Clinical Trial: Safety and Efficacy of Lithium Carbonate in Patients With Spinocerebellar Ataxia Type 3

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Randomized Clinical Trial to Assess the Safety and Efficacy of Lithium Carbonate in Patients With Spinocerebellar Ataxia Type 3

Brief Summary:

Design: Phase II-III, double-blind, parallel, placebo controlled randomized Clinical trial

Background: Spinocerebellar ataxia type 3 (SCA-3) is an autosomal dominant adult-onset neurodegenerative disorder for which there is no current treatment. Patients will invariably become dependent from others and unable to walk during the disease course.

Hypothesis: Lithium Carbonate is safe and effective in treating neurological symptoms and improving quality of life of patients with SCA3.

Outcomes:

Primary

  • Phase 2 - To assess safety and tolerability of Lithium Carbonate in patients with SCA3 after 6 months of follow-up
  • Phase 3 (if Phase II study shows safety of therapy) - To assess efficacy of Lithium Carbonate in patients with SCA3 through the Neurological Examination Score for SCA 3 (NESSCA) after 12 months of follow-up .

Secondary

  1. - To assess efficacy on neurological function, ataxic, depressive and quality of life scores of Lithium Carbonate in patients with SCA3 through the Scale for the Assessment and Rating of Ataxia (SARA), 9-Hole Peg Board test, 8m walking time, PATA repetition rate, Click Test, SCA Functional Index (SCAFI), Composite Cerebellar Functional Score (CCFS), Beck Depression Inventory, Barthel Index and WHOQol after 6 and 12 months of follow-up.
  2. - To assess the effect of Lithium Carbonate in peripheral levels and expression of treatment biomarkers (BDNF, NSE, HDAC, GSK-3Beta)

  • Phase 2 - Safety and tolerability of Lithium Carbonate treatment in patients with SCA3 [ Time Frame: 6 months ]
    According to the Common toxicity criteria manual, version 2.0
  • Phase 3 - Efficacy of Lithium Carbonate treatment in patients with SCA3 [ Time Frame: 12 months ]
    Application of the Neurological Examination Score for SCA 3 (NESSCA)


    • Original Primary Outcome:

    • Safety and tolerability of Lithium Carbonate treatment in patients with SCA3 [ Time Frame: 12 months ]
      According to the Common toxicity criteria manual, version 2.0
    • Efficacy of Lithium Carbonate treatment in patients with SCA3 [ Time Frame: 12 months ]
      Application of the Scale for the Assessment and Rating of Ataxia (SARA)


    Current Secondary Outcome:

    • Efficacy of Lithium Carbonate in patients with SCA3 on neurological function, ataxic, depressive and quality of life scores [ Time Frame: 6 and 12 months ]
      Scale for the Assessment and Rating of Ataxia (SARA), 9-Hole Peg Board test, 8m walking time, PATA repetition rate, Click Test, SCA Functional Index (SCAFI), Composite Cerebellar Functional Score (CCFS), Beck Depression Inventory, Barthel Index and WHOQol
    • Effect of Lithium Carbonate treatment in peripheral levels and expression of treatment biomarkers [ Time Frame: 3 and 6 months ]
      BDNF, NSE, HDAC, GSK-3Beta


    Original Secondary Outcome:

    • Efficacy of Lithium Carbonate in patients with SCA3 on neurological function, ataxic, depressive and quality of life scores [ Time Frame: 12 months ]
      Neurological Examination Score for SCA 3 (NESSCA), 9-Hole Peg Board test, Beck Depression Inventory and WHOQol
    • Effect of Lithium Carbonate treatment in peripheral levels and expression of treatment biomarkers [ Time Frame: 12 months ]
      BDNF, NSE, HDAC, GSK-3Beta


    Information By: Hospital de Clinicas de Porto Alegre

    Dates:
    Date Received: March 29, 2010
    Date Started: May 2011
    Date Completion:
    Last Updated: January 29, 2013
    Last Verified: January 2013