Safety and Tolerability of Lithium in Spinocerebellar Ataxia 2 (SCA2) (LISCA2)

Federico II University

Status and phase: Completed; Phase 2

Conditions: SPINOCEREBELLAR ATAXIA 2

Treatments: Drug: LITHIUM CARBONATE

Study type: Interventional

Funder types: Other

Identifiers

NCT00998634

SCA_LITIO_12

EUDRACT N°2009-016317-20

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 20 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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20 participants in 2 patient groups, including a placebo group

LITHIUM CARBONATE 150 and/or 300 mg

Experimental group

Treatment:

Drug: LITHIUM CARBONATE

PLACEBO

Placebo Comparator group

Treatment:

Drug: LITHIUM CARBONATE