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

Phase 2

Completed

• Conditions: SPINOCEREBELLAR ATAXIA 2
• Interventions: Drug: LITHIUM CARBONATE

• Registration Number: NCT00998634
• Lead Sponsor: Federico II University

Brief Summary

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.

Detailed 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.

Study Timeline

• Study Start: 2009-10
• Study First Submitted: 2009-10-16
• Study First Submitted QC: 2009-10-19
• Study First Posted: 2009-10-20
• Primary Completion: 2012-11
• Study Completion: 2012-11
• Status Verified: 2013-01
• Last Update Submitted: 2013-01-07
• Last Update Posted: 2013-01-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
LITHIUM CARBONATE 150 and/or 300 mg	LITHIUM CARBONATE	-
PLACEBO	LITHIUM CARBONATE	-

Primary Outcome Measures

Name	Time	Method
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.	the endpoint will be recorded at all visits	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.

Secondary Outcome Measures

Name	Time	Method
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.	48 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.	48 weeks
The effect of Lithium on mood will be explored with the Beck depression inventory.	48 weeks
Effect of Lithium on quality of life will be assessed with the EQ-5D scale.	48 weeks

Trial Locations

Locations (1)

Dipartimento di Scienze Neurologiche; 🇮🇹 Napoli, Italy