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

Federico II University20 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

SPINOCEREBELLAR ATAXIA 2

Interventions

LITHIUM CARBONATEDRUG

Lithium Carbonate will be dosed based on lithiemy, which will be in the range 0.9-1.2 mEq/L. Maximum allowed dose will be 1500mg/day.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 80 Years

Medical Language ↔ Plain English

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

Locations (1)

Dipartimento di Scienze Neurologiche

Naples, Italy

Outcomes

Primary Outcomes

Primary endpoint of the study will be the difference in the number and frequency of Severe Adverse Events (SAE) and Non Severe Adverse Events (nSAE) recorded during the study, between treatment and placebo group.

Adverse events and Severe Adverse events will be recorded during the trial at each visit starting from Baseline to Visit 8 at 48 weeks of treatment.

Time frame: the endpoint will be recorded at all visits

Secondary Outcomes

Secondary outcome will be the Scale for the Assessment and Rating of Ataxia (SARA). Statistical analysis will be performed to compare the effect of treatment on both groups.

Time frame: 0 weeks

Micro- and macrostructural Magnetic Resonance parameters will be compared before and after treatment. This will include Voxel Based Morphometry, resting functional MRI, Diffusion tensor imaging and MRI spectroscopy.

Time frame: 0 weeks

The effect of Lithium on mood will be explored with the Beck depression inventory.

Time frame: 0 weeks

Effect of Lithium on quality of life will be assessed with the EQ-5D scale.

Time frame: 0 weeks

Secondary outcome will be the Scale for the Assessment and Rating of Ataxia (SARA). Statistical analysis will be performed to compare the effect of treatment on both groups.

Time frame: 24 weeks

Secondary outcome will be the Scale for the Assessment and Rating of Ataxia (SARA). Statistical analysis will be performed to compare the effect of treatment on both groups.

Time frame: 48 weeks

Data from ClinicalTrials.gov

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