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

The NINDS NET-PD Investigators

Research output: Contribution to journalArticle

45 Citations (Scopus)

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

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title = "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",
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.",
keywords = "Clinical trial, Creatine, Global statistical test, Neuroprotection, Parkinson’s disease",
author = "{The NINDS NET-PD Investigators} and Elm, {Jordan J.} and Robert Hauser and Tilley, {Barbara C.} and Karl Kieburtz and Aminoff, {Michael J.} and Erika Augustine and Susan Bennett and Bodis-Wollner, {Ivan G.} and Franca Cambi and Carter, {Julie H.} and Kelvin Chou and Christine, {Chadwick W.} and Rohit Dhall and Dewey, {Richard B.} and Elble, {Rodger J.} and John Fang and Andrew Feigin and Wendy Galpern and Irenita Gardiner and Jennifer Harman and John Goudreau and Juncos, {Jorge L.} and Maureen Leehey and Cornelia Kamp and Lew, {Mark F.} and {Lin Liang}, {Grace S.} and Zoltan Mari and Wayne Martin and Martha Nance and Sotirios Parashos and Rajesh Pahwa and Lyons, {Kelly E.} and Helen Petrovitch and Racette, {Brad A.} and Bernard Ravina and {Webster Ross}, G. and Sage, {Jacob I.} and Lisa Shulman and Simon, {David K.} and Tanya Simuni and Carlos Singer and Carlos Singer and Oksana Suchowersky and Tanner, {Caroline M.} and Aleksandar Videnovic and Voss, {Tiffini S.} and Harrison Walker and Wills, {Anne Marie} and Richard Zweig",
year = "2012",
doi = "10.1002/mds.25175",
language = "English (US)",
volume = "27",
pages = "1513--1521",
journal = "Movement Disorders",
issn = "0885-3185",
publisher = "John Wiley and Sons Inc.",
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T1 - Design innovations and baseline findings in a long-term parkinson’s trial

T2 - The national institute of neurological disorders and stroke exploratory trials in parkinson’s disease long-term study-1

AU - The NINDS NET-PD Investigators

AU - Elm, Jordan J.

AU - Hauser, Robert

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 - Singer, Carlos

AU - Suchowersky, Oksana

AU - Tanner, Caroline M.

AU - Videnovic, Aleksandar

AU - Voss, Tiffini S.

AU - Walker, Harrison

AU - Wills, Anne Marie

AU - Zweig, Richard

PY - 2012

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AB - 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.

KW - Clinical trial

KW - Creatine

KW - Global statistical test

KW - Neuroprotection

KW - Parkinson’s disease

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JF - Movement Disorders

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