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Clinical Trials and Natural History study . Vikram Shakkottai , MD, PhD University of Michigan. Ataxia trials. 82 listed studies on www.clinicaltrials.gov. Sporadic ataxia. Friedreich Ataxia. 20 studies. Dominant ataxias. Riluzole in ataxia.

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clinical trials and natural history study

Clinical Trials and Natural History study

VikramShakkottai, MD, PhD

University of Michigan

ataxia trials
Ataxia trials
  • 82 listed studies on www.clinicaltrials.gov
riluzole in ataxia
Riluzole in ataxia
  • In a randomized, double-blind, placebo-controlled pilot trial, 40 patients presenting with cerebellar ataxias of different etiologies were randomly assigned to riluzole (100 mg/day) or placebo for 8 weeks.
  • Outcome measure: 5 points in the International Cooperative Ataxia Rating Scale (ICARS).
  • The number of patients with a 5-point ICARS drop was significantly higher in the riluzole group than in the placebo group after 8 weeks (13/19 vs 1/19). The mean change in the riluzole group ICARS after treatment revealed a decrease (p < 0.001) in the total score -7.05 vs 0.16.
  • Sporadic, mild adverse events occurred.

Ristori et. Al., Neurology. 2010;74:839-45.

riluzole in ataxia7
Riluzole in ataxia

Ristori et. Al., Neurology. 2010;74:839-45.

natural history study of and genetic modifiers in spinocerebellar ataxias
Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias
  • To establish a new multidisciplinary consortium that provides the infrastructure for future clinical trials to test safety and efficacy of therapeutic interventions for spinocerebellar ataxias.
  • Rare Disease = # of Patient <200,000 in US
participating sites of sca crc
Participating Sites of SCA-CRC

Original Sites ( )

  • University of Florida: Ashizawa, Subramony
  • UCLA: Perlman
  • University of Chicago: Gomez
  • Emory University: Wilmot
  • University of Michigan: Paulson
  • University of Minnesota: Bushara
  • University of South Florida: Zesiewicz
  • University of Utah: Pulst

Voluntary Participants ( )

  • Johns Hopkins University: Ying
  • Harvard University: Schmarmann
  • UCSF: Kang
  • Columbia University: Kuo/Fahn
  • NIH, NINDS: Galpern, ORDR: Ferguson
  • EuroSCA, Brazil, Japan

Patient Support Organizations

  • National Ataxia Foundation: S. Hagan
  • Sparkman Fund
specific aims of sca crc
Specific Aims of SCA-CRC
  • Aim 1. Establish the organizational foundations for the CRC-SCA
  • Aim 2. Recruit patients, obtain longitudinal clinical data for future clinical trials, and develop novel methods for clinical trials for a small sample size.
  • Aim 3. Initiate a pilot study to determine genetic modifiers of SCA 1, 2, 3 and 6
  • Aim 4. Establish a training program for cultivating physician-scientist investigators for clinical and translational research of SCA
future directions
Future directions

Spin-off clinical trials:

  • Varenicline for SCA3
  • Lithium for SCA1
  • CoQ10 for SAOA
  • Riluzole for SCAs
acknowledgements
Acknowledgements
  • Tetsuo Ashizawa
  • H. Subramony
  • National Ataxia Foundation
  • NIH 5RC1NS068897 (PI Ashizawa)
  • Training component of the NIH 5RC1NS068897