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Clinical Trials and Natural History study

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

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  1. Clinical Trials and Natural History study VikramShakkottai, MD, PhD University of Michigan

  2. Ataxia trials • 82 listed studies on www.clinicaltrials.gov

  3. Sporadic ataxia

  4. Friedreich Ataxia • 20 studies

  5. Dominant ataxias

  6. 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.

  7. Riluzole in ataxia Ristori et. Al., Neurology. 2010;74:839-45.

  8. 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

  9. 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

  10. 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

  11. Current enrollment

  12. Future directions Spin-off clinical trials: • Varenicline for SCA3 • Lithium for SCA1 • CoQ10 for SAOA • Riluzole for SCAs

  13. Acknowledgements • Tetsuo Ashizawa • H. Subramony • National Ataxia Foundation • NIH 5RC1NS068897 (PI Ashizawa) • Training component of the NIH 5RC1NS068897

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