick W. ; Dhall, Rohit ; Dewey, Richard B. ; Elble, Rodger J. ; Fang, John ; Feigin, Andrew ; Galpern, Wendy ; Gardiner, Irenita ; Harman, Jennifer ; Goudreau, John ; Juncos, Jorge L. ; Leehey, Maureen ; Kamp, Cornelia ; Lew, Mark F. ; Lin Liang, Grace S. ; Mari, Zoltan ; Martin, Wayne ; Nance, Martha ; Parashos, Sotirios ; Pahwa, Rajesh ; Lyons, Kelly E. ; Petrovitch, Helen ; Racette, Brad A. ; Ravina, Bernard ; Webster Ross, G. ; Sage, Jacob I. ; Shulman, Lisa ; Simon, David K. ; Simuni, Tanya ; Singer, Carlos ; Slevin, John T. ; Suchowersky, Oksana ; Tanner, Caroline M. ; Videnovic, Aleksandar ; Voss, Tiffini S. ; Walker, Harrison ; Wills, Anne Marie ; Zweig, Richard ; The NINDS NET-PD Investigators. / Design innovations and baseline findings in a long-term parkinson’s trial : The national institute of neurological disorders and stroke exploratory trials in parkinson’s disease long-term study-1. In: Movement Disorders. 2012 ; Vol. 27, No. 12. pp. 1513-1521.
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abstract = "Based on the preclinical data and the results of a phase II futility study, creatine was Based on the preclinical data and the results of a phase II futility study, creatine was selected for an efficacy trial in Parkinson’s disease (PD). We present the design rationale and a description of the study cohort at baseline. A randomized, multicenter, double-blind, parallel-group, placebo-controlled phase III study of creatine (10 g daily) in participants with early, treated PD, the Long-term Study-1 (LS-1), is being conducted by the National Institute of Neurological Disorders and Stroke Exploratory Trials in Parkinson’s Disease network. The study utilizes a global statistical test (GST) encompassing five clinical rating scales to provide a multidimensional assessment of disease progression. A total of 1,741 PD participants from 45 sites in the United States and Canada were randomized 1:1 to either 10 g of creatine/day or matching placebo. Participants are being evaluated for a minimum of 5 years. The LS-1 baseline cohort includes participants treated with dopaminergic therapy and generally mild PD. LS-1 represents the largest cohort of patients with early treated PD ever enrolled in a clinical trial. The GST approach should provide high power to test the hypothesis that daily administration of creatine (10 g/day) is more effective than placebo in slowing clinical decline in PD between baseline and the 5-year follow-up visit against the background of dopaminergic therapy and best PD care.",
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AU - Tilley, Barbara C.

AU - Kieburtz, Karl

AU - Aminoff, Michael J.

AU - Augustine, Erika

AU - Bennett, Susan

AU - Bodis-Wollner, Ivan G.

AU - Cambi, Franca

AU - Carter, Julie H.

AU - Chou, Kelvin

AU - Christine, Chadwick W.

AU - Dhall, Rohit

AU - Dewey, Richard B.

AU - Elble, Rodger J.

AU - Fang, John

AU - Feigin, Andrew

AU - Galpern, Wendy

AU - Gardiner, Irenita

AU - Harman, Jennifer

AU - Goudreau, John

AU - Juncos, Jorge L.

AU - Leehey, Maureen

AU - Kamp, Cornelia

AU - Lew, Mark F.

AU - Lin Liang, Grace S.

AU - Mari, Zoltan

AU - Martin, Wayne

AU - Nance, Martha

AU - Parashos, Sotirios

AU - Pahwa, Rajesh

AU - Lyons, Kelly E.

AU - Petrovitch, Helen

AU - Racette, Brad A.

AU - Ravina, Bernard

AU - Webster Ross, G.

AU - Sage, Jacob I.

AU - Shulman, Lisa

AU - Simon, David K.

AU - Simuni, Tanya

AU - Singer, Carlos

AU - Slevin, John T.

AU - Suchowersky, Oksana

AU - Tanner, Caroline M.

AU - Videnovic, Aleksandar

AU - Voss, Tiffini S.

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AU - Wills, Anne Marie

AU - Zweig, Richard

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