Randomized, Placebo-controlled Trial to Test Safety, Tolerability and Efficacy of Lithium Carbonate in Spinocerebellar Ataxia 2
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Enrollment
- 20
- Locations
- 1
- Primary Endpoint
- 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.
Study Overview
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 Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 18 Years to 80 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
PLACEBO
Intervention: LITHIUM CARBONATE (Drug)
LITHIUM CARBONATE 150 and/or 300 mg
Intervention: LITHIUM CARBONATE (Drug)
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.
Time Frame: 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 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.(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)
Investigators
Alessandro Filla
Principal Investigator
Federico II University