Spinocerebellar Ataxia Type 3 Clinical Trial
Official title:
Randomized Clinical Trial to Assess the Safety and Efficacy of Lithium Carbonate in Patients With Spinocerebellar Ataxia Type 3
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)
Study Duration: 12 months
- Final analysis of phase 2 (safety study) at 6 months with continuous monitoring until
the end of phase 3 (efficacy study).
- Preliminary analysis of efficacy on ataxia scales at 6 months of study and final
analysis of phase 3 at 12 months.
Obs: A futility analysis will be performed after 12 months of therapy if no statistically
significant difference between groups were found. This analysis will define if the study
will continue until 18 or 24 months of follow-up or will be ended at 12 months.
Location: Hospital de Clínicas de Porto Alegre
Subjects: 60 molecularly diagnosed SCA3 patients from the outpatient unit of the Medical
Genetics Service of Hospital de Clínicas de Porto Alegre
Intervention: Lithium Carbonate tablets of 300mg. Starting dose will be 300mg/day with drug
titration during 49 days or until achieving the defined target lithium serum level of 0.5 to
0.8 mEq/L
n/a
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
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