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
- - 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.
- - To assess the effect of Lithium Carbonate in peripheral levels and expression of treatment biomarkers (BDNF, NSE, HDAC, GSK-3Beta)
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