Safety and Tolerability of Lithium in Spinocerebellar Ataxia 2 (SCA2)

Randomized, Placebo-controlled Trial to Test Safety, Tolerability and Efficacy of Lithium Carbonate in Spinocerebellar Ataxia 2

The purpose of this study is to determine safety and tolerability of the treatment with lithium in Spinocerebellar Ataxia 2. Moreover, clinical symptoms, neuronal loss, quality of life and depressive symptoms, will be considered to further investigate the effect of lithium therapy.

Patients will be progressively enrolled in the study and undergo a screening visit to test for inclusion/exclusion criteria. Patients will then be randomized to receive either Lithium carbonate or placebo. Patients will visit study center at 2, 4, 8, 12, 24, 36 and 48 weeks, for endpoint and laboratory assessments.

Inclusion Criteria:

• Molecular diagnosis of SCA2 (≥34 CAG in the ataxin-2 gene)
• Age ≥18, <80
• SARA score ≥8

Exclusion Criteria:

• SARA score >32
• Heart failure
• Liver disease
• Kidney failure
• Thyroid disease
• Sick sinus syndrome and/or significant ECG alterations
• Hyposodemia
• Treatment with diuretics
• Treatment with haloperidol and/or other antipsychotics
• Treatment with NSAIDs or corticosteroids
• Treatment with ACE inhibitors
• Treatment with aminophyllines
• Treatment with mannitol
• Pregnancy and/or breastfeeding
• Acute diseases that might interfere with the trial